Long-Term Follow-up Study for Patients From AVXS-101-CL-101 (START)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03421977|
Recruitment Status : Active, not recruiting
First Posted : February 5, 2018
Last Update Posted : November 15, 2021
|Condition or disease||Intervention/treatment|
|Spinal Muscular Atrophy 1||Biological: Onasemnogene Abeparvovec-xioi|
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|
|Actual Enrollment :||13 participants|
|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|
|Actual Study Start Date :||September 21, 2017|
|Estimated Primary Completion Date :||December 2033|
|Estimated Study Completion Date :||December 2033|
- Biological: Onasemnogene Abeparvovec-xioi
Patients received treatment with onasemnogene abeparvovec-xioi in the parent study, AVXS-101-CL-101Other Name: Zolgensma
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03421977
|United States, Ohio|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|