Official 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If you notice that this information is out of date, please let us know at website@canpku.org.

According 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.

Current 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.

Pegtibatinase 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.

For the most current status, site list, and eligibility details, use the official study record.

This 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 program

Publications and scientific presentations to date

• Design of the HARMONY Phase 3 Study of an Investigational Enzyme Replacement Therapy, Pegtibatinase, for the Treatment of Classical Homocystinuria.
  View poster
• Travere announcement on initiation of the pivotal Phase 3 HARMONY study, including COMPOSE background and topline data timing guidance.
  Read company announcement
• Sponsor pipeline page for pegtibatinase.
  View sponsor page

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.