Clinical trial NCT06431893: 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)
Official title: An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Homocystinuria
Keywords: Classical homocystinuria
HCU
Cystathionine Beta Synthase Deficiency
Lead sponsor: Travere Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2024-05-29
Enrollment: 100
Sex: ALL
Minimum age (years): 5
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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

Detailed description: 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 ENSEMBLE study, an optional protein tolerance modification (PTM) sub-study will also be conducted for eligible participants.

Eligibility criteria: Inclusion Criteria:

* Participants active in the COMPOSE study or who completed the 24-week blinded period on the full target dose of study intervention in the Phase 3 HARMONY study

Exclusion Criteria:

* Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance

Primary outcomes:
measure: Treatment-emergent AEs
timeFrame: Week 1 - Week 108
measure: Hypermethioninemia
timeFrame: Week 1 - Week 108
measure: Hypomethioninemia
timeFrame: Week 1 - Week 108
measure: Dietary Protein Rescue
timeFrame: Week 1 - Week 108
Secondary outcomes:
measure: Total Homocysteine (tHcy) Levels
timeFrame: Week 1 - Week 108
Other outcomes:
Locations:
city: Portland
country: United States
facility: Travere Investigational Site
state: Maine
city: New York
country: United States
facility: Travere Investigational Site
state: New York
city: Morrisville
country: United States
facility: Travere Investigational Site - Virtual Site
state: North Carolina
city: Philadelphia
country: United States
facility: Travere Investigational Site
state: Pennsylvania
Study officials:
affiliation: Travere Therapeutics, Inc.
name: Michael Imperiale, MD
role: STUDY_DIRECTOR
References: