NCT02078427

Brief Summary

The purpose of the study is to document the natural history of hemophilia A disease and long-term outcomes in terms of effectiveness, safety and quality of life in participants receiving Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) in routine clinical practice

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
951

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jun 2011

Longer than P75 for all trials

Geographic Reach
21 countries

108 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 28, 2011

Completed
2.7 years until next milestone

First Submitted

Initial submission to the registry

March 3, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 5, 2014

Completed
9.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 16, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 16, 2024

Completed
Last Updated

June 17, 2024

Status Verified

June 1, 2024

Enrollment Period

12.6 years

First QC Date

March 3, 2014

Last Update Submit

June 14, 2024

Conditions

Outcome Measures

Primary Outcomes (1)

  • Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale

    The World Federation of Hemophilia developed a musculoskeletal evaluation system, commonly referred to as the Gilbert test, to measure hemophilia joint health status.The Gilbert test needs to be performed in the absence of acute bleed, acute pain, and acute inflammation into the evaluated joint. Four parameters are used in each Gilbert test: pain (score: 0-3), bleeding (score: 0-3), physical exam (score: 0-12), and X-ray evaluation (score: 0-13) Scores of 0, represent no pain, no bleeding, no physical exam issues, and/or no x-ray issues. Higher scores for each of these categories represents worsening conditions.

    Up to approximately 12 years

Secondary Outcomes (32)

  • Annualized Bleed Rate, All Joints

    Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit

  • Annualized Bleed Rate, All Bleeds

    Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit

  • Annualized bleed rate, pre-existing target joints at baseline

    Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit

  • Incidence of New Target Joints

    Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit

  • Status of joint health by X-ray by Pettersson scale

    Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit.

  • +27 more secondary outcomes

Study Arms (3)

rAHF-PFM

Participants treated with rAHF-PFM alone

Biological: ADVATE

rAHF-PEG

Participants treated with rAHF-PEG alone

Biological: ADYNOVI

rAHF-PFM then rAHF-PEG

Participants treated with rAHF-PFM and subsequently switched to rAHF-PEG

Biological: ADVATEBiological: ADYNOVI

Interventions

ADVATEBIOLOGICAL

Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Also known as: octocog alfa, rAHF-PFM
rAHF-PFMrAHF-PFM then rAHF-PEG
ADYNOVIBIOLOGICAL

Antihemophilic Factor (Recombinant) Pegylated

Also known as: rurioctocog alfa pegol, rAHF-PEG
rAHF-PEGrAHF-PFM then rAHF-PEG

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

The study population will include male and female participants of any race and age who have a diagnosis of hemophilia A (Factor VIII (FVIII) =5%). Participants must have been prescribed rAHF-PFM or rAHF-PEG for the management of hemophilia A by the treating physician prior to the decision to enroll in the study.

You may qualify if:

  • Participant has hemophilia A {FVIII lesser than or equal to (\<=)5%}
  • Participant is prescribed Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) by the treating physician
  • Participant or participant's legally authorized representative provides informed consent

You may not qualify if:

  • Participant has known hypersensitivity to the active substance or any of the excipients
  • Participant has known allergic reaction to mouse or hamster proteins
  • Participant has participated in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment or is scheduled to participate in another clinical study involving another FVIII concentrate or device during the course of this study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (110)

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

Location

South Metropolitan Health Service trading as Fiona Stanley Hospital

Murdoch, Western Australia, 6050, Australia

Location

Landes Frauen und Kinderklinik Linz (LFKK Linz)

