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

StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary completion moved at least 12 months later Jun 2026 → Jul 2027
Primary endpoints added 3 → 6 primary endpoints
See other at-risk trials from Travere Therapeutics, Inc.

Record History

See every version on ClinicalTrials.gov ↗

  1. Primary completion pushed from Jun 2026 to Jul 2027v18 Show 10 more changesShow less
    • Primary endpoint(s) modified
    • Enrollment reopened
    • Enrollment increased: 32 → 39 participants
    • Trial sites expanded: 8 → 12 locations
    • Study completion pushed from Dec 2026 to Jul 2027
    • Trial arms changed: 2 → 3
    • Eligibility criteria shortened, about 22 words
    • Secondary endpoints revised (12)
    • Study description expanded, about 1,052 words
    • Study title expanded, about 1 word
  2. Study Identification, Study Status, Oversightv17
  3. Re-verified, no change to tracked fieldsv16
  4. Primary completion date: Feb 2023 to Jun 2026
    • Trial status: Recruiting to Active, Not Recruiting
    • Study completion date: Jun 2025 to Dec 2026
    • Study sites revised (8)
    v15
  5. Study sites: 7 to 8
    • Primary completion date: Oct 2022 to Feb 2023
    v14
Show 12 earlier versionsHide 12 earlier versions
  1. Study sites: 6 to 7v13
  2. Enrollment target: 40 to 32
    • Primary completion date: Jul 2022 to Oct 2022
    • Eligibility criteria expanded, about 73 words
    • Secondary endpoints: 11 to 12
    v12
  3. Primary completion date: Dec 2021 to Jul 2022v11 Show 7 more changesShow less
    • Primary endpoints: 1 to 3
    • Study completion date: Dec 2021 to Jun 2025
    • Interventions: OT-58 (Drug), Placebo (Drug) to Pegtibatinase (Drug), Placebo (Drug)
    • Eligibility criteria expanded, about 25 words
    • Secondary endpoints: 8 to 11
    • Study sites revised (6)
    • Study title expanded, about 1 word
  4. Study sites: 4 to 6v10
  5. Enrollment target: 20 to 40
    • Primary and study completion dates: Jun 2020 to Dec 2021
    • Eligibility criteria revised
    • Study title expanded, about 1 word
    v9
  6. Enrollment target: 12 to 20
    • Secondary endpoints revised (8)
    • Study title shortened, about 1 word
    v8
  7. Primary and study completion dates: Oct 2019 to Jun 2020
    • Start date: Dec 13, 2018 to Jan 22, 2019
    v7
  8. Start date changed from estimate of Oct 2018 to Dec 13, 2018
    • Site details revised at 1 of 4 sites
    v6
  9. Start date: Sep 2018 to Oct 2018
    • Site details revised at 2 of 4 sites
    v5
  10. Trial status: Not Yet Recruiting to Recruiting
    • Primary and study completion dates: Sep 2019 to Oct 2019
    • Start date: Aug 2018 to Sep 2018
    • Site details revised at 4 of 4 sites
    v4
  11. Study sites: 0 to 4v3
  12. Enrollment target: 20 to 12v2 Show 6 more changesShow less
    • Start date: Apr 2018 to Aug 2018
    • Primary completion date: Apr 2020 to Sep 2019
    • Study completion date: Jul 2020 to Sep 2019
    • Interventions: OT-58 (Drug), Placebos (Drug) to OT-58 (Drug), Placebo (Drug)
    • Eligibility criteria revised
    • Secondary endpoints revised (8)
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally. People with HCU can experience problems with vision, bones, blood vessels, and cognitive function (the ability to think, learn, and remember). Treatments available for HCU, such as a low protein diet and betaine (Cystadane®), help reduce homocysteine levels. The diet is a low methionine diet and a methionine-free protein supplement (a product that provides extra protein to help meet daily protein needs). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was developed by scientists to be a version of the CBS enzyme that can be given to people with HCU. Researchers believe that giving pegtibatinase to people with HCU already getting medical treatment (or "standard of care") may reduce their homocysteine levels. This study is split into 7 different groups getting different amounts of drug. The first 6 groups have already finished the study. Group 7 plans to enroll participants from the US (virtual and in-person), France, and Qatar.
Trial Details
NCT Number NCT03406611
Lead Sponsor Travere Therapeutics, Inc.
Conditions
(from sponsor)		 Homocystinuria
Related terms Homocystinuria
Enrollment 39 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov