Factor VIII DeficiencyPedNetNCT02979119
The European Paediatric Network for Haemophilia Management ( PedNet Registry)
An observational study of Factor VIII Deficiency and Factor IX Deficiency, run by PedNet Haemophilia Research Foundation at 39 sites in 19 countries.
Trial summary
Rationale: Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Factor VIII Deficiency, Factor IX Deficiency
- Type of treatment
- Injection or infusion
- How many people
- 4,000 participants
- Where
- 39 sitesin 19 countries
- Study dates
- Jun 2014to Dec 2039
- How long will I take part?
- 18 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2000 until January 1st 2030 who have been or are to be treated in one of the participating centres
Who may be able to join
- Diagnosed with Haemophilia A or B
- Factor VIII/ IX activity of <1 to 25%
- Complete records of Factor treatment and bleeds
- Treated in one of the participating centres
Who cannot join
- Patients referred because of an inhibitor*
- Informed consent not obtained
Trial locations and contacts
39 sites in 19 countries. Contact the study team or a site near you to ask about taking part.
Hermeling contact
39 sites in 19 countries
Germany7 sites, 7 recruiting
- Recruiting
Charité Campus Virchow Klinikum, Klinik für Pädiatrie m.S. Onkologie und Hämatologie
Berlin
Susanne Holzhauer, MD
- Recruiting
Department of Internal Medicine, Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain
Berlin
Robert Klamroth, MD, PhD
- Recruiting
Institut für Experimentelle Hämatologie und Transfusionsmedizin Universitätsklinikum Bonn
Bonn, D-53127
Johannes Oldenburg, MD, PhD
- Recruiting
Klinik Bremen-Mitte Prof.-Hess-Kinderklinik
Bremen, D 28177
Martina Bührlen, MD
- Recruiting
Hämophilie Zentrum Rhein Main
Frankfurt, D-60596
Carmen Escuriola Ettingshausen, MD
- Recruiting
University Hospital Frankfurt & Goethe University - Clinical and Molecular Hemostasis, Department of Pediatrics
Frankfurt am Main
Christoph Königs, MD, PhD
- Recruiting
Dr. v. Haunersches Kinderspital University of Munich
Munich, D-80337
Martin Olivieri, MD
Spain4 sites, 3 recruiting
- Recruiting
Unitat Hemofilia Hospital Vall d'Hebron
Barcelona, 08035
Olga Benítez Hidalgo, MD
- Recruiting
Unidad de Coagulopatías Hospital Universitario La Paz
Madrid, 28046
Maria Theresa Alvarez Roman, MD
- Completed
Hospital General Unidad de Hemofilia 1 Sur Hospitales Universitarios Virgen del Rocio
Seville, 41013
- Recruiting
Unidad de Coagulopatias Congenitas Hospital Universitario la Fe
Valencia, 46009
Ana Rosa Cid, MD
United Kingdom4 sites, 2 recruiting
- Recruiting
Birmingham Children's Hospital NHS Trust - Department of Haematology
Birmingham
Jayashree Motwani, MD
- Completed
Royal Hospital for Sick Children
Edinburgh, EH9 1LF
- Recruiting
Department of Haematology Royal Hospital for Sick Children
Glasgow, G 3885
Fernando Pinto, MD
- Completed
Haemophila Center Great Ormond Street Hospital for Children
London, WC1N3JH
France3 sites, 3 recruiting
- Recruiting
Service Hématologique Centre Regional Traitement d'Hemophilie Bicetre
Le Kremlin-Bicêtre, F-94270
Roseline d'Oiron, MD
- Recruiting
Service d'Hématologie Pédiatrique Hôpital Universitaire La Timone
Marseille Cedex-05, F-13385
Hervé Chambost, MD, PhD
- Recruiting
Centre de traitement des hémophiles Hôpital Universitaire Purpan
Toulouse, F-31052
Caroline Oudot, MD
Italy3 sites, 2 recruiting
- Recruiting
Azienda Ospedaliero Universitaria Careggi
Florence
Giancarlo Castaman, MD
- Recruiting
Gaslini Hospital
Genova, I-16184
Angelo Claudio Molinari, MD
- Completed
A. Bianchi Bonomi Hemophilia and Thrombosis Centre IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
