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Safety, Immunogenicity and Hemostatic Efficacy of PEGylated Recombinant FVIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years of Age With Severe Hemophilia A

IDENTIFIERS
Trial Protocol: 261203
Clinicaltrials.gov: NCT02615691
EudraCT: 2015-002136-40
PURPOSE

The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion.

Recruiting
US
AT
BE
21+
BG
CA
CZ
DK
FI
FR
DE
HK
HU
IT
KR
MY
NL
NO
SG
ES
TW
TH
TR
UA
GB

Trial at a glance

What medical condition is being studied?   Hemophilia A
What is the trial testing?   PEGylated Recombinant Factor VIII
How many participants are being enrolled?   120
When is the trial being conducted?    - 

Key requirements

Sex   
All
Age   
Up to 5 Years
Accepts Healthy Volunteers?   
No

Trial Details / Participation Requirements

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Trial phase:   Phase 3
Trial Type:   Interventional
Trial Arms/Groups or Cohorts:   
Previously untreated patients (PUPs): <6 years of age with severe hemophilia A (baseline Factor VIII (FVIII) level < 1%)
Intervention:   PEGylated Recombinant Factor VIII
Primary Outcome Measure(s):   
Incidence of FVIII inhibitor development [Time Frame: Throughout Part A of the study, approximately 5 years]
Success rate of Immune tolerance induction (ITI) [Time Frame: Up to 33 months]
Secondary Outcome Measure(s):   
Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) antibodies [Time Frame: Throughout Part A of the study, approximately 5 years]
Clinically significant adverse events (AEs) and serious adverse events (SAEs) [Time Frame: Throughout Part A and Part B of the study, approximately 7 years]
Clinically significant changes in vital signs [Time Frame: Throughout Part A and Part B of the study, approximately 7 years]
Clinically significant changes in clinical laboratory parameters [Time Frame: Throughout Part A and Part B of the study, approximately 7 years]
Annualized bleeding rate (ABR) for prophylactic and on-demand treatment [Time Frame: Throughout Part A of the study, approximately 5 years]
Number of BAX 855 infusions per bleeding episode [Time Frame: Throughout Part A of the study, approximately 5 years]
Overall hemostatic efficacy rating [Time Frame: 24 h after initiation of treatment and at resolution of bleed]
Weight-adjusted consumption of BAX 855 per month, per year and per event [Time Frame: Throughout Part A of the study, approximately 5 years]
Number of infusions per month and per year [Time Frame: Throughout Part A of the study, approximately 5 years]
Assessment of intra-, post- and perioperative hemostatic efficacy in case of surgery [Time Frame: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)]
Intra- and postoperative blood loss in case of surgery [Time Frame: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)]
Pharmacokinetics- Incremental recovery (IR) at baseline and over time [Time Frame: Pre-infusion within 30 minutes; and post-infusion at 15-30 minutes and 24-48 hours]
Pharmacokinetics- Half-life (T1/2) at baseline (optional) [Time Frame: Post-infusion: 15-30 minutes and 24-48 hours]
Immune tolerance induction (ITI) - Rate of partial success and failure of ITI [Time Frame: Up to 33 months]
Immune tolerance induction (ITI) - annualized bleeding rate (ABR) during ITI [Time Frame: Up to 33 months]
Immune tolerance induction (ITI) - Weight-adjusted consumption of BAX 855 per month and per year for each ITI regimen employed [Time Frame: Up to 33 months]
Immune tolerance induction (ITI) - Catheter-related complications [Time Frame: Up to 33 months]
Immune tolerance induction (ITI) -Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) antibodies [Time Frame: Up to 33 months]
Entry Criteria:
Inclusion Criteria 1. Participant is < 6 years old at the time of screening 2. Participant is previously untreated with < 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion at any time prior to screening 3. Participant has severe hemophilia A (Factor VIII (FVIII) < 1%) as determined by the central laboratory, or a historical FVIII level < 1% as determined at any local laboratory, optionally supported by an additional FVIII gene mutation consistent with severe hemophilia A 4. Participant is immune competent with a CD4+ count > 200 cells/mm^3, as confirmed by the central laboratory at screening 5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocol Additional inclusion criteria for Part B (immune tolerance induction (ITI)) 1. Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion 2. Participant has a confirmed positive high titer inhibitor (> 5.00 Bethesda unit (BU)) or has a positive confirmed low titer inhibitor (≥ 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with a. poorly controlled bleeding despite increased BAX 855 doses, or b. requires bypassing agents to treat bleeding Exclusion Criteria 1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening 2. Participant has a history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening 3. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease) 4. Participant has been previously treated with any type of FVIII concentrate other than ADVATE or BAX 855, or was administered ADVATE, BAX 855 or plasma transfusion for ≥ 3 EDs at any time prior to screening 5. Participant receives >2 EDs of ADVATE in total during the periods prior to enrollment and during the screening period, until the baseline infusion. 6. The participant’s weight is anticipated to be < 5 kg at the baseline visit 7. Participant’s platelet count is < 100,000/mL 8. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG) or Tween 80 9. Participant has severe chronic hepatic dysfunction [eg, > 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a documented international normalized ratio (INR) > 1.5] in his medical history or at the time of screening 10. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal) 11. Participant has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation 12. Participant is scheduled to receive during the course of the study a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day or α-interferon) other than anti-retroviral chemotherapy 13. Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study 14. Parent or legally authorized representative has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance 15. Parent, legally authorized representative or participant are a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study. Additional exclusion criteria for Part B (ITI) 1. Spontaneous disappearance of the inhibitor prior to ITI 2. FVIII inhibitor titer ≥ 0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory 3. Inability or unwillingness to comply with the protocol

Trial Locations

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RESULTS
Phoenix Childrens Hospital

1919 E Thomas Rd, Phoenix, AZ 85016, USA

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Kaiser Permanente Oakland M.C.

10050 N Wolfe Rd, Cupertino, CA 95014, USA

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Kaiser Permanente Oakland M.C.

3600 Broadway, Oakland, CA 94611, USA

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Kaiser Permanente Oakland M.C.

1600 Eureka Rd, Roseville, CA 95661, USA

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UC Davis Health System

2315 Stockton Blvd, Sacramento, CA 95817, USA

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Connecticut Children's Med Ctr

282 Washington St, Hartford, CT 06106, USA

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Univ Florida College Medicine

2015 SW 16th Ave, Gainesville, FL 32608, USA

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Center for Advanced Pediatrics

1400 Tullie Rd NE, Atlanta, GA 30329, USA

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Ann & Robert H. Lurie Children's H

225 E Chicago Ave, Chicago, IL 60611, USA

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Bleeding and Clotting Dis.Inst.

9128 N Lindbergh Dr, Peoria, IL 61615, USA

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UMHS

2903 Baxter Rd, Ann Arbor, MI 48109, USA

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New York Presbyterian Hospital

525 E 68th St, New York, NY 10065, USA

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Novant Health Presbyterian Medical Center

200 Hawthorne Ln, Charlotte, NC 28204, USA

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Wake Forest Baptist Medical Center

1 Medical Center Blvd, Winston-Salem, NC 27157, USA

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Cincinnati Children's Hospital

3333 Burnet Ave, Cincinnati, OH 45229, USA

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Rainbow Babies/Childrens Htl

11100 Euclid Ave, Cleveland, OH 44106, USA

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Penn State MS Hershey Med Ctr

500 University Dr, Hershey, PA 17033, USA

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Texas Tech University Health Sciences Center

4801 Alberta Ave 3rd fl, El Paso, TX 79905, USA

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Texas Children's Hospital

6621 Fannin St, Houston, TX 77030, USA

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Primary Children's Hospital

100 Mario Capecchi Dr, Salt Lake City, UT 84113, USA

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HUDERF

Avenue Jean Joseph Crocq 15, 1020 Bruxelles, Belgium

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Cliniques Uni Saint-Luc

Avenue Hippocrate 10, 1200 Bruxelles, Belgium

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Univ. Ziekenhuis Gent Apotheek

Zwijnaardsesteenweg 566, 9000 Gent, Belgium

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Universitair Ziekenhuis Leuven

