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
NCT ID: NCT05386680
Last Updated: 2026-01-13
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
27 participants
INTERVENTIONAL
2023-01-12
2024-11-29
Brief Summary
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Detailed Description
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Participants were admitted to the hospital on Day -1 for pre-treatment baseline procedures. After receiving OAV101B on Day 1, participants underwent in-patient safety monitoring over the next 48 hours, after which the participant could be discharged, based on Investigator judgment.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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OAV-101
Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose
OAV101
Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose
Interventions
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OAV101
Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
2 Years
17 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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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
Countries
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References
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Kwon JM, Munell F, Le Goff L, Yuge K, Kato T, Cances C, De Waele L, Woodcock IR, Mercuri EM, Proud CM, Darras BT, Hayes LH, Oskoui M, Visootsak J, Williams G, Ilic A, Yang L, van der Pol WL. Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial. Nat Med. 2025 Dec 8. doi: 10.1038/s41591-025-04119-2. Online ahead of print.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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A Plain Language Trial Summary is available on www.novctrd.com
A Pediatric Plain Language Trial Summary is available on www.novctrd.com
Other Identifiers
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COAV101B12302
Identifier Type: -
Identifier Source: org_study_id
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