Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
Completed · Phase 3
Conditions studied: Spinal Muscular Atrophy
In brief
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to \<18 years after the discontinuation of treatment with nusinersen or risdiplam. The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to \<6 years, and 6 to \<18 years).
Key facts
- Study ID
- NCT05386680
- Run by
- Novartis Pharmaceuticals
- People needed
- 27
- Starts
- 2023-01-12
- Expected to finish
- 2024-11-29
- 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.
You may qualify if…
- SMA diagnosis
- Aged 2 to < 18 years
- Have had at least four loading doses of nusinersen (Spinraza®) or at least 3 months of treatment with risdiplam (Evrysdi®) at Screening
- Must have symptoms of SMA as defined in the protocol
You may not qualify if…
- Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated
- Clinically significant abnormalities in test results during screening
- Contraindications for lumbar puncture procedure
- At Baseline, participants are excluded if they received:
- nusinersen (Spinraza®) or
- risdiplam (Evrysdi®) within a defined timeframe
- Vaccinations 2 weeks prior to administration of OAV101
- Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.
- Presence of an infection or febrile illness up to 30 days prior to administration of OAV101
- Requiring invasive ventilation
Where it is running
- Boston Childrens Hospital — Boston, Massachusetts, United States
- Child Hosp Of The Kings Daughters — Norfolk, Virginia, United States
- University of Wisconsin Madison Medical School — Madison, Wisconsin, United States
- Novartis Investigative Site — Parkville, Victoria, Australia
- Novartis Investigative Site — Leuven, Belgium
- Novartis Investigative Site — Montreal, Quebec, Canada
- Novartis Investigative Site — Bron, France
- Novartis Investigative Site — Toulouse, France
- Novartis Investigative Site — Roma, RM, Italy
- Novartis Investigative Site — Kurume, Fukuoka, Japan
- Novartis Investigative Site — Shinjuku Ku, Tokyo, Japan
- Novartis Investigative Site — Utrecht, Netherlands
- Novartis Investigative Site — Barcelona, Catalonia, Spain
Full record on ClinicalTrials.gov
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