TerminatedPhase 2

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

Stopped: The decision to terminate the study was based on business considerations by the Sponsor.

United States26 participantsStarted 2021-03-15

Plain-language 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.

Who can participate

Age range2 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Confirmed diagnosis of symptomatic PA or MMA (Mutase) * Ages ≥ 2 years old. * History of Inadequate metabolic control while receiving standard of care (SoC). * Plasma MCA concentration \> 3x upper limit of normal of the reference range at screening. * Stable supplementation dose of carnitine for at least 1 week prior to the entry in the study. Exclusion Criteria: * Moderate-to-severely impaired cardiac function with LVEF \< 45% by ECHO. * Clinically significant arrhythmia by Holter monitor. * QTcF \> 450 msec * Moderate to severe chronic kidney disease with estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2. * Exposure to any investigational therapy, apart for a COVID-19 vaccine, within the past 6 months prior to study entry. * Exposure to gene therapy for PA or MMA at any time prior to study entry. * History of organ transplantation (Part A and B only) * History of severe allergic or anaphylactic reactions to any of the components of HST5040.

What they're measuring

Change in plasma 2-methylcitric acid (MCA) levels

Timeframe: 6 months

Trial details

NCT IDNCT04732429

SponsorHemoShear Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-10-20