Long-Term Follow-up Study for Patients From AVXS-101-CL-101

NCT03421977 | Active Not Recruiting DMC FDA Drug

• 2 other identifiers: AVXS-101-LT-001COAV101A12401
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.

Conditions

Spinal Muscular Atrophy 1

Outcome Measures

Primary Outcomes (1)

• Long-Term Safety

  The primary objective is to collect long term safety data of patients with SMA Type 1 who were treated with onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial by assessing incidence of SAEs and Adverse Events of Special Interest

  15 years

Interventions

Onasemnogene Abeparvovec-xioi BIOLOGICAL

Patients received treatment with onasemnogene abeparvovec-xioi in the parent study, AVXS-101-CL-101

Also known as: Zolgensma

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi will roll over from the previous study into the AVXS-101-LT-001 study for continuous safety monitoring for up to 15 years.

You may qualify if:

• Patient who received onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1.
• Parent/legal guardian willing and able to complete the informed consent process, comply with study procedures and visit schedule.

You may not qualify if:

• \. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.

Sponsors & Collaborators

• Novartis Gene Therapies: lead

Study Sites (1)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Related Publications (2)

• Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12.

  PMID: 34383289 DERIVED

• Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 2021 Jul 1;78(7):834-841. doi: 10.1001/jamaneurol.2021.1272.

  PMID: 33999158 DERIVED

MeSH Terms

Conditions

Spinal Muscular Atrophies of Childhood

Interventions

Zolgensma

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 30, 2018
• First Posted: February 5, 2018
• Study Start: September 21, 2017
• Primary Completion (Estimated): December 2, 2030
• Study Completion (Estimated): December 2, 2030
• Last Updated: April 25, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)