Austria2 sites, 1 recruiting
- Completed
Universitäts-Klinik für Kinder- und Jugendheilkunde
Graz, A-8036
- Recruiting
Medical University of Vienna - Department of Paediatrics
Vienna
Christoph Male, MD
Canada2 sites, 2 recruiting
- Recruiting
Division of Hematology/Oncology Hôpital St Justine
Montreal, H3T 1C5
Natalie Mathews, MD
- Recruiting
Division of Haematology/Oncology Hospital for Sick Children
Toronto, M5G-1X8
Manuel Carcao, MD, PhD
Czechia2 sites, 2 recruiting
- Recruiting
Haemophilia Comprehensive Care Centre, Centre for Thrombosis and Haemostasis Children's University Hospital Brno
Brno, 613 00
Jan Blatny, MD, PhD
- Recruiting
Department of Paediatric Haematology/oncology - University Hospital Motol
Prague, 150 06
Ester Zapotocka, MD
Sweden2 sites, 2 recruiting
- Recruiting
Lund University Hospital
Malmö, S-20502
Nadine Gretenkort Andersson, MD, PhD
- Recruiting
Department of Pediatrics, Clinic of Coag. Disorders Karolinska Hospital
Stockholm, S-17176
Susanna Ranta, MD
Belgium1 site, 1 recruiting
- Recruiting
Service of Pediatric Haematology University Hospital Leuven
Leuven, B-3000
Christel Van Geet, MD, PhD
Denmark1 site, 1 recruiting
- Recruiting
Department of Pediatrics Århus Kommunehospital Skejby Sygehus
Aarhus, DK-8200
Torben Stamm Mikkelsen, MD
Finland1 site, 1 recruiting
- Recruiting
Children's Hospital Helsinki University Hospital
Helsinki, FIN-00029
Satu Langstrom, MD, PhD
Greece1 site, 1 recruiting
- Recruiting
Haemophilia-Haemostasis Unit St. Sophia Children's Hospital
Athens, GR-11527
Helen Pergantou, MD
Ireland1 site, 1 recruiting
- Recruiting
Children's Health Ireland (CHI) at Crumlin
Dublin
Beatrice Nolan, MD
Israel1 site, 1 recruiting
- Recruiting
The National Hemophilia Center Sheba Medical Center, Tel Hashomer
Ramat Gan, 52621
Sarina Levy Mendelovich, MD
Netherlands1 site, 1 recruiting
- Recruiting
Van Creveld Kliniek University Medical Center Utrecht
Utrecht, 3508 GA
Corien Eckhardt, MD, PhD
Norway1 site, 1 recruiting
- Not yet recruiting
Oslo University Hospital
Oslo
Heidi Knüdsen, MD
Portugal1 site, 1 recruiting
- Recruiting
Centro Hospitalar São João, S. Imuno-hemoterapia
Porto
Manuela Carvalho, MD
Switzerland1 site, 1 recruiting
- Recruiting
Inselspital Bern, University Children's Hospital
Bern
Mutlu Kartal-Kaess, MD
About this study
Official title: The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry
Rationale:
Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.
Objective:
To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.
Read the detailed description
Design: Multicenter Prospective Observational Birth Cohort Study
Population:
Patients with haemophilia A and B with FVIII/IX levels of <1 to 25% born between 1-1-2000 and 1-1-2040.
Intervention:
No intervention; only documentation of patient characteristics and parameters of routine patient care and outcome
Main outcome parameters:
Outcome: clinically relevant inhibitor development, bleeding pattern and joint status on physical examination and imaging.
Determinants: baseline FVIII/IX levels, measurement of inhibitory antibodies, family history, FVIII/IX gene mutation, details on replacement therapy (according to each infusion for the first 50 treatment days, and annually thereafter) and surgeries.
Nature and extent of the burden and risks associated with participation, benefit and group relatedness:
- No burden for the patients. Well-defined clinical data will be collected from the medical files. Participating in this registry will not change the number of visits to the clinic. All outcome parameters that are collected (including laboratory results) are part of routine clinical care.
