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)
← catalyst calendarNCT06431893 · readout ≤ 1,473 d
Sponsored by Travere Therapeutics, Inc. (industry) · TVTX — their whole pipeline →.
Phase
Phase 3
Status
Enrolling by invitation
Study type
Interventional
Design
Na · None · Treatment
Enrollment
100estimated
Sites
52
Countries
Australia, Belgium, France +11
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-04-30 | actual | When the study began enrolling |
| Primary completion | Aug 2030 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Aug 2030 | estimated | The whole study's end, after follow-up |
| First posted | 2024-05-29 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-05-28 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Homocystinuria
Intervention
- Drug: Pegtibatinase — also filed as OT-58, TVT-058
Primary outcomes
what the primary-completion date above is the date OFTreatment-emergent AEs
measured Week 1 - Week 108
Hypermethioninemia
measured Week 1 - Week 108
Hypomethioninemia
measured Week 1 - Week 108
Dietary Protein Rescue
measured Week 1 - Week 108
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