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

Completed · Phase 2 · Has a placebo group

Conditions studied: Muscular Atrophy, Spinal

In brief

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.

Key facts

Study ID

NCT02908685

Run by

Hoffmann-La Roche

People needed

231

Starts

2016-10-19

Expected to finish

2023-10-02

Last updated by the study team

2024-04-24

Who can join

Age: 2 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• 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
• Children's Hospital of Fudan University — Shanghai, China
• Clinical Medical Center Zagreb; University Hospital Rebro Department of Paediatrics — Zagreb, Croatia
• Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique ? L?ESCALE — Bron, France
• Hopital Roger Salengro — Lille, France
• CHU de Nantes - Hotel Dieu — Nantes, France
• Hôpital Necker-Enfants Malades; Service de neuropédiatrie — Paris, France
• Hopital Armand Trousseau — Paris, France
• Universitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen — Freiburg im Breisgau, Germany
• IRCCS Ospedale Pediatrico Bambino Gesù; U.O. Malattie Neuromuscolari e Neurodegenerative — Rome, Lazio, Italy
• Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile — Rome, Lazio, Italy
• IRCCS Istituto Giannina Gaslini; U.O.S.D. Centro di Miologia e Patologie Neurodegenerative — Genoa, Liguria, Italy
• Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Unità Operativa Complessa di Neurologia — Milan, Lombardy, Italy
• Fondazione IRCCS Istituto Neurologico ?Carlo Besta?; UO di Neurologia dello Sviluppo — Milan, Lombardy, Italy
• Fukuoka Children's Hospital — Fukuoka, Japan
• Hiroshima University Hospital — Hiroshima, Japan

Full record on ClinicalTrials.gov

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