A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

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

This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855. This includes the buildup of antibodies against FVIII which may stop BAX855 from working properly. Another aim is to learn how well BAX855 controls bleeding. In this study, the children can receive BAX855 either as preventative treatment (prophylaxis), or as needed to treat bleeding (on-demand). In case a participant develops antibodies, treatment will be provided as part of the study.

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, ITI
How many participants are being enrolled?   120
When is the trial being conducted?    - 
How long is participation in the trial?   Participants will stay in the study for up to 2 years. Participants may be contacted by their study doctor for up to 3 months after they have completed the study for additional information. Participants who benefit from BAX 855 may stay in the study for longer and continue to receive this medicine until the medicine is available commercially or has not been licensed in the participant’s country at the time the study is completed globally.

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): Part A (Main Study): Participants age <3 years and not experienced two joint bleeds will receive on- demand treatment of 10-80 international unit per kilogram (IU/kg) of BAX 855 intravenously depending on the severity of the bleeding episode and age <3 years or after a maximum of two joint bleeds will receive prophylaxis treatment with dose of 25-80 IU/kg of BAX855 IV (based on investigator discretion) once weekly for up to 100 EDs. Part A (Surgery): In participants, the administration of BAX 855 will be individualized based on the participants IR and half-life. For major surgery to achieve the target level of 80-100% FVIII in plasma of normal FVIII level and for minor surgery >=30-60% FVIII levels for dental or other invasive surgery. Part B (Immune tolerance induction [ITI]): Participants will receive prophylaxis treatment of 100-200 IU/kg BAX 855 IV daily or 50 IU/kg three time in a week and will be reduced to twice weekly to maintain FVIII trough level of 1% for further 3 months.
Intervention:   PEGylated Recombinant Factor VIII, ITI
Primary Outcome Measure(s):   
Number of Participants With 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]
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Throughout Part A and Part B of the study, approximately 7 years]
Number of Participants With Clinically Significant Changes in Vital Signs [Time Frame: Throughout Part A and Part B of the study, approximately 7 years]
Number of Participants With 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 Needed for the Treatment of Bleeding Episodes [Time Frame: Throughout Part A of the study, approximately 5 years]
Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Initiation of Treatment [Time Frame: 24 hours after study drug administration]
Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Bleed Resolution [Time Frame: From start of study treatment up to bleed resolution (approximately 5 years)]
Number of Participants With Weight-adjusted Consumption of BAX 855 [Time Frame: Throughout Part A of the study, approximately 5 years]
Number of Infusions During Weight-adjusted Consumption of BAX 855 [Time Frame: Throughout Part A of the study, approximately 5 years]
Number of Participants With Hemostatic Efficacy in Case of Surgery [Time Frame: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)]
Blood Loss Per Participant in Case of Surgery [Time Frame: Surgery Day 0 up to postoperative Day 14 or discharge (whichever occurs first)]
Incremental Recovery (IR) of BAX 855 [Time Frame: Pre-infusion within 30 minutes; and post-infusion at 15-30 minutes and 24-48 hours]
Half-life (T1/2) of BAX 855 [Time Frame: Pre-infusion, 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) [Time Frame: Up to 33 months]
Immune Tolerance Induction (ITI) - Weight-adjusted Consumption of BAX 855 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:
- Children 6 years or younger can take part. - Must have severe hemophilia A - Must not have been previously treated with BAX855 or other FVIII concentrates. - Cannot have been diagnosed with a bleeding condition other than hemophilia A Additional entry criteria will be discussed with the study doctor.

Trial Locations

Any
Recruiting
Suspended
Withdrawn
Any
RESULTS
Phoenix Childrens Hospital

1919 E Thomas Rd, Phoenix, AZ 85016, USA

Recruiting
1,557 miles (2,506 km)  awayfrom
Canada
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Kaiser Permanente Oakland M.C.

10050 N Wolfe Rd, Cupertino, CA 95014, USA

Site Contact
Recruiting
1,472 miles (2,369 km)  awayfrom
Canada
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Kaiser Permanente Oakland M.C.

3600 Broadway, Oakland, CA 94611, USA

Site Contact
Recruiting
1,447 miles (2,329 km)  awayfrom
Canada
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Kaiser Permanente Oakland M.C.

1600 Eureka Rd, Roseville, CA 95661, USA

Site Contact
Recruiting
1,366 miles (2,198 km)  awayfrom
Canada
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UC Davis Health System

2315 Stockton Blvd, Sacramento, CA 95817, USA

Recruiting
1,382 miles (2,225 km)  awayfrom
Canada
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Connecticut Children's Med Ctr

282 Washington St, Hartford, CT 06106, USA

Site Contact
Recruiting
1,761 miles (2,834 km)  awayfrom
Canada
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Univ Florida College Medicine

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

Recruiting
2,130 miles (3,427 km)  awayfrom
Canada
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Center for Advanced Pediatrics

1400 Tullie Rd NE, Atlanta, GA 30329, USA

Recruiting
1,818 miles (2,926 km)  awayfrom
Canada
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Ann & Robert H. Lurie Children's H

225 E Chicago Ave, Chicago, IL 60611, USA

Recruiting
1,248 miles (2,009 km)  awayfrom
Canada
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Bleeding and Clotting Dis.Inst.

9128 N Lindbergh Dr, Peoria, IL 61615, USA

Recruiting
1,257 miles (2,023 km)  awayfrom
Canada
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UMHS

2903 Baxter Rd, Ann Arbor, MI 48109, USA

Recruiting
1,347 miles (2,168 km)  awayfrom
Canada
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New York Presbyterian Hospital

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

Recruiting
1,763 miles (2,837 km)  awayfrom
Canada
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Novant Health Presbyterian Medical Center

200 Hawthorne Ln, Charlotte, NC 28204, USA

Recruiting
1,835 miles (2,954 km)  awayfrom
Canada
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Wake Forest Baptist Medical Center

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

Recruiting
1,801 miles (2,899 km)  awayfrom
Canada
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Cincinnati Children's Hospital

3333 Burnet Ave, Cincinnati, OH 45229, USA

Recruiting
1,498 miles (2,410 km)  awayfrom
Canada
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Rainbow Babies/Childrens Htl

11100 Euclid Ave, Cleveland, OH 44106, USA

Recruiting
1,458 miles (2,347 km)  awayfrom
Canada
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Penn State MS Hershey Med Ctr

500 University Dr, Hershey, PA 17033, USA

Recruiting
1,691 miles (2,722 km)  awayfrom
Canada
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Texas Tech University Health Sciences Center

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

Recruiting
1,646 miles (2,648 km)  awayfrom
Canada
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Texas Children's Hospital

6621 Fannin St, Houston, TX 77030, USA

Recruiting
1,859 miles (2,992 km)  awayfrom
Canada
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Primary Children's Hospital

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

Recruiting
1,060 miles (1,706 km)  awayfrom
Canada
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HUDERF

Avenue Jean Joseph Crocq 15, 1020 Bruxelles, Belgium

Recruiting
4,064 miles (6,540 km)  awayfrom
Canada
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Cliniques Uni Saint-Luc

Avenue Hippocrate 10, 1200 Bruxelles, Belgium

Recruiting
4,069 miles (6,549 km)  awayfrom
Canada
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Univ. Ziekenhuis Gent Apotheek

Zwijnaardsesteenweg 566, 9000 Gent, Belgium

Recruiting
4,040 miles (6,502 km)  awayfrom
Canada
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Universitair Ziekenhuis Leuven

Herestraat 49, 3000 Leuven, Belgium

Recruiting
4,074 miles (6,556 km)  awayfrom
Canada
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UMHAT Sv. Georgi, EAD

4000 Plovdiv, Bulgaria

Recruiting
5,089 miles (8,190 km)  awayfrom
Canada
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SHAT Oncohaematology Diseases

Sofia, Bulgaria

Recruiting
5,022 miles (8,082 km)  awayfrom
Canada
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MHAT Sv. Marina, EAD

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

Recruiting
5,092 miles (8,195 km)  awayfrom
Canada
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Kaye Edmonton Clinic

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

Recruiting
343 miles (551 km)  awayfrom
Canada
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McMaster Health Science

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

Recruiting
1,426 miles (2,295 km)  awayfrom
Canada
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Rigshospitalet Copenhagen

Blegdamsvej 9, 2100 København, Denmark

Recruiting
4,005 miles (6,445 km)  awayfrom
Canada
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Helsinki Univ Hospital

Haartmaninkatu 4 Rakennus 1, 00290 Helsinki, Finland

Recruiting
3,994 miles (6,428 km)  awayfrom
Canada
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CHU CAEN Hopital Cote de Nacre

Avenue de la Côte de Nacre, 14000 Caen, France

Recruiting
4,023 miles (6,474 km)  awayfrom
Canada
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Essais cliniques CHU Rennes

