3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details


臨床試験数 : 217 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80

  
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PhaseCountries
1NCT05115110
(ClinicalTrials.gov)
March 31, 20221/11/2021A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulatory Children With Spinal Muscular AtrophyA Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulant Patients With Spinal Muscular AtrophySpinal Muscular Atrophy (SMA)Drug: RO7204239;Drug: Placebo;Drug: RisdiplamHoffmann-La RocheNULLNot yet recruiting2 Years10 YearsAll180Phase 2/Phase 3United States;Belgium;Germany;Italy;Netherlands;Poland;United Kingdom
2NCT05232929
(ClinicalTrials.gov)
March 31, 202231/1/2022Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam TreatmentSpinal Muscular AtrophyDrug: RisdiplamGenentech, Inc.NULLRecruiting2 MonthsN/AAll500Phase 4United States
3NCT05272969
(ClinicalTrials.gov)
March 31, 202228/1/2022Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseasePompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD)Pompe Disease (Late-onset);Inclusion Body Myositis;Spinal Muscular Atrophy Type 3;FSHDDiagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire);Diagnostic Test: German Pain Inventory (Questionnaire);Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire);Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire);Diagnostic Test: R-PAct (Questionnaire);Diagnostic Test: Quick Motor Function Test;Diagnostic Test: Handheld Dynamometry (HHD);Diagnostic Test: Six-minute walk test (6MWT);Diagnostic Test: Pressure pain threshold;Diagnostic Test: Muscle ultrasound;Diagnostic Test: Vital signs;Diagnostic Test: Borg Scale;Diagnostic Test: Laboratory assessment: Creatine kinase;Diagnostic Test: Laboratory assessment: Vitamin D Level;Diagnostic Test: Laboratory assessment: calcium;Diagnostic Test: Laboratory assessment: magnesium;Diagnostic Test: Laboratory assessment: phosphate;Genetic: Genetic test: ACE polymorphism;Genetic: Genetic test: ACTN3 polymorphism;Genetic: Blood draw for optional genetic exome sequencingLMU KlinikumNULLNot yet recruiting18 YearsN/AAll95Germany
4NCT05067790
(ClinicalTrials.gov)
January 21, 202224/9/2021A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With RisdiplamA Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With RisdiplamSpinal Muscular AtrophyDrug: NusinersenBiogenNULLRecruiting5 Years39 YearsAll135Phase 3United States
5EUCTR2021-003417-19-PL
(EUCTR)
18/01/202225/10/2021A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: N/A
Product Code: RO7204239/F01-01
INN or Proposed INN: N/A
Other descriptive name: GYM329
Trade Name: Evrysdi
INN or Proposed INN: Risdiplam
F.Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 2;Phase 3United States;Belgium;Poland;Netherlands;United Kingdom;Italy
6EUCTR2021-003417-19-IT
(EUCTR)
03/01/202218/10/2021A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY - NA Spinal Muscular Atrophy (SMA)
MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850
MedDRA version: 20.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079413;Term: Spinal muscular atrophy type I;Classification code 10079416;Term: Spinal muscular atrophy type II;Classification code 10079417;Term: Spinal muscular atrophy infantile onset;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;Classification code 10079418;Term: Spinal muscular atrophy later onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: N/A
Product Code: [RO7204239/F01-01]
Trade Name: Evrysdi
Product Name: Risdiplam
Product Code: [RO7034067]
INN or Proposed INN: Risdiplam
F. HOFFMANN - LA ROCHE LTD.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 2;Phase 3United States;Poland;Belgium;Netherlands;United Kingdom;Italy
7EUCTR2020-005995-37-PT
(EUCTR)
15/10/202119/07/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion
Product Name: OAV101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 3United States;France;Portugal;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy
8EUCTR2020-005995-37-IT
(EUCTR)
28/09/202117/08/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Prednisolone
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Trade Name: Prednisolone
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Product Name: Prednisolone
Product Code: [-]
INN or Proposed INN: PREDNISOLONE
Trade Name: Zolgensma
Product Name: OAV101
Product Code: [AVXS-101]
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: Previously termed sc.AAV9.CB.SMN and AVXS-101
NOVARTIS PHARMA AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy
9EUCTR2020-005995-37-BE
(EUCTR)
03/09/202128/06/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion
Product Name: OAV101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 3Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy
10EUCTR2020-005995-37-FR
(EUCTR)
25/08/202121/06/2021Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma
Product Name: OAV101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3Portugal;United States;France;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy
11JPRN-jRCT2061200040
03/08/202125/12/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Spinal Muscular Atrophy- Part A: Participants with later-onset SMA will receive loading doses of 28 mg of nusinersen intrathecally on Days 1, 15 and 29 followed by maintenance doses of 28 mg on Days 149 and 269.

- Active Comparator
Part B: Participants with infantile- or later-onset SMA will receive loading doses of 12 mg of nusinersen intrathecally on Days 1, 15, 29, and 64 followed by maintenance doses of 12 mg on Days 183 and 279. Sham procedure will be administered on Day 135.

- 50/28 mg Group
Part B: Participants with infantile- or later-onset SMA will receive loading doses of 50 mg of nusinersen intrathecally on Days 1 and 15 followed by maintenance doses of 28 mg on Days 135 and 279. Sham procedure will be administered on Days 29, 64 and 183.

- Part C: Participants who have been receiving the approved dose for at least 1 year prior to entry in this study, will receive a single bolus dose of 50 mg of nusinersen intrathecally on Day 1 (4 months after their most recent maintenance dose) followed by maintenance doses of 28 mg on Days 121 and 241.
Berger ZdenekNULLRecruitingNot applicableNot applicableBoth152Phase 2-3Spain;Taiwan;United States;Japan;Estonia;Ireland;Latvia;Colombia
12EUCTR2020-004708-32-HU
(EUCTR)
15/06/202117/04/2021Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With NusinersenA Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 3United States;Taiwan;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
13EUCTR2020-003492-18-DE
(EUCTR)
07/06/202118/03/2021A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecA Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 4United States;Spain;Israel;Germany;Italy
14EUCTR2020-003492-18-ES
(EUCTR)
02/06/202101/09/2021A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecA Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 4United States;Spain;Israel;Germany;Italy
15EUCTR2020-004708-32-ES
(EUCTR)
05/05/202109/02/2021Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With NusinersenA Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 3United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
16EUCTR2020-003492-18-IT
(EUCTR)
26/04/202108/06/2021A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecA Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Trade Name: Spinraza
Product Name: .
Product Code: [.]
