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Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
NCT03406611 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03406611
The ClinicalTrials.gov registry entry for NCT03406611 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Travere Therapeutics, which has 9 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03406611 reports 8 study locations spanning 8 distinct geographic areas — top geographies include California, Colorado, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03406611?
The interventions under investigation include: Placebo (DRUG), Pegtibatinase (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03406611?
This trial is sponsored by Travere Therapeutics, which has 9 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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