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NCT04042025 · ClinicalTrials.gov registry record · Phase 3

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

A Phase 3 study of SMA and Spinal Muscular Atrophy Type II, sponsored by Novartis Gene Therapies.

Active
Registry status
Phase 3
Development phase
85
Enrollment target
20
Study locations

NCT04042025 is a Phase 3 study of SMA and Spinal Muscular Atrophy Type II that is active but no longer recruiting, run by Novartis Gene Therapies. The registered enrollment target is 85 participants, below the 135-participant average among 6 other SMA trials with a reported enrollment target (37% lower). The trial reports 20 study locations across 17 states.

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The verdict

NCT04042025, a Phase 3 study of SMA and Spinal Muscular Atrophy Type II, is active but no longer recruiting, sponsored by Novartis Gene Therapies.

ACTIVE NOT RECRUITING
Registry status
Phase 3
Development phase
85 participants
Enrollment target
20
Study locations

Study Summary

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

Conditions Studied

SMA Spinal Muscular Atrophy Type II Spinal Muscular Atrophy Type I Spinal Muscular Atrophy Type III

Interventions

  • BIOLOGICAL Onasemnogene Abeparvovec-xioi

Study Locations (20)

Massachusetts

  • Massachusetts General Hospital - Boston
  • Boston Children's Hospital - Boston

Pennsylvania

  • Children's Hospital of Philadelphia - Philadelphia
  • Clinic for Special Children - Strasburg

Virginia

  • Children's Hospital of The King's Daughters - Norfolk
  • Virginia Commonwealth University - Richmond

California

  • Stanford University Medical Center - Palo Alto

Colorado

  • Children's Hospital Colorado - Aurora

Illinois

  • Ann Robert H. Lurie Children's Hospital of Chicago - Chicago

Maryland

  • John Hopkins Hospital - David M. Rubenstein Child Health Building - Baltimore

Michigan

  • Spectrum Health Hospitals Helen DeVos Children's Hospital - Grand Rapids

Trial Details

FieldValue
Enrollment Target 85 participants
Start Date 2020-02-10
Est. Completion 2035-12-31
Phase Phase 3

Sponsor

Novartis Gene Therapies

6 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04042025

The ClinicalTrials.gov registry entry for NCT04042025 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 85 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 135-participant average among 6 other SMA trials with a reported enrollment target (37% lower). The listed sponsor is Novartis Gene Therapies, which has 6 total studies on file at ClinicalTrials.gov.

The record links to 4 conditions, with SMA appearing as the primary indexed condition, and to 1 intervention - of which Onasemnogene Abeparvovec-xioi is the first listed.

NCT04042025 reports 20 study locations spanning 17 distinct geographic areas - top geographies include Massachusetts, Pennsylvania, Virginia.

Frequently Asked Questions

What is clinical trial NCT04042025 about?

NCT04042025 is a clinical study titled "Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi". This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitor...

What is the current status of trial NCT04042025?

This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 85 participants. The study started on 2020-02-10. Estimated completion is 2035-12-31.

What conditions does trial NCT04042025 study?

This clinical trial studies the following conditions: SMA, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type III.

What interventions are being tested in trial NCT04042025?

The interventions under investigation include: Onasemnogene Abeparvovec-xioi (BIOLOGICAL).

Who is sponsoring clinical trial NCT04042025?

This trial is sponsored by Novartis Gene Therapies, which has 6 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04042025 being conducted?

This trial has 20 study locations across California, Colorado, Illinois, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.