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
BIOGEN IDEC RESEARCH LIMITEDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 4United States;Spain;Israel;Germany;Italy
17NCT04729907
(ClinicalTrials.gov)
April 19, 202111/12/2020Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With NusinersenA Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With NusinersenMuscular Atrophy, SpinalDrug: NusinersenBiogenNULLEnrolling by invitationN/AN/AAll172Phase 3United States;Brazil;Colombia;Estonia;Hungary;Spain;Taiwan;Argentina;Australia;Canada;Chile;France;Germany;Greece;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Turkey;United Kingdom
18EUCTR2019-002663-10-DE
(EUCTR)
18/02/202123/10/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 2;Phase 3United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
19NCT04488133
(ClinicalTrials.gov)
January 7, 202120/7/2020A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecA Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene AbeparvovecMuscular Atrophy, SpinalDrug: NusinersenBiogenNULLRecruiting2 Months36 MonthsAll60Phase 4United States;Germany;Israel;Italy;Spain
20ChiCTR2000041208
2020-12-302020-12-22A randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophyA randomized, double-blind, placebo parallel controlled clinical trial on the efficacy and safety of salbutamol sulfate tablets in the treatment of children with spinal muscular atrophy spinal muscular atrophyexperimental group: salbutamol sulfate tablets ;control group:placebo;The Children's Hospital, Zhejiang University School of MedicineNULLRecruiting217Bothexperimental group:60;control group:30;Phase 4China
21EUCTR2019-002663-10-FR
(EUCTR)
23/12/202023/10/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
22EUCTR2019-002663-10-GB
(EUCTR)
17/12/202001/09/2020Study of Nusinersen (BIIB058) in Participants With Spinal MuscularAtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nursinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: Nursinersen
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
23NCT04576494
(ClinicalTrials.gov)
December 202029/9/2020Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms): a Multicenter Single-case Experimental Design in Multiple Baselines Across Subjects, Randomized, Single-blinded EvaluationSpinal Muscular AtrophyOther: Monthly assessments of functional motor abilities by a trained therapist;Drug: NusinersenCHU de ReimsNULLNot yet recruiting18 YearsN/AAll24N/AFrance
24EUCTR2016-000750-35-GB
(EUCTR)
25/11/202023/06/2016A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RISDIPLAM (RO7034067) IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
231Phase 2United States;Serbia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Bulgaria;Germany;China;Japan
25EUCTR2019-002611-26-FR
(EUCTR)
16/09/202010/03/2020not applicableA Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
308Phase 3Korea, Republic of;Italy;Japan;United Kingdom;Australia;Belgium;Spain;Canada;Taiwan;France;United States
26NCT04404764
(ClinicalTrials.gov)
May 27, 202019/5/2020Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational StudyCharacterization of the Clinical-epidemiological Profile of Patients With Spinal Muscular Atrophy (SMA) 5q Types II and III in Follow-up in the Brazilian Unified Public Health System: A Cross-sectional Observational Study (Registry)Spinal Muscular AtrophyDrug: Nusinersen Injectable ProductHospital Israelita Albert EinsteinMinistry of Health, BrazilCompleted6 MonthsN/AAll155Brazil
27EUCTR2019-002663-10-ES
(EUCTR)
06/05/202010/05/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
28EUCTR2019-002611-26-IT
(EUCTR)
27/04/202003/08/2021not applicableA Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 - not applicable Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: AVXS-101
Product Code: [AVXS-101]
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
AVEXIS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
308Phase 3France;United States;Taiwan;Canada;Spain;Belgium;Australia;United Kingdom;Japan;Italy;Korea, Republic of
29EUCTR2019-002663-10-HU
(EUCTR)
23/04/202005/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
30EUCTR2019-002611-26-GB
(EUCTR)
30/03/202031/12/2019not applicableA Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
308Phase 3United States;France;Taiwan;Canada;Spain;Belgium;Australia;Japan;Italy;United Kingdom;Korea, Republic of
31EUCTR2019-002663-10-LV
(EUCTR)
27/03/202012/02/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2;Phase 3United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of
32NCT04089566
(ClinicalTrials.gov)
March 26, 202011/9/2019Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyMuscular Atrophy, SpinalDrug: NusinersenBiogenNULLRecruitingN/AN/AAll172Phase 2/Phase 3United States;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;United Kingdom
33EUCTR2019-002663-10-IT
(EUCTR)
05/03/202017/06/2021Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: [ ISIS 396443, BIIB058]
Other descriptive name: NUSINERSEN SODIUM
Trade Name: Spinraza
Product Name: Nusinersen
Product Code: [ISIS 396443, BIIB058]
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
BIOGEN IDEC RESEARCH LIMITEDNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
152Phase 2;Phase 3United States;Taiwan;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
34EUCTR2019-002611-26-ES
(EUCTR)
28/02/202014/02/2020not applicableA Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
308Phase 3Italy;Korea, Republic of;United States;France;Taiwan;Canada;Belgium;Japan;United Kingdom;Australia;Spain
35NCT04042025
(ClinicalTrials.gov)
February 10, 202031/7/2019Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiA Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III;SMABiological: Onasemnogene Abeparvovec-xioiNovartis Gene TherapiesNULLEnrolling by invitationN/AN/AAll308Phase 4United States;Australia;Belgium;Canada;France;Italy;Japan;Taiwan;United Kingdom;Korea, Republic of;Spain
36EUCTR2018-004383-65-DE
(EUCTR)
21/11/201926/07/2019A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular AtrophyPhase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study Later Onset Spinal Muscular Atrophy (SMA)
MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: SRK-015
INN or Proposed INN: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Product Name: SRK-015
INN or Proposed INN: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Scholar Rock, Inc.NULLNot Recruiting Female: yes
Male: yes
55 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Spain;Netherlands;Germany;Italy
37EUCTR2018-004383-65-NL
(EUCTR)
30/10/201903/04/2019A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular AtrophyPhase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study Later Onset Spinal Muscular Atrophy (SMA)
MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: SRK-015
INN or Proposed INN: Apitegromab
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Product Name: SRK-015
INN or Proposed INN: Apitegromab
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Scholar Rock, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
58Phase 2United States;Spain;Germany;Netherlands;Italy
38NCT03779334
(ClinicalTrials.gov)
August 7, 201917/12/2018A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyMuscular Atrophy, SpinalDrug: RisdiplamHoffmann-La RocheNULLActive, not recruitingN/A6 WeeksAll25Phase 2United States;Australia;Belgium;Brazil;Canada;China;Italy;Poland;Russian Federation;Saudi Arabia;Taiwan
39NCT04050852
(ClinicalTrials.gov)
July 17, 20197/8/2019Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen TreatmentsPulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen TreatmentsSpinal Muscular AtrophyDrug: Nusinersen TreatmentsNYU Langone HealthWinthrop University HospitalWithdrawn5 Years21 YearsAll0Early Phase 1United States
40NCT03988907
(ClinicalTrials.gov)
June 18, 201914/6/2019A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy ParticipantsA Phase I, 2-Part, Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of Risdiplam and the Effect of Risdiplam on the Pharmacokinetics of Midazolam Following Oral Administration in Healthy ParticipantsSpinal Muscular AtrophyDrug: Risdiplam;Drug: MidazolamHoffmann-La RocheNULLCompleted18 Years55 YearsAll35Phase 1United States
41EUCTR2018-004383-65-IT
(EUCTR)
16/06/201925/01/2021A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular AtrophyPhase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study Later Onset Spinal Muscular Atrophy (SMA)
MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: SRK-015
Product Code: [NA]
INN or Proposed INN: SRK-015
Product Name: SRK-015
Product Code: [NA]
INN or Proposed INN: SRK-015
Scholar Rock, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
55Phase 2United States;Spain;Germany;Netherlands;Italy
42EUCTR2018-004383-65-ES
(EUCTR)
10/06/201912/04/2019A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular AtrophyPhase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study Later Onset Spinal Muscular Atrophy (SMA)
MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: SRK-015
INN or Proposed INN: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Product Name: SRK-015
INN or Proposed INN: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Scholar Rock, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
55 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Spain;Netherlands;Germany;Italy
43NCT03837184
(ClinicalTrials.gov)
May 15, 20198/2/2019Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 CopiesPhase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionSpinal Muscular Atrophy Type IBiological: Onasemnogene Abeparvovec-xioiNovartis Gene TherapiesPRA Health SciencesCompletedN/A6 MonthsAll2Phase 3Japan;Korea, Republic of;Taiwan
44EUCTR2016-004184-39-NL
(EUCTR)
08/05/201920/11/2018A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophyAN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RISDIPLAM (RO7034067) IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 2France;United States;Poland;Belgium;Germany;Netherlands;United Kingdom;Switzerland;Italy
45NCT03921528
(ClinicalTrials.gov)
April 22, 201916/4/2019An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular AtrophyPhase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)Spinal Muscular Atrophy;Spinal Muscular Atrophy Type 3;Spinal Muscular Atrophy Type 2;SMA;Neuromuscular Diseases;Muscular Atrophy;Atrophy;Muscular Atrophy, Spinal;Neuromuscular ManifestationsBiological: SRK-015Scholar Rock, Inc.NULLActive, not recruiting2 Years21 YearsAll58Phase 2United States;Italy;Netherlands;Spain
