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 Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-30

Study Completion Date

2027-01-31

Brief Summary

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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

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Detailed Description

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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.

Conditions

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Homocystinuria

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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pegtibatinase

Group Type EXPERIMENTAL

Pegtibatinase

Intervention Type DRUG

Full Target Dose of pegtibatinase BIW

Interventions

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Pegtibatinase

Full Target Dose of pegtibatinase BIW

Intervention Type DRUG

Other Intervention Names

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OT-58 TVT-058

Eligibility Criteria

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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