Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia (HERO)
- Conditions
- Methylmalonic Acidemia (MMA)Propionic Acidemia (PA)Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2023-000785-33-Outside-EU/EEA
- Lead Sponsor
- HemoShear Therapeutics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active
- Sex
- All
- Target Recruitment
- Not specified
* 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 8
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
* Moderate-to-severely impaired cardiac function with LVEF < 40% 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 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
* History of severe allergic or anaphylactic reactions to any of the components of HST5040.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method