NCT04732429

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: HemoShear Therapeutics · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Methylmalonic Acidemia
• Propionic Acidemia

Interventions

Type	Name	Description
DRUG	HST5040	Liquid solution
DRUG	Placebo	Liquid solution

Timeline

• Start date: 2021-03-15
• Primary completion: 2023-10-20
• Completion: 2023-10-20
• First posted: 2021-02-01
• Last updated: 2024-01-05

Locations

16 sites across 3 countries: United States, Australia, Saudi Arabia

Regulatory

• FDA-regulated drug study