2 Rue Henri le Guilloux, 35000 Rennes, France

Recruiting
4,041 miles (6,504 km)  awayfrom
Canada
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Hopital Necker Enfants Malades

149 Rue de Sèvres, 75015 Paris, France

Recruiting
4,119 miles (6,628 km)  awayfrom
Canada
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Hopital Jeanne de Flandre - CHU Lille

Avenue Eugène Avinée, 59000 Lille, France

Recruiting
4,044 miles (6,508 km)  awayfrom
Canada
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CHU de Rouen

37 Boulevard Gambetta, 76000 Rouen, France

Recruiting
4,053 miles (6,522 km)  awayfrom
Canada
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Werlhof-Institut GmbH

Schillerstraße 23, 30159 Hannover, Germany

Recruiting
4,123 miles (6,635 km)  awayfrom
Canada
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Inst. f. Experimentelle

Sigmund-Freud-Straße 25, 53127 Bonn, Germany

Recruiting
4,148 miles (6,676 km)  awayfrom
Canada
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Klinik F.Haematologie,Onkologie

Moorenstraße 5, 40225 Düsseldorf, Germany

Recruiting
4,113 miles (6,619 km)  awayfrom
Canada
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Poliklinik PaediaHaematologie

Hamburg, Germany

Recruiting
4,064 miles (6,540 km)  awayfrom
Canada
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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

Recruiting
6,570 miles (10,573 km)  awayfrom
Canada
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Chinese University Of Hong Kong

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

Recruiting
6,558 miles (10,555 km)  awayfrom
Canada
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Belgyogyaszat Onkohaematologia

Budapest, Hungary

Recruiting
4,631 miles (7,452 km)  awayfrom
Canada
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Debreceni Egyetem

Debrecen, Egyetem tér 1, 4032 Hungary

Recruiting
4,687 miles (7,543 km)  awayfrom
Canada
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Presidio Ospedaliero F. Alessi

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

Recruiting
5,125 miles (8,248 km)  awayfrom
Canada
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Azienda Ospedaliera Universitaria Careggi

Largo Piero Palagi, 1, 50139 Firenze FI, Italy

Recruiting
4,650 miles (7,483 km)  awayfrom
Canada
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Ospedale Maggiore Policlinico

Via Francesco Sforza, 35, 20122 Milano MI, Italy

Recruiting
4,499 miles (7,240 km)  awayfrom
Canada
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Umberto I Pol. di Roma-Università di Roma La Sapienza

Piazzale Aldo Moro, 5, 00185 Roma RM, Italy

Recruiting
4,795 miles (7,717 km)  awayfrom
Canada
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Eulji University Hospital

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

Recruiting
5,356 miles (8,620 km)  awayfrom
Canada
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Severance Hospital, Yonsei

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

Recruiting
5,294 miles (8,519 km)  awayfrom
Canada
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Kyung Hee University Hospital

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

Recruiting
5,289 miles (8,512 km)  awayfrom
Canada
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Ulsan University Hospital

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

Recruiting
5,354 miles (8,616 km)  awayfrom
Canada
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Hospital Ampang

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

Recruiting
8,090 miles (13,020 km)  awayfrom
Canada
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Hospital HRPB

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

Recruiting
8,006 miles (12,884 km)  awayfrom
Canada
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Hospital Pulau Pinang

Jalan Residensi, 10990 George Town, Pulau Pinang, Malaysia

Recruiting
7,967 miles (12,822 km)  awayfrom
Canada
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Hospital Kuala Lumpur

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

Recruiting
8,089 miles (13,017 km)  awayfrom
Canada
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Hospital Umum Sarawak

Jalan Hospital, 93586 Kuching, Sarawak, Malaysia

Recruiting
7,997 miles (12,869 km)  awayfrom
Canada
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Hospital Sultanah Nur Zahirah

20400 Kuala Terengganu, Terengganu, Malaysia

Recruiting
7,917 miles (12,741 km)  awayfrom
Canada
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Universitair Medisch Centrum Groningen (UMCG)

Hanzeplein 1, 9713 GZ Groningen, Netherlands

Recruiting
3,997 miles (6,432 km)  awayfrom
Canada
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Oslo Universitetssykehus - Rikshospitalet

Sognsvannsveien 20, 0372 Oslo, Norway

Recruiting
3,730 miles (6,003 km)  awayfrom
Canada
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NUS YLL School of Medicine

10 Medical Dr, Singapore 117597

Recruiting
8,168 miles (13,146 km)  awayfrom
Canada
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KKH

