RECRUITING

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.

Official Title

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials

Quick Facts

Study Start:2022-12-19
Study Completion:2030-06-26
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05335876

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:0 Years to 100 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Participated in an OAV101 clinical trial.
  2. 2. Written informed consent must be obtained before any assessment is performed.
  3. 3. Patient/Parent/legal guardian willing and able to comply with study procedures.
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Novartis Pharmaceuticals
CONTACT
1-888-669-6682
medinfo.gtx@novartis.com
Novartis Pharmaceuticals
CONTACT
+41613241111
medinfoemea.gtx@novartis.com

Study Locations (Sites)

Child Hosp Of The Kings Daughters
Norfolk, Virginia, 23507
United States

Collaborators and Investigators

Sponsor: Novartis Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-12-19
Study Completion Date2030-06-26

Study Record Updates

Study Start Date2022-12-19
Study Completion Date2030-06-26

Terms related to this study

Keywords Provided by Researchers

  • Zolgensma
  • OAV101
  • AVXS 101
  • gene therapy
  • Muscle atrophy
  • SBMA
  • spinal and bulbar muscular atrophy
  • spinal muscular atrophy
  • bulbar muscular atrophy
  • muscle function
  • myopathy
  • muscle wasting
  • atrophied muscle
  • loss of muscle strength
  • Spinal Muscular Atrophy (SMA)
  • survival motor neuron 1 gene (SMN1)
  • SMN protein depletion
  • survival motor neuron 2 gene (SMN2)
  • chromosome 5q13
  • neurogenetic disorder
  • onasemnogene abeparvovec

Additional Relevant MeSH Terms

  • Spinal Muscular Atrophy (SMA)