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

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Travere Therapeutics, Inc.
Study ID
NCT06247085
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • Homocystinuria

Eligibility Criteria

Sex
ALL
Age
12 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • Pegtibatinase — DRUG
    Pegtibatinase BIW
  • Placebo — OTHER
    volume-matched saline SC BIW

Study Details

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 Dates

Start date
Dec 28, 2023
Status verified
May 2026
Primary completion
Aug 31, 2027
Completion
Sep 30, 2027

Study Design

Enrollment
70 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: pegtibatinase
  • Placebo Comparator: placebo

Primary Outcome Measure

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

Central Contacts

Locations (13)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85006-
Yale University School of MedicineNew HavenConnecticut06519-
Emory UniversityAtlantaGeorgia30322-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
Maine Health - Maine Medical CenterPortlandMaine04102-
Uncommon CuresChevy ChaseMaryland20815-
The Mount Sinai HospitalNew YorkNew York10029-
Science 37 - Virtual SiteMorrisvilleNorth Carolina27560-
Oregon Health and Science UniversityPortlandOregon97239-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
University of Pittsburgh Medical Center - Children's Hospital of PittsburghPittsburghPennsylvania15224-
University of Texas Southwestern Medical CenterDallasTexas75390-
Utah Health - The University of Utah Primary Children's HospitalSalt Lake CityUtah84113-

Find similar trials in Phoenix, AZ

By research site
Phoenix Children's Hospital· Phoenix, AZYale University School of Medicine· New Haven, CTEmory University· Atlanta, GAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILMaine Health - Maine Medical Center· Portland, MEUncommon Cures· Chevy Chase, MD

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