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

Study Identifier:
BP39055
ClinicalTrials.gov Identifier:
NCT02908685
EudraCT Identifier:
2016-000750-35
EU CT Number:
N/A
Sponsor:
Hoffmann-La Roche
Study Complete

Trial Results

Study Protocol
Available Languages: English
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Statistical Analysis Plan
Available Languages: English
View PDF

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Placebo
  • Drug: Risdiplam
Date
Oct 2016 - Sep 2019
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 2 - 25 Years
Requirements Information
Inclusion and Exclusion Criteria
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
  • 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

Protocol 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.

Trial Locations

Location
Status
Location
Stanford University Medical Center
Palo Alto, California, United States, 94304
Status
N/A
Location
Columbia University Medical Center; The Neurological Institute of New York
New York, New York, United States, 10032
Status
N/A
Location
UZ Gent
Ghent, Belgium, 9000
Status
N/A
Location
UZ Leuven Gasthuisberg
Leuven, Belgium, 3000
Status
N/A
Location
Chr de La Citadelle
Liège, Belgium, 4000
Status
N/A
Location
Instituto de Puericultura E Pediatria Martagão Gesteira
Rio de Janeiro, Rio de Janeiro, Brazil, CEP 21941-912
Status
N/A
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