A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy … (NCT02908685) | Clinical Trial Compass
CompletedPhase 2
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
United States, Belgium231 participantsStarted 2016-10-19
Plain-language 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.
Who can participate
Age range2 Years – 25 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 examina…
What they're measuring
1
Part 1: Selected Part 2 Dose of Risdiplam for Participants With a Body Weight (BW) of >/=20kg
Timeframe: Day 1 up to at least 4 weeks on study (Up to CCOD of 25 July 2017)
2
Part 1: Selected Part 2 Dose of Risdiplam for Participants With BW of <20kg
Timeframe: Day 1 up to at least 4 weeks on study (Up to CCOD of 25 July 2017)
3
Part 2: Change From Baseline in the Total Motor Function Measure 32 (MFM-32) Total Score at Month 12