A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
Travere Therapeutics, Inc.
Study ID
NCT06431893
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • Homocystinuria

Eligibility Criteria

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

Interventions

  • Pegtibatinase — DRUG
    Pegtibatinase BIW

Study Details

The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patients who are active in the Phase 1/2 COMPOSE study or those who complete the 24 weeks of blinded treatment in the Phase 3 HARMONY are eligible to participate. Participants will be in this clinical study for a minimum of 108 weeks including: * a treatment period of up to 104 weeks * a 4-week safety follow-up period

Key Dates

Start date
Apr 30, 2024
Status verified
May 2026
Primary completion
Aug 31, 2030
Completion
Aug 31, 2030

Study Design

Enrollment
100 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: pegtibatinase

Primary Outcome Measure

Treatment-emergent AEs [ Time Frame: Week 1 - Week 108 ]

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 Pediatric Specialty CarePortlandMaine04012-
Uncommon CuresChevy ChaseMaryland20815-
Icahn School of Medicine at Mount SinaiNew YorkNew York10029-
Science 37 - Virtual SiteMorrisvilleNorth Carolina27560-
Oregon Health and Science UniversityPortlandOregon97239-
The 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 Pediatric Specialty Care· Portland, MEUncommon Cures· Chevy Chase, MD

Related Studies