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ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD) (AHEAD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02078427
Recruitment Status : Recruiting
First Posted : March 5, 2014
Last Update Posted : July 8, 2019
Sponsor:
Collaborator:
Baxalta Innovations GmbH, now part of Shire
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

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

Condition or disease Intervention/treatment
Hemophilia A Biological: ADVATE Biological: ADYNOVI

Layout table for study information
Study Type : Observational
Estimated Enrollment : 630 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: ADVATE/ ADYNOVI Hemophilia A Outcome Database
Actual Study Start Date : June 29, 2011
Estimated Primary Completion Date : January 15, 2025
Estimated Study Completion Date : January 15, 2025


Group/Cohort Intervention/treatment
rAHF-PFM
Participants treated with rAHF-PFM alone
Biological: ADVATE
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Other Names:
  • octocog alfa
  • rAHF-PFM

rAHF-PEG
Participants treated with rAHF-PEG alone
Biological: ADYNOVI
Antihemophilic Factor (Recombinant) Pegylated
Other Names:
  • rurioctocog alfa pegol
  • rAHF-PEG

rAHF-PFM then rAHF-PEG
Participants treated with rAHF-PFM and subsequently switched to rAHF-PEG
Biological: ADVATE
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Other Names:
  • octocog alfa
  • rAHF-PFM

Biological: ADYNOVI
Antihemophilic Factor (Recombinant) Pegylated
Other Names:
  • rurioctocog alfa pegol
  • rAHF-PEG




Primary Outcome Measures :
  1. Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale [ Time Frame: Up to approximately 12 years ]
    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.


Secondary Outcome Measures :
  1. Annualized Bleed Rate, All Joints [ Time Frame: 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 ]
    The annualized bleed rate for all joints will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.

  2. Annualized Bleed Rate, All Bleeds [ Time Frame: 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 ]
    The annualized bleed rate for all bleeds will be calculated per participant and summarized over the set of available participants.with a minimum observation period of 90 days per treatment regimen.

  3. Annualized bleed rate, pre-existing target joints at baseline [ Time Frame: 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 ]
    The annualized bleed rate for pre-existing target joints at baseline will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.

  4. Incidence of New Target Joints [ Time Frame: 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 ]
    The incidence of new target joints will be calculated as the total number of new target joints in all participants divided by the total number of observation days.

  5. Status of joint health by X-ray by Pettersson scale [ Time Frame: 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. ]
    The status of joint health by X-ray by Pettersson score will be summarized for each observational year.

  6. Status of Joint Health by Magnetic Resonance Imaging (MRI) Scoring System- Using The Lund Scoring System (LSS) [ Time Frame: 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 ]

    LSS score format= A(e:s:h). Sum of values for Subchondral Cyst (score: 1-6), irregularity/erosion of Subchondral Cortex (score: 1-4), and Chondral Destruction (score: 1-6) gives value for the A component of score. e, s, h components represent effusion/hemarthrosis, hypertrophic synovial, & hemosiderin deposition (score: 0-4 for each). Max. score is 16(4:4:4).

    Subchondral Cyst:

    • ≥1 bone
    • ≥2 bones
    • >3 cysts in ≥1 bone
    • >3 cysts ≥2 bones
    • Largest size >4 mm: ≥1 bone
    • Largest size >4 mm: ≥2 bones

    Subchondral Cortex

    • ≥1 bone
    • ≥2 bones
    • Involve > half joint surface: ≥1 bone
    • Involve > half of joint surface: ≥2 bones

    Chondral Destruction

    • ≥1 bone
    • ≥2 bones
    • Full thickness defect (FTD): ≥1 bone
    • FTD: ≥2 bones
    • FTD involves >1/3 of joint surface: ≥1 bone
    • FTD involves >1/3 of joint surface: ≥2 bones

    Effusion/hemarthrosis (e): Hypertrophic synovial (s): Hemosiderin (h): (0-4 for each):

    • 0 absent
    • 1 equivocal
    • 2 small
    • 3 moderate
    • 4 large

  7. Status of joint health using the Hemophilia Joint Health Score (HJHS) [ Time Frame: 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 ]

    The International Prophylaxis Study Group (IPSG) developed a scoring system for musculoskeletal evaluation, the HJHS, optimized for use in children with no or minimal joint disease.

