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
HARMONY
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)
1 other identifier
interventional
70
14 countries
52
Brief Summary
The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standard of care. Study details include:
- Total Study duration: up to 38 weeks
- Screening:
- Initial Screening duration: up to 4 weeks
- Pre-treatment Diet Standardization Period duration: up to 6 weeks
- Blinded Treatment Duration: 24 weeks
- 2-week blinded dose titration period
- 22-week blinded assessment period
- Safety Follow-Up: 4 weeks after last dose (as applicable for those not enrolling in the long term extension study, ENSEMBLE)
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Dec 2023
Typical duration for phase_3
52 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 28, 2023
CompletedFirst Submitted
Initial submission to the registry
January 30, 2024
CompletedFirst Posted
Study publicly available on registry
February 7, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 1, 2027
May 8, 2026
May 1, 2026
3.6 years
January 30, 2024
May 5, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from baseline in plasma tHcy levels - Weeks 6 to 12
Change between baseline and average of 6 to 12 week (6, 8, 10, and 12 week) plasma tHcy levels in participants receiving pegtibatinase vs. placebo. Baseline tHcy level defined as average of Week -3, Week -1, and Day 1 pre-dose plasma tHcy
Weeks 6 - 12
Secondary Outcomes (1)
Change from baseline in plasma tHcy levels - Weeks 16 to 24
Weeks 16 - 24
Study Arms (2)
pegtibatinase
EXPERIMENTALplacebo
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- Must be ≥12 to ≤65 years of age, at the time of signing the informed consent
- Must have a diagnosis of classical HCU based on clinical, biochemical, and/or molecular genetic testing
- Plasma tHcy ≥80 µM at Screening visit, with allowance for up to 18 participants who may be enrolled with a Screening plasma tHcy ≥50 to \<80 µM
- Participants who can become pregnant must have a negative pregnancy test before starting the study and must use a highly effective form of birth control (less than 1% risk of pregnancy per year) during the study and for at least 4 weeks after the last dose.
- Willing to maintain a generally stable diet for the duration of the study (unless changes are required based on medical/safety reasons)
- Willing to maintain generally stable intake and doses of betaine, pyridoxine, and medical food for the duration of the study (unless changes are required based on medical/safety reasons)
You may not qualify if:
- Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorder of cobalamin metabolism
- Concurrent disease or condition (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that would interfere with study participation or safety (excluding complications of HCU).
- History of major thrombotic event (eg, cerebrovascular accident, myocardial infarction, pulmonary embolism) in the previous 6 months.
- Body weight ≥160 kg.
- Use or planned use of any injectable drugs containing PEG (excluding PEG-containing vaccines)
- Any previous exposure to pegtibatinase and/or previous participation in a clinical study that included administration of pegtibatinase or pegtarviliase
- Prior severe immune reaction to a PEG-containing product
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (52)
Phoenix Children's Hospital
Phoenix, Arizona, 85006, United States
Yale University School of Medicine
New Haven, Connecticut, 06519, United States
Emory University
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Maine Health - Maine Medical Center
Portland, Maine, 04102, United States
Uncommon Cures
Chevy Chase, Maryland, 20815, United States
The Mount Sinai Hospital
New York, New York, 10029, United States
Science 37 - Virtual Site
Morrisville, North Carolina, 27560, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Utah Health - The University of Utah Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
Royal Adelaide Hospital
Adelaide, Australia
Royal Children's Hospital Melbourne
Parkville, Australia
Westmead Hospital
Westmead, Australia
Cliniques Universitaires Saint-Luc
Brussels, Belgium
Universitair Ziekenhuis Antwerpen (UZA)
Edegem, Belgium
Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades
Paris, France
CHRU Hôpitaux de Tours - Hôpital Bretonneau
Tours, France
Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants
Vandœuvre-lès-Nancy, France
Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
Universitätsklinikum Leipzig
Leipzig, Germany
Universitätsklinikum Münster
Münster, Germany
Children's Health Ireland (CHI) at Temple Street
Dublin, Ireland
Ospedale Pediatrico Giovanni XXIII
Bari, Italy
IRCCS Istituto Giannina Gaslini
Genova, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano)
Milan, Italy
Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo
Monza, Italy
Azienda Ospedaliera di Padova
Padova, Italy
SP ZOZ Szpital Uniwersytecki w Krakowie
Krakow, Poland
Instytut Matki i Dziecka
Warsaw, Poland
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Poland
Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra
Coimbra, Portugal
Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria
Lisbon, Portugal
Unidade Local de Saúde de São João, EPE - Hospital São João
Porto, Portugal
Hamad General Hospital
Doha, Qatar
Sidra Hospital
Doha, Qatar
King Abdullah International Medical Research Center
Riyadh, Saudi Arabia
King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh
Riyadh, Saudi Arabia
Hospital Sant Joan de Déu
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Hospital Universitario Ramón y Cajal
Madrid, Spain
Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario
Santiago de Compostela, Spain
Hospital Universitario Virgen del Rocío
Seville, Spain
Gazi University Hospital
Ankara, Turkey (Türkiye)
Ege Üniversitesi Hastanesi Bornova
Bornova, Turkey (Türkiye)
Istanbul University Cerrahpasa Hospital
Istanbul, Turkey (Türkiye)
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery
London, United Kingdom
Salford Royal NHS Foundation Trust - Salford Royal Hospital
Salford, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Michael Imperiale, MD
Travere Therapeutics, Inc.
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 30, 2024
First Posted
February 7, 2024
Study Start
December 28, 2023
Primary Completion (Estimated)
August 1, 2027
Study Completion (Estimated)
September 1, 2027
Last Updated
May 8, 2026
Record last verified: 2026-05
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication.
- Access Criteria
- Requires submission and approval of intended use and a data sharing agreement.
Requests for clinical trial data, including language stating its intended use, should be directed to datarequest@travere.com. If approved, the requested information will be provided to the requestor after signing a data access agreement. Requests can be made following completion of the study and full publication of the study data in a peer reviewed journal for up to 36 months following its publication. Travere reserves the right to decline or recommend modifications to a request if it does not comply with the data sharing policy or if it is determined that the request is made by a biased source.