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 3. 脊髄性筋萎縮症 [臨床試験数:75,薬物数:73(DrugBank:24),標的遺伝子数:46,標的パスウェイ数:20] 

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04089566February 26, 20204 November 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: NusinersenBiogenNot recruitingN/AN/AAll125Phase 2/Phase 3
2NCT04042025November 4, 201911 November 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 IIIBiological: Onasemnogene Abeparvovec-xioiAveXis, Inc.Not recruitingN/AN/AAll308Phase 4United States;Australia;Belgium;Canada;France;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom
3NCT03779334August 8, 20194 November 2019A 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 RocheRecruitingN/A6 WeeksAll25Phase 2United States;Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia
4NCT03988907June 18, 20194 November 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 RocheNot recruiting18 Years55 YearsAll27Phase 1United States
5NCT03837184May 15, 201926 August 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-xioiAveXis, Inc.PRA Health SciencesRecruitingN/A6 MonthsAll6Phase 3Japan;Korea, Republic of;Taiwan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03921528April 22, 20192 September 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.Recruiting2 Years21 YearsAll55Phase 2United States;Italy;Spain
7NCT03878030April 1, 201911 June 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 HealthRecruiting18 Years60 YearsAll40Phase 3United States
8NCT03819660March 7, 20198 April 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.Recruiting6 Years50 YearsAll12Phase 2Italy
9NCT03648658February 18, 201911 March 2019Paracetamol 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
10NCT02876094January 29, 201926 August 2019Effect 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 FoundationRecruiting2 Years80 YearsAll12Phase 2Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03781479January 21, 20194 February 2019Controlled 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: Placebo Oral Tablet;Drug: Amifampridine PhosphateCatalyst Pharmaceuticals, Inc.Recruiting6 Years50 YearsAll12Phase 2Italy
12EUCTR2018-002087-12-BE07/12/201830 April 2019A 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 ATROPHYSpinal 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
Pharmaceutical Form: Powder for oral solution
INN or Proposed INN: Risdiplam
CAS Number: 1825352-65-5
Current Sponsor code: RO7034067/F13
Other descriptive name: RO7034067
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 60-
Pharmaceutical form of the placebo: Powder for oral solution
Route of administration of the placebo: Oral use
Product Name: RO7034067
Product Code: RO7034067/F12
Pharmaceutical Form: Powder for oral solution
INN or Proposed INN: Risdiplam
CAS Number: 1825352-65-5
Current Sponsor code: RO7034067/F12
Other descriptive name: RO7034067
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
F. Hoffmann-La Roche LtdAuthorised Female: yes
Male: yes
25Phase 2United States;Saudi Arabia;Poland;Brazil;Belgium;Australia;Russian Federation;Italy;China
13EUCTR2018-002295-40-DK11/09/201824 September 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 massSpinal muscular atrophy type II (SMA II) Cerebral palsy (CP)
MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1 Level: PT Classification code 10008129 Term: Cerebral palsy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Paracetamol
Other descriptive name: PARACETAMOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 24-
Copenhagen Neuromuscular CenterAuthorisedFemale: yes
Male: yes
24Phase 1;Phase 4Denmark
14NCT03461289August 29, 20187 October 2019Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1European, Phase 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-xioiAveXis, Inc.Not recruitingN/A6 MonthsAll33Phase 3Belgium;Italy;United Kingdom;France;Germany;Netherlands;Spain;Sweden
15JPRN-JapicCTI-18389101/6/201816 July 2019Investigate 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 AtrophySpinal Muscular AtrophyIntervention name : RO7034067
INN of the intervention : risdiplam
Dosage And administration of the intervention : po
CHUGAI PHARMACEUTICAL CO., LTD.Not RecruitingBOTH40Phase 2
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT03505099April 10, 201826 August 2019Pre-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-xioiAveXis, Inc.PRA Health SciencesRecruitingN/A42 DaysAll27Phase 3United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom
17EUCTR2018-004383-65-NL30 April 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 studyLater 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 Level: LLT 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
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: SRK-015
