A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy

NCT ID: NCT06821542

Last Updated: 2025-09-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-04

Study Completion Date

2032-11-30

Brief Summary

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This study will be conducted to compare Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.

Detailed Description

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Conditions

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Chronic Graft-versus-host-disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Axatilimab

Axatilimab at the protocol-defined dose.

Group Type EXPERIMENTAL

INCA034176

Intervention Type DRUG

IV infusion

Best available Treatment (BAT)

Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.

Group Type EXPERIMENTAL

Best Available Therapy (BAT)

Intervention Type DRUG

Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.

Interventions

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INCA034176

IV infusion

Intervention Type DRUG

Best Available Therapy (BAT)

Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.

Intervention Type DRUG

Other Intervention Names

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Axatilimab BAT could include: • Calcineurin inhibitor (cyclosporine or tacrolimus) • Extracorporeal photopheresis (ECP) • Mycophenolate mofetil (MMF) • A mammalian target of rapamycin (mTOR) inhibitor (everolimus or sirolimus) • Rituximab • Pentostatin • Proteasome inhibitors • Imatinib • Ibrutinib

Eligibility Criteria

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Inclusion Criteria

* Age ≥ 12 years at the time of signing the ICF.
* Active, moderate to severe cGVHD, requiring systemic immune suppression.
* Participants with refractory or recurrent cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib.
* Concomitant use of systemic corticosteroids is allowed. Participants on systemic corticosteroids must be on a stable dose of corticosteroids for at least 2 weeks prior to C1D1. Topical and inhaled corticosteroid agents are allowed.
* Participants must accept to be treated with one of the following BAT options on C1D1: CNI (cyclosporine or tacrolimus), ECP, MMF, an mTOR inhibitor (everolimus or sirolimus), rituximab, pentostatin, proteasome inhibitors, imatinib, or ibrutinib.
* History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood). Recipients of myeloablative, nonmyeloablative, or reduced-intensity conditioning are eligible.

Exclusion Criteria

* Receipt of more than 1 prior allo-HCT. Prior autologous HCT is allowed.
* Evidence of relapse of hematologic disease or treatment for relapse after the allo-SCT was performed, including DLI for the treatment of molecular relapse. Note: Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.
* Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
* Severe renal impairment, that is, estimated creatinine clearance \< 30 mL/min measured or calculated by Cockcroft-Gault equation in adults and Schwartz formula in pediatric participants, or end-stage renal disease on dialysis.
* Impaired liver function, defined as total bilirubin \> 1.5 × ULN and/or ALT and AST \> 3 × ULN in participants with no evidence of liver cGVHD.
* History of acute or chronic pancreatitis.
* Active, symptomatic myositis.
* Pregnant or breastfeeding.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Incyte Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Incyte Medical Monitor

Role: STUDY_DIRECTOR

Incyte Corporation

Locations

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Irccs Ospedale Pediatrico Bambino Gesu

Rome, , Italy

Site Status

Irccs Istituto Clinico Humanitas

Rozzano, , Italy

Site Status

Fondazione Ircss Casa Sollievo Della Sofferenza

San Giovanni Rotondo, , Italy

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A.O.U. Citta Della Salute E Della Scienza Di Torino

Torino, , Italy

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Ospedale Santa Maria Di Ca' Foncello

Treviso, , Italy

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Medical University of Graz

Graz, , Austria

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Medizinische Universitaet Innsbruck - Universitaetsklinik Fuer Innere Medizin Iii

Innsbruck, , Austria

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Krankenhaus Der Elisabethinen Linz Gmbh

Linz, , Austria

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Medizinische Universitat Wien, Universitatsklinik Fur Innere Medizin I

Vienna, , Austria

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St. Anna Childrens Hospital

Vienna, , Austria

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Az Sint-Jan Brugge Av

Bruges, , Belgium

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Chu Ucl Namur Site Godinne

Godinne-mont, , Belgium

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Universitair Ziekenhuis Leuven

Leuven, , Belgium

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Chu Liege -Centre Hospitalier Universitaire Sart Tilman

Liège, , Belgium

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Algemeen Ziekenhuis Delta

Roeselare, , Belgium

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Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, , Belgium

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Fakultni Nemocnice Hradec Kralove

Hradec Králové, , Czechia

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Fakultni Nemocnice Ostrava

Ostrava-poruba, , Czechia

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Ustav Hematologie A Krevni Transfuze (Uhkt), the Institute of Hematology and Blood Transfusion (Ihbt

Prague, , Czechia

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Helsinki University Central Hospital

Helsinki, , Finland

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New Children'S Hospital, University of Helsinki and Helsinki University Hospital

Helsinki, , Finland

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Turku University Hospital

Turku, , Finland

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Chu D'Amiens-Picardie - Hopital Sud

Amiens, , France

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CHU CAEN

Caen, , France

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Centre Hospitalier Regional Universitaire (Chru) de Lille - Hopital Claude Huriez

Lille, , France

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Institut Paoli Calmettes

Marseille, , France

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Chu de Nantes

Nantes, , France

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Centre Hospitalier Universitaire de Nice,Hopital L Archet

Nice, , France

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Hospital Saint-Louis - Aphp

Paris, , France

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Hopital Universitaire Robert Debre

Paris, , France

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Centre Hospitalier Universitaire de Bordeaux - Hopital Haut-Leveque

Pessac, , France

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Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

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Centre Henri Becquerel

Rouen, , France

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Institut Claudius Regaud Cancer Comprehensive Center - Iuct Oncopole

Toulouse, , France

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Chru Nancy

Vandœuvre-lès-Nancy, , France

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Gustave Roussy Cancer Campus Grand Paris- (Institut de Cancerologie Gustave-Roussy)

Villejuif, , France

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Uniklinik Rwth Aachen Medizinische Klinik Iv

Aachen, , Germany

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Charite - Universitaetsmedizin Berlin

Berlin, , Germany

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Universitaetsklinikum Koeln

Cologne, , Germany

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University Hospital Carl Gustav Carus

Dresden, , Germany

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Universitatsklinikum Erlangen

Erlangen, , Germany

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Klinikum Der Johann Wolfgang Goethe-Universitaet

Frankfurt am Main, , Germany

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Universitaetsklinikum Freiburg

Freiburg im Breisgau, , Germany

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Universitaetsmedizin Goettingen

Göttingen, , Germany

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Universitaetsklinikum Halle

Halle, , Germany

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Universitaetsklinikum Des Saarlandes

Homburg, , Germany

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Universitaetsklinikum Schleswig-Holstein, Uksh-Campus Kiel

Kiel, , Germany

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University Hospital Leipzig

Leipzig, , Germany

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Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii

Mainz, , Germany

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Universitaetsklinikum Muenster

Münster, , Germany

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University Clinic Regensburg

Regensburg, , Germany

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University Hospital of Ulm, Universitatsklinikum Ulm

Ulm, , Germany

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University of Wuerzburg

Würzburg, , Germany

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General Hospital of Athens Evangelismos - Eye Hospital

Athens, , Greece

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Attikon Hospital and Cutaneous Lymphoma Clinic-Athens University Medical School

Athens, , Greece

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University Hospital of Patras

RIO Patras, , Greece

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General Hospital of Thessalonikis George Papanikolaou - Gene and Cell Therapy Center

Thessaloniki, , Greece

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St. James Hospital

Dublin, , Ireland

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Ss Antonio & Biagio and C. Arrigo Hospital

Alessandria, , Italy

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Azienda Ospedaliero Universitaria Delle Marche Clinica Ematologica

Ancona, , Italy

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Azienda Ospedaliera Papa Giovanni Xxiii

Bergamo, , Italy

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Azienda Ospedaliera Sant'Orsola Malpighi

Bologna, , Italy

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Azienda Ospedaliero Universitaria Di Bologna - Policlinico S. Orsola-Malpighi - Istituto Di Ematolog

Bologna, , Italy

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Universita Degli Studi Di Brescia - Azienda Ospedaliera Spedali Civili Di Brescia

Brescia, , Italy

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Azienda Policlinico Vittorio Emanuele, Presidio G Rodolico, Universita Di Catania

Catania, , Italy

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Azienda Ospedaliero Universitaria Careggi-S.O.D. Patologia Medica

Florence, , Italy

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Irccs - Azienda Ospedaliera Universitaria - Ist San Martino

Genova, , Italy

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Pia Fondazione Cardinale Giovanni Panico Azienda Ospedaliera

Lecce, , Italy

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Fondazione Irccs Ca' Granda - Ospedale Maggiore Policlinico

Milan, , Italy

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Istituto Di Ricovero E Cura A Carattere Scientifico (Irccs) - Ospedale San Raffaele (Hsr) (Istituto

Milan, , Italy

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Asst Grande Ospedale Metropolitano Niguarda

Milan, , Italy

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Azienda Ospedaliera San Gerardo Di Monza

Monza, , Italy

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Azienda Ospedaliero Universitaria Federico Ii Di Napoli

Naples, , Italy

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Azienda Ospedaliera Di Rilievo Nazionale (Aorn) 'Antonio Cardarelli'

Napoli, , Italy

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Azienda Ospedale Universita Di Padova

Padua, , Italy

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Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello

Palermo, , Italy

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Irccs Fondazione Policlinico San Matteo

Pavia, , Italy

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Presidio Ospedaliero Spirito Santo Azienda U.S.L. Pescara

Pescara, , Italy

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Morelli Hospital

Reggio Calabria, , Italy

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Aou Policlinico Umberto I

Roma, , Italy

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A. Gemelli University Hospital, Catholic University of the Sacred Heart

Roma, , Italy

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Fondazione Ptv Policlinico Tor Vergata

Rome, , Italy

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Clinica Ematologica, Dism Azienda Ospedaliero Universitaria

Udine, , Italy

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Uoc Ematologia E Centro Trapianti Midollo Osseo Br, Ospedale Borgo Roma

Verona, , Italy

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Amsterdam University Medical Center (Amsterdam Umc), Vrije University Medical Center (Vumc)

Amsterdam, , Netherlands

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University Medical Center Groningen

Groningen, , Netherlands

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Leiden University Medical Center (Leids Universitair Medisch Centrum (Lumc))

Leiden, , Netherlands

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Maastricht University Medical Center (Mumc)

Maastricht, , Netherlands

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Erasmus Medisch Centrum 1

Rotterdam, , Netherlands

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Universitair Medisch Centrum Utrecht (Umc Utrecht)

Utrecht, , Netherlands

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Haukeland University Hospital

Bergen, , Norway

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Rikshospitalet University Hospital

Oslo, , Norway

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Ipo Lisboa Francisco Gentil

Lisbon, , Portugal

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Centro Hospitalar Universitario Lisboa Norte Epe

Lisbon, , Portugal

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Instituto Portugues de Oncologia Do Porto Francisco Gentil E.P.E

Porto, , Portugal

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Institut Catala D'Oncologia Badalona, Hospital Germans Trias I Pujol

Badalona, , Spain

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Hospital Universitario Vall D'Hebron

Barcelona, , Spain

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Hospital Clinic de Barcelona (Hospital Clinic I Provincial)

Barcelona, , Spain

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Hospital de La Santa Creu I Sant Pau

Barcelona, , Spain

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Institut Catala D Oncologia

Barcelona, , Spain

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Hospital Clinico Universitario Virgen de La Arrixaca

El Palmar, , Spain

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Hospital Sant Joan de Deu Barcelona - Children'S Hospital

Esplugues de Llobregat, , Spain

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Hospital Universitario Virgen de Las Nieves

Granada, , Spain

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Hospital Universitario de Gran Canaria Doctor Negrín

Las Palmas de Gran Canaria, , Spain

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Hospital General Universitario Gregorio Maranon (Hgugm)

Madrid, , Spain

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Hospital Universitario Infantil Nino Jesus

Madrid, , Spain

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Hospital Universitario Ramon Y Cajal

Madrid, , Spain

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Hospital Universitario 12 de Octubre

Madrid, , Spain

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Hospital Regional Universitario de Malaga

Málaga, , Spain

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Hospital Clinico Universitario de Salamanca

Salamanca, , Spain

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Hospital Universitario de Donostia

SAN Sebasttian, , Spain

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Hospital Universitario Marques de Valdecilla

Santander, , Spain

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Hospital Universitario Virgen Del Rocio

Seville, , Spain

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Hospital Clinico Universitario de Valencia (Instituto de Investigacion Sanitaria Incliva)

Valencia, , Spain

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Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia

Valencia, , Spain

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Hospital Universitario Miguel Servet

Zaragoza, , Spain

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Sahlgrenska University Hospital

Gothenburg, , Sweden

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University of Gothenburg and Queen Silvia Children S Hospital At Sahlgrenska University Hospital

Gothenburg, , Sweden

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Skanes Universitetssjukhus - Universitetssjukhuset I Lund

Lund, , Sweden

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Karolinska Universitetssjukhuset, Centrum For Cellterapi Och Allogen Stamcellstransplantation

Stockholm, , Sweden

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Uppsala University Hospital

Uppsala, , Sweden

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University Hospital Basel

Basel, , Switzerland

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Hopitaux Universitaires de Geneve (Hug) - Centre de Recherche Clinique (Crc)

Geneva, , Switzerland

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Universitatsspital Zurich

Zurich, , Switzerland

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Universitats-Kinderspital Zurich Eleonorenstiftung

Zurich, , Switzerland

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Cardiff and Vale Nhs Trust - University Hospital of Wales (Uhw)

Cardiff, , United Kingdom

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Queen Elizabeth University Hospital

Glasgow, , United Kingdom

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St James'S University Hospital - Leeds Teaching Hospitals Nhs Trust

Leeds, , United Kingdom

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University Hospitals of Leicester Nhs Trust-Leicester Royal Infirmary

Leicester, , United Kingdom

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King'S College Hospital Nhs Foundation Trust

London, , United Kingdom

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Countries

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Austria Belgium Czechia Finland France Germany Greece Ireland Italy Netherlands Norway Portugal Spain Sweden Switzerland United Kingdom

Related Links

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Other Identifiers

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2024-518973-32-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

INCA034176-355

Identifier Type: -

Identifier Source: org_study_id

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