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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Homocystinuria

In brief

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)

Key facts

Study ID

NCT06247085

Run by

Travere Therapeutics, Inc.

People needed

70

Starts

2023-12-28

Expected to finish

2027-09-01

Last updated by the study team

2026-08-07

Who can join

Age: 12 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Yale University School of Medicine — New Haven, Connecticut, United States (enrolling)
• Uncommon Cures — Chevy Chase, Maryland, United States (enrolling)
• Maine Health - Maine Medical Center — Portland, Maine, United States (enrolling)
• Hamad General Hospital — Doha, Qatar (enrolling)
• The Mount Sinai Hospital — New York, New York, United States (enrolling)
• Phoenix Children's Hospital — Phoenix, Arizona, United States (enrolling)
• Sidra Hospital — Doha, Qatar (enrolling)
• University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
• University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
• Emory University — Atlanta, Georgia, United States (enrolling)
• Utah Health - The University of Utah Primary Children's Hospital — Salt Lake City, Utah, United States (enrolling)
• Royal Adelaide Hospital — Adelaide, Australia (enrolling)
• Royal Children's Hospital Melbourne — Parkville, Australia (enrolling)
• Westmead Hospital — Westmead, Australia (enrolling)
• Science 37 - Virtual Site — Morrisville, North Carolina, United States (enrolling)
• Children's Health Ireland (CHI) at Temple Street — Dublin, Ireland
• Universitätsklinikum Münster — Münster, Germany
• Universitätsklinikum Hamburg-Eppendorf — Hamburg, Germany
• Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants — Vandœuvre-lès-Nancy, France
• CHRU Hôpitaux de Tours - Hôpital Bretonneau — Tours, France
• Universitätsklinikum Leipzig — Leipzig, Germany
• Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades — Paris, France
• Universitair Ziekenhuis Antwerpen (UZA) — Edegem, Belgium
• Cliniques Universitaires Saint-Luc — Brussels, Belgium

Full record on ClinicalTrials.gov

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