A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
Public ClinicalTrials.gov record NCT06431893. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patients who are active in the Phase 1/2 COMPOSE study or those who complete the 24 weeks of blinded treatment in the Phase 3 HARMONY are eligible to participate. Participants will be in this clinical study for a minimum of 108 weeks including: * a treatment period of up to 104 weeks * a 4-week safety follow-up period
Study identification
- NCT ID
- NCT06431893
- Recruitment status
- Enrolling by invitation
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 100 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 5 Years to 65 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 29, 2024
- Primary completion
- Jul 31, 2030
- Completion
- Jul 31, 2030
- Last update posted
- May 27, 2026
2024 – 2030
United States locations
- U.S. sites
- 13
- U.S. states
- 12
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85006 | — |
| Yale University School of Medicine | New Haven | Connecticut | 06519 | — |
| Emory University | Atlanta | Georgia | 30322 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Maine Health Pediatric Specialty Care | Portland | Maine | 04012 | — |
| Uncommon Cures | Chevy Chase | Maryland | 20815 | — |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | — |
| Science 37 - Virtual Site | Morrisville | North Carolina | 27560 | — |
| Oregon Health and Science University | Portland | Oregon | 97239 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | — |
| Utah Health - The University of Utah Primary Children's Hospital | Salt Lake City | Utah | 84113 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 39 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06431893, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 27, 2026 · Synced Sep 7, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06431893 live on ClinicalTrials.gov.