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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 : Enrolling by invitation
First Posted : February 5, 2018
Last Update Posted : February 7, 2019
Sponsor:
Information provided by (Responsible Party):
AveXis, Inc.

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 AVXS 101. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.

Condition or disease Intervention/treatment
Spinal Muscular Atrophy 1 Biological: AVXS-101

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 AVXS 101. 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.


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Study Type : Observational
Estimated Enrollment : 15 participants
Observational Model: Other
Time Perspective: Prospective
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 : August 15, 2017
Estimated Primary Completion Date : December 2033
Estimated Study Completion Date : December 2033



Intervention Details:
  • Biological: AVXS-101
    Patients received treatment with AVXS-101 in the parent study, AVXS-101-CL-101


Primary Outcome Measures :
  1. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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 AVXS 101 will roll over from the previous study into the AVXS-101-LT-001 study for continuous safety monitoring for up to 15 years.
Criteria

Inclusion Criteria:

  1. Patient who received AVXS-101 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.


Information from the National Library of Medicine

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


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
AveXis, Inc.

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Responsible Party: AveXis, Inc.
ClinicalTrials.gov Identifier: NCT03421977     History of Changes
Other Study ID Numbers: AVXS-101-LT-001
First Posted: February 5, 2018    Key Record Dates
Last Update Posted: February 7, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Additional relevant MeSH terms:
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Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Spinal Muscular Atrophies of Childhood
Spinal Cord Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn