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

PHASE3Active, not recruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

December 28, 2023

Primary Completion Date

December 31, 2025

Study Completion Date

January 31, 2026

Conditions

Homocystinuria

Interventions

DRUG

Pegtibatinase

Full Target Dose of pegtibatinase BIW

OTHER

Placebo

volume-matched saline SC BIW

Trial Locations (1)

Travere Investigational Site - Virtual Site, Morrisville

All Listed Sponsors

lead

Travere Therapeutics, Inc.

INDUSTRY