46NCT03878030
(ClinicalTrials.gov)
April 1, 201910/3/2019Effect of Nusinersen on Adults With Spinal Muscular AtrophyEffect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3Adult Spinal Muscular AtrophyDrug: nusinersenNorthwell HealthNULLActive, not recruiting18 Years60 YearsAll12United States
47EUCTR2017-000266-29-NL
(EUCTR)
25/03/201905/06/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot Recruiting Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Belgium;Spain;Germany;Netherlands;Italy;United Kingdom;Sweden
48EUCTR2018-002087-12-PL
(EUCTR)
21/03/201905/12/2018A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Saudi Arabia;Belgium;Brazil;Poland;Australia;Russian Federation;Italy;China
49NCT03819660
(ClinicalTrials.gov)
March 7, 201923/1/2019Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3Muscle AtrophyDrug: Amifampridine Phosphate 10 MG Oral TabletCatalyst Pharmaceuticals, Inc.NULLEnrolling by invitation6 Years50 YearsAll12Phase 2Italy
50EUCTR2017-004087-35-NL
(EUCTR)
28/02/201909/07/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
44 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Netherlands;Germany;Japan
51NCT03648658
(ClinicalTrials.gov)
February 18, 20198/8/2018Paracetamol Study in Patients With Low Muscle MassPharmacokinetics and Safety of Treatment With Paracetamol in Children and Adults With Spinal Muscular Atrophy and Cerebral PalsySMA II;Cerebral PalsyDrug: Paracetamol 120Mg/5mL Oral SuspensionMette Cathrine OerngreenElsass FoundationRecruiting6 Years45 YearsAll48Phase 4Denmark
52EUCTR2018-002087-12-IT
(EUCTR)
05/02/201917/06/2021A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY - RAINBOWFISH Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: [RO7034067/F13]
INN or Proposed INN: Risdiplam
Product Name: RO7034067
Product Code: [RO7034067/F12]
INN or Proposed INN: Risdiplam
F. HOFFMANN - LA ROCHE LTD.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Saudi Arabia;Poland;Belgium;Brazil;Australia;Russian Federation;China;Italy
53EUCTR2017-004087-35-BE
(EUCTR)
31/01/201916/05/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
29Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan
54EUCTR2017-000266-29-BE
(EUCTR)
31/01/201923/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;United Kingdom;Italy;Sweden
55NCT02876094
(ClinicalTrials.gov)
January 29, 20199/8/2016Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular AtrophyA Pilot, Open-Label, Dose Response Study Investigating the Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (SMA)Spinal Muscular Atrophy (SMA)Drug: celecoxibHugh McMillanFamilies of Spinal Muscular Atrophy;Gwendolyn Strong FoundationTerminated2 Years80 YearsAll1Phase 2Canada
56NCT03781479
(ClinicalTrials.gov)
January 21, 201918/12/2018Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 PatientsA Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3Muscular Atrophy, SpinalDrug: Amifampridine Phosphate;Drug: Placebo Oral TabletCatalyst Pharmaceuticals, Inc.NULLCompleted6 Years50 YearsAll13Phase 2Italy
57EUCTR2017-004087-35-IT
(EUCTR)
24/12/201823/04/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
44Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan
58EUCTR2017-004087-35-ES
(EUCTR)
21/12/201820/04/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
44 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noAustralia;Germany;Netherlands;Japan;Belgium;Canada;Italy;United Kingdom;Israel;Korea, Democratic People's Republic of;Spain;Taiwan;United States
59EUCTR2017-000266-29-ES
(EUCTR)
18/12/201828/05/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy;Sweden
60EUCTR2018-002087-12-BE
(EUCTR)
07/12/201823/10/2018A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10079419;Term: Spinal muscular atrophy pre-symptomatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
Trade Name: EVRYSDI
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Saudi Arabia;Poland;Brazil;Belgium;Australia;Russian Federation;Italy;China
61EUCTR2018-000160-28-IT
(EUCTR)
06/11/201824/05/2021Clinical study evaluating the long term safety of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3Long term safety study of amifampridine phosphate in ambulatory patients with Spinal Muscular Atrophy (SMA) type 3 - SMA-002 Spinal Muscular Atrophy (SMA) Type 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE
Product Name: Amifampridina fosfato
Product Code: [Amifampridina fosfato]
INN or Proposed INN: AMIFAMPRIDINA
CATALYST PHARMACEUTICALS INC.NULLNot RecruitingFemale: yes
Male: yes
12Phase 2Italy
62EUCTR2017-000266-29-DE
(EUCTR)
05/11/201805/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3France;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Sweden
63EUCTR2014-002053-19-BG
(EUCTR)
04/10/201831/07/2018Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Not established
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Hungary;Czech Republic;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy
64NCT04591678
(ClinicalTrials.gov)
October 1, 201823/10/2019Adults With SMA Treated With NusinersenCharacterizing Longitudinal Outcomes in Adults With SMA Treated With NusinersenSpinal Muscular AtrophyDrug: nusinersenOhio State UniversityBiogen;Cure SMACompleted18 Years60 YearsAll15United States
65NCT04174157
(ClinicalTrials.gov)
September 25, 201812/9/2019Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)Spinal Muscular Atrophy (SMA)Other: Prospective observational registry;Drug: ZolgensmaAveXis, Inc.United BioSource, LLCRecruitingN/AN/AAll500United States;Ireland;Israel;Portugal;Japan
66EUCTR2014-002053-19-HU
(EUCTR)
17/09/201817/07/2018Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Czech Republic;Hungary;Poland;Belgium;Denmark;Russian Federation;Bulgaria;Netherlands;Germany;Italy
67EUCTR2018-002295-40-DK
(EUCTR)
11/09/201811/07/2018Is treatment with the painkiller paracetamol safe in patients with spinal muscular atrophy and cerebral palsy?Pharmacokinetics and safety of treatment with paracetamol in children and adults with spinal muscular atrophy and cerebral palsy - Paracetamol study in patients with low muscle mass Spinal muscular atrophy type II (SMA II)Cerebral palsy (CP);Therapeutic area: Diseases [C] - Nervous System Diseases [C10]INN or Proposed INN: Paracetamol
Other descriptive name: PARACETAMOL
Copenhagen Neuromuscular CenterNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 1;Phase 4Denmark
68EUCTR2017-004087-35-GB
(EUCTR)
24/08/201808/05/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
Novartis Gene Therapies, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
29Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan
69NCT03461289
(ClinicalTrials.gov)
August 16, 20184/3/2018Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionSMABiological: Onasemnogene Abeparvovec-xioiNovartis Gene TherapiesNULLCompletedN/A6 MonthsAll33Phase 3Belgium;France;Italy;United Kingdom;Germany;Netherlands;Spain;Sweden
70EUCTR2016-000778-40-HR
(EUCTR)
15/08/201807/12/2018A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular AtrophyA TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
64Phase 2United States;Serbia;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Germany;China;Japan
71EUCTR2016-000778-40-ES
(EUCTR)
10/08/201805/08/2016A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular AtrophyA TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: n/a
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLNot Recruiting Female: yes
Male: yes
64 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noSerbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China
72EUCTR2016-000778-40-PL
(EUCTR)
27/06/201822/05/2018A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular AtrophyA TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: n/a
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
64Phase 2Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China
73EUCTR2016-000750-35-BG
(EUCTR)
20/06/201804/06/2018A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: n.a
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: n.a
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
219Phase 2Serbia;United States;Spain;Ukraine;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;Japan;China
74EUCTR2017-000266-29-GB
(EUCTR)
12/06/201812/02/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
33Phase 3France;Spain;Belgium;Netherlands;Italy;United Kingdom;Sweden
75JPRN-JapicCTI-183891
12/6/201808/03/2018Investigate Safety, Tolerability, PK, PD and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (FIREFISH)A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy Spinal Muscular AtrophyIntervention name : RO7034067
INN of the intervention : risdiplam
Dosage And administration of the intervention : po
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -
CHUGAI PHARMACEUTICAL CO., LTD.NULLcompleteBOTH40Phase 2Japan, Asia except Japan, North America, South America, Europe
76EUCTR2017-000266-29-IT
(EUCTR)
21/05/201809/06/2020Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion - N/A Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
AVEXIS, INC.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
77NCT03505099
(ClinicalTrials.gov)
April 10, 201813/4/2018Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2Spinal Muscular AtrophyBiological: onasemnogene abeparvovec-xioiNovartis Gene TherapiesPRA Health SciencesCompletedN/A42 DaysAll30Phase 3United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom
78EUCTR2017-004600-22-IT
(EUCTR)
04/04/201807/04/2021Clinical study evaluating the effect of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3.A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients with Spinal Muscular Atrophy (SMA) Type 3 - SMA-001 Spinal Muscular Atrophy (SMA) Type 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE
Product Name: Amifampridina fosfato
Product Code: [Amifampridina fosfato]
INN or Proposed INN: AMIFAMPRIDINA
Other descriptive name: 3,4-Diaminopyridine Phosphate
CATALYST PHARMACEUTICALS INC.NULLNot RecruitingFemale: yes
Male: yes
12Phase 2Italy
79JPRN-JapicCTI-173722
03/4/201804/10/2017A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish)A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients Spinal Muscular AtrophyIntervention name : RO7034067
