Skip to main content
TrialFinder
TrialFinder is for informational purposes only and does not provide medical advice. Always talk to your doctor about whether a trial is right for you.
RECRUITINGOBSERVATIONAL

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

A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 Who Should NOT Join This Trial: \- 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * 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 Exclusion Criteria: \- 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.

Treatments Being Tested

OTHER

Prospective observational registry

This prospective observational registry will assess long-term outcomes of patients with a diagnosis of SMA.

DRUG

Zolgensma

Zolgensma will be given to patients as per normal clinical practice and clinical care will not be mandated by the protocol. As such, the decision to prescribe Zolgensma is separate from the decision to include the patient in this study

Locations (20)

Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Loma Linda University Health
Loma Linda, California, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
University of California Los Angeles Health
Los Angeles, California, United States
Valley Children's Healthcare
Madera, California, United States
Children's Hospital of Orange County
Madera, California, United States
University of California Davis Health System
Sacramento, California, United States
Rady Children's Hospital San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Connecticut Children's Medical Center
Farmington, Connecticut, United States
Yale-New Haven Health System
New Haven, Connecticut, United States
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Adventist Health System - Florida
Orlando, Florida, United States
Jackson South Medical Center
Palmetto Bay, Florida, United States
Indiana University Health University Hospital (IUHUH)
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Louisville
Louisville, Kentucky, United States