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A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

NCT02908685 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 231

Last updated 2024-04-24

Study results available
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Summary

Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part (Part 2) of Risdiplam for 24 months.

Conditions

  • Muscular Atrophy, Spinal

Interventions

DRUG

Placebo

Placebo will be administered orally (via mouth) or through a feeding tube (naso-gastric or gastrostomy tube).

DRUG

Risdiplam

Risdiplam will be administered orally (via mouth) or through a feeding tube (naso-gastric or gastrostomy tube).

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-10-19
Primary Completion
2019-09-06
Completion
2023-10-02

Countries

  • United States
  • Belgium
  • Brazil
  • Canada
  • China
  • Croatia
  • France
  • Germany
  • Italy
  • Japan
  • Poland
  • Russia
  • Serbia
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02908685 on ClinicalTrials.gov