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)

ID#: NCT06431893

Age: 5 - 65 years

Gender: All

Healthy Subjects: No

Study Phase: Phase 3

Recruitment Status: Enrolling by invitation

Start Date: April 30, 2024

End Date: August 01, 2030

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:

Inclusion Criteria:

- Participants active in the COMPOSE study or who completed the 24-week blinded period on study intervention (pegtibatinase or placebo) in the Phase 3 HARMONY study

Exclusion Criteria:

- Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance
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