Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
Stopped early · Phase 2 · Has a placebo group
Conditions studied: Methylmalonic Acidemia, Propionic Acidemia
In brief
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.
Key facts
- Study ID
- NCT04732429
- Run by
- HemoShear Therapeutics
- People needed
- 26
- Starts
- 2021-03-15
- Expected to finish
- 2023-10-20
- Last updated by the study team
- 2024-01-05
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- Rady Children's Hospital — San Diego, California, United States
- Yale — New Haven, Connecticut, United States
- Children's National Health System — Washington D.C., District of Columbia, United States
- Emory University School of Medicine — Atlanta, Georgia, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- University of Minnesota — Minneapolis, Minnesota, United States
- Children's Mercy Hospital Kansas City — Kansas City, Missouri, United States
- University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
- Nationwide Children's Hospital — Columbus, Ohio, United States
- University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Vanderbilt University Medical Center — Nashville, Tennessee, United States
- John P. and Kathrine G. McGovern Medical School — Houston, Texas, United States
- University of Utah Hospital — Salt Lake City, Utah, United States
- Royal Children's Hospital Melbourne — Parkville, Victoria, Australia
- King Faisal Specialist Hospital and Research Centre — Riyadh, Riyadh Region, Saudi Arabia
Full record on ClinicalTrials.gov
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