Trial Profile

An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)

Status: Recruiting

Phase of Trial: Phase III

Latest Information Update: 16 Apr 2025

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At a glance

20 Mar 2025 Time frame of primary endpoint changed from Week 1 - Week 56 to Week 1 - Week 108.
20 Mar 2025 Planned number of patients changed from 90 to 100.
22 Aug 2024 According to a Travere Therapeutics media release, the trial design from this study will be presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal, September 3-6, 2024.

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