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

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