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HERO: HST5040 in Propionic or Methylmalonic Acidemia
NCT04732429Phase 2Terminated
- Sponsor
- HemoShear Therapeutics
- Start
- Mar 2021
- Primary completion
- Oct 2023
- Modality
- Small Molecules
- Countries
- 3
01
Overview
Phase 2, randomised, double-blind trial of 2,2-dimethylbutanoic acid in propionic acidemia (PA) and methylmalonic acidemia (MMA) (2 arms), sponsored by HemoShear Therapeutics. Primary endpoint: Change in plasma 2-methylcitric acid at 6 months. Started Mar 2021; status: terminated.
02
Drugs
2,2-dimethylbutanoic acid
03
Conditions
propionic acidemia (PA)methylmalonic acidemia (MMA)
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