Linz, 4017, Austria

Location

Kepler Universitätsklinikum Klinik für Kinder-und Jugendheilkunde

Linz, 4020, Austria

Location

Medizinische Universitaet Wien

Vienna, 1090, Austria

Location

Cliniques Universitaires Saint-Luc

Brussels, B-1200, Belgium

Location

Hemoce - Ce

Fortaleza, Ceará, 60431086, Brazil

Location

Hemocentro do Espírito Santo

Vitória, Espírito Santo, 29040-090, Brazil

Location

Hemepar - Pr

Curitiba, Paraná, 80045-145, Brazil

Location

Hemorgs - Rs

Porto Alegre, Parthenon, 90650-000, Brazil

Location

Fundação HEMOPA

Belém, Pará, 66033-000, Brazil

Location

Hemocentro da UNICAMP

Campinas, São Paulo, 13083-970, Brazil

Location

Hemocentro Ribeirão Preto - SP

Ribeirão Preto, São Paulo, 14051140, Brazil

Location

Hemorio - Rj

Rio de Janeiro, 20211030, Brazil

Location

Faculdade de Medicina da Universidade de São Paulo

São Paulo, 5403000, Brazil

Location

Stollery Children's Hospital, University of Alberta

Edmonton, Alberta, T6G 2B7, Canada

Location

The Moncton City Hospital

Moncton, New Brunswick, E1C 6Z8, Canada

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

St-Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada

Location

Sick Kids Hospital

Toronto, Ontario, M5G 1X8, Canada

Location

Royal University Hospital

Saskatoon, Saskatchewan, S7N 0W8, Canada

Location

Shenzhen Children's Hospital

Shenzhen, Futian, China

Location

Nanfang Hospital Affiliated to Nanfang Medical University

Guangzhou, Guangdong, China

Location

Institute of Hematology, Blood Disease Hospital, PUMC&CAMS

Tianjin, Heping, China

Location

Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology

Wuhan, Hubei, China

Location

The Affiliated Hospital of Xuzhou Medical University

Xuzhou, Jiangsu, 221006, China

Location

The Blood Center of Shandong Province

Jinan, Shandong, China

Location

Beijing Children's Hospital Affiliated to Capital University of Medical Sciences

Beijing, 100045, China

Location

Nanjing Drum Tower Hospital The Affiliated Hospital of Nanjing University Medical School

Nanjing, China

Location

Shenzhen Second People's Hospital

Shenzhen, 518037, China

Location

IPS FUSA SAS Centro Integral de Coagulacion

Atlántico, Colombia

Location

Integral Solutions SD SAS

Bogotá, Colombia

Location

Fundación Oftalmológica de Santander FOSCAL

Floridablanca, 681004, Colombia

Location

Fakultní nemocnice Brno

Brno, 613 00, Czechia

Location

Fakultní nemocnice Ostrava, Oddělení dětské hematologie a hematoonkologie

Ostrava, 70852, Czechia

Location

Fakultní nemocnice Ostrava

Ostrava, 70852, Czechia

Location

Fakultní nemocnice v Motole

Prague, 150 06, Czechia

Location

Rigshospitalet

Copenhagen, 2100, Denmark

Location

"Hôpital Morvan CHRU de Brest"

Brest, 29200, France

Location

CHU Côte de Nacre - CRTH

Caen, 14033, France

Location

Centre Hospitalier Générale - CTH

Chambéry, 73011, France

Location

CTRH - CHU Bocage

Dijon, 21079, France

Location

Hôpital André Mignot

Le Chesnay, 78157, France

Location

Hôpital de la Mère et de l'Enfant - CHU de LIMOGES

Limoges, 87043, France

Location

CHRU Hôtel Dieu - CRTH

Nantes, 44093, France

Location

Hôpital Cochin

Paris, 75014, France

Location

CHU de Reims Hôpital Maison Blanche - CRTH

Reims, 51092, France

Location

Centre Régional de Traitement de l'Hémophilie et des Maladies hémorragiques. CHU de Rennes - Hôpital Pontchaillou

Rennes, 35033, France

Location

CHRU Charles Nicolle

Rouen, 76031, France

Location

CIC

Saint-Priest-en-Jarez, 42270, France

Location

CHRU Purpan CRTH - Pavillon Sénac

Toulouse, 31059, France

Location

Aghia Sofia Children's Hospital

Goudi, Athens, 11527, Greece

Location

Laikon General Hospital

Goudi, Athens, 11527, Greece

Location

General Hospital of Thessaloniki "Ippokratio"

Thessaloniki, 546 42, Greece

Location

Heim Pál Gyermekkórház

Budapest, H-1089, Hungary

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, H-4032, Hungary

Location

Mohács City Hospital

Mohács, H-7700, Hungary

Location

Jósa András County Hospital

Nyíregyháza, H-4400, Hungary

Location

Markusovszky Hospital

Szombathely, H-9700, Hungary

Location

Azienda Ospedaliera Policlinico Consorziale Di Bari

Bari, 70124, Italy

Location

Policlinico S Orsola Malpighi

Bologna, 40138, Italy

Location

Azienda Ospedaliera Universitaria Vittorio Emanuele, Ferrarotto, S. Bambino

Catania, 95120, Italy

Location

Ospedale Pugliese -Ciaccio

Catanzaro, 88100, Italy

Location

Az. Osp. Univ. Careggi

Florence, 50134, Italy

Location

Ospedale di Macerata

Macerata, 62100, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Azienda Ospedaliera Universitaria Federico II

Napoli, 80131, Italy

Location

Azienda Ospedaliera di Padova Clinica Medica II

Padua, 35100, Italy

Location

AOUP P. Giaccone

Palermo, 90133, Italy

Location

Azienda Ospedaliera Bianchi Melacrino Morelli

Reggio Calabria, 89123, Italy

Location

Università degli studi di Roma "La Sapienza"

