Pegtibatinase for Classical Homocystinuria

Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

Recruiting Phase 1 Phase 2 Interventional Study
Homocystinuria
Prior Safety Data This treatment has already been tested in at least one earlier human trial.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
5 – 65
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
39 (estimated)
Sponsor
Travere Therapeutics, Inc. · Industry
Who this trial is looking for

This trial is looking for people with classical homocystinuria (HCU) to see if pegtibatinase can help reduce their homocysteine levels. Participants will receive different amounts of the drug while continuing their usual treatments and diets for HCU.

Are You a Good Fit for This Trial?
Rules you out Marfan syndromeMTHFR deficiencyCobalamin metabolism disorder

You may be able to join if

  • I am between 5 and 65 years old.
  • I have been diagnosed with classical homocystinuria (HCU).
  • My plasma total homocysteine level is at least 50 μM.
  • I can provide consent to join this study.
  • I am willing to stick to my usual HCU treatments and diet.

You may not be able to join if

  • I have a major thrombotic event in the last 6 months.
  • My body weight is less than 15 kg.
  • I have been treated with pegtibatinase before.
  • I have an active infection with HIV, hepatitis B, or hepatitis C.
  • I am pregnant or breastfeeding.
  • I have had a major surgery planned during the study.
  • I cannot comply with study procedures.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

Think this trial could be right for you?

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Check Your Eligibility
About This Trial
Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in prot…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Age * Cohort 7 (currently enrolling): ≥5 to \<12 years of age. * Completed Cohorts 1-6: ≥12 to 65 years of age. * Diagnosis of classical homocystinuria (HCU) * Cohort 7 (currently enrolling): Diagnosis based on clinical, biochemical, and/or molecular genetic testing. …
Contacts

Travere Call Center

1-877-659-5518

medinfo@travere.com

CONTACT