INN of the intervention : risdiplam
Dosage And administration of the intervention : po
Control intervention name : Placebo
INN of the control intervention : -
Dosage And administration of the control intervention : po
CHUGAI PHARMACEUTICAL CO., LTD.NULLcomplete225BOTH168Phase 2Japan, Asia except Japan, North America, South America, Europe
80EUCTR2016-000750-35-HR
(EUCTR)
27/03/201812/04/2018A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
219Phase 2United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan
81EUCTR2014-002053-19-CZ
(EUCTR)
14/02/201830/10/2017Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Not established
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
82NCT03381729
(ClinicalTrials.gov)
December 14, 201713/12/2017Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular AtrophyPhase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular AtrophySpinal Muscular AtrophyBiological: Onasemnogene Abeparvovec-xioiNovartis Gene TherapiesNULLCompleted6 Months60 MonthsAll32Phase 1United States
83NCT03306277
(ClinicalTrials.gov)
October 24, 20172/10/2017Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionSMA - Spinal Muscular Atrophy;Gene TherapyBiological: Onasemnogene Abeparvovec-xioiNovartis Gene TherapiesNULLCompletedN/A180 DaysAll22Phase 3United States
84NCT04825119
(ClinicalTrials.gov)
October 1, 20172/3/2021Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With NusinersenHyperkinetic Movements in Patients With Motor Neuron Disease and in Patients With Spinal Muscular Atrophy and Their Response to Treatment With NusinersenTremor;Involuntary Movements;SMA;MND (Motor Neurone DIsease)Drug: NusinersenUniversity Medical Centre LjubljanaNULLRecruitingN/AN/AAll110Slovenia
85NCT03421977
(ClinicalTrials.gov)
September 21, 201730/1/2018Long-Term Follow-up Study for Patients From AVXS-101-CL-101A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101Spinal Muscular Atrophy 1Biological: Onasemnogene Abeparvovec-xioiNovartis Gene TherapiesNULLActive, not recruitingN/AN/AAll13United States
86EUCTR2016-004184-39-GB
(EUCTR)
05/09/201704/05/2017A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophyAN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 2France;United States;United Kingdom;Switzerland;Italy;Germany;Netherlands;Belgium;Poland
87EUCTR2016-004184-39-IT
(EUCTR)
30/05/201706/02/2018A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy.AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY - A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 + solvente (RO
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067/F07 + solvente (RO
Other descriptive name: RO7034067
F. HOFFMANN - LA ROCHE LTD.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 2United States;United Kingdom;Switzerland;Italy
88EUCTR2016-000778-40-FR
(EUCTR)
21/04/201724/10/2016A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular AtrophyA TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 with solvent (RO7034067/F08)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
64Phase 2France;Belgium;Germany;Italy
89NCT03032172
(ClinicalTrials.gov)
March 3, 201724/1/2017A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular AtrophyAn Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: RisdiplamHoffmann-La RocheNULLActive, not recruiting6 Months60 YearsAll174Phase 2United States;Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom
90EUCTR2016-000778-40-DE
(EUCTR)
05/01/201721/07/2016A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular AtrophyA TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 with solvent (RO7034067/F08)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067 / F12
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067 / F13
INN or Proposed INN: n/a
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
64Phase 2Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China
91NCT04587492
(ClinicalTrials.gov)
January 1, 20177/10/2020Metabolomics of Children With SMAMetabolome of Children With Spinal Muscular Atrophy Treated With NusinersenSpinal Muscular AtrophyDrug: NusinersenUniversity Medical Centre LjubljanaNULLCompletedN/A21 YearsAll35Slovenia
92NCT02913482
(ClinicalTrials.gov)
December 24, 201621/9/2016Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular AtrophyA Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular AtrophyMuscular Atrophy, SpinalDrug: RisdiplamHoffmann-La RocheNULLActive, not recruiting1 Month7 MonthsAll62Phase 2/Phase 3United States;Belgium;Brazil;China;Croatia;France;Italy;Japan;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;Australia;Germany;Taiwan
93EUCTR2016-000778-40-BE
(EUCTR)
09/12/201613/09/2016A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants with Type1 Spinal Muscular AtrophyA TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
64Phase 2United States;Serbia;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Germany;China;Japan
94EUCTR2016-000750-35-DE
(EUCTR)
09/11/201613/07/2016A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy PatientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: Risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
219Phase 2Serbia;United States;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Bulgaria;Germany;China;Japan
95NCT02908685
(ClinicalTrials.gov)
October 20, 201619/9/2016A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) ParticipantsA Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy PatientsMuscular Atrophy, SpinalDrug: Placebo;Drug: RisdiplamHoffmann-La RocheNULLActive, not recruiting2 Years25 YearsAll231Phase 2/Phase 3United States;Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;United Kingdom;Argentina;Australia;Sweden;Switzerland;Taiwan
96EUCTR2016-000778-40-IT
(EUCTR)
04/10/201616/08/2016A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular AtrophyA TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 with solvent (RO7034067/F08)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
64Phase 2France;Belgium;Germany;Italy
97EUCTR2015-001870-16-GB
(EUCTR)
04/10/201629/10/2015A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
98NCT02865109
(ClinicalTrials.gov)
September 13, 201610/8/2016Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)Infantile-onset Spinal Muscular AtrophyDrug: NusinersenBiogenNULLNo longer availableN/AN/AAllKorea, Republic of;Mexico;Netherlands;Norway;Poland;Portugal;Slovenia;Spain;Sweden;Switzerland;Taiwan;United Kingdom;United States;Colombia;New Zealand;Turkey;Australia;Austria;Belgium;Canada;China;Denmark;Finland;France;Germany;Greece;Ireland;Israel;Italy
99EUCTR2016-000750-35-BE
(EUCTR)
06/09/201612/07/2016A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA) Type 2 and 3
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
219Phase 2United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan
100EUCTR2016-000750-35-IT
(EUCTR)
29/08/201628/06/2016A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy PatientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 with solvent (RO7034067/F08)
INN or Proposed INN: n.a
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
INN or Proposed INN: n.a
F. Hoffmann-La Roche LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
186Phase 2United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden
101JPRN-JMA-IIA00259
16/08/201610/08/2016Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trialMulticenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial spinal muscular atrophyIntervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper..Institute of Medical Genetics, Tokyo Women's Medical UniversityNULLRecruiting>=1 YEARS<8 YEARSBOTH28Phase 2BJapan
102EUCTR2015-001870-16-BE
(EUCTR)
29/07/201623/05/2016A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
103EUCTR2015-001870-16-SE
(EUCTR)
26/05/201622/01/2016A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of
104EUCTR2015-001589-25-NL
(EUCTR)
05/04/201607/10/2015A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular AtrophyMulticenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL, REFINED
Other descriptive name: SESAME OIL, REFINED
F. Hoffmann-La Roche LtdNULLNot Recruiting Female: yes
Male: yes
173 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Poland;Belgium;Germany;Netherlands;Italy;United Kingdom
105EUCTR2015-001870-16-ES
(EUCTR)
08/03/201603/02/2016An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
274United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
106EUCTR2015-001870-16-DE
(EUCTR)
08/03/201628/10/2015An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
107JPRN-JMA-IIA00231
29/01/201601/12/2015Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory TrialMulticenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial spinal muscular atrophyIntervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper.. Control intervention1:Placebo, Dose form:GRANULES, Route of administration:ORAL, Intended dose regimen:Placebo is to be taken once a day after supper..Institute of Medical Genetics, Tokyo Women's Medical UniversityNULLCompleted>=1 YEARS<7 YEARSBOTH28Phase 2BJapan
108NCT02628743
(ClinicalTrials.gov)
January 20, 20161/12/2015A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)Multicenter, Open-Label, Single-Arm Study to Evaluate Long-Term Safety, Tolerability, and Effectiveness of 10 mg/kg BID Olesoxime in Patients With Spinal Muscular AtrophyMuscular Atrophy, SpinalDrug: OlesoximeHoffmann-La RocheNULLCompletedN/AN/AAll131Phase 2Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom
109NCT02644668
(ClinicalTrials.gov)
January 14, 201623/12/2015A Study of CK-2127107 in Patients With Spinal Muscular AtrophyA Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Placebo;Drug: Reldesemtiv 150 mg;Drug: Reldesemtiv 450 mgCytokineticsAstellas Pharma Global Development, Inc.Completed12 YearsN/AAll70Phase 2United States;Canada
110NCT02633709
(ClinicalTrials.gov)
January 7, 201615/12/2015A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy VolunteersA Single-Center, Randomized, Investigator/Subject-Blind, Adaptive Single-Ascending-Dose(SAD), Placebo-Controlled, Parallel Study to Investigate the Safety, Tolerability, Pharmacokinetics (Including the Effect of Food and the Effect of Itraconazole on the Pharmacokinetics of a Single Oral Dose of RO7034067), and Pharmacodynamics of RO7034067 Following Oral Administration in Healthy SubjectsSpinal Muscular AtrophyDrug: Itraconazole;Other: Placebo;Drug: RisdiplamHoffmann-La RocheNULLCompleted18 Years45 YearsMale33Phase 1Netherlands
