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

  
36 trials found
No.TrialIDDate_
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PhaseCountries
1NCT05232929
(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
2NCT05115110
(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
3NCT05067790
(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
4EUCTR2021-003417-19-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
5EUCTR2021-003417-19-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
6EUCTR2016-000750-35-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
7NCT03779334
(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
8NCT03988907
(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
9EUCTR2016-004184-39-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
10EUCTR2018-002087-12-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
11EUCTR2018-002087-12-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
12EUCTR2018-002087-12-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
13EUCTR2016-000778-40-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
14JPRN-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
15JPRN-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
16EUCTR2016-000750-35-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
17EUCTR2016-004184-39-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
18NCT03032172
(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
19NCT02913482
(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
20EUCTR2016-000778-40-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
21EUCTR2016-000750-35-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
22NCT02908685
(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
23EUCTR2016-000750-35-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
24NCT02633709
(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
25NCT04256265
(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
26EUCTR2021-005314-34-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
27EUCTR2016-004184-39-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
28EUCTR2021-003417-19-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
29EUCTR2016-004184-39-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
30EUCTR2021-001294-23-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
31EUCTR2016-004184-39-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
32EUCTR2016-004184-39-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
33EUCTR2016-000750-35-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
34EUCTR2021-001294-23-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
35EUCTR2021-003417-19-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
36EUCTR2021-005314-34-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