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NCT04174157 · ClinicalTrials.gov registry record
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
A clinical trial of Spinal Muscular Atrophy (SMA), sponsored by Novartis Pharmaceuticals.
- Recruiting
- Registry status
- 700
- Enrollment target
- 20
- Study locations
NCT04174157: Recruiting study of Spinal Muscular Atrophy (SMA), sponsored by Novartis Pharmaceuticals.
NCT04174157 is a study of Spinal Muscular Atrophy (SMA) that is actively recruiting participants, run by Novartis Pharmaceuticals. The registered enrollment target is 700 participants, above the 143-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (390% higher). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04174157, a study of Spinal Muscular Atrophy (SMA), is actively recruiting participants, sponsored by Novartis Pharmaceuticals.
- RECRUITING
- Registry status
- 700 participants
- Enrollment target
- 20
- Study locations
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 |
What NCT04174157 shows while recruiting
NCT04174157 is an observational study that tracks outcomes without assigning an intervention. The registered 700 participants enrollment target is mid-sized for trials with a published cap, above the 143-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (390% higher).
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.
NCT04174157 names 20 study sites across 10 states, led by California, Florida, Connecticut.
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).
What interventions are being tested in trial NCT04174157?
The interventions under investigation include: Prospective observational registry (OTHER), Zolgensma (DRUG).
Who is sponsoring clinical trial NCT04174157?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
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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