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 | 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] | Trade Name: pyridostigmine bromide Product Name: Mestinon Product Code: RVG 03820 | Universtiy Medical Center Utrecht | NULL | Not Recruiting | Female: yes Male: yes | 45 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Netherlands | ||
| 2 | NCT02227823 (ClinicalTrials.gov) | July 2014 | 20/8/2014 | Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 | Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3. | Spinal Muscular Atrophy Type 3 | Drug: Pyridostigmine Bromide | Centre Hospitalier Régional de la Citadelle | NULL | Recruiting | 6 Years | N/A | Both | 20 | Phase 2 | Belgium |