111EUCTR2015-001589-25-FR
(EUCTR)
01/12/201507/12/2015A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular AtrophyMulticenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA Spinal Muscular Atrophy
MedDRA version: 18.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL, REFINED
Other descriptive name: SESAME OIL, REFINED
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
173Phase 2;Phase 3France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom
112NCT02941328
(ClinicalTrials.gov)
December 201530/9/2016SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4A Phase II, Mono-center, Placebo-controlled, Double-blind, Crossover Trial to Investigate Effect and Efficacy of Pyridostigmine in Dutch Patients With Spinal Muscular Atrophy Types 2, 3 and 4Spinal Muscular Atrophy;SMA;Kugelberg-Welander DiseaseDrug: Pyridostigmine;Drug: PlaceboUMC UtrechtNULLCompleted12 YearsN/AAll39Phase 2Netherlands
113EUCTR2015-001589-25-BE
(EUCTR)
20/11/201525/09/2015A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular AtrophyMulticenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL, REFINED
Other descriptive name: SESAME OIL, REFINED
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
173Phase 2;Phase 3France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom
114EUCTR2015-001589-25-GB
(EUCTR)
19/11/201504/08/2015A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular AtrophyMulticenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL, REFINED
Other descriptive name: SESAME OIL, REFINED
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
173Phase 2;Phase 3France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom
115EUCTR2014-003657-33-GB
(EUCTR)
17/11/201519/06/2015A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA).A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Spinraza
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLNot Recruiting Female: yes
Male: yes
16 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Germany;United Kingdom
116NCT02594124
(ClinicalTrials.gov)
November 4, 201530/10/2015A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational StudiesAn Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443Spinal Muscular AtrophyDrug: nusinersenBiogenNULLActive, not recruitingN/AN/AAll292Phase 3United States;Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Taiwan
117EUCTR2013-004422-29-BE
(EUCTR)
22/10/201504/08/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
118EUCTR2015-001589-25-IT
(EUCTR)
05/10/201511/08/2015Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA Spinal Muscular Atrophy
MedDRA version: 18.0;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL, REFINED
Other descriptive name: SESAME OIL, REFINED
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: yes
Male: yes
173Phase 2;Phase 3France;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy
119EUCTR2014-003657-33-DE
(EUCTR)
21/09/201505/05/2015A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA).A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLNot RecruitingFemale: yes
Male: yes
16Phase 2United States;Germany;United Kingdom
120NCT02462759
(ClinicalTrials.gov)
August 19, 201514/5/2015A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).A Phase 2, Randomized, Double-blind, Sham-procedure Controlled Study to Assess the Safety and Tolerability and Explore the Efficacy of ISIS 396443 (BIIB058) Administered Intrathecally in Subjects With Spinal Muscular Atrophy Who Are Not Eligible to Participate in the Clinical Studies ISIS 396443-CS3B or ISIS 396443-CS4Spinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham ProcedureBiogenNULLTerminatedN/AN/AAll21Phase 2United States;Germany
121EUCTR2014-002098-12-DE
(EUCTR)
05/08/201509/04/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Qatar;Taiwan;Canada;Turkey;Australia;Germany;United Kingdom;Italy
122EUCTR2014-001947-18-DE
(EUCTR)
07/07/201502/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
123EUCTR2014-002098-12-GB
(EUCTR)
10/06/201530/03/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: Spinraza
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: Nusinersen
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;Qatar;Taiwan;Turkey;Australia;Germany;Italy;United Kingdom
124NCT02855112
(ClinicalTrials.gov)
June 201522/7/2016Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman PatientsThe Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman PatientsInfantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]Biological: Adipose derived mesenchymal stem cellTehran University of Medical SciencesNULLRecruiting5 Months12 MonthsBoth10Phase 1/Phase 2Iran, Islamic Republic of
125NCT02386553
(ClinicalTrials.gov)
May 20, 201527/2/2015A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophySpinal Muscular AtrophyDrug: NusinersenBiogenNULLActive, not recruitingN/A6 WeeksAll25Phase 2United States;Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;Argentina;Israel;United Kingdom
126EUCTR2014-002098-12-IT
(EUCTR)
12/05/201524/03/2015A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular AtrophyAn Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443 (BIIB058)
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443 (BIIB058)
Biogen Idec Research LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25United States;Qatar;Taiwan;Argentina;Turkey;Australia;Israel;Germany;United Kingdom;Italy
127EUCTR2011-004369-34-NL
(EUCTR)
22/04/201503/11/2014 SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4 - SPACE trial Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive.
MedDRA version: 17.1;Level: LLT;Classification code 10068209;Term: Spinal muscular atrophy adult onset;System Organ Class: 100000004850
MedDRA version: 17.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: pyridostigmine bromide
Product Name: Mestinon
Product Code: RVG 03820
Universtiy Medical Center UtrechtNULLNot Recruiting Female: yes
Male: yes
45 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noNetherlands
128EUCTR2014-002246-41-ES
(EUCTR)
15/04/201503/03/2015A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH)A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH Spinal Muscular Atrophy
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: RO6885247/F03
INN or Proposed INN: n.a.
Other descriptive name: n.a.
Product Code: RO6885247/F02
INN or Proposed INN: n.a.
Other descriptive name: n.a.
Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd.NULLNot RecruitingFemale: yes
Male: yes
64Phase 1France;United States;Spain;Turkey;Netherlands;Italy;United Kingdom;Switzerland;Sweden
129EUCTR2014-001947-18-ES
(EUCTR)
08/04/201509/02/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
130NCT02268552
(ClinicalTrials.gov)
April 2, 20151/10/2014An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular AtrophySpinal Muscular AtrophyDrug: branaplamNovartis PharmaceuticalsNULLActive, not recruitingN/A182 DaysAll40Phase 1/Phase 2Belgium;Bulgaria;Denmark;Germany;Italy;Poland;Russian Federation;Czechia;Hungary;Netherlands
131EUCTR2014-002053-19-IT
(EUCTR)
01/04/201527/10/2014Clinical trial of LMI070 given my mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: NA
Product Code: LMI070
INN or Proposed INN: NA
Other descriptive name: NA
NOVARTIS FARMA S.p.A.NULLNot RecruitingFemale: yes
Male: yes
22Phase 1;Phase 2United States;Hungary;Czech Republic;European Union;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy
132EUCTR2014-001947-18-IT
(EUCTR)
12/03/201512/05/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;United Kingdom;Italy;Korea, Republic of;Sweden
133EUCTR2014-001947-18-SE
(EUCTR)
03/02/201523/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
134EUCTR2014-002053-19-DE
(EUCTR)
02/02/201530/10/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Not established
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
44 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noHungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy
135EUCTR2013-004422-29-ES
(EUCTR)
20/01/201508/09/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3Korea, Republic of;United States;Taiwan;Hong Kong;Spain;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Sweden
136EUCTR2013-004422-29-DE
(EUCTR)
08/01/201527/11/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
137EUCTR2014-002053-19-DK
(EUCTR)
16/12/201415/10/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Not established
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNot RecruitingFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
138EUCTR2014-002053-19-BE
(EUCTR)
15/12/201404/11/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Branaplam
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNot RecruitingFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
139EUCTR2013-004422-29-SE
(EUCTR)
09/12/201423/10/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
140EUCTR2013-004422-29-GB
(EUCTR)
01/12/201429/07/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy - Study to assess the Clinical Efficacy and Safety of ISIS 396443 Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
141NCT02292537
(ClinicalTrials.gov)
November 24, 201412/11/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham procedureBiogenNULLCompleted2 Years12 YearsAll126Phase 3United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom
142EUCTR2013-004422-29-IT
(EUCTR)
13/11/201423/07/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with infantile-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
111Phase 3United States;Taiwan;Hong Kong;Spain;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of
143NCT02240355
(ClinicalTrials.gov)
November 201411/9/2014A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH).Muscular Atrophy, SpinalDrug: RO6885247;Drug: placeboHoffmann-La RocheNULLTerminatedN/A55 YearsBoth9Phase 1United States;Canada;France;Italy;Netherlands;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic
144EUCTR2014-002246-41-IT
(EUCTR)
23/10/201409/09/2014A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH)A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH Spinal Muscular Atrophy
MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: RO6885247/F03
INN or Proposed INN: n.a.
