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

ClinicalTrials.gov ID: NCT06247085

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.

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ClinicalTrials.gov public records Last synced May 9, 2026, 6:11 PM EDT

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)

Study identification

NCT ID: NCT06247085
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 3
Lead sponsor: Travere Therapeutics, Inc.; Industry
Enrollment: 70 participants

Conditions and interventions

Conditions

Homocystinuria

Interventions

Pegtibatinase Drug
Placebo Other

Drug · Other

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 27, 2023
Primary completion: Jul 31, 2027
Completion: Aug 31, 2027
Last update posted: May 7, 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	Not yet 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	Not yet 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	Not yet 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 May 7, 2026 · Synced May 9, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT06247085 live on ClinicalTrials.gov.

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