    The HJHS includes the following parameters: swelling, duration of swelling, muscle atrophy, joint pain, crepitus on motion, flexion loss, extension loss, strength and global gait.


  8. Overall effectiveness assessment for prophylaxis therapy [ Time Frame: 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 ]
    • Excellent: Same or lower breakthrough bleed rate (BBR) within last 12 months (M) compared with prior prophylaxis; if participant did not receive prior prophylaxis with rAHF-PFM, rAHF-PEG or other Factor VIII (FVIII), same or better than expected outcome according to investigator's expectation
    • Good: Minor increase in BBR within last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, slightly less than expected outcome according to investigator's expectation
    • Fair: Moderate increase in BBR in last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, somewhat less than expected outcome according to investigator's expectation
    • Poor: Significant increase in BBR in the 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, little to no benefit according to investigator's expectation

  9. Compliance with the dosing prescribed and its relationship with effectiveness [ Time Frame: 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 ]
    Evaluation of patients´ compliance to prescribed prophylactic treatment will be performed by the treating physician. Compliance will be categorized according to a 4-point table (Highly compliant, Fairly compliant, Moderately compliant, Poorly compliant)

  10. Overall effectiveness assessment for on-demand treatment [ Time Frame: 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 ]
    • Excellent: Bleed episodes typically respond to same or fewer number of infusion and same or lower dose as compared with previous on-demand treatment or investigator's expectation
    • Good: Most bleed episodes typically respond to same number of infusion and dose but some require more infusions or higher dose as compared with previous on-demand treatment or investigator's expectation
    • Fair: Bleed episodes typically require more infusions and/or higher dose than expected as compared with previous on-demand treatment or investigator's expectation
    • Poor: Bleed episodes routinely fail to respond to same number of infusion and dose and require additional or different factor concentrate for hemostatic control as compared with previous on-demand treatment or investigator's expectation

  11. Global effectiveness assessment for on-demand treatment [ Time Frame: 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 ]
    • Excellent: Full relief of pain and cessation of bleeding as evidenced by objective signs (e.g., swelling, tenderness, irritability, inconsolability, and decreased range of motion in the case of musculoskeletal hemorrhage) within approximately 8 hours of a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring.
    • Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after the infusion. Possibly requires more than 1 infusion for complete resolution.
    • Fair: Probable or slight relief of pain and slight improvement in signs of bleeding within approximately 8 hours after the infusion. Requires more than 1 infusion for complete resolution.
    • Poor: No improvement or condition worsens.

  12. Number of rAHF-PFM or rAHF-PEG units required for bleed cessation [ Time Frame: 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 ]
    Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)

  13. Number of rAHF-PFM or rAHF-PEG infusions required for bleed cessation [ Time Frame: 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 ]
    Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)

  14. Incidence of target joint intervention, including surgery, radiosynovectomy, and chemosynovectomy [ Time Frame: 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 ]
  15. Incidence of pseudo tumor development [ Time Frame: 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; Termination visit ]
  16. Quality of Life: HAL questionnaire - for adult patients [ Time Frame: Enrollment visit; 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; Termination visit ]
    The the lHAL measures activities involving the upper extremities, basic activities involving ower extremities and complex activities involving the lower extremities as well as an overall physical activity score for adults.

  17. Quality of Life: SF-12v2 questionnaire - for adult patients [ Time Frame: Enrollment visit; 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; Termination visit ]
    The SF-12v2 measures generic health-related quality of life for adults.

  18. Quality of Life: EQ-5D questionnaire - for adult patients [ Time Frame: Enrollment visit;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; Termination visit ]
    The EQ-5D measures health utility in adult participants.

  19. Quality of Life: PedHAL questionnaire - for pediatric patients [ Time Frame: Enrollment visit; 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; Termination visit ]

    The PedHAL measures activities involving the upper extremities, basic activities involving the lower extremities and complex activities involving the lower extremities as well as an overall physical activity score for children.

    For participants 4-13 years of age:

    - PedHAL (parent version)

    For participants 14-17 years of age:

    - PedHAL (child version)


  20. Quality of Life: SF-10 questionnaire - for pediatric patients [ Time Frame: Enrollment visit; 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; Termination visit ]
    The SF-10 measures generic health-related quality of life for children and is parent-completed.

  21. Quality of Life: EQ-5D (14 and up) questionnaire - for pediatric patients [ Time Frame: Enrollment visit; 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; Termination visit ]
    The EQ-5D measures health utility in subjects aged 14 and up.

  22. Chronic pain associated with hemophilia, as measured over a period of 4 weeks on an annual basis, using the visual analog scale (VAS) [ Time Frame: Enrollment visit; 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; Termination visit ]

    The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain).

    During screening visit and on an annual basis, the investigators shall ask participants to rate the average level of chronic pain associated with hemophilia over the period of 4 weeks prior to visit date using the VAS.


  23. Acute pain associated with hemophilia, as measured with individual bleeding episodes, using the visual analog scale (VAS) [ Time Frame: Enrollment visit; 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; Termination visit ]

    The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain).

    Participants will be asked to provide ratings on level of acute pain associated with each bleeding episode using the VAS. The VAS scores will be recorded in the participant diary.


  24. Number of days lost from school or work due to bleeding episodes [ Time Frame: Enrollment visit; 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; Termination visit ]
  25. Modalities of switching from a standard FVIII product to rAHF-PEG - 1 [ Time Frame: Enrollment visit; 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; Termination visit ]
    Difference in number of weekly prophylactic infusions between previous regimen and rAHF-PEG

  26. Modalities of switching from a standard FVIII product to rAHF-PEG - 2 [ Time Frame: Enrollment visit; 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; Termination visit ]
    Difference in number of weekly doses between previous regimen and rAHFPEG

  27. Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels Lesser than (<)1%, Lesser Than or Equal to (<=) 2%, and <= 5% without history of inhibitor [ Time Frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) ]
  28. Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5% with history of inhibitor [ Time Frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) ]
  29. Incidence of Inhibitors in Previously Untreated Patient (PUPs) and Minimally Treated Patients (MTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5% [ Time Frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) ]
  30. Incidence of therapy-related serious adverse events [ Time Frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) ]
  31. Incidence of therapy-related non-serious adverse events [ Time Frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) ]
  32. Incidence of inhibitors after switching to rAHF-PEG [ Time Frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM) ]
    Incidence of inhibitors after switching to rAHF-PEG in the same subgroups of patients



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-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.
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02078427