Other descriptive name: n.a.
F. Hoffmann-La Roche Ltd.NULLNot RecruitingFemale: yes
Male: yes
48Phase 1United States;Spain;Netherlands;United Kingdom;Switzerland;Italy
145NCT02193074
(ClinicalTrials.gov)
August 19, 201414/7/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular AtrophyA Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersen;Procedure: Sham procedureBiogenNULLTerminatedN/A210 DaysAll122Phase 3United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan
146JPRN-JMA-IIA00190
22/07/201401/08/2014Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophyMulticenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy spinal muscular atrophyIntervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA is to be taken once a day after supper.
Administration of VPA starts with an initial dosage (standard dose: 12.5 mg / kg) for 4 weeks.
The dosage then increases to a maintenance dosage (standard dose: 25 mg/kg) from the 5th week to the 12th week. Administration, however, could be increased to an additional 50 mg/day in case of a blood concentration of VPA below 50 mcg/mL
From the 13th week, administered doses are to be decreased (standard dose: 12.5 mg/kg) and continued to the 14th week. . Control intervention1:No.
Institute of Medical Genetics, Tokyo Women's Medical UniversityNULLCompletedNo Limit<8 YEARSBOTH13Phase 2AJapan
147NCT02227823
(ClinicalTrials.gov)
July 201420/8/2014Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.Spinal Muscular Atrophy Type 3Drug: Pyridostigmine BromideCentre Hospitalier Régional de la CitadelleNULLRecruiting6 YearsN/ABoth20Phase 2Belgium
148NCT02122952
(ClinicalTrials.gov)
May 5, 201423/4/2014Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101Spinal Muscular Atrophy 1Biological: AVXS-101Novartis Gene TherapiesNULLCompletedN/A6 MonthsAll15Phase 1United States
149NCT04256265
(ClinicalTrials.gov)
April 20143/2/2020An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular AtrophyMuscular Atrophy, SpinalDrug: RisdiplamGenentech, Inc.NULLApproved for marketing2 MonthsN/AAllUnited States
150NCT02052791
(ClinicalTrials.gov)
January 31, 201430/1/2014An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10Spinal Muscular AtrophyDrug: nusinersenBiogenNULLCompletedN/AN/AAll47Phase 1United States
151NCT01671384
(ClinicalTrials.gov)
August 201313/8/2012Valproate and Levocarnitine in Children With Spinal Muscular AtrophyRandomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 YearsSpinal Muscular AtrophyDrug: Valproate, Levocarnitine;Drug: PlaceboAll India Institute of Medical Sciences, New DelhiNULLRecruiting2 Years15 YearsBoth60Phase 3India
152NCT01839656
(ClinicalTrials.gov)
May 8, 201322/4/2013A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersenBiogenNULLCompletedN/A210 DaysAll21Phase 2United States;Canada
153NCT01780246
(ClinicalTrials.gov)
January 31, 201328/1/2013An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1Spinal Muscular AtrophyDrug: nusinersenBiogenNULLCompleted2 Years15 YearsAll18Phase 1United States
154NCT01703988
(ClinicalTrials.gov)
October 20128/10/2012An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular AtrophyAn Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: NusinersenBiogenNULLCompleted2 Years15 YearsAll34Phase 1/Phase 2United States
155NCT01645787
(ClinicalTrials.gov)
June 20125/7/2012Short and Long Term Treatment With 4-AP in Ambulatory SMA PatientsColumbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of ActionSpinal Muscular AtrophyDrug: 4-aminopyridine;Drug: PlaceboColumbia UniversityNULLCompleted18 Years50 YearsBoth11Phase 2/Phase 3United States
156NCT01494701
(ClinicalTrials.gov)
November 30, 201113/12/2011An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersenBiogenNULLCompleted2 Years14 YearsAll28Phase 1United States
157NCT01422200
(ClinicalTrials.gov)
August 201122/8/2011Flu Vaccine Study in Neuromuscular Patients 2011Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular DiseasesDuchenne Muscular Dystrophy;Spinal Muscular Atrophy;Congenital Muscular DystrophyBiological: 2011-2012 seasonal flu vaccine Subcutaneous;Biological: 2011-2012 seasonal flu vaccine IntramuscularChildren's Hospital Medical Center, CincinnatiNULLCompleted3 Years35 YearsAll22Phase 4United States
158EUCTR2010-020386-24-GB
(EUCTR)
31/01/201108/10/2010Safety and Efficacy of Olesoxime (TRO19622) in 3-25 yrs SMA patientsPhase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 14.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot Recruiting Female: yes
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Belgium;Germany;Netherlands;Italy;United Kingdom
159NCT01522079
(ClinicalTrials.gov)
January 201124/1/2012Spinal Muscular Atrophy and Cardiac Autonomic FunctionSpinal Muscular Atrophy and Cardiac Autonomic FunctionSpinal Muscular AtrophyProcedure: Air stacking manueverCentro Universitário Augusto MottaNULLCompleted5 Years25 YearsBoth9N/ANULL
160EUCTR2010-020386-24-DE
(EUCTR)
29/12/201028/07/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot RecruitingFemale: yes
Male: yes
150Phase 2Belgium;Netherlands;Germany;United Kingdom;Italy
161EUCTR2010-020386-24-NL
(EUCTR)
18/11/201028/06/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot RecruitingFemale: yes
Male: yes
150Phase 2Belgium;Germany;Netherlands;United Kingdom;Italy
162NCT01302600
(ClinicalTrials.gov)
November 201018/2/2011Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.Phase II, Multicenter, Randomized, Adaptive, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients.Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III Non AmbulantDrug: Olesoxime;Drug: PlaceboHoffmann-La RocheAssociation Française contre les Myopathies (AFM), ParisCompleted3 Years25 YearsBoth165Phase 2Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom
163EUCTR2010-020386-24-BE
(EUCTR)
29/10/201027/07/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNot RecruitingFemale: yes
Male: yes
150Phase 2Belgium;Germany;Netherlands;United Kingdom;Italy
164ChiCTR-TRC-10001093
2010-10-012010-11-02Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trialRat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial children with spinal muscular atrophyA:Rat nerve growth factor injection for six monthes,;B:Rat nerve growth factor injection for six monthes,;PLA General HospitalNULLCompleted38BothA:20;B:20;China
165EUCTR2010-020386-24-IT
(EUCTR)
14/09/201001/09/2010Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patientsPhase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients Spinal Muscular Atrophy - type II or III in non ambulant patients aged 3-25 years
MedDRA version: 9.1;Level: LLT;Classification code 10032950
Product Name: olesoxime
Product Code: TRO19622
INN or Proposed INN: olesoxime
TROPHOSNULLNot RecruitingFemale: yes
Male: yes
150Phase 2Belgium;Germany;Netherlands;United Kingdom;Italy
166EUCTR2008-003915-11-DE
(EUCTR)
24/09/200925/06/2009Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type IPhase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I Spinal Muscular Atrophy Type I in infants
MedDRA version: 9.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital
Trade Name: Orfiril Saft
INN or Proposed INN: VALPROATE SODIUM
Trade Name: Biocarn
INN or Proposed INN: LEVOCARNITINE
University of UtahNULLNot RecruitingFemale: yes
Male: yes
36Phase 1/2Germany
167EUCTR2007-001088-32-IT
(EUCTR)
29/07/200818/07/2008A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - NDA PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - ND Patients affected by SMA
MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Trade Name: VOLMAX*30CPR 4MG R.P.
