Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Part of paid clinical trials in Norfolk, Virginia.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT05335876
Phase
PHASE3
Status
Recruiting
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Conditions

  • Spinal Muscular Atrophy (SMA)

Eligibility Criteria

Sex
ALL
Age
0 Years - 100 Years
Healthy Volunteers
Not accepted

Interventions

  • onasemnogene abeparvovec — BIOLOGICAL
    Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.

Study Details

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.

Key Dates

Start date
Dec 19, 2022
Status verified
Jan 2026
Primary completion
Sep 26, 2030
Completion
Feb 27, 2031

Study Design

Enrollment
175 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec
    Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)

Primary Outcome Measure

Number of participants with treatment-emergent serious adverse events (SAEs) [ Time Frame: Up to Year 5 ]

Central Contacts

  • Novartis Pharmaceuticals
    1-888-669-6682
    [email protected]
  • Novartis Pharmaceuticals
    +41613241111

Locations (1)

FacilityCityStateZIPSite coordinators
Child Hosp Of The Kings DaughtersNorfolkVirginia23507
Holly Casper
[email protected]
757-668-9026
Crystal Proud (PRINCIPAL_INVESTIGATOR)

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By research site
Child Hosp Of The Kings Daughters· Norfolk, VA

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