Current Sponsor code: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Product Name: SRK-015
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: SRK-015
Current Sponsor code: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Scholar Rock, Inc.Not Available Female: yes
Male: yes
55Phase 2United States;Canada;Spain;Germany;Netherlands;Italy
18NCT03381729December 14, 201726 August 2019Study 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-xioiAveXis, Inc.Recruiting6 Months60 MonthsAll51Phase 1United States
19JPRN-JapicCTI-17372201/12/201716 July 2019A 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 PatientsSpinal Muscular AtrophyIntervention name : RO7034067
INN of the intervention : risdiplam
Dosage And administration of the intervention : po
Control intervention name : Placebo
Dosage And administration of the control intervention : po
CHUGAI PHARMACEUTICAL CO., LTD.Not Recruiting225BOTH168Phase 2
20NCT03306277October 24, 201726 August 2019Gene 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 InfusionSMA - Spinal Muscular Atrophy;Gene TherapyBiological: Onasemnogene Abeparvovec-xioiAveXis, Inc.Not recruitingN/A180 DaysAll20Phase 3United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT03421977August 15, 201726 August 2019Long-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-xioiAveXis, Inc.RecruitingN/AN/AAll15N/AUnited States
22NCT03032172March 3, 20174 November 2019A 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 RO7034067 in Adult and Pediatric Patients With Spinal Muscular AtrophySpinal Muscular AtrophyDrug: RisdiplamHoffmann-La RocheRecruiting6 Months60 YearsAll180Phase 2United States;Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom
23EUCTR2016-000778-40-DE05/01/20175 June 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 ATROPHYType 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)
Pharmaceutical Form: Powder and solvent for oral solution
INN or Proposed INN: n/a
Current Sponsor code: RO7034067/F06
Other descriptive name: RO7034067
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
Pharmaceutical Form: Powder and solvent for oral solution
INN or Proposed INN: n/a
Current Sponsor code: RO7034067/F07
Other descriptive name: RO7034067
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 120-
Product Name: RO7034067
Product Code: RO7034067 / F12
Pharmaceutical Form: Powder for oral solution
INN or Proposed INN: n/a
Current Sponsor code: RO7034067/F12
Other descriptive name: RO7034067
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Product Name: RO7034067
Product Code: RO7034067 / F13
Pharmaceutical Form: Powder for oral solution
INN or Proposed INN: n/a
Current Sponsor code: RO7034067 / F13
Other descriptive name: RO7034067
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 60-
F. Hoffmann-La Roche LtdAuthorisedFemale: 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
24EUCTR2017-004087-35-DE19 November 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 - SPR1NTSpinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
Pharmaceutical Form: Infusion
INN or Proposed INN: onasemnogene abeparvovec
CAS Number: 1922968-73-7
Current Sponsor code: AVXS-101
Concentration unit: vector genomes (vg)/mL
Concentration type: range
Concentration number: 20000000000000-60000000000000
AveXis, Inc.Not AvailableFemale: yes
Male: yes
27Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan
25EUCTR2017-004087-35-NL23 July 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 - SPR1NTSpinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: AVXS-101 (previously known as scAAV9.CB.SMN)
Product Code: AVXS-101
Pharmaceutical Form: Infusion
INN or Proposed INN: onasemnogene abeparvovec
CAS Number: 1922968-73-7
Current Sponsor code: AVXS-101
Concentration unit: vector genomes (vg)/mL
Concentration type: range
Concentration number: 20000000000000-60000000000000
AveXis, Inc.Not AvailableFemale: yes
Male: yes
44Phase 3United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Netherlands;Germany;Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT02913482December 24, 201616 September 2019Investigate 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 RocheNot recruiting1 Month7 MonthsAll48Phase 2/Phase 3United States;Belgium;Brazil;China;Croatia;France;Italy;Japan;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Australia;Germany;Taiwan
27NCT02908685October 20, 201616 September 2019A 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 RO7034067 in Type 2 and 3 Spinal Muscular Atrophy PatientsMuscular Atrophy, SpinalDrug: Placebo;Drug: RisdiplamHoffmann-La RocheNot recruiting2 Years25 YearsAll231Phase 2/Phase 3United States;Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;Argentina;Australia;Sweden;Switzerland;Taiwan;United Kingdom
