Randomized, placebo-controlled, double-blind trial of intrathecal (IT) OAV101 administration in patients with later onset Type 2 spinal muscular atrophy (SMA), to evaluate the efficacy and safety.

Phase 3

• Conditions: Health Condition 1: G121- Other inherited spinal muscular atrophy

• Registration Number: CTRI/2021/11/037956
• Lead Sponsor: ovartis Healthcare Pvt Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 22 January 2024

Recruitment & Eligibility

• Status: Closed to Recruitment of Participants
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

Diagnostic confirmation during screening period of SMA caused by biallelic SMN1 pathogenic variants affecting SMN1 and 2-4 copies of SMN2

- The patient must be treatment naive for all SMN dependent therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).

- = 2 years and < 18 years of age at time of screening

- Onset of clinical signs and symptoms at = 6 months of age

- Patient must have a complete HFMSE assessment, with available total score as administered by qualified clinical evaluator during the screening period for trial eligibility

- Able to sit independently at screening, but has never had the ability to walk independently.

--Definition of sitting independently: Child sits up straight with the head erect for at least 10 seconds without using arms or hands to balance body or support position (Wijnhoven et al 2004).

--Definition of walking independently: The child is able to balance the body and control forward stepping movements without assistance (Wijnhoven et al 2004).

Exclusion Criteria

- Anti-adeno-associated virus serotype 9 (AAV9) antibody titers > 1:50 as determined by enzyme-linked immunosorbent assay (ELISA) binding immunoassay. NOTE: A negative anti-AAV9 antibody titer is defined as = 1:50.

- Presence of the following:

. An active infectious process requiring systemic antiviral or antimicrobial therapy at any time between onset of screening and dosing of OAV101 or the sham procedure

· An active but untreated viral or bacterial infectious process at any time between onset of screening and dosing of OAV101 or the sham procedure

· Any febrile illness within two weeks prior to start of screening, during screening period or during baseline period up to OAV101 treatment or sham procedure

· Hepatic dysfunction (i.e., aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), > upper limit of normal (ULN) (Common Terminology Criteria for Adverse Events (CTCAE) grade1 or greater) at Screening Visit 1. NOTE: In the absence of other liver laboratory abnormalities, isolated AST elevation is not considered exclusionary

· Requiring invasive or awake noninvasive ventilation for > 6 hours during a 24-hour period, invasive or noninvasive ventilation for > 12 hours during a 24-hour period, or requiring tracheostomy during the 4 weeks prior to screening or baseline.

· Complications at screening, as determined by theInvestigator, that would interfere with motor efficacy assessments

. Body mass index (BMI) < 3rd percentile

Study & Design

• Study Type: Interventional
• Study Design: Not specified