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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2016-000750-35-GB (EUCTR) | 25/11/2020 | 23/06/2016 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients | A 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 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 231 | Phase 2 | United States;Serbia;Spain;Turkey;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Croatia;Bulgaria;Germany;China;Japan | ||
| 2 | NCT03781479 (ClinicalTrials.gov) | January 21, 2019 | 18/12/2018 | Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients | A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 | Muscular Atrophy, Spinal | Drug: Amifampridine Phosphate;Drug: Placebo Oral Tablet | Catalyst Pharmaceuticals, Inc. | NULL | Completed | 6 Years | 50 Years | All | 12 | Phase 2 | Italy |
| 3 | EUCTR2016-000750-35-BG (EUCTR) | 20/06/2018 | 04/06/2018 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients | 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 Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F12 INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067/F13 INN or Proposed INN: n.a | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 219 | Phase 2 | Serbia;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 | ||
| 4 | EUCTR2016-000750-35-HR (EUCTR) | 27/03/2018 | 12/04/2018 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients | 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 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 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 219 | Phase 2 | United 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 | ||
| 5 | JPRN-JapicCTI-173722 | 01/12/2017 | 04/10/2017 | A 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 Atrophy | Intervention 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. | NULL | complete | 2 | 25 | BOTH | 168 | Phase 2 | NULL |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2016-000750-35-DE (EUCTR) | 09/11/2016 | 13/07/2016 | A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients | 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 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 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 219 | Phase 2 | Serbia;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 | ||
| 7 | NCT02908685 (ClinicalTrials.gov) | October 20, 2016 | 19/9/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants | A 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 | Muscular Atrophy, Spinal | Drug: Placebo;Drug: Risdiplam | Hoffmann-La Roche | NULL | Active, not recruiting | 2 Years | 25 Years | All | 231 | Phase 2;Phase 3 | United States;Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;Argentina;Australia;Sweden;Switzerland;Taiwan;United Kingdom |
| 8 | EUCTR2016-000750-35-BE (EUCTR) | 06/09/2016 | 12/07/2016 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of Risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients | 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 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 Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 219 | Phase 2 | United 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 | ||
| 9 | EUCTR2016-000750-35-IT (EUCTR) | 29/08/2016 | 28/06/2016 | A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | 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 Atrophy (SMA) MedDRA version: 19.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n.a | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 186 | Phase 2 | United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 10 | NCT02644668 (ClinicalTrials.gov) | January 14, 2016 | 23/12/2015 | A Study of CK-2127107 in Patients With Spinal Muscular Atrophy | A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Placebo;Drug: Reldesemtiv 150 mg;Drug: Reldesemtiv 450 mg | Cytokinetics | Astellas Pharma Global Development, Inc. | Completed | 12 Years | N/A | All | 70 | Phase 2 | United States;Canada |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT02633709 (ClinicalTrials.gov) | January 7, 2016 | 15/12/2015 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers | A 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 Subjects | Spinal Muscular Atrophy | Drug: Itraconazole;Other: Placebo;Drug: Risdiplam | Hoffmann-La Roche | NULL | Completed | 18 Years | 45 Years | Male | 33 | Phase 1 | Netherlands |
| 12 | NCT02941328 (ClinicalTrials.gov) | December 2015 | 30/9/2016 | SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 | A 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 4 | Spinal Muscular Atrophy;SMA;Kugelberg-Welander Disease | Drug: Pyridostigmine;Drug: Placebo | UMC Utrecht | NULL | Completed | 12 Years | N/A | All | 39 | Phase 2 | Netherlands |
| 13 | EUCTR2011-004369-34-NL (EUCTR) | 22/04/2015 | 03/11/2014 | SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 | SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4 - SPACE trial | Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive. MedDRA version: 17.1;Level: LLT;Classification code 10068209;Term: Spinal muscular atrophy adult onset;System Organ Class: 100000004850 MedDRA version: 17.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Universtiy Medical Center Utrecht | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 2 | Netherlands | |||
| 14 | EUCTR2014-002246-41-ES (EUCTR) | 15/04/2015 | 03/03/2015 | A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH) | A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH | Spinal Muscular Atrophy MedDRA version: 18.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: RO6885247/F03 INN or Proposed INN: n.a. Other descriptive name: n.a. Product Code: RO6885247/F02 INN or Proposed INN: n.a. Other descriptive name: n.a. | Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 64 | Phase 1 | France;United States;Spain;Turkey;Netherlands;Italy;United Kingdom;Switzerland;Sweden | ||
