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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 | Clinical Trials | Clareo Health

COMPLETEDPHASE2

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

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

NCT02908685•Hoffmann-La Roche

View on ClinicalTrials.gov

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.

Eligibility

Age

2 - 25 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Confirmed diagnosis of 5q-autosomal recessive SMA
•Negative blood pregnancy test at screening and agreement to comply with measures to prevent pregnancy and restrictions on sperm donation
•For Part 1: Type 2 or 3 SMA ambulant or non-ambulant
•For Part 2: 1) Type 2 or 3 SMA non-ambulant; 2) RULM entry item A greater than or equal to 2; 3) ability to sit independently as assessed by item 9 of the MFM

Exclusion Criteria

•Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer
•Concomitant or previous administration of a SMN2-targeting antisense oligonucleotide, SMN2 splicing modifier or gene therapy either in a clinical study or as part of medical care
•Any history of cell therapy
•Hospitalization for a pulmonary event within the last 2 months or planned at time of screening
•Surgery for scoliosis or hip fixation in the one year preceding screening or planned within the next 18 months
•Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator
•Presence of clinically significant electrocardiogram abnormalities before study drug administration from average of triplicate measurement or cardiovascular disease indicating a safety risk for participants as determined by the Investigator
•Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration
•Recently initiated treatment (within less than \[\<\] 6 months prior to randomization) with oral salbutamol or another beta 2-adrenergic agonist taken orally
•Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine, is not allowed
+3 more criteria

Locations (45)

Stanford University Medical Center

Palo Alto, California, United States

Columbia University Medical Center; The Neurological Institute of New York

New York, New York, United States

UZ Gent

Ghent, Belgium

UZ Leuven Gasthuisberg

Leuven, Belgium

Chr de La Citadelle

Liège, Belgium

Instituto de Puericultura E Pediatria Martagão Gesteira

Rio de Janeiro, Rio de Janeiro, Brazil

Alberta Children's Hospital Division of Pediatric Neurology

Calgary, Alberta, Canada

London Health Sciences Centre; Children's Hospital; Pediatrics

London, Ontario, Canada

McGill University Health Centre - Glen Site

Montreal, Quebec, Canada

Peking University First Hospital

Beijing, China

Key Dates

Start Date

October 19, 2016

Primary Completion Date

September 6, 2019

Completion Date

October 2, 2023

Last Updated

April 24, 2024

Enrollment

231

ACTUAL participants

Conditions

Muscular Atrophy, Spinal

Interventions

Placebo

DRUG

Risdiplam

DRUG

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

NCT05861986

Muscular Atrophy, Spinal

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RECRUITINGPHASE2

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

NCT05808764

Muscular Atrophy, Spinal

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RECRUITING

Natural History of SMA

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 24, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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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

Inclusion Criteria

Exclusion Criteria

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Natural History of SMA

Adult Spinal Muscular Atrophy (SMA) China Registry

Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen

Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection