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NCT Number: NCT06431893

A 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)

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

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Key information

Conditions

Homocystinuria Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Hyperhomocysteinemia Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

5 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Royal Adelaide Hospital, Adelaide, Australia

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants active in the COMPOSE study or who completed the 24-week blinded period on study intervention (pegtibatinase or placebo) 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

Treatment and study plan

Pegtibatinase

Drug

Pegtibatinase BIW

Other names: OT-58, TVT-058

Primary outcomes

  1. Treatment-emergent AEs

    Time frame: Week 1 - Week 108

    Incidence of treatment-emergent AEs

  2. Hypermethioninemia

    Time frame: Week 1 - Week 108

    Incidence of hypermethioninemia

  3. Hypomethioninemia

    Time frame: Week 1 - Week 108

    Incidence of hypomethioninemia

  4. Dietary Protein Rescue

    Time frame: Week 1 - Week 108

    Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia

Secondary outcomes

  1. Maximum Drug Concentration

    Time frame: Pharmacokinetic Assessment Period - 4 weeks & 8 weeks

    Pegtibatinase pharmacokinetic parameters at steady state - maximum drug concentration [Cmax]

  2. Area under the Curve within the Dosing Interval

    Time frame: Pharmacokinetic Assessment Period - 4 weeks & 8 weeks

    Pegtibatinase pharmacokinetic parameters at steady state within the - area under the curve within the dosing interval [AUC0-tau]

  3. Total Homocysteine (tHcy) Levels

    Time frame: Week 1 - Week 108

    Relative and absolute changes from baseline in tHcy levels

Sponsors and collaborators

Lead sponsor

Travere Therapeutics, Inc.

Industry

Registry information

Official study 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)

Acronym: ENSEMBLE

Important dates

Study start
2024
Primary completion
2030
Study completion
2030
First posted
May 29, 2024
Registry last updated
May 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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