| 15 | NCT02240355 (ClinicalTrials.gov) | November 2014 | 11/9/2014 | A 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, Spinal | Drug: RO6885247;Drug: placebo | Hoffmann-La Roche | NULL | Terminated | N/A | 55 Years | Both | 9 | Phase 1 | United States;Canada;France;Italy;Netherlands;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | EUCTR2014-002246-41-IT (EUCTR) | 23/10/2014 | 09/09/2014 | A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH) | A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH | Spinal Muscular Atrophy MedDRA version: 17.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: RO6885247/F03 INN or Proposed INN: n.a. Other descriptive name: n.a. | F. Hoffmann-La Roche Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 1 | United States;Spain;Netherlands;United Kingdom;Switzerland;Italy | ||
| 17 | NCT01671384 (ClinicalTrials.gov) | August 2013 | 13/8/2012 | Valproate and Levocarnitine in Children With Spinal Muscular Atrophy | Randomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 Years | Spinal Muscular Atrophy | Drug: Valproate, Levocarnitine;Drug: Placebo | All India Institute of Medical Sciences, New Delhi | NULL | Recruiting | 2 Years | 15 Years | Both | 60 | Phase 3 | India |
| 18 | NCT01645787 (ClinicalTrials.gov) | June 2012 | 5/7/2012 | Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients | Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action | Spinal Muscular Atrophy | Drug: 4-aminopyridine;Drug: Placebo | Columbia University | NULL | Completed | 18 Years | 50 Years | Both | 11 | Phase 2;Phase 3 | United States |
| 19 | EUCTR2010-020386-24-GB (EUCTR) | 31/01/2011 | 08/10/2010 | Safety and Efficacy of Olesoxime (TRO19622) in 3-25 yrs 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: 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 SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | France;Belgium;Germany;Netherlands;Italy;United Kingdom | |||
| 20 | EUCTR2010-020386-24-DE (EUCTR) | 29/12/2010 | 28/07/2010 | 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. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | EUCTR2010-020386-24-NL (EUCTR) | 18/11/2010 | 28/06/2010 | 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. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 22 | NCT01302600 (ClinicalTrials.gov) | November 2010 | 18/2/2011 | Safety 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 Ambulant | Drug: Olesoxime;Drug: Placebo | Hoffmann-La Roche | Association Française contre les Myopathies (AFM), Paris | Completed | 3 Years | 25 Years | Both | 165 | Phase 2 | Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom |
| 23 | EUCTR2010-020386-24-BE (EUCTR) | 29/10/2010 | 27/07/2010 | 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. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 24 | EUCTR2007-001088-32-IT (EUCTR) | 29/07/2008 | 18/07/2008 | A 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 | A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - ND | Patients affected by SMA MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Trade Name: VOLMAX*30CPR 4MG R.P. INN or Proposed INN: Salbutamol | ISTITUTO NEUROLOGICO CARLO BESTA | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | Italy | |||
| 25 | NCT00533221 (ClinicalTrials.gov) | October 2007 | 12/9/2007 | Pilot Study of Growth Hormon to Treat SMA Typ II and III | Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III? | Muscular Atrophy, Spinal | Drug: somatotropin;Drug: Placebo | University Hospital Freiburg | Novo Nordisk A/S | Completed | 6 Years | 35 Years | Both | 20 | Phase 2 | Germany |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT00481013 (ClinicalTrials.gov) | July 2007 | 30/5/2007 | Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy | Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study | Spinal Muscular Atrophy | Drug: Valproic Acid (VPA);Drug: Placebo | University of Utah | Families of Spinal Muscular Atrophy;Abbott | Completed | 18 Years | 60 Years | Both | 33 | Phase 2 | United States |
| 27 | NCT00485511 (ClinicalTrials.gov) | June 2007 | 11/6/2007 | A Trial of Hydroxyurea in Spinal Muscular Atrophy | A Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Hydroxyurea | Kaohsiung Medical University Chung-Ho Memorial Hospital | NULL | Completed | 4 Years | N/A | Both | Phase 2;Phase 3 | Taiwan | |
| 28 | NCT00774423 (ClinicalTrials.gov) | January 2006 | 16/10/2008 | Study 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) | SMA | Drug: Riluzole | Assistance Publique - Hôpitaux de Paris | NULL | Completed | 6 Years | 20 Years | Both | 141 | Phase 2;Phase 3 | France |
| 29 | NCT00227266 (ClinicalTrials.gov) | September 2005 | 23/9/2005 | Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy | Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial) | Spinal Muscular Atrophy | Drug: Valproic Acid and Levocarnitine;Drug: Placebo | University of Utah | Families of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.;Abbott | Completed | 2 Years | 17 Years | All | 94 | Phase 2 | United States;Canada |
| 30 | NCT00568698 (ClinicalTrials.gov) | January 2004 | 4/12/2007 | A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients | A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea | Stanford University | NULL | Completed | N/A | 2 Years | All | 29 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT00568802 (ClinicalTrials.gov) | January 2004 | 4/12/2007 | A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients | A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea | Stanford University | NULL | Completed | 1 Year | 10 Years | All | 27 | Phase 1;Phase 2 | United States |
| 32 | EUCTR2016-000750-35-PL (EUCTR) | 20/02/2018 | A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients | 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 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 Ltd | NULL | NA | Female: yes Male: yes | 219 | Phase 2 | United 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 | |||
| 33 | EUCTR2010-020386-24-FR (EUCTR) | 01/06/2010 | 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. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years) MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime | TROPHOS SA | NULL | NA | Female: yes Male: yes | Phase 2 | France;Belgium;Germany;Netherlands;United Kingdom;Italy | ||||
| 34 | EUCTR2016-000750-35-FR (EUCTR) | 26/01/2018 | A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | 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 Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n.a | F. Hoffmann-La Roche Ltd | NULL | NA | Female: yes Male: yes | 186 | Phase 2 | United States;Spain;Turkey;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden |