NCT06247085

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

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)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 70 (estimated)
• Sponsor: Travere Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

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 who have not achieved tHcy target levels. 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)

Detailed 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 2. Pre-treatment Diet Standardization Period (DSP): After meeting initial eligibility criteria, participants will begin a pre treatment DSP of up to 6 weeks. The DSP is intended to help minimize variability in protein intake and supplements throughout the randomized portion of the study. The baseline diet and compliance with HCU-related treatments will be recorded by the dietitian using the HCU-specific diet monitoring tool, called SING (Simplified Ingested Nutrients Guide). The baseline diet and treatment compliance will be used as a reference for future evaluation of daily intact protein intake (DIPI) and HCU treatments. Blinded Treatment Period: During the 24-week blinded treatment period, study visits will occur at regular intervals including home visits for study drug administration. Some study visits may be conducted remotely. Dietary protein intake and compliance with HCU treatments will continue to be monitored and recorded to ensure a stable diet and HCU treatment compliance.

Conditions

• Homocystinuria

Interventions

Type	Name	Description
DRUG	Pegtibatinase	Full Target Dose of pegtibatinase BIW
OTHER	Placebo	volume-matched saline SC BIW

Timeline

• Start date: 2023-12-28
• Primary completion: 2025-12-01
• Completion: 2026-01-01
• First posted: 2024-02-07
• Last updated: 2025-04-09

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study