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Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

NCT02913482 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 62

Last updated 2024-08-07

Study results available
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Summary

Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.

Conditions

  • Muscular Atrophy, Spinal

Interventions

DRUG

Risdiplam

Risdiplam will be administered orally.

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Month
Max Age
7 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-12-23
Primary Completion
2019-11-14
Completion
2023-12-22
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Brazil
  • China
  • Croatia
  • France
  • Italy
  • Japan
  • Poland
  • Russia
  • Saudi Arabia
  • Serbia
  • Spain
  • Switzerland
  • Turkey (Türkiye)
  • Ukraine

Study Locations

More Related Trials

Entities

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 NCT02913482 on ClinicalTrials.gov