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

Recruiting now

Conditions studied: Spinal Muscular Atrophy (SMA)

In brief

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. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.

Key facts

Study ID: NCT04174157
Run by: Novartis Pharmaceuticals
People needed: 700
Starts: 2018-09-25
Expected to finish: 2038-06-30
Last updated by the study team: 2025-01-31

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.
Appropriate consent/assent has been obtained for participation in the registry

You may not qualify if…

Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA.
Note: Patients who are participating in a Compassionate Use Program (CUP) for OAV-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 a genetic or clinical diagnosis of SMA.

Where it is running

Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
Duke Health — Durham, North Carolina, United States (enrolling)
Children's Hospital of Los Angeles — Los Angeles, California, United States (enrolling)
University of California Los Angeles Health — Los Angeles, California, United States (enrolling)
Valley Children's Healthcare — Madera, California, United States (enrolling)
The State University of New York — Stony Brook, New York, United States (enrolling)
University of California Davis Health System — Sacramento, California, United States (enrolling)
Rady Children's Hospital San Diego — San Diego, California, United States (enrolling)
Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
Connecticut Children's Medical Center — Farmington, Connecticut, United States (enrolling)
Yale-New Haven Health System — New Haven, Connecticut, United States (enrolling)
Golisano Children's Hospital of Southwest Florida — Fort Myers, Florida, United States (enrolling)
Children's Hospital of Orange County — Madera, California, United States (enrolling)
Phoenix Children's Hospital — Phoenix, Arizona, United States (enrolling)
Jackson South Medical Center — Palmetto Bay, Florida, United States (enrolling)
Indiana University Health University Hospital (IUHUH) — Indianapolis, Indiana, United States (enrolling)
University of Iowa — Iowa City, Iowa, United States (enrolling)
University of Kansas Medical Center — Kansas City, Kansas, United States (enrolling)
University of Louisville — Louisville, Kentucky, United States (enrolling)
University of Minnesota — Minneapolis, Minnesota, United States (enrolling)
University of Missouri Health System — Columbia, Missouri, United States (enrolling)
Washington University School of Medicine in St. Louis — St Louis, Missouri, United States (enrolling)
Atlantic Health System — Morristown, New Jersey, United States (enrolling)
University of Rochester Medical Center — Rochester, New York, United States (enrolling)
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)

Full record on ClinicalTrials.gov

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