Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Study Identifier:
AVXS-101-RG-001
ClinicalTrials.gov Identifier:
NCT04174157
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
AveXis, Inc.
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Zolgensma
Date
Sep 2018 - Jun 2038
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Patients with SMA, genetically confirmed on or after 24 May 2018.
  • Appropriate consent/assent has been obtained for participation in the registry
Exclusion Criteria
  • Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA.
  • Note: Patients that are participating in a Compassionate Use Program (CUP) for AVXS-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of genetic confirmation of SMA.

Protocol Summary

Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases.

Until recently, the mainstay of treatment for these patients was supportive medical care. However, advances in medical treatment focusing on gene replacement, gene enhancement, motor neuron protection and muscle enhancement is likely to change the management and prognosis of these patients in the future.

The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options.

Trial Locations

Location
Status
Location
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Status
Recruiting
Location
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Status
Recruiting
Location
Loma Linda University Health
Loma Linda, California, United States, 92350
Status
Recruiting
Location
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Status
Recruiting
Location
University of California Los Angeles Health
Los Angeles, California, United States, 90095
Status
Recruiting
Location
Valley Children's Healthcare
Madera, California, United States, 93636
Status
Recruiting
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