Actively Recruiting
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
Led by Travere Therapeutics, Inc. · Updated on 2026-05-08
70
Participants Needed
52
Research Sites
191 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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)
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 12 to 65 years at the time of informed consent
- Diagnosed with classical homocystinuria (HCU) based on clinical, biochemical, or genetic testing
- Plasma total homocysteine (tHcy) level 80 µM or higher at Screening, with up to 18 participants allowed with levels 50 to less than 80 µM
- Negative pregnancy test if capable of becoming pregnant and agreement to use effective birth control during and for 4 weeks after the study
- Willing to maintain a generally stable diet during the study unless medically required to change
- Willing to maintain stable intake and doses of betaine, pyridoxine, and medical food during the study unless medically required to change
You will not qualify if you...
- Diagnosis of Marfan syndrome, MTHFR deficiency, or disorder of cobalamin metabolism
- Presence of any significant disease that could interfere with participation or safety (excluding HCU complications)
- History of major thrombotic event such as stroke, heart attack, or pulmonary embolism within the past 6 months
- Body weight of 160 kg or more
- Use or planned use of injectable drugs containing PEG (except PEG-containing vaccines)
- Previous exposure to pegtibatinase or participation in studies involving pegtibatinase or pegtarviliase
- History of severe immune reaction to PEG-containing products
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 52 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85006
Not Yet Recruiting
2
Yale University School of Medicine
New Haven, Connecticut, United States, 06519
Actively Recruiting
3
Emory University
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Not Yet Recruiting
5
Maine Health - Maine Medical Center
Portland, Maine, United States, 04102
Actively Recruiting
6
Uncommon Cures
Chevy Chase, Maryland, United States, 20815
Actively Recruiting
7
The Mount Sinai Hospital
New York, New York, United States, 10029
Actively Recruiting
8
Science 37 - Virtual Site
Morrisville, North Carolina, United States, 27560
Actively Recruiting
9
Oregon Health and Science University
Portland, Oregon, United States, 97239
Not Yet Recruiting
10
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
11
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
12
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
13
Utah Health - The University of Utah Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Not Yet Recruiting
14
Royal Adelaide Hospital
Adelaide, Australia
Not Yet Recruiting
15
Royal Children's Hospital Melbourne
Parkville, Australia
Not Yet Recruiting
16
Westmead Hospital
Westmead, Australia
Not Yet Recruiting
17
Cliniques Universitaires Saint-Luc
Brussels, Belgium
Not Yet Recruiting
18
Universitair Ziekenhuis Antwerpen (UZA)
Edegem, Belgium
Not Yet Recruiting
19
Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades
Paris, France
Not Yet Recruiting
20
CHRU Hôpitaux de Tours - Hôpital Bretonneau
Tours, France
Not Yet Recruiting
21
Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants
Vandœuvre-lès-Nancy, France
Not Yet Recruiting
22
Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
Not Yet Recruiting
23
Universitätsklinikum Leipzig
Leipzig, Germany
Not Yet Recruiting
24
Universitätsklinikum Münster
Münster, Germany
Not Yet Recruiting
25
Children's Health Ireland (CHI) at Temple Street
Dublin, Ireland
Not Yet Recruiting
26
Ospedale Pediatrico Giovanni XXIII
Bari, Italy
Not Yet Recruiting
27
IRCCS Istituto Giannina Gaslini
Genova, Italy
Not Yet Recruiting
28
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano)
Milan, Italy
Not Yet Recruiting
29
Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo
Monza, Italy
Not Yet Recruiting
30
Azienda Ospedaliera di Padova
Padova, Italy
Not Yet Recruiting
31
SP ZOZ Szpital Uniwersytecki w Krakowie
Krakow, Poland
Not Yet Recruiting
32
Instytut Matki i Dziecka
Warsaw, Poland
Not Yet Recruiting
33
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Poland
Not Yet Recruiting
34
Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra
Coimbra, Portugal
Not Yet Recruiting
35
Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria
Lisbon, Portugal
Not Yet Recruiting
36
Unidade Local de Saúde de São João, EPE - Hospital São João
Porto, Portugal
Not Yet Recruiting
37
Hamad General Hospital
Doha, Qatar
Not Yet Recruiting
38
Sidra Hospital
Doha, Qatar
Not Yet Recruiting
39
King Abdullah International Medical Research Center
Riyadh, Saudi Arabia
Not Yet Recruiting
40
King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh
Riyadh, Saudi Arabia
Not Yet Recruiting
41
Hospital Sant Joan de Déu
Barcelona, Spain
Not Yet Recruiting
42
Hospital Universitario 12 de Octubre
Madrid, Spain
Not Yet Recruiting
43
Hospital Universitario Ramón y Cajal
Madrid, Spain
Not Yet Recruiting
44
Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario
Santiago de Compostela, Spain
Not Yet Recruiting
45
Hospital Universitario Virgen del Rocío
Seville, Spain
Not Yet Recruiting
46
Gazi University Hospital
Ankara, Turkey (Türkiye)
Not Yet Recruiting
47
Ege Üniversitesi Hastanesi Bornova
Bornova, Turkey (Türkiye)
Not Yet Recruiting
48
Istanbul University Cerrahpasa Hospital
Istanbul, Turkey (Türkiye)
Not Yet Recruiting
49
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
Not Yet Recruiting
50
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
Not Yet Recruiting
51
University College London Hospitals NHS Foundation Trust - National Hospital for Neurology and Neurosurgery
London, United Kingdom
Not Yet Recruiting
52
Salford Royal NHS Foundation Trust - Salford Royal Hospital
Salford, United Kingdom
Not Yet Recruiting
Research Team
T
Travere Call Center
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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