Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-25 @ 1:23 AM
Ignite Modification Date:
2025-12-25 @ 1:23 AM
NCT ID:
NCT06431893
Brief Summary:
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 treatment in the Phase 3 HARMONY are eligible to participate.
Participants will be in this clinical study for up to about 13 months including:
* a treatment period of up to 104 weeks
* a 4-week safety follow-up period
Detailed Description:
Overall Design:
This is a global, multicenter, single-arm, open-label study. Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety, efficacy, and clinical assessments of pegtibatinase treatment.
All participants will follow a protocol of self-administration of pegtibatinase unless exempted. During the ENSEMBLE study, an optional protein tolerance modification (PTM) sub-study will also be conducted for eligible participants.
Study:
NCT06431893
Study Brief:
Protocol Section:
NCT06431893