3. Spinal muscular atrophy Clinical trials / Disease details
Clinical trials : 217 / Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 80
| 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 | EUCTR2016-000778-40-FR (EUCTR) | 21/04/2017 | 24/10/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 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 Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | France;Belgium;Germany;Italy | ||
| 2 | EUCTR2016-000778-40-DE (EUCTR) | 05/01/2017 | 21/07/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 / F12 INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 / F13 INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China | ||
| 3 | EUCTR2016-000778-40-IT (EUCTR) | 04/10/2016 | 16/08/2016 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 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 Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067 | F. Hoffmann-La Roche Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 64 | Phase 2 | France;Belgium;Germany;Italy | ||
| 4 | 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 | ||
| 5 | 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 |