©2024 Stanford Medicine
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Not Recruiting
Trial ID: NCT02908685
Purpose
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
Stanford Investigator(s)
John W. Day, MD, PhD
Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology
Eligibility
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
Intervention(s):
drug: Placebo
drug: Risdiplam
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305