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Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

NCT04732429 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2024-01-05

No results posted yet for this study

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:

* 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.

Conditions

Interventions

DRUG

HST5040

Liquid solution

DRUG

Placebo

Liquid solution

Sponsors & Collaborators

  • HemoShear Therapeutics

    lead INDUSTRY

Principal Investigators

  • Patrick Horn, MD PhD · HemoShear Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-15
Primary Completion
2023-10-20
Completion
2023-10-20
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Saudi Arabia

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04732429 on ClinicalTrials.gov