Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment
Public ClinicalTrials.gov record NCT06247085. 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
A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Researchers are looking for a better way to treat people living with classical homocystinuria (HCU), a rare inherited 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 helps break down homocysteine, which is produced when the body processes methionine from protein-containing foods. This helps keep homocysteine and methionine at healthy levels. When this enzyme is not working, homocysteine and methionine build up in the blood and then spread into different tissues of the body and stop these body tissues from working normally. People living with HCU can experience problems with their vision, bones, blood vessels, thinking, learning, and memory. Available treatments for HCU include a low-methionine diet (typically achieved by limiting protein-containing foods), a methionine-free protein supplement to help meet daily protein needs, and betaine (Cystadane®). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was designed by researchers to act like the CBS enzyme, which is missing or not working properly in people living with HCU. Researchers are studying whether adding pegtibatinase to standard treatment can lower homocysteine levels. Researchers want to learn how safe and effective pegtibatinase is, how it moves through and affects the body, and how it may affect the daily lives of people with HCU. The study plans to enroll participants from the US, Australia, Belgium, France, Germany, Ireland, Italy, Poland, Portugal, Qatar, Saudi Arabia, Spain, Turkey, and the United Kingdom. Many study visits may happen at home, depending on where you live and local regulations. Some visits may include a home nurse and telemedicine appointments with the study doctor.
Study identification
- NCT ID
- NCT06247085
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 70 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 12 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
- Dec 28, 2023 (Actual)
- Primary completion
- Aug 2027 (Estimated)
- Completion
- Sep 2027 (Estimated)
- Last update posted
- Oct 8, 2026
2023 – 2027
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 | Recruiting |
| Yale University School of Medicine | New Haven | Connecticut | 06519 | Recruiting |
| Emory University | Atlanta | Georgia | 30322 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | Recruiting |
| Maine Health - Maine Medical Center | Portland | Maine | 04102 | Recruiting |
| Uncommon Cures | Chevy Chase | Maryland | 20815 | Recruiting |
| The Mount Sinai Hospital | New York | New York | 10029 | Recruiting |
| Science 37 - Virtual Site | Morrisville | North Carolina | 27560 | Recruiting |
| Oregon Health and Science University | Portland | Oregon | 97239 | Not yet recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Recruiting |
| University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | Recruiting |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | Recruiting |
| Utah Health - The University of Utah Primary Children's Hospital | Salt Lake City | Utah | 84113 | Recruiting |
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 NCT06247085, 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 Oct 8, 2026 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT06247085 live on ClinicalTrials.gov.