- Direct benefit is not to be expected. However, the direct interaction between centres that treat patients with rare diseases improves both clinical care and will result in better guidelines and as such may provide indirect benefit.
- Multicentre participation: haemophilia is a very rare condition. Therefore, collecting data on a multi-centre observational cohort is the only way to study this specific population.
- The registry concerns young boys and girls with haemophilia and cannot be performed in older patients, as >90% of inhibitors occur develop during the first 50 exposure days, and the results of prophylactic replacement therapy are highly dependent on the initiation of this treatment.
- Conditions
- Factor VIII Deficiency, Factor IX Deficiency
- Keywords
- Hemophilia, Children, Inhibitors, Prophylaxis, Long-term outcome, Congenital
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cohort I | Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2000 until December 31st 2009 who have been or are to be treated with coagulation proteins in one of the participating centres | |
| Cohort II | Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2010 until December 31st 2019 who have been or are to be treated with coagulation proteins in one of the participating centres | |
| Cohort III | Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2020 until December 31st 2029 who have been or are to be treated with coagulation proteins in one of the participating centres | |
| Cohort IV | Cohort IV Group/Cohort Description: Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2030 until December 31st 2039 who have been or are to be treated with coagulation proteins in one of the participating centres |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with antibody development to exogenous clotting factors
Allo-antibodies against Factor VIII and IX; Blood test: measurement in Bethesda units (BU), positive according to local standards, for most labs >0.5 BU
Measured: Until patient reaches age of 18
Secondary outcomes (2)
Long term outcome of haemophilia on joint status using the Hemophilia Joint Health Score (HJHS) and MRI techniques.
Effect of different prophylactic regimen on bleeding and joint damage
Measured: From diagnose every 5 years until patient reaches age of 18
Long term outcome different Immune Tolerance Induction (ITI) therapies in patients with inhibitor.
Effect of different ITI therapies on bleeding and joint damage. Joint damage is assessed using the HJHS and MRI.
Measured: From date first positive inhibitor titer preferably every 3 years until patient reaches age of 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2016 (estimated)
- Study startJun 2014
- Primary completionDec 2039 (estimated)
- Study completionDec 2039 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (34)Papers the sponsor has linked to this study.
- Other trials for Factor VIII DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 4,000 participants (estimated)
- Target duration
- 18 Years
Sponsor and investigators
- Lead sponsor
- PedNet Haemophilia Research Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Gili Kenet, PedNet Haemophilia Research Foundation
- Investigators
- Gili Kenet, PhD, MD, Study director, The National Hemophilia Center Ministry of Health Sheba Medical Center Ramat Gan, IsraelChristoph Male, MD, Study chair, Medical University of ViennaGili Kenet, PhD, MD, Principal investigator, The National Hemophilia Center Ministry of Health Sheba Medical Center Ramat Gan, Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02979119
- Sponsor's protocol ID
- Version 6.4 November 2022
- Acronym
- PedNet
- Status last verified
- Sep 2025
- First submitted
- 21 Oct 2014
- Last updated
- 16 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
de Kovel M, van Haaster AC, Carcao M, Ranta S, Glosli H, Rivard GE, Kenet G, Kurnik K, Van Geet C, Carvalho M, Andersson NG, Kartal-Kaess M, Ljung R, van den Berg HM; PedNet Study Group. Blood Group O Does Not Increase the Risk of Inhibitors in Severe Haemophilia A: Data from the PedNet Study Group. Haemophilia. 2025 May;31(3):419-423. doi: 10.1111/hae.70035. Epub 2025 Mar 23.
PubMed 40123267Carcao M, Konigs C, Andersson NG, de Kovel M, de Boer-Verdonk E, Motwani J, Blatny J, Olivieri M, van den Berg M, Fischer K. Predictors of immune tolerance induction success in 231 children with severe hemophilia A with high-titer inhibitors - lessons learned from the PedNet prospective cohort study. J Thromb Haemost. 2025 Oct;23(10):3134-3147. doi: 10.1016/j.jtha.2025.07.010. Epub 2025 Jul 22.
