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Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

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NCT03406611 · readout ≤ 346 d

Sponsored by Travere Therapeutics, Inc. (industry) · TVTX — their whole pipeline →.

Phase
Phase 1/2
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
39estimated
Sites
12
Countries
France, Qatar, United States

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
DateFiledBasisWhat it is
Start2019-01-22actualWhen the study began enrolling
Primary completionJul 2027estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completionJul 2027estimatedThe whole study's end, after follow-up
First posted2018-01-23actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-08-10actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

  • Homocystinuria

Interventions

  • Drug: Pegtibatinase — also filed as TVT-058, OT-58, PEG modified CBS, PEG htCBS C15S, htCBS C15S ME-200GS
  • Drug: Placebo

Primary outcomes

what the primary-completion date above is the date OF
Incidence of AEs
measured • Cohorts 1-6: Through double-blind study completion, approximately 10 months per patient • Cohort 7: First dose through End of Treatment (up to 32 weeks)
Anti-pegtibatinase antibodies
measured • Cohorts 1-6: Through double-blind study completion, approximately 10 months per patient • Cohort 7: First dose through End of Treatment (up to 32 weeks)
Anti-PEG antibodies
measured • Cohorts 1-6: Through double-blind study completion, approximately 10 months per patient • Cohort 7: First dose through End of Treatment (up to 32 weeks)
Incidence of hypermethioninemia (Cohort 7 only)
measured First dose through End of Treatment (up to Week 32)
Incidence of hypomethioninemia (Cohort 7 only)
measured First dose through End of Treatment (up to Week 32)
The proportion of participants requiring dietary protein rescue (Cohort 7 only)
measured First dose through End of Treatment (up to Week 32)

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