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

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Locations
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Australia, New South Wales
Royal Prince Alfred Hospital Recruiting
Camperdown, New South Wales, Australia, 2050
Australia, Western Australia
Fiona Stanley Hospital Recruiting
Murdoch, Western Australia, Australia, 6150
Austria
Landes Frauen und Kinderklinik Linz (LFKK Linz) Completed
Linz, Austria, 4017
Medizinische Universitaet Wien Completed
Wien, Austria, 1090
Belgium
Cliniques Universitaires Saint-Luc Completed
Bruxelles, Belgium, B-1200
Brazil
Hemoce - Ce Completed
Fortaleza, Ceará, Brazil, 60431086
Hemocentro do Espírito Santo Completed
Vitória, Espírito Santo, Brazil, 29040-090
Fundação HEMOPA Completed
Belém, Pará, Brazil, 66033-000
Hemocentro da UNICAMP Completed
Campinas, São Paulo, Brazil, 13083-970
Hemocentro Ribeirão Preto - SP Completed
Ribeirão Preto, São Paulo, Brazil, 14051140
Hemepar - Pr Completed
Curitiba, Brazil, 80045-145
Hemorgs - Rs Completed
Porto Alegre, Brazil, 90650-000
Hemorio - Rj Completed
Rio de Janeiro, Brazil, 20211030
Bulgaria
UMHAP Sveti Georgï EAD Withdrawn
Plovdiv, Bulgaria, 4000
Canada, Alberta
Stollery Children's Hospital, University of Alberta Suspended
Edmonton, Alberta, Canada, T6G 2B7
Canada, New Brunswick
The Moncton City Hospital Terminated
Moncton, New Brunswick, Canada, E1C 6Z8
Canada, Ontario
Children's Hospital of Eastern Ontario Completed
Ottawa, Ontario, Canada, K1H 8L1
St-Michael's Hospital Completed
Toronto, Ontario, Canada, M5B 1W8
Sick Kids Hospital Completed
Toronto, Ontario, Canada, M5G 1X8
Canada, Saskatchewan
Royal University Hospital Completed
Saskatoon, Saskatchewan, Canada, S7N 0W8
Colombia
Fundación Oftalmológica de Santander FOSCAL Recruiting
Floridablanca, Colombia
Czechia
Fakultní nemocnice Brno Completed
Brno, Czechia, 613 00
Fakultní nemocnice Ostrava, Krevní centrum Completed
Ostrava, Czechia, 70852
Fakultní nemocnice Ostrava, Oddělení dětské hematologie a hematoonkologie Completed
Ostrava, Czechia, 70852
Fakultní nemocnice v Motole Completed
Praha, Czechia, 150 06
Denmark
Rigshospitalet Completed
Copenhagen, Denmark, 2100
France
CHRU Pellegrin Withdrawn
Bordeaux, France, 33076
Hôpital Morvan CHRU de Brest Completed
Brest, France, 29200
CHU Côte de Nacre - CRTH Completed
CAEN Cedex, France, 14033
Centre Hospitalier Générale - CTH Completed
Chambery, France, 73011
CTRH - CHU Bocage Completed
Dijon, France, 21079
Hôpital André Mignot Completed
Le Chesnay, France, 78157
Hôpital de la Mère et de l'Enfant - CHU de LIMOGES Completed
LIMOGES cedex, France, 87043
CHRU Hôtel Dieu - CRTH Completed
Nantes, France, 44093
Hôpital Cochin Completed
Paris, France, 75014
CHU de Reims Hôpital Maison Blanche - CRTH Completed
Reims, France, 51092
Centre Régional de Traitement de l'Hémophilie et des Maladies hémorragiques. CHU de Rennes - Hôpital Pontchaillou Completed
Rennes, France, 35033
CHRU Charles Nicolle Completed
Rouen, France, 76031
CIC Completed
Saint Priest en Jarez, France, 42270
CHRU Purpan CRTH - Pavillon Sénac Completed
Toulouse Cedex 9, France, 31059
Greece
Aghia Sofia Children's Hospital Completed
Goudi, Athens, Greece, 11527
Laikon General Hospital Completed
Goudi, Athens, Greece, 11527
General Hospital of Thessaloniki "Ippokratio" Completed
Thessaloniki, Greece, 546 42
Hungary
Heim Pál Gyermekkórház - Madarász utcai telephely Completed
Budapest, Hungary, H-1131
Debreceni Egyetem Klinikai Kozpont Completed
Debrecen, Hungary, H-4032
Mohács City Hospital Completed
Mohács, Hungary, H-7700
Jósa András County Hospital Terminated
Nyíregyháza, Hungary, H-4400
Jósa András County Hospital Completed
Nyíregyháza, Hungary, H-4400
Markusovszky Hospital Terminated
Szombathely, Hungary, H-9700
Italy
Azienda Ospedaliera Policlinico Consorziale Di Bari Completed
Bari, Italy, 70124
Policlinico S Orsola Malpighi Completed
