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NCT03406611 · ClinicalTrials.gov registry record · Phase 1
Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
A Phase 1 study of Homocystinuria, sponsored by Travere Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 32
- Enrollment target
- 8
- Study locations
NCT03406611: Active Phase 1 study of Homocystinuria, sponsored by Travere Therapeutics.
NCT03406611 is a Phase 1 study of Homocystinuria that is active but no longer recruiting, run by Travere Therapeutics. The registered enrollment target is 32 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (47% lower). The trial reports 8 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03406611, a Phase 1 study of Homocystinuria, is active but no longer recruiting, sponsored by Travere Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 32 participants
- Enrollment target
- 8
- Study locations
Study Summary
Homocystinuria caused by Cystathionine Beta-Synthase (CBS) Deficiency is a rare autosomal-recessive metabolic condition characterized by an excess of homocysteine (Hcy) in the plasma, tissues and urine. It is due to reduced or absent activity of the CBS enzyme, and is also known as classical homocystinuria. The symptoms associated with homocystinuria are variable in severity and time of onset across patients. Some affected individuals may have mild signs of the disorder; others may have multi-systemic involvement including potentially life-threatening complications. Homocystinuria can affect many different organ systems of the body; the four most commonly involved are the eyes, central nervous system, skeleton, and the vascular system. The current approaches to treatment of homocystinuria patients include a highly restrictive diet and use of dietary supplements. Lifetime compliance with this diet is poor. Pegtibatinase (TVT-058) represents a novel therapeutic approach that incorporates the use of a modified version of the native, human CBS (hCBS) enzyme. The goal of treatment is to introduce the CBS enzyme into circulation, resulting in reduced Hcy levels, increased cystathionine (Cth) and cysteine (Cys) levels.
Primary Outcome
Incidence of AEs (by type, severity and relationship to study drug)
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Pegtibatinase
Study Locations (8)
California
- Travere Investigational Site - Virtual Site - Culver City
Colorado
- Travere Investigational Site - Aurora
Florida
- Travere Investigational Site - Miami
Indiana
- Travere Investigational Site - Indianapolis
Maine
- Travere Investigational Site - Portland
Massachusetts
- Travere Investigational Site - Boston
New York
- Travere Investigational Site - New York
Pennsylvania
- Travere Investigational Site - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2019-01-22 |
| Est. Completion | 2026-12 |
| Phase | Phase 1 |
What the registry record for NCT03406611 still lists
NCT03406611 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (47% 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.
NCT03406611 lists 8 locations in 8 states (California, Colorado, Florida).
Frequently Asked Questions
What is clinical trial NCT03406611 about?
NCT03406611 is a clinical study titled "Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)". Homocystinuria caused by Cystathionine Beta-Synthase (CBS) Deficiency is a rare autosomal-recessive metabolic condition characterized by an excess of homocysteine (Hcy) in the plasma, tissues and urine. It is due to reduced or absent activity of the CBS enzyme, and is also known as classical homocys...
What is the current status of trial NCT03406611?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 32 participants. The study started on 2019-01-22. Estimated completion is 2026-12.
What conditions does trial NCT03406611 study?
This clinical trial studies the following conditions: Homocystinuria.
What interventions are being tested in trial NCT03406611?
The interventions under investigation include: Placebo (DRUG), Pegtibatinase (DRUG).
Who is sponsoring clinical trial NCT03406611?
This trial is sponsored by Travere Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03406611 being conducted?
This trial has 8 study locations across California, Colorado, Florida, Indiana, Maine. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Homocystinuria
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 NCT03406611's enrollment target sits among peer trials
32 1296th of 2000 higher than 679 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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