NCT06431893
A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
Enrolling by Invitation
PHASE3Ages 5–65InterventionalTreatmentTravere Therapeutics, Inc.
~100 participants
Updated 2026-05-28 on ClinicalTrials.gov
What's tested:Pegtibatinase
At a glance
Recruiting sites
0 of 52 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Treatment-emergent AEs
Measured over Week 1 - Week 108
+3 more outcomes measured
Conditions
Where it's being run
52 sites across 25 statesItaly5
Spain5
United Kingdom4
Australia3
France3
Germany3
Poland3
Portugal3
Study leadership
- Michael Imperiale, MD · STUDY_DIRECTOR · Travere Therapeutics, Inc.
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Participants active in the COMPOSE study or who completed the 24-week blinded period on study intervention (pegtibatinase or placebo) in the Phase 3 HARMONY study
Exclusion
Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance
What this trial measures
- Treatment-emergent AEsWeek 1 - Week 108
Incidence of treatment-emergent AEs
- HypermethioninemiaWeek 1 - Week 108
Incidence of hypermethioninemia
- HypomethioninemiaWeek 1 - Week 108
Incidence of hypomethioninemia
- Dietary Protein RescueWeek 1 - Week 108
Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia