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

Stopped early · Phase 1

Conditions studied: Spinal Muscular Atrophy

In brief

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.

Key facts

Study ID

NCT03381729

Run by

Novartis Gene Therapies

People needed

32

Starts

2017-12-14

Expected to finish

2021-11-18

Last updated by the study team

2026-01-26

Who can join

Age: 1 and older, up to 5. Sex: any. Healthy volunteers: not accepted.

Where it is running

• UCLA — Los Angeles, California, United States
• Stanford University — Stanford, California, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Ann & Robert H. Lurie Children's Hospital — Chicago, Illinois, United States
• Johns Hopkins — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Washington University — St Louis, Missouri, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• UT Southwestern — Dallas, Texas, United States
• University of Utah — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.