3. Spinal muscular atrophy
179 clinical trials,   102 drugs   (DrugBank: 26 drugs),   52 drug target genes,   78 drug target pathways
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 |
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1 | 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 | ||
2 | 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 |
3 | 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 |