drugset / Trial / NCT03306277
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
NaSingle-groupOpen-labelTreatment
Summary
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
Timeline
- Start
- 2017-10-24
- Primary completion
- 2019-11-12
- Completion
- 2019-11-12
Outcome
Met primary endpoint
paper 13 (59%, 97·5% CI 36-100) of 22 patients achieved functional independent sitting for 30 s or longer PMID 33743238 ↗
paper at the 18 month of age study visit (vs 0 of 23 patients in the untreated PNCR cohort; p<0·0001). PMID 33743238 ↗
Publications
- Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12.
- Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, Iannaccone ST, Kuntz NL, Pena LDM, Shieh PB, Smith EC, Kwon JM, Zaidman CM, Schultz M, Feltner DE, Tauscher-Wisniewski S, Ouyang H, Chand DH, Sproule DM, Macek TA, Mendell JR. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Onasemnogene Abeparvovec-xioi | Gene therapy (AAV / viral vector) | 1.1e+14 vg/kg | Intravenous |