Recruiting
Phase 3

Pegtibatinase

Sponsor:

Travere Therapeutics, Inc.

Code:

NCT06247085

Conditions

Homocystinuria

Eligibility Criteria

Sex: All

Age: 12 - 65

Healthy Volunteers: Not accepted

Interventions

Pegtibatinase

Placebo

Study Details

Brief 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)

Conditions

Homocystinuria

Study ID

NCT06247085

Start date

Dec 28, 2023

Status verified date

Jul, 2026

Completion date

Sep, 2027

Anticipated

Primary completion date

Aug, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 65

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

70 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: pegtibatinase

placebo comparator: placebo

Interventions

Pegtibatinase

Pegtibatinase BIW

Placebo

volume-matched saline SC BIW

Primary outcome measure

  • Change from baseline in plasma tHcy levels - Weeks 6 to 12 [ Time Frame: Weeks 6 - 12 ]

Central Contacts and Locations

Central contacts

Travere Call Center

1-877-659-5518medinfo@travere.com

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85006

Yale University School of Medicine

Recruiting

New Haven, Connecticut, United States, 06519

Emory University

Recruiting

Atlanta, Georgia, United States, 30322

Maine Health - Maine Medical Center

Recruiting

Portland, Maine, United States, 04102

Uncommon Cures

Recruiting

Chevy Chase, Maryland, United States, 20815

The Mount Sinai Hospital

Recruiting

New York, New York, United States, 10029

Science 37 - Virtual Site

Recruiting

Morrisville, North Carolina, United States, 27560

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

Utah Health - The University of Utah Primary Children's Hospital

Recruiting

Salt Lake City, Utah, United States, 84113

More Information

Sponsor

Travere Therapeutics, Inc.

Last update posted

Aug 7, 2026

Last verified

Jul, 2026

Keywords

  • HCU
  • cystathionine beta synthase deficiency
  • Classical Homocystinuria

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Travere Therapeutics, Inc. on 2026-08-07.