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Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A

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ClinicalTrials.gov Identifier: NCT01863758
Recruitment Status : Completed
First Posted : May 29, 2013
Results First Posted : January 30, 2018
Last Update Posted : January 30, 2018
Sponsor:
Information provided by (Responsible Party):
Octapharma

Brief Summary:
To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.

Condition or disease Intervention/treatment Phase
Severe Haemophilia A Biological: Human-cl rhFVIII Phase 3

Detailed Description:
There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or 3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing interval were determined individually from data gathered in the initial PK assessment. The maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01 IU/mL was determined. Participants were treated for 6 months.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 66 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Prospective, Open-label, Multicenter Phase 3b Study to Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A
Study Start Date : August 2013
Actual Primary Completion Date : January 2015
Actual Study Completion Date : January 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
Drug Information available for: Chlorine

Arm Intervention/treatment
Experimental: Human-cl rhFVIII
Up to 60-80 IU/kg of intravenous Human-cl rhFVIII was administered at an individually determined dose and dose interval.
Biological: Human-cl rhFVIII
Human-cl rhFVIII was provided as a freeze-dried concentrate to be reconstituted in water for injection.




Primary Outcome Measures :
  1. Annualized Number of Bleeding Episodes (BE) in Phase II [ Time Frame: Beginning to the end of Phase II (6 months) ]
    The annualized number of total BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196).


Secondary Outcome Measures :
  1. Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II [ Time Frame: Beginning to the end of Phase II (6 months) ]
    The annualized number of spontaneous BEs was calculated for each participant as follows: d*y/t, where y = the number of spontaneous BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A spontaneous bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery and BEs due to trauma or due to other causes were not included.

  2. Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week [ Time Frame: Beginning to the end of Phase II (6 months) ]
    The annualized number of BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery were not included.

  3. Median Dosing Interval During Individually Tailored Prophylaxis [ Time Frame: Beginning to the end of Phase II (6 months) ]
    The median time between 2 prophylactic doses of Human-cl rhFVIII in the prophylactic treatment phase II were determined per patient

  4. Dosage Per Week in Phase II [ Time Frame: Beginning to the end of Phase II (6 months) ]
    The mean dosage per week during Phase II of the study are reported.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Severe haemophilia A (FVIII:C < 1%) according to medical history.
  • Male patients ≥ 18 years old.
  • Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 exposure days (EDs).
  • Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start.
  • Immunocompetence (CD4+ count > 200/microliter).
  • HIV-negative, if positive, viral load < 200 particles/microliter or < 400,000 copies/mL.
  • Freely given written informed consent

Exclusion Criteria:

  • Any coagulation disorder other than haemophilia A.
  • Present or past FVIII inhibitor activity (> 0.6 Bethesda Unit [BU])
  • Severe liver or kidney disease.

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): NCT01863758


Locations
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Austria
Medical University Vienna
Vienna, Austria
Bulgaria
University Multiprofile Hospital for Active Treatment
Plovdiv, Bulgaria
Specialized Hospital for Active Treatment
Sofia, Bulgaria
Multiprofile Hospital for Active Treatment
Varna, Bulgaria
Germany
Vivantes Hospital in Friedrichshain
Berlin, Germany
SRH Kurpfalzklinik Heidelberg GMBH
Heidelberg, Germany
Hungary
Hungarian National Healthcare Center
Budapest, Hungary
University of Debrecen, Medical and Health Science Center
Debrecen, Hungary
Poland
University Teaching Hospital in Bialystok, Teaching Department of Hematology with a Subdepartment of Vascular Diseases
Białystok, Poland
University Clinical Center, Teaching Department of Hematology and Transplantology
Gdańsk, Poland
Nicolaus Copernicus Municipal Specialist Hospital, Department of Hematology
Torun, Poland
Institute of Hematology and Transfusion Medicine, Depart. of Hemostatic Disorders and Internal Diseases
Warsaw, Poland
Romania
Sanador SRL
Bucharest, Romania
Louis Turcanu Emergency Clinical Children's Hospital
Timisoara, Romania
Slovakia
University Hospital Saint Cyril and Metod
Bratislava, Slovakia
University Hospital Martin, Department of Hematology and Transfusiology
Martin, Slovakia
United Kingdom
Basingstoke and North Hampshire Hospital, Hemophilia, Hemostasis and Thrombosis Center
Basingstoke, United Kingdom
Royal London Hospital, Barts and the London Hemophilia Center
London, United Kingdom
Manchester Royal Infirmary, Department of Clinical Hematology
Manchester, United Kingdom
Royal Hallamshire Hospital
Sheffield, United Kingdom
Sponsors and Collaborators
Octapharma

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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT01863758     History of Changes
Other Study ID Numbers: GENA-21
First Posted: May 29, 2013    Key Record Dates
Results First Posted: January 30, 2018
Last Update Posted: January 30, 2018
Last Verified: July 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
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