Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia (HERO)

  • STATUS
    Recruiting
  • End date
    Jan 31, 2026
  • participants needed
    12
  • sponsor
    HemoShear Therapeutics
Updated on 7 October 2022

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.

Details
Condition Methylmalonic Acidemia, Propionic Acidemia
Treatment Placebo, HST5040
Clinical Study IdentifierNCT04732429
SponsorHemoShear Therapeutics
Last Modified on7 October 2022

Eligibility

Yes No Not Sure

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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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