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Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

NCT03381729 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2026-01-26

Study results available
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Summary

The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.

Conditions

Interventions

BIOLOGICAL

Onasemnogene Abeparvovec-xioi

Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter

Sponsors & Collaborators

  • Novartis Gene Therapies

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
6 Months
Max Age
60 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-12-14
Primary Completion
2021-11-18
Completion
2021-11-18
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03381729 on ClinicalTrials.gov