Condition: Methylmalonic Acidemia · Propionic Acidemia · Sponsor: HemoShear Therapeutics
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.
This description comes directly from the study's public registry record.
Contact details are listed per location below or on the registry record.
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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 | — |
+ 4 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT04732429