Status:
TERMINATED
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
Lead Sponsor:
HemoShear Therapeutics
Conditions:
Methylmalonic Acidemia
Propionic Acidemia
Eligibility:
All Genders
2+ years
Phase:
PHASE2
Brief 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...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 15 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 20 2023
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT04732429
Start Date
March 15 2021
End Date
October 20 2023
Last Update
January 5 2024
Active Locations (16)
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1
Rady Children's Hospital
San Diego, California, United States, 92123
2
Yale
New Haven, Connecticut, United States, 06520
3
Children's National Health System
Washington D.C., District of Columbia, United States, 20010
4
Emory University School of Medicine
Atlanta, Georgia, United States, 30322