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Study identifier: NCT06247085 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Homocystinuria  ·  Sponsor: Travere Therapeutics, Inc.

PhasePhase 3
Planned participants70
Who can joinAll sexes, 12 Years to 65 Years
Healthy volunteersNo

About this study

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)

This description comes directly from the study's public registry record.

Talk to the study team

Travere Call Center  ·  1-877-659-5518  ·  medinfo@travere.com

Always discuss trial participation with your own doctor first.

Locations (52)

Phoenix Children's HospitalPhoenix, Arizona, United StatesNot Yet Recruiting
Yale University School of MedicineNew Haven, Connecticut, United StatesRecruiting
Emory UniversityAtlanta, Georgia, United StatesRecruiting
Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois, United StatesNot Yet Recruiting
Maine Health - Maine Medical CenterPortland, Maine, United StatesRecruiting
Uncommon CuresChevy Chase, Maryland, United StatesRecruiting
The Mount Sinai HospitalNew York, New York, United StatesRecruiting
Science 37 - Virtual SiteMorrisville, North Carolina, United StatesRecruiting
Oregon Health and Science UniversityPortland, Oregon, United StatesNot Yet Recruiting
Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania, United StatesRecruiting
University of Pittsburgh Medical Center - Children's Hospital of PittsburghPittsburgh, Pennsylvania, United StatesRecruiting
University of Texas Southwestern Medical CenterDallas, Texas, United StatesRecruiting

+ 40 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06247085