Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT05115110 · ClinicalTrials.gov registry record · Phase 2

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

A Phase 2 study of Spinal Muscular Atrophy (SMA), sponsored by Hoffmann-La Roche.

Active
Registry status
Phase 2
Development phase
259
Enrollment target
20
Study locations

NCT05115110 is a Phase 2 study of Spinal Muscular Atrophy (SMA) that is active but no longer recruiting, run by Hoffmann-La Roche. The registered enrollment target is 259 participants, above the 231-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (12% higher). The trial reports 20 study locations across 13 states.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT05115110, a Phase 2 study of Spinal Muscular Atrophy (SMA), is active but no longer recruiting, sponsored by Hoffmann-La Roche.

ACTIVE NOT RECRUITING
Registry status
Phase 2
Development phase
259 participants
Enrollment target
20
Study locations

Study Summary

Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and clinical outcomes for people living with SMA. This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.

Conditions Studied

Spinal Muscular Atrophy (SMA)

Interventions

  • DRUG Placebo
  • DRUG Risdiplam
  • DRUG RO7204239

Study Locations (20)

Other

  • UZ Gent - Ghent
  • Chr de La Citadelle - Liège
  • Clinical Hospital Centre Zagreb - Zagreb
  • Kobe University Hospital - Hyōgo
  • Kagoshima University Hospital - Kagoshima
  • National Center for Global Health and Medicine - Tokyo

Lazio

  • Ospedale Pediatrico Bambino Gesù - Rome
  • Policlinico Agostino Gemelli - Rome

Lombardy

  • Fondazione IRCCS Istituto Neurologico ?Carlo Besta? - Milan
  • Asst Grande Ospedale Metropolitano Niguarda - Milan

Florida

  • Nemours Children's Hospital - Orlando

Massachusetts

  • Boston Childrens Hospital - Boston

New York

  • Columbia University Medical Center - New York

Texas

  • Neurology & Neuromuscular Care Center - Flower Mound

New South Wales

  • Sydney Children's Hospital - Randwick

Trial Details

FieldValue
Enrollment Target 259 participants
Start Date 2022-06-02
Est. Completion 2029-02-27
Phase Phase 2

Sponsor

Hoffmann-La Roche

909 total trials

Interested in This Trial?

Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT05115110

The ClinicalTrials.gov registry entry for NCT05115110 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 259 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 231-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (12% higher). The listed sponsor is Hoffmann-La Roche, which has 909 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Spinal Muscular Atrophy (SMA) appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.

NCT05115110 reports 20 study locations spanning 13 distinct geographic areas - top geographies include Other, Lazio, Lombardy.

Frequently Asked Questions

What is clinical trial NCT05115110 about?

NCT05115110 is a clinical study titled "A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy". Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an i...

What is the current status of trial NCT05115110?

This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 259 participants. The study started on 2022-06-02. Estimated completion is 2029-02-27.

What conditions does trial NCT05115110 study?

This clinical trial studies the following conditions: Spinal Muscular Atrophy (SMA).

What interventions are being tested in trial NCT05115110?

The interventions under investigation include: Placebo (DRUG), Risdiplam (DRUG), RO7204239 (DRUG).

Who is sponsoring clinical trial NCT05115110?

This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT05115110 being conducted?

This trial has 20 study locations across Florida, Massachusetts, New York, Texas, New South Wales. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Spinal Muscular Atrophy (SMA)

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Explore more on PlainTrial

Spinal Muscular Atrophy (SMA)

All registered trials for this condition

Florida

All trials with a study site in Florida

Recruiting now

Find trials open to new participants near you

Rankings

Top sponsors, conditions, and phases

Read our methodology - how this data is sourced, computed, and verified.

Related

Browse all → | Rankings →
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.