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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 : March 30, 2021
Sponsor:
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
Change History
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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
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
Removed Location Countries  
 
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