3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 237 / 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75
| 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 | EUCTR2015-001589-25-NL (EUCTR) | 05/04/2016 | 07/10/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Poland;Belgium;Germany;Netherlands;Italy;United Kingdom | ||
| 2 | NCT02628743 (ClinicalTrials.gov) | January 20, 2016 | 1/12/2015 | A 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 Atrophy | Muscular Atrophy, Spinal | Drug: Olesoxime | Hoffmann-La Roche | NULL | Completed | N/A | N/A | All | 131 | Phase 2 | Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom |
| 3 | EUCTR2015-001589-25-FR (EUCTR) | 01/12/2015 | 07/12/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA | Spinal Muscular Atrophy MedDRA version: 18.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2015-001589-25-BE (EUCTR) | 20/11/2015 | 25/09/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 5 | EUCTR2015-001589-25-GB (EUCTR) | 19/11/2015 | 04/08/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 6 | EUCTR2015-001589-25-IT (EUCTR) | 05/10/2015 | 11/08/2015 | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA | Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA | Spinal Muscular Atrophy MedDRA version: 18.0;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Phase 2;Phase 3 | France;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 7 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Belgium;Germany;Netherlands;Italy;United Kingdom | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 |
| 11 | 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 | ||
| 12 | EUCTR2010-020386-24-IT (EUCTR) | 14/09/2010 | 01/09/2010 | Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients | Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients | Spinal Muscular Atrophy - type II or III in non ambulant patients aged 3-25 years MedDRA version: 9.1;Level: LLT;Classification code 10032950 | Product Name: olesoxime Product Code: TRO19622 INN or Proposed INN: olesoxime | TROPHOS | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | Belgium;Germany;Netherlands;United Kingdom;Italy | ||
| 13 | EUCTR2015-001589-25-DE (EUCTR) | 13/08/2015 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | |||
| 14 | EUCTR2015-001589-25-PL (EUCTR) | 07/01/2016 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL, REFINED Other descriptive name: SESAME OIL, REFINED | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 173 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Poland;Netherlands;Germany;Italy;United Kingdom | |||
| 15 | 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 |