Pegtibatinase: Pegtibatinase BIW
Placebo: volume-matched saline SC BIW
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)
| Facility | City | Region | Status |
|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | Recruiting |
| Yale University School of Medicine | New Haven | Connecticut | Recruiting |
| Emory University | Atlanta | Georgia | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | Not Yet Recruiting |
| Maine Health - Maine Medical Center | Portland | Maine | Recruiting |
| Uncommon Cures | Chevy Chase | Maryland | Recruiting |
| The Mount Sinai Hospital | New York | New York | Recruiting |
| Science 37 - Virtual Site | Morrisville | North Carolina | Recruiting |
| Oregon Health and Science University | Portland | Oregon | Not Yet Recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | Recruiting |
| University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | Recruiting |
| University of Texas Southwestern Medical Center | Dallas | Texas | Recruiting |
| Utah Health - The University of Utah Primary Children's Hospital | Salt Lake City | Utah | Recruiting |
| Royal Adelaide Hospital | Adelaide | Australia | Recruiting |
| Royal Children's Hospital Melbourne | Parkville | Australia | Recruiting |
| Westmead Hospital | Westmead | Australia | Recruiting |
| Cliniques Universitaires Saint-Luc | Brussels | Belgium | Not Yet Recruiting |
| Universitair Ziekenhuis Antwerpen (UZA) | Edegem | Belgium | Not Yet Recruiting |
| Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades | Paris | France | Not Yet Recruiting |
| CHRU Hôpitaux de Tours - Hôpital Bretonneau | Tours | France | Not Yet Recruiting |
| Centre Hospitalier Régional Universitaire de Nancy (CHRU) - Hopitaux de Brabois - Hôpital d'enfants | Vandœuvre-lès-Nancy | France | Not Yet Recruiting |
| Universitätsklinikum Hamburg-Eppendorf | Hamburg | Germany | Not Yet Recruiting |
| Universitätsklinikum Leipzig | Leipzig | Germany | Not Yet Recruiting |
| Universitätsklinikum Münster | Münster | Germany | Not Yet Recruiting |
| Children's Health Ireland (CHI) at Temple Street | Dublin | Ireland | Not Yet Recruiting |
| Ospedale Pediatrico Giovanni XXIII | Bari | Italy | Not Yet Recruiting |
| IRCCS Istituto Giannina Gaslini | Genova | Italy | Not Yet Recruiting |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (Policlinico di Milano) | Milan | Italy | Not Yet Recruiting |
| Fondazione IRCCS San Gerardo Dei Tintori - Ospedale San Gerardo | Monza | Italy | Not Yet Recruiting |
| Azienda Ospedaliera di Padova | Padova | Italy | Not Yet Recruiting |
| SP ZOZ Szpital Uniwersytecki w Krakowie | Krakow | Poland | Not Yet Recruiting |
| Instytut Matki i Dziecka | Warsaw | Poland | Not Yet Recruiting |
| Instytut Pomnik-Centrum Zdrowia Dziecka | Warsaw | Poland | Not Yet Recruiting |
| Unidade Local de Saúde de Coimbra, EPE - Hospitais da Universidade de Coimbra | Coimbra | Portugal | Not Yet Recruiting |
| Unidade Local de Saúde de Santa Maria, EPE - Hospital de Santa Maria | Lisbon | Portugal | Not Yet Recruiting |
| Unidade Local de Saúde de São João, EPE - Hospital São João | Porto | Portugal | Not Yet Recruiting |
| Hamad General Hospital | Doha | Qatar | Recruiting |
| Sidra Hospital | Doha | Qatar | Recruiting |
| King Abdullah International Medical Research Center | Riyadh | Saudi Arabia | Not Yet Recruiting |
| King Faisal Specialist Hospital and Research Centre (KFSHRC) - Riyadh | Riyadh | Saudi Arabia | Not Yet Recruiting |
+ 12 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06247085 on ClinicalTrials.gov ↗ ← All trials in Ireland