28EUCTR2016-000750-35-IT29/08/201620 February 2017A 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)
Pharmaceutical Form: Powder and solvent for oral solution
INN or Proposed INN: n.a
Current Sponsor code: RO7034067/F06
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Powder and solvent for oral solution
Route of administration of the placebo: Oral use
Product Name: RO7034067
Product Code: RO7034067/F07 with solvent (RO7034067/F09)
Pharmaceutical Form: Powder and solvent for oral solution
INN or Proposed INN: n.a
Current Sponsor code: RO7034067/F07
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 120-
Pharmaceutical form of the placebo: Powder and solvent for oral solution
Route of administration of the placebo: Oral use
F. Hoffmann-La Roche LtdAuthorisedFemale: yes
Male: yes
186Phase 2United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden
29JPRN-JMA-IIA0025916/08/20162 April 2019Multicenter 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 trialspinal 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 UniversityRecruiting>=1 YEARS<8 YEARSBOTH28Phase 2BJapan
30EUCTR2015-001870-16-DE08/03/201630 April 2019An 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 396443Spinal 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Nusinersen
CAS Number: 1258984-36-9
Current Sponsor code: ISIS 396443 (BIIB058)
Other descriptive name: ISIS 396443
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2.4-
Biogen Idec Research LimitedAuthorised Female: yes
Male: yes
292Phase 3United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31JPRN-JMA-IIA0023129/01/20162 April 2019Multicenter 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 Trialspinal 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 UniversityNot Recruiting>=1 YEARS<7 YEARSBOTH28Phase 2BJapan
32NCT02628743January 20, 201618 March 2019A 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 RocheNot recruitingN/AN/AAll131Phase 2Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom
33NCT02633709January 7, 20169 October 2018A 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 RocheNot recruiting18 Years45 YearsMale33Phase 1Netherlands
34NCT02644668December 201518 March 2019A 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 Atrophy (SMA)Spinal Muscular AtrophyDrug: CK-2127107 150 mg;Drug: Placebo;Drug: CK-2127107 450 mgCytokineticsAstellas Pharma Global Development, Inc.Not recruiting12 YearsN/AAll70Phase 2United States;Canada
35NCT02941328December 201512 February 2018SPACE 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 UtrechtNot recruiting12 YearsN/AAll39Phase 2Netherlands
No.TrialIDDate_
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36EUCTR2015-001589-25-GB19/11/201511 December 2017A 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 - OLEOSSpinal 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
Pharmaceutical Form: Powder and solvent for oral suspension
INN or Proposed INN: OLESOXIME
CAS Number: 22033-87-0
Current Sponsor code: RO7090919/F01
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 7.5-
INN or Proposed INN: SESAME OIL, REFINED
Current Sponsor code: RO7090919/F02
Other descriptive name: SESAME OIL, REFINED
Concentration unit: ml millilitre(s)
Concentration type: equal
Concentration number: 67.1-
F. Hoffmann-La Roche LtdAuthorisedFemale: yes
Male: yes
173Phase 2;Phase 3Belgium;Netherlands;Germany;Italy;United Kingdom
37NCT02594124November 4, 20154 November 2019A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies.An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443Spinal Muscular AtrophyDrug: nusinersenBiogenNot recruitingN/AN/AAll292Phase 3United States;Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Taiwan
38EUCTR2014-003657-33-DE21/09/201514 January 2019A 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 - EMBRACESpinal 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)
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Nusinersen
CAS Number: 1258984-36-9
Current Sponsor code: ISIS 396443 (BIIB058)
Other descriptive name: ISIS 396443 (BIIB058)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2.4-
Biogen Idec Research LimitedNot RecruitingFemale: yes
Male: yes
16Phase 2United States;Germany;United Kingdom
39NCT02855112June 201515 August 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 SciencesRecruiting5 Months12 MonthsBoth10Phase 1/Phase 2Iran, Islamic Republic of
40NCT02386553May 20, 20154 November 2019A 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 Atrophy.Spinal Muscular AtrophyDrug: NusinersenBiogenNot recruitingN/A6 WeeksAll25Phase 2United States;Australia;Germany;Italy;Qatar;Taiwan;Turkey;Argentina;Israel;United Kingdom
No.TrialIDDate_
