A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)
Travere Therapeutics, Inc.
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)
Description
Overall Design: This global Phase 3 multi-center, multi-national, randomized, blinded, placebo-controlled study will be conducted in participants with classical CBS deficient HCU receiving standard of care who continue to have tHcy levels ≥50 μM. The total study duration will be up to 38 weeks. Approximately 70 participants (35 per arm) will be randomized to receive active drug or placebo (1:1 allocation). Screening: Participants will enter into a 2-phase Screening period of up to 10 weeks: 1. Initial Screening: up to 4-week period during which participants will be assessed for eligibility…
Eligibility
- Age range
- 12–65 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugPegtibatinase
Pegtibatinase BIW
- OtherPlacebo
volume-matched saline SC BIW
Locations (52)
- Phoenix Children's HospitalPhoenix, Arizona
- Yale University School of MedicineNew Haven, Connecticut
- Emory UniversityAtlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois
- Maine Health - Maine Medical CenterPortland, Maine
- Uncommon CuresChevy Chase, Maryland