Roma, 00161, Italy

Location

Ospedale Pediatrico Bambino Gesù

Roma, 00165, Italy

Location

Policlinico Universitario Gemelli

Roma, 00168, Italy

Location

Centro Emofilia e Coagulopatie Rare

Scorrano, 73025, Italy

Location

Ospedale Molinette

Torino, 10126, Italy

Location

Rikshospitalet

Oslo, N-0027, Norway

Location

Rikshospitalet, Oslo University Hospital

Oslo, N-0424, Norway

Location

Szpital Uniwersytecki nr 1 im. dr Andrzeja Jurasza

Bydgoszcz, 85-094, Poland

Location

Samodzielny Publiczny Szpital Kliniczny nr 1

Gdansk, 80-952, Poland

Location

Samodzielny Publiczny Dzieciecy Szpital Kliniczny

Warsaw, 02-091, Poland

Location

Uniwersytecki Szpital Kliniczny we Wrocławiu

Wroclaw, 50-556, Poland

Location

Hospital do Divino Espírito Santo

Ponta Delgada, Azores, 9500-370, Portugal

Location

Centro Hospitalar e Universitade de Coimbra

Coimbra, 3000-602, Portugal

Location

Centro Hospitalar Lisboa Norte Hospital de Sta. Maria

Lisbon, 1649-035, Portugal

Location

Centro Hospitalar de São João, E.P.E.

Porto, 4200-319, Portugal

Location

SBHI Chelyabinsk Regional Children's Clinical Hospital

Chelyabinsk, Russia

Location

Federal State Budget Institution "Hematology Research Center" of Ministry of Healthcare of Russian Federation

Moscow, 125167, Russia

Location

SBHI of the Republic of Kareliya Republican Hospital n.a. V.A. Baranov

Petrozavodsk, Russia

Location

State Budget Healthcare Institution of Saint-Petersburg "City polyclinic #37"

Saint Petersburg, 191186, Russia

Location

Clinics of FSBEI High Education Samara SMU of MoH of Russia

Samara, 443079, Russia

Location

University Medical Centre Ljubljana

Ljubljana, SI-1000, Slovenia

Location

Hospital Teresa Herrera-Materno Infantil

A Coruña, 15006, Spain

Location

Hospital Hospital Sant Joan de Déu

Barcelona, 08035, Spain

Location

Hospital Universitari Vall d'Hebron (HUVH)

Barcelona, 08035, Spain

Location

Hospital Universitario Son Espases

Palma de Mallorca, 07120, Spain

Location

Sahlgrenska University Hospital

Gothenburg, SE 41345, Sweden

Location

Kliniska studier i Sverige - Forum Söder

Malmo, SE-205 02, Sweden

Location

Karolinska Universitetssjukhuset Solna

Stockholm, 171 76, Sweden

Location

Universitätsspital Basel

Basel, CH-4031, Switzerland

Location

Inselspital Bern

Bern, 3010, Switzerland

Location

Centre Hospitalier Universitaire Vaudois

Lausanne, 1011, Switzerland

Location

Luzerner Kantonsspital

Lucerne, CH-6000, Switzerland

Location

Kantonsspital St. Gallen

Sankt Gallen, 9007, Switzerland

Location

FMH Kinder und Jugendmedizin, im Wabern-Zentrum

Wabern, CH-3084, Switzerland

Location

Kinderspital Zürich

Zurich, CH-8032, Switzerland

Location

Universitätsspital Zürich

Zurich, CH-8091, Switzerland

Location

Hampshire Hospitals NHS Foundation Trust, Basingstoke and North Hampshire Hospital

Basingstoke, Hampshire, RG24 9NA, United Kingdom

Location

Cambridge University Hospital NHS Foundation Trust, Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

East Kent Hospitals University Foundation Trust, Kent & Canterbury Hospital

Canterbury, CT1 3NG, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Trust

London, WC1N 3JH, United Kingdom

Location

Related Publications (1)

  • Ozelo MC, Hermans C, Carcao M, Guillet B, Gu J, Guerra R, Tang L, Khair K. The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study. Ther Adv Hematol. 2024 Feb 15;15:20406207231218624. doi: 10.1177/20406207231218624. eCollection 2024.

Related Links

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIIIF8 protein, humanBAX 855

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 3, 2014

First Posted

March 5, 2014

Study Start

June 28, 2011

Primary Completion

January 16, 2024

Study Completion

January 16, 2024

Last Updated

June 17, 2024

Record last verified: 2024-06

Data Sharing

IPD Sharing
Will share

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
More information

Locations