Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
Public ClinicalTrials.gov record NCT04732429. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04732429
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 26 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 2 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 15, 2021 (Actual)
- Primary completion
- Oct 20, 2023 (Actual)
- Completion
- Oct 20, 2023 (Actual)
- Last update posted
- Jan 5, 2024
2021 – 2023
United States locations
- U.S. sites
- 14
- U.S. states
- 13
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Rady Children's Hospital | San Diego | California | 92123 | — |
| Yale | New Haven | Connecticut | 06520 | — |
| Children's National Health System | Washington D.C. | District of Columbia | 20010 | — |
| Emory University School of Medicine | Atlanta | Georgia | 30322 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Children's Mercy Hospital Kansas City | Kansas City | Missouri | 64108 | — |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| John P. and Kathrine G. McGovern Medical School | Houston | Texas | 77030 | — |
| University of Utah Hospital | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 2 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04732429, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 5, 2024 · Synced Oct 23, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04732429 live on ClinicalTrials.gov.