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

A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Spinal Muscular Atrophy 1
• Intervention / Treatment: Biological: Onasemnogene Abeparvovec-xioi

Detailed Description

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. The last visit of the parent study or early discontinuation from the parent study may serve as the visit at which the informed consent form process is conducted for the AVXS 101-LT-001 long term follow-up safety study. Patients will return annually for follow up study visits for five (5) years, and then will be contacted via phone annually for ten (10) years. Additionally, patient record transfers from their local physician and/or neurologist will be requested in conjunction with the annual study visits and phone contacts for review by the investigator.

If the patient is unable to return to the original investigative site, the sponsor will arrange with the patients' local established physician to serve as an additional investigator to conduct the required assessments.

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

• Study Type: Observational
• Enrollment (Actual): 13

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Novartis Gene Therapies

Publications and helpful links

General Publications

• 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. Erratum In: Drug Saf. 2022 Feb;45(2):191-192.
• 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.

Study record dates

Study Major Dates

• Study Start (Actual): September 21, 2017
• Primary Completion (Estimated): December 2, 2030
• Study Completion (Estimated): December 2, 2030

Study Registration Dates

• First Submitted: January 30, 2018
• First Submitted That Met QC Criteria: February 2, 2018
• First Posted (Actual): February 5, 2018

Study Record Updates

• Last Update Posted (Actual): February 29, 2024
• Last Update Submitted That Met QC Criteria: February 27, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Motor Neuron Disease; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood
• Other Study ID Numbers: AVXS-101-LT-001; COAV101A12401 (Other Identifier: Novartis Pharmaceuticals)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No