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

• ClinicalTrials.gov Identifier: NCT03421977
• Recruitment Status: Active, not recruiting
• First Posted: February 5, 2018
• Last Update Posted: March 30, 2021
• Sponsor: Novartis Gene Therapies
• Information provided by (Responsible Party): Novartis ( Novartis Gene Therapies )

Tracking Information

• First Submitted Date: January 30, 2018
• First Posted Date: February 5, 2018
• Last Update Posted Date: March 30, 2021
• Actual Study Start Date: September 21, 2017
• Estimated Primary Completion Date: December 2033 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 13, 2019)

Long-Term Safety [ Time Frame: 15 years ]

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

Original Primary Outcome Measures (submitted: February 2, 2018)

Long-Term Safety [ Time Frame: 15 years ]

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

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Long-Term Follow-up Study for Patients From AVXS-101-CL-101
• Official Title: 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
• 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.

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.

• Study Type: Observational
• Study Design: Observational Model: Other; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.
• Condition: Spinal Muscular Atrophy 1

Intervention

Biological: Onasemnogene Abeparvovec-xioi

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

• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: June 10, 2020): 13
• Original Estimated Enrollment (submitted: February 2, 2018): 15
• Estimated Study Completion Date: December 2033
• Estimated Primary Completion Date: December 2033 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03421977
• Other Study ID Numbers: AVXS-101-LT-001
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Novartis ( Novartis Gene Therapies )
• Study Sponsor: Novartis Gene Therapies
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Novartis
• Verification Date: March 2021