Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
NCT04174157 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- OTHER Prospective observational registry
- DRUG Zolgensma
Study Locations (20)
California
- Loma Linda University Health — Loma Linda
- Children's Hospital of Los Angeles — Los Angeles
- University of California Los Angeles Health — Los Angeles
- Valley Children's Healthcare — Madera
- Children's Hospital of Orange County — Madera
- University of California Davis Health System — Sacramento
- Rady Children's Hospital San Diego — San Diego
Florida
- Golisano Children's Hospital of Southwest Florida — Fort Myers
- Nicklaus Children's Hospital — Miami
- Adventist Health System - Florida — Orlando
- Jackson South Medical Center — Palmetto Bay
Connecticut
- Connecticut Children's Medical Center — Farmington
- Yale-New Haven Health System — New Haven
Arizona
- Phoenix Children's Hospital — Phoenix
Arkansas
- Arkansas Children's Hospital — Little Rock
Colorado
- Children's Hospital Colorado — Aurora
Indiana
- Indiana University Health University Hospital (IUHUH) — Indianapolis
Iowa
- University of Iowa — Iowa City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 700 participants |
| Start Date | 2018-09-25 |
| Est. Completion | 2038-06-30 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04174157
The ClinicalTrials.gov registry entry for NCT04174157 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 700 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Novartis Pharmaceuticals, which has 792 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Spinal Muscular Atrophy (SMA) appearing as the primary indexed condition, and to 2 interventions — of which Prospective observational registry is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04174157 reports 20 study locations spanning 10 distinct geographic areas — top geographies include California, Florida, Connecticut. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04174157 about?
NCT04174157 is a clinical study titled "Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)". 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 ...
What is the current status of trial NCT04174157?
This trial is currently recruiting. The enrollment target is 700 participants. The study started on 2018-09-25. Estimated completion is 2038-06-30.
What conditions does trial NCT04174157 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy (SMA). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04174157?
The interventions under investigation include: Prospective observational registry (OTHER), Zolgensma (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04174157?
This trial is sponsored by Novartis Pharmaceuticals, which has 792 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04174157 being conducted?
This trial has 20 study locations across Arizona, Arkansas, California, Colorado, Connecticut. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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