Bologna, Italy, 40138
Azienda Ospedaliera Universitaria Vittorio Emanuele, Ferrarotto, S. Bambino Completed
Catania, Italy, 95120
Ospedale Pugliese -Ciaccio Completed
Catanzaro, Italy, 88100
Az. Osp. Univ. Careggi Completed
Firenze, Italy, 50134
Ospedale di Macerata Completed
Macerata, Italy, 62100
IRCCS Ospedale Maggiore Completed
Milano, Italy, 20122
Azienda Ospedaliera Universitaria Federico II Completed
Napoli, Italy, 80131
Azienda Ospedaliera di Padova Clinica Medica II Completed
Padova, Italy, 35100
AOUP P. Giaccone Completed
Palermo, Italy, 90133
Azienda Ospedaliera Bianchi Melacrino Morelli Completed
Reggio Calabria, Italy, 89132
Università degli studi di Roma "La Sapienza" Completed
Roma, Italy, 00161
Ospedale Bambino Gesù Completed
Roma, Italy, 00165
Policlinico Universitario Gemelli Completed
Roma, Italy, 00168
Centro Emofilia e Coagulopatie Rare Completed
Scorrano, Italy, 73025
Ospedale Molinette Completed
Torino, Italy, 10126
Norway
Rikshospitalet Completed
Oslo, Norway, N-0027
Poland
Szpital Uniwersytecki nr 1 im. dr Andrzeja Jurasza Terminated
Bydgoszcz, Poland, 85-094
Samodzielny Publiczny Szpital Kliniczny nr 1 Terminated
Gdansk, Poland, 80-952
Samodzielny Publiczny Dzieciecy Szpital Kliniczny Completed
Warszawa, Poland, 00-576
Uniwersytecki Szpital Kliniczny we Wrocławiu Terminated
Wroclaw, Poland, 50-345
Portugal
Hospital do Divino Espírito Santo Completed
Ponta Delgada, Açores, Portugal, 9500-370
Centro Hospitalar e Universitário de Coimbra, Hospital Pediátrico de Coimbra Completed
Coimbra, Portugal, 3000-602
Centro Hospitalar Lisboa Norte Hospital de Sta. Maria Completed
Lisboa, Portugal, 1649-035
Centro Hospitalar de São João, E.P.E. Terminated
Porto, Portugal, 4200-319
Russian Federation
Hematology Scientific Research Center of MoH RF Completed
Moscow, Russian Federation, 125167
City Center for hemophilia patients treatment, City out-patient clinic № 37 Completed
Saint Petersburg, Russian Federation, 191186
Slovenia
University Medical Centre Ljubljana Completed
Ljubljana, Slovenia, 1000
Spain
Hospital Teresa Herrera-Materno Infantil Completed
A Coruña, Spain, 15006
Hospital Universitari Vall d'Hebron (HUVH) Completed
Barcelona, Spain, 08035
Hospital Hospital Sant Joan de Déu Completed
Barcelona, Spain, 08950
Hospital Universitario Son Espases Completed
Palma de Mallorca, Spain, 07120
Sweden
Sahlgrenska University Hospital Completed
Gothenburg, Sweden, SE 41345
Skånes Universitetssjukhus, Malmö/Lund Completed
Malmö, Sweden, SE-205 02
Karolinska Universitetssjukhuset Solna Completed
Stockholm, Sweden, 171 76
Switzerland
Universitätsspital Basel Recruiting
Basel, Switzerland, 4031
Inselspital Bern Recruiting
Bern, Switzerland, 3010
Centre Hospitalier Universitaire Vaudois Recruiting
Lausanne, Switzerland, 1011
Luzerner Kantonsspital Recruiting
Luzern, Switzerland, 6000
Kantonsspital St. Gallen Recruiting
St. Gallen, Switzerland, 9007
FMH Kinder und Jugendmedizin, im Wabern-Zentrum Terminated
Wabern, Switzerland, 3084
Kinderspital Zürich Recruiting
Zürich, Switzerland, 8032
Universitätsspital Zürich Recruiting
Zürich, Switzerland, 8091
United Kingdom
Hampshire Hospitals NHS Foundation Trust, Basingstoke and North Hampshire Hospital Completed
Basingstoke, Hampshire, United Kingdom, RG24 9NA
Cambridge University Hospital NHS Foundation Trust, Addenbrooke's Hospital Completed
Cambridge, United Kingdom, CB2 0QQ
East Kent Hospitals University Foundation Trust, Kent & Canterbury Hospital Completed
Canterbury, United Kingdom, CT1 3NG
Great Ormond Street Hospital for Children NHS Trust Completed
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT02078427     History of Changes
Other Study ID Numbers: 061001
First Posted: March 5, 2014    Key Record Dates
Last Update Posted: July 8, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. 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.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants