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Study details
Pegtibatinase for Classical Homocystinuria(HARMONY Study)This Phase 3 study is evaluating pegtibatinase, an investigational enzyme replacement therapy for people with classical homocystinuria due to cystathionine beta synthase deficiency. The official study record currently lists the HARMONY study as active, not recruiting. Study type
Interventional, Phase 3
Recruitment status
Active, not recruiting
Recruitment geography
Global study
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Study overviewOfficial study title: A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ?12 to ?65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment (HARMONY) Condition: Classical homocystinuria due to cystathionine beta synthase deficiency Intervention / therapy: Pegtibatinase compared with placebo, in addition to standard of care Sponsor: Travere Therapeutics, Inc. Study phase: Phase 3 ClinicalTrials.gov ID: NCT06247085 Estimated enrollment: 70 participants Last checked by CanPKU+: March 10, 2026 If you notice that this information is out of date, please let us know at website@canpku.org. Who may qualifyAccording to the official study record, this study is for participants 12 to 65 years of age with classical homocystinuria due to cystathionine beta synthase deficiency who remain above target total homocysteine levels despite standard-of-care treatment. The study includes a screening period and a diet standardization period before the blinded treatment phase begins. Important: Final eligibility is always determined by the study team. Site informationCurrent CanPKU+ classification: Active, not recruiting Canadian site listed on ClinicalTrials.gov: Not confirmed in the sources reviewed Geographic note: Travere describes HARMONY as a global, multi-center study. Families interested in site availability should use the official study record and sponsor resources for the most current location information. If you are looking for current participation options: The official study record should be checked first, as recruitment status and site availability can change over time. Therapy background and related studiesPegtibatinase is an investigational PEGylated recombinant enzyme replacement therapy designed to address the underlying cause of classical homocystinuria. Travere states that the HARMONY study was launched after positive Phase 1/2 COMPOSE data, where pegtibatinase showed dose-dependent reductions in total homocysteine and was generally well tolerated. Travere has also described a long-term extension study called ENSEMBLE for participants who complete earlier pegtibatinase studies, including HARMONY. Official study record and linksFor the most current status, site list, and eligibility details, use the official study record. Development history and publicationsThis section highlights earlier studies and publications related to this treatment program. It is intended as a plain-language research history summary and does not replace the official study record. Earlier and related studies in this programCurrent Phase 3 study: HARMONY Study of Pegtibatinase in Classical Homocystinuria ClinicalTrials.gov ID: NCT06247085 This is the pivotal randomized, placebo-controlled Phase 3 study evaluating efficacy and safety of pegtibatinase in participants with classical HCU. Earlier study: COMPOSE Phase 1/2 program Travere reported positive Phase 1/2 COMPOSE data before launching HARMONY, including meaningful reductions in total homocysteine at the 2.5 mg/kg dose. Long-term extension study: ENSEMBLE Travere has described ENSEMBLE as a Phase 3b open-label long-term extension study for participants completing COMPOSE or HARMONY. Publications and scientific presentations to date
If you notice that this section is missing a publication or no longer reflects the latest study history, please let us know at website@canpku.org. |