Herestraat 49, 3000 Leuven, Belgium

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UMHAT Sv. Georgi, EAD

4000 Plovdiv, Bulgaria

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SHAT Oncohaematology Diseases

Sofia, Bulgaria

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MHAT Sv. Marina, EAD

bul. "Hristo Smirnenski" 1, 9010 Varna, Bulgaria

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Kaye Edmonton Clinic

11400 University Ave, Edmonton, AB T6G 1Z1, Canada

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McMaster Health Science

1200 Main St W, Hamilton, ON L8N 3Z5, Canada

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Rigshospitalet Copenhagen

Blegdamsvej 9, 2100 København, Denmark

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Helsinki Univ Hospital

Haartmaninkatu 4 Rakennus 1, 00290 Helsinki, Finland

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Essais cliniques CHU Rennes

2 Rue Henri le Guilloux, 35000 Rennes, France

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Hopital Necker Enfants Malades

149 Rue de Sèvres, 75015 Paris, France

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Hopital Jeanne de Flandre - CHU Lille

Avenue Eugène Avinée, 59000 Lille, France

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CHU de Rouen

37 Boulevard Gambetta, 76000 Rouen, France

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Werlhof-Institut GmbH

Schillerstraße 23, 30159 Hannover, Germany

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Inst. f. Experimentelle

Sigmund-Freud-Straße 25, 53127 Bonn, Germany

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Klinik F.Haematologie,Onkologie

Moorenstraße 5, 40225 Düsseldorf, Germany

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Poliklinik PaediaHaematologie

Hamburg, Germany

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The University of Hong Kong Queen Mary Hospital

Queen Mary Hospital Main Block, Pok Fu Lam Rd, Pok Fu Lam, Hong Kong

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Chinese University Of Hong Kong

The Chinese University of Hong Kong, Central Ave, Ma Liu Shui, Hong Kong

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Belgyogyaszat Onkohaematologia

Budapest, Hungary

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Debreceni Egyetem

Debrecen, Egyetem tér 1, 4032 Hungary

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Presidio Ospedaliero F. Alessi

Piazza Santa Maria di Gesù, 5, 95124 Catania CT, Italy

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Azienda Ospedaliera Universitaria Careggi

Largo Piero Palagi, 1, 50139 Firenze FI, Italy

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Ospedale Maggiore Policlinico

Via Francesco Sforza, 35, 20122 Milano MI, Italy

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Umberto I Pol. di Roma-Università di Roma La Sapienza

Piazzale Aldo Moro, 5, 00185 Roma RM, Italy

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Eulji University Hospital

95 Dunsanseo-ro, Dunsan-dong, Seo-gu, Daejeon, South Korea

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Severance Hospital, Yonsei

50-1 Yonsei-ro, Sinchon-dong, Seodaemun-gu, Seoul, South Korea

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Kyung Hee University Hospital

892 Dongnam-ro, Sangil-dong, Gangdong-gu, Seoul, South Korea

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Ulsan University Hospital

877 Bangeojinsunhwan-doro, Jeonha-dong, Dong-gu, Ulsan, South Korea

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Hospital Ampang

Hospital Ampang, Jalan Mewah Utara, Taman Pandan Mewah, 68000 Ampang Jaya, Selangor, Malaysia

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Hospital HRPB

Jalan Raja Ashman Shah, 30450 Ipoh, Negeri Perak, Malaysia

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Hospital Pulau Pinang

Jalan Residensi, 10990 George Town, Pulau Pinang, Malaysia

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Hospital Kuala Lumpur

23, Jalan Pahang, 50586 Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia

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Hospital Umum Sarawak

Jalan Hospital, 93586 Kuching, Sarawak, Malaysia

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Hospital Sultanah Nur Zahirah

20400 Kuala Terengganu, Terengganu, Malaysia

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Universitair Medisch Centrum Groningen (UMCG)

Hanzeplein 1, 9713 GZ Groningen, Netherlands

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Oslo Universitetssykehus - Rikshospitalet

