Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
Completed · Phase 3 · Has a placebo group
Conditions studied: Type 2 Spinal Muscular Atrophy
In brief
This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.
Key facts
- Study ID
- NCT05089656
- Run by
- Novartis Pharmaceuticals
- People needed
- 126
- Starts
- 2022-02-01
- Expected to finish
- 2025-04-29
- Last updated by the study team
- 2026-01-13
Who can join
Age: 2 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Connecticut Children's Medical Center — Farmington, Connecticut, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Clinic for Special Children — Strasburg, Pennsylvania, United States
- St Jude Children's Research Hospital — Memphis, Tennessee, United States
- Child Hosp Of The Kings Daughters — Norfolk, Virginia, United States
- Children's Specialty Group/CHKD — Norfolk, Virginia, United States
- Novartis Investigative Site — Curitiba, Paraná, Brazil
- Novartis Investigative Site — São Paulo, São Paulo, Brazil
- Novartis Investigative Site — Beijing, Beijing Municipality, China
- Novartis Investigative Site — Chongqing, Chongqing Municipality, China
- Novartis Investigative Site — Guangzhou, Guangdong, China
- Novartis Investigative Site — Shenzhen, Guangdong, China
- Novartis Investigative Site — Chengdu, Sichuan, China
- Novartis Investigative Site — Hangzhou, Zhejiang, China
- Peking University First Hospital — Beijing, China
- Novartis Investigative Site — Beijing, China
- Novartis Investigative Site — Beijing, China
- Novartis Investigative Site — Copenhagen, Denmark
- Paediatric Neurology — Copenhagen, Denmark
- Sir Ganga Ram Hospital — New Delhi, National Capital Territory of Delhi, India
- Novartis Investigative Site — New Delhi, National Capital Territory of Delhi, India
- Novartis Investigative Site — New Delhi, National Capital Territory of Delhi, India
- AIIMS, Ansari Nagar — New Delhi, New Delhi, India
- Novartis Investigative Site — Kolkata, West Bengal, India
- Novartis Investigative Site — Hyderabad, India
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.