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

NCT04174157 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 700

Last updated 2025-01-31

No results posted yet for this study

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.

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.

Conditions

  • Spinal Muscular Atrophy (SMA)

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-09-25
Primary Completion
2038-06-30
Completion
2038-06-30
FDA Drug
Yes

Countries

  • United States
  • Greece
  • Ireland
  • Israel
  • Japan
  • Poland
  • Portugal
  • Romania
  • Russia
  • South Korea
  • Taiwan

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04174157 on ClinicalTrials.gov