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RO7204239 plus risdiplam in spinal muscular atrophy
jRCT2051230159Active, not recruiting
- Sponsor
- Chugai Pharmaceutical Co., Ltd.
- Start
- Apr 2024
- Modality
- Small Molecules, Monoclonal Antibodies → Antibody-Based Therapeutics
01
Overview
Randomised, double-blind trial in spinal muscular atrophy (SMA) comparing RO7204239 + risdiplam with Placebo + risdiplam → RO7204239 + risdiplam (open-label) (2 arms). Primary endpoints: Part 1 - Percentage of participants with adverse events (AEs), Part 1 - Incidence of relevant echocardiographic parameter z scores > 2 and 16 more. Started Apr 2024; status: active, not recruiting.
02
Drugs
emugrobartrisdiplam
03
Conditions
spinal muscular atrophy (SMA)
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