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Registry for Patients with Spinal Muscular Atrophy (SMA)

This study is a registry for people with Spinal Muscular Atrophy (SMA), a genetic disorder that affects motor neurons. It aims to understand the long-term health outcomes of patients and to see how safe and effective the treatment Zolgensma (OAV-101) is over time. You can join if you have a confirmed diagnosis of SMA and have been treated with Zolgensma. The study will look at things like survival rates and changes in motor skills using tests like CHOP-INTEND and HINE, over a period of up to 15 years. The study is currently unclear on its recruitment status, but plans to enroll 700 participants.

Study design
This is an observational study, meaning participants will receive their usual medical care, and the study will collect information about their health over time. It plans to include 700 participants.
What's involved
You will be managed according to your usual clinical care, and no extra visits or tests beyond your normal medical practice are required for this study. Information will be collected at baseline, every 6 months for the first 2 years, and then annually for up to 15 years.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 15 years from when they join the study, or until death, whichever comes first.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04174157

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

Recruiting
Not specifiedAll AgesObservational
Novartis Pharmaceuticals
~700 participants
Updated 2025-01-31 on ClinicalTrials.gov
What's tested:Prospective observational registryZolgensma

At a glance

Recruiting sites
72 of 99 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change in probability of survival of all patients with SMA using Kaplan Meier method to estimate
Measured over Based on information collected at Baseline and every 6 months through 2 years of follow-up, then annually through 15 years of follow up.
+7 more outcomes measured

Conditions

Spinal Muscular Atrophy (SMA)

Explore related trials

Spinal Muscular AtrophySMN1 Gene

Where it's being run

99 sites across 52 states
California7
South Korea7
Texas5
Portugal5
Taiwan5
Florida4
Ohio4
Israel4

Study leadership

  • Novartis Pharmaceuticals · STUDY_DIRECTOR · Novartis Pharmaceuticals

Who to contact

Novartis Gene Therapies
[email protected]
1-888-669-6682
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

What this trial measures

  • Change in probability of survival of all patients with SMA using Kaplan Meier method to estimateBased on information collected at Baseline and every 6 months through 2 years of follow-up, then annually through 15 years of follow up.
  • Change from baseline Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) in infants with pre-symptomatic or type I SMABaseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up

    CHOP INTEND score ranges from 0 to 64 with higher scores indicating higher motor function

  • Change from baseline Hammersmith Infant Neurological Examination (HINE) in infants with pre-symptomatic, type I or type II SMABaseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up

    HINE score range from 0 to 26 with higher scores indicating more development.

  • Change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) for patients with type II and III SMABaseline and every 6months through 2 years of follow up, then annually through 15 years of follow up

    HFMSE score range from 0 to 66 with the higher scores indicating more development.

  • Incidence of treatment emergent adverse eventsThrough 15 years of follow up
  • Incidence of treatment emergent serious adverse eventsThrough 15 years of follow up
  • Incidence of treatment emergent adverse events related to therapyThrough 15 years of follow up
  • Incidence of treatment emergent thrombocytopenia, hepatotoxicity and cardiac adverse eventsThrough 15 years of follow up