Sognsvannsveien 20, 0372 Oslo, Norway

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NUS YLL School of Medicine

10 Medical Dr, Singapore 117597

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KKH

100 Bukit Timah Rd, Singapore 229899

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Hospital Univ. Son Espases

Carretera de Valldemossa, 79, 07120 Palma, Illes Balears, Spain

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HOSPITAL A Coruna

Xubias de Arriba, 84, 15006 A Coruña, Spain

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Hospital Universitario La Paz

Paseo de la Castellana, 261, 28046 Madrid, Spain

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Hospital Univ del Rio Hortega

Calle Dulzaina, 2, 47012 Valladolid, Spain

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Kaohsiung Chung- Ho Memorial Hosp

No. 100號, Ziyou 1st Road, Sanmin District, Kaohsiung City, Taiwan 80756

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China Medical University Hospital

No. 2, Yude Road, North District, Taichung City, Taiwan 404

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Taichung Veterans General

No. 1650號, Section 4, Taiwan Boulevard, Xitun District, Taichung City, Taiwan 40705

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Tri-Service General Hospital

No. 325號, Section 2, Chenggong Road, Neihu District, Taipei City, Taiwan 114

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Siriraj Hospital

2 Thanon Wang Lang, Khwaeng Siriraj, Khet Bangkok Noi, Krung Thep Maha Nakhon 10700, Thailand

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King Chulalongkorn Memorial

1873 Rama IV Rd, Khwaeng Pathum Wan, Khet Pathum Wan, Krung Thep Maha Nakhon 10330, Thailand

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Ramathibodi Hospital

270 Thanon Rama VI, Khwaeng Thung Phaya Thai, Khet Ratchathewi, Krung Thep Maha Nakhon 10400, Thailand

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Maharaj Nakorn Chiang Mai

110 ถนนอินทวโรรส ซอย 2 Tambon Su Thep, Amphoe Mueang Chiang Mai, Chang Wat Chiang Mai 50200, Thailand

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Srinagarind Hospital

Nai Mueang, Mueang Khon Kaen District, Khon Kaen 40000, Thailand

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Acibadem Adana Hospital

Döşeme, Cumhuriyet Cd. No:66, 01130 Seyhan/Adana, Turkey

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Hacettepe Üniversitesi

Hacettepe, Hacettepe Ünv., 06230 Altındağ/Ankara, Turkey

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Akdeniz Universitesi

Akdeniz Ünv., Konyaaltı/Antalya, Turkey

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Uludag Universitesi Tip Fakültesi

Görükle Kampüsü, Özlüce, 16059 Nilüfer/Bursa, Turkey

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Istanbul Üniversitesi Cerrahpaşa

Cerrahpaşa, 34098 Fatih/İstanbul, Turkey

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Ege Universitesi Tip Fakultesi

Kazımdirik, Ege Ünv. Hst. No:9, 35100 Bornova/İzmir, Turkey

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Erciyes Univers Tip Fakultesi

Köşk Mahallesi Prof. Dr. Turhan Feyzioğlu Cad. Erciyes Üniversitesi Sağlık Uygulama ve Araştırma Merkezi No:42, Köşk, 38039 Melikgazi/Kayseri, Turkey

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19 Mayis Universitesi

Körfez, 19 Mayıs Ünv., 55270 Atakum/Samsun, Turkey

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MI Cherkasy Reg Onc Dis of CRC

Michigan, USA

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SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

Lviv, Lviv Oblast, Ukraine, 79000

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CI Zaporizhzhia Reg CCH of ZRC

Zaporizhzhia, Zaporizhia Oblast, Ukraine, 69061

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Royal Manchester Children's Hospital

Oxford Rd, Manchester M13 9WL, UK

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Univ Hospital Southampton

Coxford Rd, Southampton SO16 5YA, UK

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Bristol Royal H. for Children

Upper Maudlin St, Bristol BS2 8BJ, UK

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Evelina Children's Hospital - St Thomas' Hospital

Westminster Bridge Rd, South Bank, London SE1 7EH, UK

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Medizinische Universitat Wien

Spitalgasse 23, 1090 Wien, Austria

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Fakultni nemocnice v Motole

V Úvalu 84, 150 06 Praha 5, Czechia

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CHU CAEN Hopital Cote de Nacre

Avenue de la Côte de Nacre, 14000 Caen, France

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Trial Protocol ID: 261203
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