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

Trial ID or NCT#



not recruiting iconNOT RECRUITING


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.

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

Eligibility Criteria

Ages Eligible for Study: 2 Years to 25 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - 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:
  1. - 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 - Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to Risdiplam or to the constituents of its formulation - Recent history (less than one year) of ophthalmological diseases - Participants requiring invasive ventilation or tracheostomy


John W. Day, MD, PhD
John W. Day, MD, PhD
Neuromuscular neurologist, Neurophysiologist
Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology