A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Hoffmann-La Roche
Age
2–25
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible 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 be 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
