A Phase 3 interventional study of Pegtibatinase in Homocystinuria, sponsored by Travere Therapeutics, Inc.. Enrolling by invitation at 52 sites in 14 countries. Open to participants aged 5 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-11-10.
Sponsored by Travere Therapeutics, Inc. · Phase 3, Interventional, and Treatment
The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patients who are active in the Phase 1/2 COMPOSE study or those who complete the 24 weeks of blinded treatment in the Phase 3 HARMONY are eligible to participate.
Participants will be in this clinical study for a minimum of 108 weeks including:
a treatment period of up to 104 weeks a 4-week safety follow-up period
Overall Design:
This is a global, multicenter, single-arm, open-label study. Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety, efficacy, and clinical assessments of pegtibatinase treatment.
All participants will follow a protocol of self-administration of pegtibatinase unless exempted. During the study, pegtibatinase pharmacokinetic assessments will be conducted. An optional protein tolerance modification (PTM) sub-study will also be conducted for eligible participants.
20 studies on the registry are indexed under Homocystinuria; 5 are open to participants now.
This study's planned enrollment of 100 is above the median of 22 across 12 interventional studies indexed under Homocystinuria.
Browse Homocystinuria studies →Travere Therapeutics, Inc. is the lead sponsor of 14 studies on the registry; 4 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 4 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Drug: Pegtibatinase
Pegtibatinase BIW
Also known as: OT-58, TVT-058
Treatment-emergent AEs
Incidence of treatment-emergent AEs
Time frame: Week 1 - Week 108
Hypermethioninemia
Incidence of hypermethioninemia
Time frame: Week 1 - Week 108
Hypomethioninemia
Incidence of hypomethioninemia
Time frame: Week 1 - Week 108
Dietary Protein Rescue
Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia
Time frame: Week 1 - Week 108
Maximum Drug Concentration
Pegtibatinase pharmacokinetic parameters at steady state - maximum drug concentration \[Cmax\]
Time frame: Pharmacokinetic Assessment Period - 4 weeks & 8 weeks
Area under the Curve within the Dosing Interval
Pegtibatinase pharmacokinetic parameters at steady state within the - area under the curve within the dosing interval \[AUC0-tau\]
Time frame: Pharmacokinetic Assessment Period - 4 weeks & 8 weeks
Total Homocysteine (tHcy) Levels
Relative and absolute changes from baseline in tHcy levels
Time frame: Week 1 - Week 108
Plan to share: Yes — Requests for clinical trial data, including language stating its intended use, should be directed to datarequest@travere.com. If approved, the requested information will be provided to the requestor after signing a data access agreement. Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication. Travere reserves the right to decline or recommend modifications to a request if it does not comply with the data sharing policy or if it is determined that the request is made by a biased source.
Time frame: Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication.
Access criteria: Requires submission and approval of intended use and a data sharing agreement.
No publications or documents are linked to this record.
Started Apr 2024 (actual). Primary completion is estimated for Aug 2030, 76 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
No contact was published for this record. The registry link below has the sponsor’s details.
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Studies of the drug tested here: Pegtibatinase
Travere Therapeutics, Inc.