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NCT03032172 · ClinicalTrials.gov registry record · Phase 2

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

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

Completed
Registry status
Phase 2
Development phase
174
Enrollment target
20
Study locations

NCT03032172 is a Phase 2 study of Spinal Muscular Atrophy that has completed, run by Hoffmann-La Roche. The registered enrollment target is 174 participants, above the 101-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (72% higher). The trial reports 20 study locations across 9 states.

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The verdict

NCT03032172, a Phase 2 study of Spinal Muscular Atrophy, has completed, sponsored by Hoffmann-La Roche.

COMPLETED
Registry status
Phase 2
Development phase
174 participants
Enrollment target
20
Study locations

Study Summary

This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.

Conditions Studied

Spinal Muscular Atrophy

Interventions

  • DRUG Risdiplam

Study Locations (20)

Other

  • UZ Gent - Ghent
  • UZ Leuven Gasthuisberg - Leuven
  • Hopital Femme Mere Enfant - Bron
  • Hopital Roger Salengro - Lille
  • CHRU de Montpellier, Hopital Gui de Chauliac - Montpellier
  • Hôpital Necker-Enfants Malades - Paris
  • Hopital des Enfants - Toulouse
  • Universitätsklinikum Essen - Essen
  • Universitätsklinikum Freiburg - Freiburg im Breisgau
  • UMC Utrecht - Utrecht
  • Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie - Warsaw

Lazio

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

California

  • Stanford University Medical Center - Palo Alto

Florida

  • Nemours Children's Hospital - Orlando

Massachusetts

  • Boston Childrens Hospital - Boston

New York

  • Columbia University Medical Center - New York

Liguria

  • IRCCS Istituto Giannina Gaslini - Genoa

Lombardy

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Milan

Trial Details

FieldValue
Enrollment Target 174 participants
Start Date 2017-03-03
Est. Completion 2025-02-07
Phase Phase 2

Sponsor

Hoffmann-La Roche

909 total trials

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What the Registry Record Tells You About NCT03032172

The ClinicalTrials.gov registry entry for NCT03032172 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 174 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 101-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (72% 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 appearing as the primary indexed condition, and to 1 intervention - of which Risdiplam is the first listed.

NCT03032172 reports 20 study locations spanning 9 distinct geographic areas - top geographies include Other, Lazio, California.

Frequently Asked Questions

What is clinical trial NCT03032172 about?

NCT03032172 is a clinical study titled "A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy". This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO...

What is the current status of trial NCT03032172?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 174 participants. The study started on 2017-03-03. Estimated completion is 2025-02-07.

What conditions does trial NCT03032172 study?

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

What interventions are being tested in trial NCT03032172?

The interventions under investigation include: Risdiplam (DRUG).

Who is sponsoring clinical trial NCT03032172?

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

Where is trial NCT03032172 being conducted?

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

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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.