Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06431893 | 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) | PHASE3 | ENROLLING BY_INVITATION | 100 | — | — | Apr 30, 2024 | Aug 1, 2030 | May 28, 2026 | 52 | United States, Australia +12 |
| NCT06247085 | A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment | PHASE3 | RECRUITING | 70 | — | — | Dec 28, 2023 | Sep 1, 2027 | May 8, 2026 | 52 | United States, Australia +12 |
| NCT03406611 | Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) | PHASE1 | ACTIVE NOT_RECRUITING | 32 | — | — | Jan 22, 2019 | Dec 1, 2026 | Nov 22, 2024 | 8 | United States |
Incidence of treatment-emergent AEs
Incidence of hypermethioninemia
Incidence of hypomethioninemia
Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia
Change between baseline and average of 6 to 12 week (6, 8, 10, and 12 week) plasma tHcy levels in participants receiving pegtibatinase vs. placebo. Baseline tHcy level defined as average of Week -3, Week -1, and Day 1 pre-dose plasma tHcy
Incidence of AEs (by type, severity and relationship to study drug)
Presence and levels of anti-pegtibatinase antibodies in plasma as measured by antibody titers
Presence and levels of anti-PEG antibodies in plasma as measured by antibody titers
| Arm | Type | Description |
|---|---|---|
| pegtibatinase | EXPERIMENTAL | - |
| placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Pegtibatinase | DRUG | Pegtibatinase BIW |
| Placebo | OTHER | volume-matched saline SC BIW |
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 pegti...