INN or Proposed INN: Salbutamol
ISTITUTO NEUROLOGICO CARLO BESTANULLNot RecruitingFemale: yes
Male: yes
Phase 2Italy
168NCT00661453
(ClinicalTrials.gov)
April 200814/4/2008CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type IPhase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I)Spinal Muscular Atrophy Type IDrug: Valproic Acid and LevocarnitineUniversity of UtahFamilies of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.CompletedN/A12 MonthsAll40Phase 1/Phase 2United States;Canada;Germany
169NCT00439218
(ClinicalTrials.gov)
January 200822/2/2007Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type IPhase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type I Spinal Muscular AtrophySpinal Muscular Atrophy Type IDrug: sodium phenylbutyrateWestatNational Institute of Neurological Disorders and Stroke (NINDS)Terminated2 Months48 MonthsAll5Phase 1/Phase 2United States
170NCT00439569
(ClinicalTrials.gov)
January 200821/2/2007Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or IIIPhase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type II/III Spinal Muscular AtrophySpinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type IIIDrug: sodium phenylbutyrateWestatNational Institute of Neurological Disorders and Stroke (NINDS)Terminated2 Years11 YearsAll9Phase 1/Phase 2United States
171NCT00533221
(ClinicalTrials.gov)
October 200712/9/2007Pilot Study of Growth Hormon to Treat SMA Typ II and IIICan Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?Muscular Atrophy, SpinalDrug: somatotropin;Drug: PlaceboUniversity Hospital FreiburgNovo Nordisk A/SCompleted6 Years35 YearsBoth20Phase 2Germany
172NCT00528268
(ClinicalTrials.gov)
July 200710/9/2007Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular AtrophyProspective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Sodium phenylbutyrate (NaPB)University of UtahEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)CompletedN/A6 MonthsAll14Phase 1/Phase 2United States
173NCT00481013
(ClinicalTrials.gov)
July 200730/5/2007Valproic Acid in Ambulant Adults With Spinal Muscular AtrophyProspective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) StudySpinal Muscular AtrophyDrug: Valproic Acid (VPA);Drug: PlaceboUniversity of UtahFamilies of Spinal Muscular Atrophy;AbbottCompleted18 Years60 YearsBoth33Phase 2United States
174NCT00485511
(ClinicalTrials.gov)
June 200711/6/2007A Trial of Hydroxyurea in Spinal Muscular AtrophyA Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular AtrophySpinal Muscular AtrophyDrug: HydroxyureaKaohsiung Medical University Chung-Ho Memorial HospitalNULLCompleted4 YearsN/ABothPhase 2/Phase 3Taiwan
175EUCTR2006-006845-14-FR
(EUCTR)
20/03/200715/02/2007Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (SMA)
MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Code: TRO19622
Other descriptive name: 4-cholesten-3-one,oxime
TROPHOSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 1France
176EUCTR2005-002822-78-DE
(EUCTR)
09/01/200621/11/2005Can treatment with human growth hormone increase strength in spinal muscular atrophy type II and III? - SMA-GHCan treatment with human growth hormone increase strength in spinal muscular atrophy type II and III? - SMA-GH Spinal muscular atrophy (SMA) is an autosomal recessive disease. Due to the genetic defect, a molecule called spinal motor neuron” (SMN) protein is lacking, resulting in muscle weakness. In SMAs muscle weakness is found most often at the level of proximal muscles. The disease is life-threatening and chronically debilitating.Trade Name: Norditropin SimpleXx
Product Name: Norditropin SimpleXx 15 mg/1.5 ml
Product Code: GH
INN or Proposed INN: Somatropin
Other descriptive name: Norditropin SimpleXx
Klinik Neuropädiatrie und MuskelkrankheitenNULLNot RecruitingFemale: yes
Male: yes
20Germany
177NCT00774423
(ClinicalTrials.gov)
January 200616/10/2008Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)Multicentric, Randomized, Double-blind Study Versus Placebo, With Two Parallel Groups Treated to Evaluate the Efficacy and the Tolerance of Riluzole in Children and Young Adults (6 to 20 Years of Age) With SMA. (Type II and Type III)SMADrug: RiluzoleAssistance Publique - Hôpitaux de ParisNULLCompleted6 Years20 YearsBoth141Phase 2/Phase 3France
178NCT00227266
(ClinicalTrials.gov)
September 200523/9/2005Valproic Acid and Carnitine in Patients With Spinal Muscular AtrophyMulti-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)Spinal Muscular AtrophyDrug: Valproic Acid and Levocarnitine;Drug: PlaceboUniversity of UtahFamilies of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.;AbbottCompleted2 Years17 YearsAll94Phase 2United States;Canada
179NCT00568698
(ClinicalTrials.gov)
January 20044/12/2007A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy PatientsA Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy PatientsMuscular Atrophy, SpinalDrug: Hydroxyurea;Drug: Placebo to match hydroxyureaStanford UniversityNULLCompletedN/A2 YearsAll29Phase 1/Phase 2United States
180NCT00568802
(ClinicalTrials.gov)
January 20044/12/2007A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy PatientsA Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy PatientsMuscular Atrophy, SpinalDrug: Hydroxyurea;Drug: Placebo to match hydroxyureaStanford UniversityNULLCompleted1 Year10 YearsAll27Phase 1/Phase 2United States
181NCT00374075
(ClinicalTrials.gov)
September 20036/9/2006Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular AtrophyIn Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Valproic AcidUniversity of UtahFamilies of Spinal Muscular Atrophy;Sigma Tau Pharmaceuticals, Inc.;AbbottCompleted2 YearsN/ABoth42Phase 1United States
182NCT00004771
(ClinicalTrials.gov)
October 199224/2/2000Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron DiseaseSpinal Muscular Atrophy;Amyotrophic Lateral Sclerosis;Spinobulbar Muscular AtrophyDrug: leuprolide;Drug: testosteroneNational Center for Research Resources (NCRR)Ohio State UniversityCompleted18 YearsN/AMale40Phase 2NULL
183EUCTR2019-002663-10-GR
(EUCTR)
02/11/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 2;Phase 3Canada;Argentina;Brazil;Poland;Germany;United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico
184EUCTR2017-000327-27-Outside-EU/EEA
(EUCTR)
26/01/2017An Open-Label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular AtrophyAn Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: nusinersen
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
34Phase 2United States
185EUCTR2016-004184-39-DE
(EUCTR)
20/09/2018A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophyAN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
F. Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
180Phase 2France;United States;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy
186EUCTR2014-002053-19-PL
(EUCTR)
03/01/2018Clinical trial of LMI070 given by mouth to Type I SMA infant patients.An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Code: LMI070
INN or Proposed INN: Branaplam
Other descriptive name: LMI070
Novartis Pharma Services AGNULLNAFemale: yes
Male: yes
44Phase 1;Phase 2United States;Hungary;Czech Republic;Belgium;Poland;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy
187EUCTR2014-002053-19-NL
(EUCTR)
27/11/2014Clinical trial of LMI070 given by mouth to Type I SMA infant patientsAn open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Code: LMI070
INN or Proposed INN: LMI070
Novartis Pharma Services AGNULLNAFemale: yes
Male: yes
22Phase 1;Phase 2United States;Belgium;Denmark;Germany;Netherlands;Italy
188EUCTR2021-001294-23-DE
(EUCTR)
27/12/2021A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with RisdiplamA Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
135Phase 3Canada;Spain;Poland;Brazil;Belgium;Australia;Germany;Switzerland;Japan
189EUCTR2021-005314-34-BE
(EUCTR)
11/02/2022Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or RisdiplamPhase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Apitegromab
Product Code: SRK-015
INN or Proposed INN: Apitegromab
Scholar Rock, Inc.NULLNAFemale: yes
Male: yes
204Phase 3United States;France;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy
190EUCTR2015-001870-16-FR
(EUCTR)
03/05/2016An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443. Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
274Phase 3United States;Taiwan;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
191EUCTR2017-000266-29-FR
(EUCTR)
05/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
192EUCTR2016-004184-39-PL
(EUCTR)
27/12/2018A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophyAN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
F. Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
180Phase 2United States;France;Belgium;Poland;Netherlands;Germany;United Kingdom;Switzerland;Italy
193EUCTR2021-001294-23-BE
(EUCTR)
16/12/2021A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with RisdiplamA Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
135Phase 3Canada;Spain;Poland;Brazil;Belgium;Australia;Germany;Switzerland;Japan
194EUCTR2016-004184-39-FR
(EUCTR)
