Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
Status:
Recruiting
Trial end date:
2026-01-31
Target enrollment:
Participant gender:
Summary
This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12
subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA). The study
consists of 3 parts:
- Part A: Open-label, within-subject, dose escalation study in PA and MMA subjects ≥ 2
years old to identify a safe and pharmacologically active (optimal) dose of HST5040 for
use in Part B. Subjects will continue in a Part A open-label extension until all
subjects complete Part A and the optimal dose of HST5040 is identified for use in Part
B.
- Part B: 6-month, randomized, double-blind, placebo-controlled, 2-period crossover in the
same subjects from Part A to evaluate safety and efficacy of the optimal dose of HST5040
in addition to standard of care (SoC).
- Part C: open-label long-term extension study in PA and MMA subjects ≥ 2 years old (N =
approximately 12, 6 each) to evaluate the long-term safety and efficacy of the optimal
dose of HST5040.
This study will determine whether HST5040 can improve levels of disease-associated toxins
that accumulate in patients with PA and MMA.