A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- drug: Placebo
- drug: Risdiplam
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
Ages Eligible for Study
2 Years - 25 Years
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Not Recruiting
Our research team includes physicians, residents, medical students, research assistants, and volunteers. Our research topics include medical imaging, device validation, mobile application development, and pharmaceutical trials.
Some of the Neuro-Opthalmic concerns we investigate include Multiple Sclerosis, Optic Neuritis, IIH, and ICP.