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

Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to 12 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)

ClinicalTrials.gov Identifier: NCT05386680

Novartis Reference Number: COAV101B12302

Last Update: Jan 20, 2023

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

This is an open-label, single arm, multi-center study. Approximately 28 participants aged 2 to 12 years will be enrolled stratified as 2 to 5 years and 6 to 12 years. The study is comprised of 3 periods, Screening (up to 45 days), Treatment (1 day), and Follow-up (52 weeks).

Condition 
Spinal Muscular Atrophy
Phase 
Phase 3
Overall status 
Recruiting
Start date 
Jan 12, 2023
Completion date 
Oct 21, 2024
Gender 
All
Age(s)
2 Years - 12 Years (Child)

Interventions

Genetic
OAV101
Single intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes

Eligibility Criteria

Inclusion Criteria

SMA diagnosis
Aged 2 to 12 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:

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
Requiring invasive ventilation

Study Locations

Canada
Novartis Investigative Site
Recruiting
Montreal, H4A 3J1
Quebec
Canada
Netherlands
Novartis Investigative Site
Recruiting
Utrecht, 3584CX
-
Netherlands

Contacts

Name: 
Novartis Gene Therapies Medical Information (US, Asia-Pacific, Latin America, and Canada)
Name: 
Novartis Gene Therapies EMEA Medical Information (Europe, Middle East and Africa)

Have a question?

Call 1-888-669-6682 or email [email protected]