Clinical Trials Logo
  1. Home
  2. Muscular Atrophy, Spinal
  3. NCT02908685

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

Muscular Atrophy, Spinal Clinical Trial

Official title:
A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02908685
Study type Interventional
Source Hoffmann-La Roche
Contact
Status Completed
Phase Phase 2
Start date October 19, 2016
Completion date October 2, 2023
View Details
See also
  Status Clinical Trial Phase
Completed NCT00533221 - Pilot Study of Growth Hormon to Treat SMA Typ II and III Phase 2
Recruiting NCT05575011 - A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115 Phase 1
Completed NCT05073133 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) Phase 4
Terminated NCT02240355 - A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) Phase 1
Recruiting NCT05042921 - Pediatric Spinal Muscular Atrophy (SMA) China Registry
Completed NCT04419233 - Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
Recruiting NCT05861986 - A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy Phase 4
Completed NCT00466349 - International SMA Patient Registry
Completed NCT03920865 - A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function Phase 1
Recruiting NCT05481164 - Newborn Screening for Spinal Muscular Atrophy
Completed NCT04089566 - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Phase 3
Recruiting NCT05861999 - A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy Phase 4
Recruiting NCT05755451 - Natural History of SMA
Active, not recruiting NCT01233817 - Progressive Strength Training in Spinal Muscular Atrophy N/A
Recruiting NCT04317794 - Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
Active, not recruiting NCT05618379 - Adult Spinal Muscular Atrophy (SMA) China Registry
Active, not recruiting NCT04488133 - A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec Phase 4
Completed NCT03781479 - Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients Phase 2
Completed NCT00568802 - A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients Phase 1/Phase 2
Completed NCT00568698 - A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients Phase 1/Phase 2