100 Bukit Timah Rd, Singapore 229899

Recruiting
8,166 miles (13,142 km)  awayfrom
Canada
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Hospital Univ. Son Espases

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

Site Contact
Recruiting
4,639 miles (7,465 km)  awayfrom
Canada
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HOSPITAL A Coruna

Xubias de Arriba, 84, 15006 A Coruña, Spain

Recruiting
4,083 miles (6,571 km)  awayfrom
Canada
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Hospital Universitario La Paz

Paseo de la Castellana, 261, 28046 Madrid, Spain

Recruiting
4,389 miles (7,064 km)  awayfrom
Canada
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Hospital Univ del Rio Hortega

Calle Dulzaina, 2, 47012 Valladolid, Spain

Recruiting
4,294 miles (6,910 km)  awayfrom
Canada
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Kaohsiung Chung- Ho Memorial Hosp

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

Recruiting
6,393 miles (10,289 km)  awayfrom
Canada
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China Medical University Hospital

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

Recruiting
6,289 miles (10,120 km)  awayfrom
Canada
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Taichung Veterans General

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

Recruiting
6,289 miles (10,121 km)  awayfrom
Canada
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Tri-Service General Hospital

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

Recruiting
6,207 miles (9,990 km)  awayfrom
Canada
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Siriraj Hospital

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

Recruiting
7,408 miles (11,923 km)  awayfrom
Canada
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King Chulalongkorn Memorial

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

Recruiting
7,409 miles (11,924 km)  awayfrom
Canada
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Ramathibodi Hospital

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

Recruiting
7,407 miles (11,921 km)  awayfrom
Canada
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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

Recruiting
7,097 miles (11,422 km)  awayfrom
Canada
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Srinagarind Hospital

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

Recruiting
7,186 miles (11,565 km)  awayfrom
Canada
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Acibadem Adana Hospital

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

Recruiting
5,641 miles (9,078 km)  awayfrom
Canada
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Hacettepe Üniversitesi

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

Recruiting
5,403 miles (8,696 km)  awayfrom
Canada
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Akdeniz Universitesi

Akdeniz Ünv., Konyaaltı/Antalya, Turkey

Recruiting
5,550 miles (8,932 km)  awayfrom
Canada
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Uludag Universitesi Tip Fakültesi

Görükle Kampüsü, Özlüce, 16059 Nilüfer/Bursa, Turkey

Recruiting
5,301 miles (8,531 km)  awayfrom
Canada
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Istanbul Üniversitesi Cerrahpaşa

Cerrahpaşa, 34098 Fatih/İstanbul, Turkey

Recruiting
5,253 miles (8,454 km)  awayfrom
Canada
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Ege Universitesi Tip Fakultesi

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

Recruiting
5,375 miles (8,650 km)  awayfrom
Canada
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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

Recruiting
5,535 miles (8,908 km)  awayfrom
Canada
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19 Mayis Universitesi

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

Recruiting
5,376 miles (8,651 km)  awayfrom
Canada
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MI Cherkasy Reg Onc Dis of CRC

Michigan, USA

Recruiting
1,179 miles (1,898 km)  awayfrom
Canada
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SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

Lviv, Lviv Oblast, Ukraine, 79000

Recruiting
4,601 miles (7,404 km)  awayfrom
Canada
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CI Zaporizhzhia Reg CCH of ZRC

Zaporizhzhia, Zaporizhia Oblast, Ukraine, 69061

Recruiting
4,941 miles (7,951 km)  awayfrom
Canada
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Royal Manchester Children's Hospital

Oxford Rd, Manchester M13 9WL, UK

Recruiting
3,749 miles (6,033 km)  awayfrom
Canada
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Univ Hospital Southampton

Coxford Rd, Southampton SO16 5YA, UK

Recruiting
3,901 miles (6,278 km)  awayfrom
Canada
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Bristol Royal H. for Children

Upper Maudlin St, Bristol BS2 8BJ, UK

Recruiting
3,841 miles (6,181 km)  awayfrom
Canada
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Evelina Children's Hospital - St Thomas' Hospital

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

Recruiting
3,909 miles (6,291 km)  awayfrom
Canada
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Medizinische Universitat Wien

Spitalgasse 23, 1090 Wien, Austria

Currently On Hold
4,524 miles (7,280 km)  awayfrom
Canada
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Fakultni nemocnice v Motole

V Úvalu 84, 150 06 Praha 5, Czechia

Terminated/Withdrawn
4,367 miles (7,028 km)  awayfrom
Canada
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View all (92)
Trial Protocol ID: 261203
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