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)

Официальное название

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)

Обзор

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

Подробное описание

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.

Вмешательства

• Препарат Pegtibatinase
  Pegtibatinase BIW

Первичные конечные точки

• Treatment-emergent AEs [Срок оценки: Week 1 - Week 108]
• Hypermethioninemia [Срок оценки: Week 1 - Week 108]
• Hypomethioninemia [Срок оценки: Week 1 - Week 108]
• Dietary Protein Rescue [Срок оценки: Week 1 - Week 108]

Вторичные конечные точки (3)

• Maximum Drug Concentration [Срок оценки: Pharmacokinetic Assessment Period - 4 weeks & 8 weeks]
• Area under the Curve within the Dosing Interval [Срок оценки: Pharmacokinetic Assessment Period - 4 weeks & 8 weeks]
• Total Homocysteine (tHcy) Levels [Срок оценки: Week 1 - Week 108]

Критерии участия

Критерии включения

• 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

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 13 центров

• Phoenix Children's Hospital — Phoenix
• Yale University School of Medicine — New Haven
• Emory University — Atlanta
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
• Maine Health Pediatric Specialty Care — Portland
• Uncommon Cures — Chevy Chase
• Icahn School of Medicine at Mount Sinai — New York
• Science 37 - Virtual Site — Morrisville
• … и ещё 5 центров

Италия · 5 центров

• Ospedale Pediatrico Giovanni XXIII — Bari
• IRCCS Istituto Giannina Gaslini — Genova
• Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano) — Milan
• Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo — Monza
• Azienda Ospedaliera di Padova — Padova

Испания · 5 центров

• Hospital Sant Joan de Déu — Barcelona
• Hospital Universitario 12 de Octubre — Madrid
• Hospital Universitario Ramón y Cajal — Madrid
• Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario — Santiago de Compostela
• Hospital Universitario Virgen del Rocío — Seville

Великобритания · 4 центра

• Birmingham Women's and Children's NHS Foundation Trust — Birmingham
• University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham — Birmingham
• University College London Hospitals NHS Foundation Trust - National Hospital for Neurology — London
• Salford Royal NHS Foundation Trust - Salford Royal Hospital — Salford

Австралия · 3 центра

• Royal Adelaide Hospital — Adelaide
• Royal Children's Hospital Melbourne — Parkville
• Westmead Hospital — Westmead

Франция · 3 центра

• Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades — Paris
• CHRU Hôpitaux de Tours - Hôpital Bretonneau — Tours
• Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital — Vandœuvre-lès-Nancy

Германия · 3 центра

• Universitätsklinikum Hamburg-Eppendorf — Hamburg
• Universitätsklinikum Leipzig — Leipzig
• Universitätsklinikum Münster — Münster

Польша · 3 центра

• SP ZOZ Szpital Uniwersytecki w Krakowie — Krakow
• Instytut Matki i Dziecka — Warsaw
• Instytut Pomnik-Centrum Zdrowia Dziecka — Warsaw

Португалия · 3 центра

• Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra — Coimbra
• Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria — Lisbon
• Unidade Local de Saúde de São João, EPE - Hospital São João — Porto

Turkey (Türkiye) · 3 центра

• Gazi University Hospital — Ankara
• Ege Üniversitesi Hastanesi Bornova — Bornova
• Istanbul University Cerrahpasa Hospital — Istanbul

Бельгия · 2 центра

• Cliniques Universitaires Saint-Luc — Brussels
• Universitair Ziekenhuis Antwerpen (UZA) — Edegem

Катар · 2 центра

• Hamad General Hospital — Doha
• Sidra Hospital — Doha

Саудовская Аравия · 2 центра

• King Abdullah International Medical Research Center — Riyadh
• King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh — Riyadh

Ирландия · 1 центр

• Children's Health Ireland (CHI) at Temple Street — Dublin

Идентификаторы

NCT: NCT06431893 · TVTX-TVT058-302

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