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NCT06247085 · ClinicalTrials.gov registry record · Phase 3
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
A Phase 3 study of Homocystinuria, sponsored by Travere Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 70
- Enrollment target
- 1
- Study location
NCT06247085: Active Phase 3 study of Homocystinuria, sponsored by Travere Therapeutics.
NCT06247085 is a Phase 3 study of Homocystinuria that is active but no longer recruiting, run by Travere Therapeutics. The registered enrollment target is 70 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06247085, a Phase 3 study of Homocystinuria, is active but no longer recruiting, sponsored by Travere Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 70 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care who have not achieved tHcy target levels. Study details include: * Total Study duration: up to 38 weeks * Screening: * Initial Screening duration: up to 4 weeks * Pre-treatment Diet Standardization Period duration: up to 6 weeks * Blinded Treatment Duration: 24 weeks * 2-week blinded dose titration period * 22-week blinded assessment period * Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)
Primary Outcome
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
Conditions Studied
Interventions
- OTHER Placebo
- DRUG Pegtibatinase
Study Locations (1)
North Carolina
- Travere Investigational Site - Virtual Site - Morrisville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 70 participants |
| Start Date | 2023-12-28 |
| Est. Completion | 2026-01 |
| Phase | Phase 3 |
What the registry record for NCT06247085 still lists
NCT06247085 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 70 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (91% lower).
The record links to 1 condition, with Homocystinuria appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT06247085 reports a single indexed study location in North Carolina.
Frequently Asked Questions
What is clinical trial NCT06247085 about?
NCT06247085 is a clinical study titled "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". The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care who have not achieved tHcy target levels. Study details include: * Total Study duration: up to 38 weeks * Screening: * Initia...
What is the current status of trial NCT06247085?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 70 participants. The study started on 2023-12-28. Estimated completion is 2026-01.
What conditions does trial NCT06247085 study?
This clinical trial studies the following conditions: Homocystinuria.
What interventions are being tested in trial NCT06247085?
The interventions under investigation include: Placebo (OTHER), Pegtibatinase (DRUG).
Who is sponsoring clinical trial NCT06247085?
This trial is sponsored by Travere Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06247085 being conducted?
This trial has 1 study location across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06247085's enrollment target sits among peer trials
70 1786th of 2000 higher than 208 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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