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41EUCTR2011-004369-34-NL22/04/201530 April 2019SPACE 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 4SPACE 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 trialProximal 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 Level: LLT 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
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Universtiy Medical Center UtrechtNot Recruiting Female: yes
Male: yes
45Phase 2Netherlands
42NCT02268552April 2, 20154 November 2019An 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 PharmaceuticalsNot recruitingN/A182 DaysAll39Phase 1/Phase 2Belgium;Bulgaria;Denmark;Germany;Italy;Poland;Russian Federation;Czechia;Hungary;Netherlands
43EUCTR2014-002053-19-IT01/04/201528 February 2019Clinical 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 - CLMI070X2201Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: NA
Product Code: LMI070
Pharmaceutical Form: Oral solution
INN or Proposed INN: NA
CAS Number: NA
Current Sponsor code: LMI070
Other descriptive name: NA
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 3.5-
NOVARTIS FARMA S.p.A.Authorised Female: yes
Male: yes
22Phase 1;Phase 2United States;Hungary;Czech Republic;European Union;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy
44NCT02292537November 201416 December 2017A 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 procedureBiogenNot recruiting2 Years12 YearsAll126Phase 3United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom
45EUCTR2014-002246-41-IT23/10/201431 January 2017A 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) - MOONFISHSpinal 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
Pharmaceutical Form: Powder for oral solution
INN or Proposed INN: n.a.
CAS Number: 1449598-06-4
Current Sponsor code: RO6885247
Other descriptive name: n.a.
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 80-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
F. Hoffmann-La Roche Ltd.Not RecruitingFemale: yes
Male: yes
48Phase 1United States;Spain;Netherlands;United Kingdom;Switzerland;Italy
No.TrialIDDate_
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46NCT02193074August 19, 201416 December 2017A 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 procedureBiogenNot recruitingN/A210 DaysAll122Phase 3United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan
47JPRN-JMA-IIA0019022/07/20142 April 2019Multicenter 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 atrophyspinal 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 UniversityNot RecruitingNo Limit<8 YEARSBOTH13Phase 2AJapan
48NCT02227823July 201419 February 2015Safety 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 CitadelleRecruiting6 YearsN/ABoth20Phase 2Belgium
49NCT02122952May 5, 201412 March 2018Gene 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-101AveXis, Inc.Not recruitingN/A6 MonthsAll15Phase 1United States
50NCT02052791January 201416 December 2017An 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: nusinersenBiogenNot recruitingN/AN/AAll47Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
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51NCT01671384August 201318 January 2016Valproate 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 DelhiRecruiting2 Years15 YearsBoth60Phase 3India
52NCT01839656May 31, 201311 June 2018A 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: nusinersenBiogenNot recruitingN/A210 DaysAll20Phase 2United States;Canada
53NCT01780246January 201316 December 2017An 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: nusinersenBiogenNot recruiting2 Years15 YearsAll18Phase 1United States
54NCT01703988October 201216 December 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 AtrophySpinal Muscular AtrophyDrug: NusinersenBiogenNot recruiting2 Years15 YearsAll34Phase 1/Phase 2United States
55NCT01645787June 201230 May 2016Short 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 UniversityNot recruiting18 Years50 YearsBoth11Phase 2/Phase 3United States
No.TrialIDDate_
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56NCT01494701November 30, 201116 December 2017An 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: nusinersenBiogenNot recruiting2 Years14 YearsAll28Phase 1United States
57NCT01422200August 201119 February 2015Flu 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 vaccineChildren's Hospital Medical Center, CincinnatiRecruiting3 Years35 YearsBoth30N/AUnited States
58ChiCTR-TRC-100010932010-10-0118 April 2017Rat 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 trialchildren with spinal muscular atrophyA:Rat nerve growth factor injection for six monthes,;B:Rat nerve growth factor injection for six monthes,;PLA General HospitalNot Recruiting38BothA:20;B:20;Post-marketChina
59EUCTR2010-020386-24-FR2 October 2017Phase 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: olesoxime
CAS Number: 2203-87-0
Current Sponsor code: TRO19622
Other descriptive name: 4-cholesten-3-one, oxime
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
TROPHOS SANot AvailableFemale: yes
Male: yes
Phase 2France;Belgium;Germany;Netherlands;United Kingdom;Italy
60EUCTR2008-003915-11-DE24/09/200917 December 2012Phase 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 ISpinal Muscular Atrophy Type I in infants
MedDRA version: 9.1 Level: LLT Classification code 10051203 Term: Spinal muscular atrophy congenital
Trade Name: Orfiril Saft
Pharmaceutical Form: Oral solution
INN or Proposed INN: VALPROATE SODIUM
CAS Number: 1069665
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 60-
Trade Name: Biocarn
Pharmaceutical Form: Oral solution
INN or Proposed INN: LEVOCARNITINE
CAS Number: 541151
Concentration unit: g/ml gram(s)/millilitre
Concentration type: equal
Concentration number: 0.303030-
University of UtahNot RecruitingFemale: yes
Male: yes
36Phase 1/2Germany
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61EUCTR2007-001088-32-IT29/07/200819 March 2012A 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. - NDPatients affected by SMA
MedDRA version: 9.1 Level: LLT Classification code 10041582 Term: Spinal muscular atrophy
Trade Name: VOLMAX*30CPR 4MG R.P.
Pharmaceutical Form: Modified-release tablet
INN or Proposed INN: Salbutamol
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 4-
Pharmaceutical form of the placebo: Modified-release tablet
Route of administration of the placebo: Oral use
ISTITUTO NEUROLOGICO CARLO BESTANot RecruitingFemale: yes
Male: yes
Phase 2Italy
62NCT00661453April 200819 October 2017CARNIVAL 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.Not recruitingN/A12 MonthsAll40Phase 1/Phase 2United States;Canada;Germany
63NCT00439218January 200819 October 2017Clinical 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)Not recruiting2 Months48 MonthsAll5Phase 1/Phase 2United States
64NCT00439569January 200819 October 2017Clinical 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)Not recruiting2 Years11 YearsAll9Phase 1/Phase 2United States
65NCT00481013July 20079 January 2017Valproic 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;AbbottNot recruiting18 Years60 YearsBoth33Phase 2United States
No.TrialIDDate_
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66NCT00528268July 200719 October 2017Study 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)Not recruitingN/A6 MonthsAll14Phase 1/Phase 2United States
67NCT00485511June 200719 February 2015A 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 HospitalNot recruiting4 YearsN/ABothPhase 2/Phase 3Taiwan
68EUCTR2006-006845-14-FR20/03/200719 March 2012Open-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
Pharmaceutical Form: Capsule*
CAS Number: 22033-87-0
Current Sponsor code: TRO19622
Other descriptive name: 4-cholesten-3-one,oxime
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 125-250
TROPHOSAuthorisedFemale: yes
Male: yes
Phase 1bFrance
69EUCTR2005-002822-78-DE09/01/200619 March 2012Can 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-GHSpinal 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 Code: GH
Pharmaceutical Form: Injection*
INN or Proposed INN: Somatropin
Other descriptive name: Norditropin SimpleXx
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Injection*
Route of administration of the placebo: Subcutaneous use
Klinik Neuropädiatrie und MuskelkrankheitenNot RecruitingFemale: yes
Male: yes
20Germany
70NCT00774423January 200619 February 2015Study 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 ParisNot recruiting6 Years20 YearsBoth141Phase 2/Phase 3France
No.TrialIDDate_
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71NCT00227266September 200519 October 2017Valproic 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.;AbbottNot recruiting2 Years17 YearsAll94Phase 2United States;Canada
72NCT00568698January 200419 February 2015A 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: HydroxyureaStanford UniversityNot recruitingN/A2 YearsBoth18Phase 1/Phase 2United States
73NCT00568802January 200416 September 2019A 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 UniversityNot recruiting1 Year10 YearsAll27Phase 1/Phase 2United States
74NCT00374075September 20035 September 2016Study 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.;AbbottNot recruiting2 YearsN/ABoth42Phase 1United States
75NCT00004771October 199219 February 2015Phase 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 UniversityNot recruiting18 YearsN/AMale40Phase 2

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