PubMed 40706963Mendoza A, Rivas I, Hidalgo OB, Cid AR, Olivieri M, Ranta S, Labarque V, Andersson NG, de Kovel M, Alvarez-Roman MT. Impact of Family History of Haemophilia on Diagnosis, Management and Outcomes in Severe Haemophilia. Haemophilia. 2025 Jul;31(4):679-686. doi: 10.1111/hae.70018. Epub 2025 May 30.
PubMed 40444652Ranta S, Zapotocka E, Andersson NG, Fischer K, Kenet G, de Kovel M, Konigs C, Labarque V, Male C, Olivieri M, Motwani J. A survey on clinical practice in monitoring and management of bleeding in children with haemophilia A on emicizumab prophylaxis in the PedNet centres. Thromb Res. 2025 May;249:109307. doi: 10.1016/j.thromres.2025.109307. Epub 2025 Mar 19. No abstract available.
PubMed 40120320van den Berg HM, Gouw SC, van der Bom JG. Factor VIII products and inhibitors in severe hemophilia A. N Engl J Med. 2013 Apr 11;368(15):1457. doi: 10.1056/NEJMc1301995. No abstract available.
PubMed 23574131Gouw SC, van den Berg HM, Fischer K, Auerswald G, Carcao M, Chalmers E, Chambost H, Kurnik K, Liesner R, Petrini P, Platokouki H, Altisent C, Oldenburg J, Nolan B, Garrido RP, Mancuso ME, Rafowicz A, Williams M, Clausen N, Middelburg RA, Ljung R, van der Bom JG; PedNet and Research of Determinants of INhibitor development (RODIN) Study Group. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013 May 16;121(20):4046-55. doi: 10.1182/blood-2012-09-457036. Epub 2013 Apr 3.
PubMed 23553768Carcao MD, van den Berg HM, Ljung R, Mancuso ME; PedNet and the Rodin Study Group. Correlation between phenotype and genotype in a large unselected cohort of children with severe hemophilia A. Blood. 2013 May 9;121(19):3946-52, S1. doi: 10.1182/blood-2012-11-469403. Epub 2013 Mar 12.
PubMed 23482934Clausen N, Petrini P, Claeyssens-Donadel S, Gouw SC, Liesner R; PedNet and Research of Determinants of Inhibitor development (RODIN) Study Group. Similar bleeding phenotype in young children with haemophilia A or B: a cohort study. Haemophilia. 2014 Nov;20(6):747-55. doi: 10.1111/hae.12470. Epub 2014 Jun 3.
PubMed 24893572Fischer K, Ljung R, Platokouki H, Liesner R, Claeyssens S, Smink E, van den Berg HM. Prospective observational cohort studies for studying rare diseases: the European PedNet Haemophilia Registry. Haemophilia. 2014 Jul;20(4):e280-6. doi: 10.1111/hae.12448. Epub 2014 May 2.
PubMed 24784937Gouw SC, van der Bom JG, Ljung R, Escuriola C, Cid AR, Claeyssens-Donadel S, van Geet C, Kenet G, Makipernaa A, Molinari AC, Muntean W, Kobelt R, Rivard G, Santagostino E, Thomas A, van den Berg HM; PedNet and RODIN Study Group. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013 Jan 17;368(3):231-9. doi: 10.1056/NEJMoa1208024.
PubMed 23323899Andersson NG, Auerswald G, Barnes C, Carcao M, Dunn AL, Fijnvandraat K, Hoffmann M, Kavakli K, Kenet G, Kobelt R, Kurnik K, Liesner R, Makipernaa A, Manco-Johnson MJ, Mancuso ME, Molinari AC, Nolan B, Perez Garrido R, Petrini P, Platokouki HE, Shapiro AD, Wu R, Ljung R. Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment. Br J Haematol. 2017 Oct;179(2):298-307. doi: 10.1111/bjh.14844. Epub 2017 Jul 12.
