By InvitationPhase 3

A Phase 3 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)

United States100 participantsStarted 2024-04-30

Plain-language 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

Who can participate

Age range5 Years – 65 Years

SexALL

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Inclusion Criteria: * Participants active in the COMPOSE study or who completed the 24-week blinded period on the full target dose of study intervention 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

What they're measuring

Treatment-emergent AEs

Timeframe: Week 1 - Week 108

Hypermethioninemia

Timeframe: Week 1 - Week 108

Hypomethioninemia

Timeframe: Week 1 - Week 108

Dietary Protein Rescue

Timeframe: Week 1 - Week 108

Trial details

NCT IDNCT06431893

SponsorTravere Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-01

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