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