Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
NCT ID: NCT04732429
Last Updated: 2024-01-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE2
26 participants
INTERVENTIONAL
2021-03-15
2023-10-20
Brief Summary
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* 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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
QUADRUPLE
Study Groups
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Active Drug
Part B is the 6-month, randomized, double-blind (Subject/Investigator/Sponsor), placebo-controlled, 2-period crossover study consisting of 2 intervention periods of 12 weeks each to evaluate the safety and efficacy of the optimal dose of HST5040 in PA and MMA subjects ≥ 2 years old (N = minimum 12) in addition to SoC determined in Part A (within-subject dose escalation).
HST5040
Liquid solution
Placebo
Placebo in addition to standard of care.
Placebo
Liquid solution
Interventions
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HST5040
Liquid solution
Placebo
Liquid solution
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
2 Years
ALL
No
Sponsors
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HemoShear Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Patrick Horn, MD PhD
Role: STUDY_CHAIR
HemoShear Therapeutics, Inc.
Locations
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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
Countries
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Other Identifiers
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HST20-CL01
Identifier Type: -
Identifier Source: org_study_id
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