3. 脊髄性筋萎縮症 [臨床試験数:179,薬物数:102(DrugBank:26),標的遺伝子数:52,標的パスウェイ数:78

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
34 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2016-000750-35-GB25/11/202023/06/2016A 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: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
2NCT03781479January 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 YearsAll12Phase 2Italy
3EUCTR2016-000750-35-BG20/06/201804/06/2018A 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: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
4EUCTR2016-000750-35-HR27/03/201812/04/2018A 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: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
5JPRN-JapicCTI-17372201/12/201704/10/2017A 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
Dosage And administration of the control intervention : po
CHUGAI PHARMACEUTICAL CO., LTD.NULLcomplete225BOTH168NULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2016-000750-35-DE09/11/201613/07/2016A 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: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
7NCT02908685October 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;Argentina;Australia;Sweden;Switzerland;Taiwan;United Kingdom
8EUCTR2016-000750-35-BE06/09/201612/07/2016A 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: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
9EUCTR2016-000750-35-IT29/08/201628/06/2016A 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: 19.0Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
10NCT02644668January 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT02633709January 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
12NCT02941328December 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
13EUCTR2011-004369-34-NL22/04/201503/11/2014 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 Level: LLT Classification code 10051203 Term: Spinal muscular atrophy congenital System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Universtiy Medical Center UtrechtNULLNot Recruiting Female: yes
Male: yes
45 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noNetherlands
14EUCTR2014-002246-41-ES15/04/201503/03/2015A 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: 18.0Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
15NCT02240355November 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2014-002246-41-IT23/10/201409/09/2014A 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: 17.0Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
17NCT01671384August 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
18NCT01645787June 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
19EUCTR2010-020386-24-GB31/01/201108/10/2010Safety 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]
TROPHOS SANULLNot Recruiting Female: yes
Male: yes
150 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance;Belgium;Germany;Netherlands;Italy;United Kingdom
20EUCTR2010-020386-24-DE29/12/201028/07/2010Phase 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: 12.1Level: LLTClassification code 10041582Term: 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2010-020386-24-NL18/11/201028/06/2010Phase 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: 12.1Level: LLTClassification code 10041582Term: 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
22NCT01302600November 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
23EUCTR2010-020386-24-BE29/10/201027/07/2010Phase 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: 12.1Level: LLTClassification code 10041582Term: 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
24EUCTR2007-001088-32-IT29/07/200818/07/2008A 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: 9.1Level: LLTClassification code 10041582Term: 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
25NCT00533221October 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT00481013July 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
27NCT00485511June 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
28NCT00774423January 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
29NCT00227266September 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
30NCT00568698January 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT00568802January 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
32EUCTR2016-000750-35-PL20/02/2018A 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: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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
33EUCTR2010-020386-24-FR01/06/2010Phase 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: 12.1Level: LLTClassification code 10041582Term: 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
34EUCTR2016-000750-35-FR26/01/2018A 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: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem 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