01/10/2018A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophyAN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
Other descriptive name: RO7034067
F. Hoffmann-La Roche LtdNULLNA Female: yes
Male: yes
125 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom;Switzerland
195EUCTR2014-001947-18-GB
(EUCTR)
19/12/2014A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 17.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
117Phase 3France;United States;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
196EUCTR2020-004708-32-DE
(EUCTR)
18/11/2021Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With NusinersenA Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Lebanon;Ireland;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan
197EUCTR2021-005314-34-NL
(EUCTR)
10/03/2022Phase 3 Active Treatment Trial to Evaluate the Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy Who Are Being Treated with Nusinersen or RisdiplamPhase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy - SAPPHIRE Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: LLT;Classification code 10041583;Term: Spinal muscular atrophy, unspecified;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Apitegromab
Product Code: SRK-015
INN or Proposed INN: Apitegromab
Scholar Rock, Inc.NULLNAFemale: yes
Male: yes
204Phase 3France;United States;Spain;Poland;Belgium;Germany;Netherlands;United Kingdom;Italy
198EUCTR2021-003474-31-DK
(EUCTR)
02/11/2021Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER Spinal Muscular Atrophy
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zolgensma
Product Name: OAV101
Product Code: OAV101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101
Trade Name: Okrido 6mg/mL oral solution
Product Name: Okrido
INN or Proposed INN: prednisolone sodium phosphate
Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE
Novartis Pharma AGNULLNAFemale: yes
Male: yes
125Phase 3United States;United Arab Emirates;Saudi Arabia;Taiwan;Thailand;Russian Federation;Colombia;Italy;Vietnam;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Denmark;South Africa;China
199EUCTR2019-002663-10-IE
(EUCTR)
04/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
172Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany
200EUCTR2020-004708-32-PL
(EUCTR)
21/12/2021Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With NusinersenA Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Lebanon;Ireland;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan
201EUCTR2021-003417-19-NL
(EUCTR)
11/01/2022A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: N/A
Product Code: RO7204239/F01-01
INN or Proposed INN: N/A
Other descriptive name: GYM329
Trade Name: Evrysdi
INN or Proposed INN: Risdiplam
F.Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
180Phase 2;Phase 3United States;Poland;Belgium;Netherlands;United Kingdom;Italy
202EUCTR2015-001589-25-DE
(EUCTR)
13/08/2015A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular AtrophyMulticenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
F. Hoffmann-La Roche LtdNULLNot Recruiting Female: yes
Male: yes
173 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom
203EUCTR2014-001947-18-FR
(EUCTR)
18/06/2015A controlled clinical study to assess the effectiveness and safety of ISIS 396443 in patients with later-onset Spinal Muscular AtrophyA Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
Isis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
117Phase 3United States;France;Hong Kong;Canada;Spain;Germany;Japan;Italy;United Kingdom;Korea, Republic of;Sweden
204EUCTR2019-002611-26-BE
(EUCTR)
20/01/2020not applicableA Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101
Product Code: AVXS-101
INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC
Novartis Gene Therapies, Inc.NULLNAFemale: yes
Male: yes
308Phase 3Japan;Italy;United Kingdom;Australia;Belgium;Spain;Canada;Taiwan;United States;France
205EUCTR2015-001870-16-IT
(EUCTR)
22/01/2021An extension study for patients with Spinal Muscular Atrophy whoparticipated to the previous ISIS 396443 studiesAn Open-label Extension Study for Patients with Spinal Muscular Atrophywho Previously Participated in Investigational Studies of ISIS 396443 - An Open-label Extension Study for Patients with Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
Product Name: na
Product Code: [0000]
INN or Proposed INN: ISIS 396443
Other descriptive name: ISIS 396443
BIOGEN IDEC RESEARCH LIMITEDNULLNAFemale: yes
Male: yes
292Phase 3United States;Hong Kong;Taiwan;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of
206EUCTR2019-002663-10-PL
(EUCTR)
11/03/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
172Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan
207EUCTR2016-004184-39-BE
(EUCTR)
30/11/2018A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam in adult and pediatric patients with spinal muscular atrophyAN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: Risdiplam
F. Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
180Phase 2United States;France;Poland;Belgium;Netherlands;Germany;United Kingdom;Switzerland;Italy
208EUCTR2019-002663-10-NL
(EUCTR)
05/02/2022Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Spinraza
INN or Proposed INN: Nusinersen
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
172Phase 2;Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Chile;Israel;Russian Federation;Colombia;Italy;France;Australia;Netherlands;Latvia;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan
209EUCTR2017-000621-12-Outside-EU/EEA
(EUCTR)
15/02/2017A Study to Assess the Efficacy, Safety and Pharmacokinetics of IONIS SMNRx in Infants With Spinal Muscular AtrophyA Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide
Product Code: ISIS 396443
INN or Proposed INN: NUSINERSEN
Other descriptive name: ISIS 396443
Ionis Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
20Phase 2United States;Canada
210EUCTR2016-000750-35-PL
(EUCTR)
20/02/2018A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F12
INN or Proposed INN: risdiplam
Product Name: RO7034067
Product Code: RO7034067/F13
INN or Proposed INN: risdiplam
F. Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
219Phase 2United States;Serbia;Spain;Ukraine;Turkey;Russian Federation;United Kingdom;Switzerland;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Bulgaria;Germany;China;Japan
211EUCTR2010-020386-24-FR
(EUCTR)
01/06/2010Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years)
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy
Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime
TROPHOS SANULLNAFemale: yes
Male: yes
Phase 2France;Belgium;Germany;Netherlands;United Kingdom;Italy
212EUCTR2016-000750-35-FR
(EUCTR)
26/01/2018A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy PatientsA TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: RO7034067
Product Code: RO7034067/F06 with solvent (RO7034067/F08)
INN or Proposed INN: n.a
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
INN or Proposed INN: n.a
F. Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
186Phase 2United States;Spain;Turkey;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden
213EUCTR2017-004087-35-DE
(EUCTR)
06/04/2018Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular AtrophyA Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNAFemale: yes
Male: yes
27Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan
214EUCTR2020-004708-32-EE
(EUCTR)
02/02/2021Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With NusinersenA Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD Muscular Atrophy, Spinal
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Product Name: Nusinersen
Product Code: ISIS 396443, BIIB058
INN or Proposed INN: NUSINERSEN
Other descriptive name: NUSINERSEN SODIUM
Biogen Idec Research LimitedNULLNAFemale: yes
Male: yes
152Phase 3United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Netherlands;China;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Germany;Japan
215EUCTR2017-000266-29-SE
(EUCTR)
12/03/2018Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
INN or Proposed INN: onasemnogene abeparvovec
AveXis, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 3France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
216EUCTR2021-003417-19-BE
(EUCTR)
15/12/2021A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Ambulant Patients with Spinal Muscular AtrophyA TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY Spinal Muscular Atrophy (SMA);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: N/A
Product Code: RO7204239/F01-01
INN or Proposed INN: N/A
Other descriptive name: GYM329
Trade Name: Evrysdi
INN or Proposed INN: Risdiplam
F.Hoffmann-La Roche LtdNULLNAFemale: yes
Male: yes
180Phase 2;Phase 3Belgium;Netherlands;United Kingdom;Italy;Poland;United States
217EUCTR2015-001589-25-PL
(EUCTR)
07/01/2016A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular AtrophyMulticenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL, REFINED
Other descriptive name: SESAME OIL, REFINED
F. Hoffmann-La Roche LtdNULLNot Recruiting Female: yes
Male: yes
173 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Belgium;Poland;Netherlands;Germany;Italy;United Kingdom