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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)

NCT06431893 · Status: ENROLLING_BY_INVITATION · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 100

Last updated 2025-04-11

No results posted yet for this 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

DRUG

Pegtibatinase

Full Target Dose of pegtibatinase BIW

Sponsors & Collaborators

  • Travere Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Michael Imperiale, MD · Travere Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-30
Primary Completion
2027-01-31
Completion
2027-01-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06431893 on ClinicalTrials.gov