Trial Profile
Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy
Status:
Discontinued
Phase of Trial:
Phase I/II
Latest Information Update: 26 Apr 2023
Price :
$35
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At a glance
- Drugs Onasemnogene-abeparvovec (Primary)
- Indications Spinal muscular atrophy
- Focus Adverse reactions; Proof of concept; Therapeutic Use
- Acronyms STRONG
- Sponsors Novartis Gene Therapies; Novartis Pharmaceuticals
- 04 Jan 2022 Status changed from recruiting to completed.
- 03 Aug 2021 According to a Novartis media release, the U.S. Food and Drug Administration (FDA) has lifted partial clinical hold based on data from Novartis comprehensive nonclinical toxicology study in non-human primates (NHP) that addressed all issues identified, including questions of dorsal root ganglia (DRG) injury following IT administration.
- 03 Aug 2021 Status changed from suspended to recruiting, according to a Novartis media release.