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Clinical Trials in Ireland / NCT06247085
Recruiting Phase 3

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

NCT06247085 · tracked via the Priya Life Science Ireland tracker
Sponsor
Travere Therapeutics, Inc.
Phase
Phase 3
Started
2023-12-28
Last updated
2026-08-07

Condition(s) studied

Homocystinuria

Investigational drug(s) / intervention(s)

PegtibatinasePlacebo

Pegtibatinase: Pegtibatinase BIW

Placebo: volume-matched saline SC BIW

Study summary

The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care. Study details include:

* Total Study duration: up to 38 weeks
* Screening:

* Initial Screening duration: up to 4 weeks
* Pre-treatment Diet Standardization Period duration: up to 6 weeks
* Blinded Treatment Duration: 24 weeks

* 2-week blinded dose titration period
* 22-week blinded assessment period
* Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)

Eligibility

Sex
ALL
Min age
12 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria: * Must be ≥12 to ≤65 years of age, at the time of signing the informed consent * Must have a diagnosis of classical HCU based on clinical, biochemical, and/or molecular genetic testing * Plasma tHcy ≥80 µM at Screening visit, with allowance for up to 18 participants who may be enrolled with a Screening plasma tHcy ≥50 to \<80 µM * Participants who can become pregnant must have a negative pregnancy test before starting the study and must use a highly effective form of birth control (less than 1% risk of pregnancy per year) during the study and for at least 4 weeks after the last dose. * Willing to maintain a generally stable diet for the duration of the study (unless changes are required based on medical/safety reasons) * Willing to maintain generally stable intake and doses of betaine, pyridoxine, and medical food for the duration of the study (unless changes are required based on medical/safety reasons) Exclusion Criteria: * Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorder of cobalamin metabolism * Concurrent disease or condition (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that would interfere with study participation or safety (excluding complications of HCU). * History of major thrombotic event (eg, cerebrovascular accident, myocardial infarction, pulmonary embolism) in the previous 6 months. * Body weight ≥160 kg. * Use or planned use of any injectable drugs containing PEG (excluding PEG-containing vaccines) * Any previous exposure to pegtibatinase and/or previous participation in a clinical study that included administration of pegtibatinase or pegtarviliase * Prior severe immune reaction to a PEG-containing product

Primary outcome measure(s)

Trial sites (52)

FacilityCityRegionStatus
Phoenix Children's Hospital Phoenix Arizona Recruiting
Yale University School of Medicine New Haven Connecticut Recruiting
Emory University Atlanta Georgia Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Not Yet Recruiting
Maine Health - Maine Medical Center Portland Maine Recruiting
Uncommon Cures Chevy Chase Maryland Recruiting
The Mount Sinai Hospital New York New York Recruiting
Science 37 - Virtual Site Morrisville North Carolina Recruiting
Oregon Health and Science University Portland Oregon Not Yet Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh Pittsburgh Pennsylvania Recruiting
University of Texas Southwestern Medical Center Dallas Texas Recruiting
Utah Health - The University of Utah Primary Children's Hospital Salt Lake City Utah Recruiting
Royal Adelaide Hospital Adelaide Australia Recruiting
Royal Children's Hospital Melbourne Parkville Australia Recruiting
Westmead Hospital Westmead Australia Recruiting
Cliniques Universitaires Saint-Luc Brussels Belgium Not Yet Recruiting
Universitair Ziekenhuis Antwerpen (UZA) Edegem Belgium Not Yet Recruiting
Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades Paris France Not Yet Recruiting
CHRU Hôpitaux de Tours - Hôpital Bretonneau Tours France Not Yet Recruiting
Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants Vandœuvre-lès-Nancy France Not Yet Recruiting
Universitätsklinikum Hamburg-Eppendorf Hamburg Germany Not Yet Recruiting
Universitätsklinikum Leipzig Leipzig Germany Not Yet Recruiting
Universitätsklinikum Münster Münster Germany Not Yet Recruiting
Children's Health Ireland (CHI) at Temple Street Dublin Ireland Not Yet Recruiting
Ospedale Pediatrico Giovanni XXIII Bari Italy Not Yet Recruiting
IRCCS Istituto Giannina Gaslini Genova Italy Not Yet Recruiting
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano) Milan Italy Not Yet Recruiting
Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo Monza Italy Not Yet Recruiting
Azienda Ospedaliera di Padova Padova Italy Not Yet Recruiting
SP ZOZ Szpital Uniwersytecki w Krakowie Krakow Poland Not Yet Recruiting
Instytut Matki i Dziecka Warsaw Poland Not Yet Recruiting
Instytut Pomnik-Centrum Zdrowia Dziecka Warsaw Poland Not Yet Recruiting
Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra Coimbra Portugal Not Yet Recruiting
Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria Lisbon Portugal Not Yet Recruiting
Unidade Local de Saúde de São João, EPE - Hospital São João Porto Portugal Not Yet Recruiting
Hamad General Hospital Doha Qatar Recruiting
Sidra Hospital Doha Qatar Recruiting
King Abdullah International Medical Research Center Riyadh Saudi Arabia Not Yet Recruiting
King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh Riyadh Saudi Arabia Not Yet Recruiting

+ 12 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06247085 on ClinicalTrials.gov ↗ ← All trials in Ireland