Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia (HERO)
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- STATUS
- Recruiting
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- End date
- Jan 31, 2026
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- participants needed
- 12
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- sponsor
- HemoShear Therapeutics
Sara Elsbecker, MS, RN, CPNP
Primary Contact
University of Minnesota (1.9 mi away) Contact
+10 other locationSummary
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.
Details
| Condition | Methylmalonic Acidemia, Propionic Acidemia |
|---|---|
| Treatment | Placebo, HST5040 |
| Clinical Study Identifier | NCT04732429 |
| Sponsor | HemoShear Therapeutics |
| Last Modified on | 7 October 2022 |
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