Spinal muscular atrophies are characterized by muscle weakness and hypotonia due to the degeneration and loss of motor neurons. SMA type II affects one person in 70 000. Its onset is between 6 and 18 months of age. The children have difficulty in sitting alone and cannot stand or walk. The muscle weakness affects mainly the muscles of the legs and the trunk.
Trials currently conducted on these diseases at the Institute:
- AVXS-101-LT-002: A Long-term Follow-up Study of Patients in the Clinical Trials for SMA Receiving AVXS-101
- ONYX: An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab
- PHENO SMART: Characterization of New Phenotypes of Patients with Spinal Muscular Atrophy Treated with SMN Restoring Therapy
- Manatee Roche: A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy
- SRK-015-005 – OPALE: A Phase 2 Study Evaluating the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old with Spinal Muscular Atrophy (SMA)
- R-SMA: French Register of Patients with SMA
- NH-SMA / SMOB: Outcome measures and biomarkers in a cohort of type III and IV SMA
Acronym |
AVXS-101-LT-002 |
ONYX |
| Intervention | AVX-101 | Apitegromab |
| Principal investigator | Andreea Seferian | Andreea Seferian |
| Sponsor | AveXis | Scholar Rock |
| Study status | Active | Active |
| Recruitment status | Completed | Completed |
| Population | Child | Child |
| + infos on clinicaltrials.gov | + infos on clinicaltrials.gov |
Acronym |
PHENO SMART |
Manatee Roche |
SRK-015-005 – OPALE |
| Intervention | – | RO7204239 | Apitegromab |
| Principal investigator | Silvana De Lucia | Silvana De Lucia | Andreea Seferian |
| Sponsor | UCL | Roche | Scholar Rock |
| Study status | In preparation | In preparation | In preparation |
| Recruitment status | Not started | Not started | Not started |
| Population | Child | Child | Child |
| + infos on clinicaltrials.gov | + infos on clinicaltrials.gov |
Acronym |
R-SMA |
NH-SMA / SMOB |
| Intervention | – | Nusibersen |
| Principal investigator | Tanya Stojkovic | Tanya Stojkovic |
| Sponsor | APHP | APHM |
| Study status | Active | Active |
| Recruitment status | Ongoing | Completed |
| Population | Adult | Adult |
| + infos on clinicaltrials.gov | + infos sur clinicaltrials.gov |
Contact for adults clinical trials: essais-adultes@institut-myologie.org