PubMed 28699675Mancuso ME, Fischer K, Santagostino E, Oldenburg J, Platokouki H, Konigs C, Escuriola-Ettingshausen C, Rivard GE, Cid AR, Carcao M, Ljung R, Petrini P, Altisent C, Kenet G, Liesner R, Kurnik K, Auerswald G, Chambost H, Makipernaa A, Molinari AC, Williams M, van den Berg HM; European Pediatric Network for Haemophilia Management (PedNet) the REMAIN (REal life MAnagement of children with INhibitors) Study Group. Risk Factors for the Progression from Low to High Titres in 260 Children with Severe Haemophilia A and Newly Developed Inhibitors. Thromb Haemost. 2017 Dec;117(12):2274-2282. doi: 10.1160/TH17-01-0059. Epub 2017 Dec 6.
PubMed 29212115Andersson NG, Chalmers EA, Kenet G, Ljung R, Makipernaa A, Chambost H; PedNet Haemophilia Research Foundation. Mode of delivery in hemophilia: vaginal delivery and Cesarean section carry similar risks for intracranial hemorrhages and other major bleeds. Haematologica. 2019 Oct;104(10):2100-2106. doi: 10.3324/haematol.2018.209619. Epub 2019 Feb 21.
PubMed 30792204van den Berg HM, Fischer K, Carcao M, Chambost H, Kenet G, Kurnik K, Konigs C, Male C, Santagostino E, Ljung R; PedNet Study Group. Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A. Blood. 2019 Jul 18;134(3):317-320. doi: 10.1182/blood.2019000658. Epub 2019 Jun 11. No abstract available.
PubMed 31186271Male C, Andersson NG, Rafowicz A, Liesner R, Kurnik K, Fischer K, Platokouki H, Santagostino E, Chambost H, Nolan B, Konigs C, Kenet G, Ljung R, Van den Berg M. Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica. 2021 Jan 1;106(1):123-129. doi: 10.3324/haematol.2019.239160.
PubMed 31919092
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry
- ClinicalTrials.gov ID
- NCT02979119
- Acronym / sponsor ID
- PedNet / Version 6.4 November 2022
- Conditions
- Factor VIII Deficiency, Factor IX Deficiency
- Keywords
- Hemophilia, Children, Inhibitors, Prophylaxis, Long-term outcome, Congenital
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 4,000 participants (estimated)
- Lead sponsor
- PedNet Haemophilia Research FoundationUniversity, hospital or other
- Study start
- Jun 2014
- Primary completion
- Dec 2039 (estimated)
- Study completion
- Dec 2039 (estimated)
- First posted
- 1 Dec 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Rationale:
Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.
Objective:
To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.
Detailed description
Design: Multicenter Prospective Observational Birth Cohort Study
Population:
Patients with haemophilia A and B with FVIII/IX levels of <1 to 25% born between 1-1-2000 and 1-1-2040.
Intervention:
No intervention; only documentation of patient characteristics and parameters of routine patient care and outcome
Main outcome parameters:
Outcome: clinically relevant inhibitor development, bleeding pattern and joint status on physical examination and imaging.
Determinants: baseline FVIII/IX levels, measurement of inhibitory antibodies, family history, FVIII/IX gene mutation, details on replacement therapy (according to each infusion for the first 50 treatment days, and annually thereafter) and surgeries.
Nature and extent of the burden and risks associated with participation, benefit and group relatedness:
- No burden for the patients. Well-defined clinical data will be collected from the medical files. Participating in this registry will not change the number of visits to the clinic. All outcome parameters that are collected (including laboratory results) are part of routine clinical care.
- Direct benefit is not to be expected. However, the direct interaction between centres that treat patients with rare diseases improves both clinical care and will result in better guidelines and as such may provide indirect benefit.
- Multicentre participation: haemophilia is a very rare condition. Therefore, collecting data on a multi-centre observational cohort is the only way to study this specific population.
