A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects with Propionic or Methylmalonic Acidemia Followed by a 6-Month, Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study

drug protocol number
HST20-CL01
drug sponsor
HemoShear Therapeutics,
drug study
ST5040 (2,2-dimethylbutanoic acid)
drug trial site
King Faisal Specialist Hospital and Research Center (Riyadh)
drug status
Terminated
drug phase