A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

Condition(s) studied

Investigational drug(s) / intervention(s)

Pegtibatinase: Pegtibatinase BIW

Study 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 blinded treatment in the Phase 3 HARMONY are eligible to participate.

Participants will be in this clinical study for a minimum of 108 weeks including:

* a treatment period of up to 104 weeks
* a 4-week safety follow-up period

Eligibility

Primary outcome measure(s)

• Treatment-emergent AEs — Week 1 - Week 108
  Incidence of treatment-emergent AEs
• Hypermethioninemia — Week 1 - Week 108
  Incidence of hypermethioninemia
• Hypomethioninemia — Week 1 - Week 108
  Incidence of hypomethioninemia
• Dietary Protein Rescue — Week 1 - Week 108
  Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia

Trial sites (52)

+ 12 more sites — see the full list on the official registry below.

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