- The registry concerns young boys and girls with haemophilia and cannot be performed in older patients, as >90% of inhibitors occur develop during the first 50 exposure days, and the results of prophylactic replacement therapy are highly dependent on the initiation of this treatment.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 18 Years
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort I | – | –Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2000 until December 31st 2009 who have been or are to be treated with coagulation proteins in one of the participating centres |
| Cohort II | – | –Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2010 until December 31st 2019 who have been or are to be treated with coagulation proteins in one of the participating centres |
| Cohort III | – | –Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2020 until December 31st 2029 who have been or are to be treated with coagulation proteins in one of the participating centres |
| Cohort IV | – | –Cohort IV Group/Cohort Description: Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2030 until December 31st 2039 who have been or are to be treated with coagulation proteins in one of the participating centres |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2000 until January 1st 2030 who have been or are to be treated in one of the participating centres
Key inclusion criteria (4 listed)
- Diagnosed with Haemophilia A or B
- Factor VIII/ IX activity of <1 to 25%
- Complete records of Factor treatment and bleeds
- Treated in one of the participating centres
Key exclusion criteria (2 listed)
- Patients referred because of an inhibitor*
- Informed consent not obtained
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of patients with antibody development to exogenous clotting factors
Time frame: Until patient reaches age of 18
Allo-antibodies against Factor VIII and IX; Blood test: measurement in Bethesda units (BU), positive according to local standards, for most labs >0.5 BU
2 secondary outcomes
Long term outcome of haemophilia on joint status using the Hemophilia Joint Health Score (HJHS) and MRI techniques.
Time frame: From diagnose every 5 years until patient reaches age of 18
Long term outcome different Immune Tolerance Induction (ITI) therapies in patients with inhibitor.
Time frame: From date first positive inhibitor titer preferably every 3 years until patient reaches age of 18
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2025.
- Results publication: PubMed 40123267de Kovel M, van Haaster AC, Carcao M, Ranta S, Glosli H, Rivard GE, Kenet G, Kurnik K, Van Geet C, Carvalho M, Andersson NG, Kartal-Kaess M, Ljung R, van den Berg HM; PedNet Study Group. Blood Group O Does Not Increase the Risk of Inhibitors in Severe Haemophilia A: Data from the PedNet Study Group. Haemophilia. 2025 May;31(3):419-423. doi: 10.1111/hae.70035. Epub 2025 Mar 23.
- Results publication: PubMed 40706963Carcao M, Konigs C, Andersson NG, de Kovel M, de Boer-Verdonk E, Motwani J, Blatny J, Olivieri M, van den Berg M, Fischer K. Predictors of immune tolerance induction success in 231 children with severe hemophilia A with high-titer inhibitors - lessons learned from the PedNet prospective cohort study. J Thromb Haemost. 2025 Oct;23(10):3134-3147. doi: 10.1016/j.jtha.2025.07.010. Epub 2025 Jul 22.
- Results publication: PubMed 40444652Mendoza A, Rivas I, Hidalgo OB, Cid AR, Olivieri M, Ranta S, Labarque V, Andersson NG, de Kovel M, Alvarez-Roman MT. Impact of Family History of Haemophilia on Diagnosis, Management and Outcomes in Severe Haemophilia. Haemophilia. 2025 Jul;31(4):679-686. doi: 10.1111/hae.70018. Epub 2025 May 30.
- Results publication: PubMed 40120320Ranta S, Zapotocka E, Andersson NG, Fischer K, Kenet G, de Kovel M, Konigs C, Labarque V, Male C, Olivieri M, Motwani J. A survey on clinical practice in monitoring and management of bleeding in children with haemophilia A on emicizumab prophylaxis in the PedNet centres. Thromb Res. 2025 May;249:109307. doi: 10.1016/j.thromres.2025.109307. Epub 2025 Mar 19. No abstract available.
- Results publication: PubMed 23574131van den Berg HM, Gouw SC, van der Bom JG. Factor VIII products and inhibitors in severe hemophilia A. N Engl J Med. 2013 Apr 11;368(15):1457. doi: 10.1056/NEJMc1301995. No abstract available.
- Results publication: PubMed 23553768Gouw SC, van den Berg HM, Fischer K, Auerswald G, Carcao M, Chalmers E, Chambost H, Kurnik K, Liesner R, Petrini P, Platokouki H, Altisent C, Oldenburg J, Nolan B, Garrido RP, Mancuso ME, Rafowicz A, Williams M, Clausen N, Middelburg RA, Ljung R, van der Bom JG; PedNet and Research of Determinants of INhibitor development (RODIN) Study Group. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013 May 16;121(20):4046-55. doi: 10.1182/blood-2012-09-457036. Epub 2013 Apr 3.
