← Back to Search

Other

HST5040 for Methylmalonic and Propionic Acidemia (HERO Trial)

Phase 2

Waitlist Available

Research Sponsored by HemoShear Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

HERO Trial Summary

This trial is testing a new drug, HST5040, to see if it can improve levels of toxins that accumulate in patients with Methylmalonic Acidemia (MMA) or Propionic Acidemia (PA). The trial has three parts: (1) an initial safety and dose-finding phase in a small group of patients, (2) a larger placebo-controlled phase to test efficacy, and (3) a long-term extension phase.

Who is the study for?

This trial is for people over 2 years old with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA), who have high levels of certain toxins despite standard treatments. They must not have severe heart, kidney issues, a history of organ transplants, exposure to gene therapy for PA/MMA, or recent investigational therapies.Check my eligibility

What is being tested?

The study tests HST5040's safety and effectiveness in reducing disease-related toxins in patients with PA and MMA. It includes an initial dose-finding phase, followed by a double-blind comparison with placebo and then a long-term open-label extension to monitor ongoing effects.See study design

What are the potential side effects?

While the specific side effects are not listed here, participants will be monitored for any adverse reactions to HST5040 during the trial phases. This could include allergic reactions as individuals with known severe allergies to components of HST5040 are excluded from participation.

HERO Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in plasma 2-methylcitric acid (MCA) levels

Secondary outcome measures

Acute Metabolic Decompensations

Anion Gap

Change in 3-OH propionate

+10 more

HERO Trial Design

2Treatment groups

Experimental Treatment

Group I: PlaceboExperimental Treatment1 Intervention

Placebo in addition to standard of care.

Group II: Active DrugExperimental Treatment1 Intervention

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).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

HemoShear TherapeuticsLead Sponsor

1 Previous Clinical Trials

11 Total Patients Enrolled

Gerald F CoxStudy ChairHemoShear Therapeutics, Inc.

Patrick Horn, MD PhDStudy ChairHemoShear Therapeutics, Inc.

Media Library

HST5040 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04732429 — Phase 2

Methylmalonic Acidemia Research Study Groups: Active Drug, Placebo

Methylmalonic Acidemia Clinical Trial 2023: HST5040 Highlights & Side Effects. Trial Name: NCT04732429 — Phase 2

HST5040 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04732429 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many locations have access to this research study?

"This clinical trial is being held in three locations: Yale University (New Haven, Connecticut), the University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh (Pittsburgh, Pennsylvania) and Ann & Robert H. Lurie Children's Hospital of Chicago (Chicago Illinois). Additionally, there are 12 additional sites participating."

Answered by AI

What is the population size of this research investigation?

"In order to carry out the research, 12 suitable volunteers need to be recruited. The sponsor of this trial is HemoShear Therapeutics and will oversee it from multiple locations including Yale in New Haven, Connecticut and University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh in Pennsylvania."

Answered by AI

Is enrollment open for this experiment?

"Affirmative. According to the clinicaltrials.gov information, this trial is actively seeking enrolment with an initial posting date of March 15th 2021 and a last editing on September 6th 2022. 12 participants are being sought from 12 distinct locations."

Answered by AI

Has HST5040 been given the go-ahead by the Food and Drug Administration?

"Our internal risk assessment assigned HST5040 a value of 2, since there is some data confirming the drug's safety but no evidence for its efficacy yet."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

2021. "Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04732429.