Home›Trial›NCT06431893
NCT06431893Phase 3ENROLLING BY INVITATION

A Phase 3 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)

Study Summary

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 treatment in the Phase 3 HARMONY are eligible to participate. Participants will be in this clinical study for up to about 13 months including: * a treatment period of up to 104 weeks * a 4-week safety follow-up period

Conditions
Homocystinuria
Interventions / Drugs
Pegtibatinase
View on ClinicalTrials.gov ↗
Sponsor
TVTX
Travere Therapeutics
Nephrology
C61/100
Study Details
Enrollment100 participants
Primary Completion—
Lead SponsorTravere Therapeutics, Inc.
Data Retrieved2026-04-11 04:30:13.942984+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Current status: ENROLLING BY INVITATION.

Enrolled: 100 participants
Sponsor Catalyst Profile

Travere Therapeutics (TVTX) is the sponsoring company for this trial. BiotechSign currently grades this company C (61/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

TVTX Full Pipeline →All Companies →
Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.