- Results publication: PubMed 23482934Carcao MD, van den Berg HM, Ljung R, Mancuso ME; PedNet and the Rodin Study Group. Correlation between phenotype and genotype in a large unselected cohort of children with severe hemophilia A. Blood. 2013 May 9;121(19):3946-52, S1. doi: 10.1182/blood-2012-11-469403. Epub 2013 Mar 12.
- Results publication: PubMed 24893572Clausen N, Petrini P, Claeyssens-Donadel S, Gouw SC, Liesner R; PedNet and Research of Determinants of Inhibitor development (RODIN) Study Group. Similar bleeding phenotype in young children with haemophilia A or B: a cohort study. Haemophilia. 2014 Nov;20(6):747-55. doi: 10.1111/hae.12470. Epub 2014 Jun 3.
- Results publication: PubMed 24784937Fischer K, Ljung R, Platokouki H, Liesner R, Claeyssens S, Smink E, van den Berg HM. Prospective observational cohort studies for studying rare diseases: the European PedNet Haemophilia Registry. Haemophilia. 2014 Jul;20(4):e280-6. doi: 10.1111/hae.12448. Epub 2014 May 2.
- Results publication: PubMed 23323899Gouw SC, van der Bom JG, Ljung R, Escuriola C, Cid AR, Claeyssens-Donadel S, van Geet C, Kenet G, Makipernaa A, Molinari AC, Muntean W, Kobelt R, Rivard G, Santagostino E, Thomas A, van den Berg HM; PedNet and RODIN Study Group. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013 Jan 17;368(3):231-9. doi: 10.1056/NEJMoa1208024.
- Results publication: PubMed 28699675Andersson NG, Auerswald G, Barnes C, Carcao M, Dunn AL, Fijnvandraat K, Hoffmann M, Kavakli K, Kenet G, Kobelt R, Kurnik K, Liesner R, Makipernaa A, Manco-Johnson MJ, Mancuso ME, Molinari AC, Nolan B, Perez Garrido R, Petrini P, Platokouki HE, Shapiro AD, Wu R, Ljung R. Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment. Br J Haematol. 2017 Oct;179(2):298-307. doi: 10.1111/bjh.14844. Epub 2017 Jul 12.
- Results publication: PubMed 29212115Mancuso ME, Fischer K, Santagostino E, Oldenburg J, Platokouki H, Konigs C, Escuriola-Ettingshausen C, Rivard GE, Cid AR, Carcao M, Ljung R, Petrini P, Altisent C, Kenet G, Liesner R, Kurnik K, Auerswald G, Chambost H, Makipernaa A, Molinari AC, Williams M, van den Berg HM; European Pediatric Network for Haemophilia Management (PedNet) the REMAIN (REal life MAnagement of children with INhibitors) Study Group. Risk Factors for the Progression from Low to High Titres in 260 Children with Severe Haemophilia A and Newly Developed Inhibitors. Thromb Haemost. 2017 Dec;117(12):2274-2282. doi: 10.1160/TH17-01-0059. Epub 2017 Dec 6.
- Website of the PedNet RegistryLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Hermeling contact
Gili Kenet, PhD, MD study director
The National Hemophilia Center Ministry of Health Sheba Medical Center Ramat Gan, Israel
Christoph Male, MD study chair
Medical University of Vienna
Gili Kenet, PhD, MD principal investigator
The National Hemophilia Center Ministry of Health Sheba Medical Center Ramat Gan, Israel
39 sites in 19 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Factor VIII Deficiency and Factor IX Deficiency, run by PedNet Haemophilia Research Foundation at 39 sites in 19 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in Germany, Spain, United Kingdom, France, Italy and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Angelique Hermeling (+31850299993, a.hermeling@pednet.eu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with antibody development to exogenous clotting factors (time frame: Until patient reaches age of 18). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02979119, which also lists 34 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2025.