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Trial record 1 of 1 for:    NCT02020369
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Phase III Study of Coagulation FVIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors

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ClinicalTrials.gov Identifier: NCT02020369
Recruitment Status : Completed
First Posted : December 24, 2013
Results First Posted : June 14, 2017
Last Update Posted : June 14, 2017
Sponsor:
Collaborator:
Laboratoire français de Fractionnement et de Biotechnologies
Information provided by (Responsible Party):
rEVO Biologics

Brief Summary:
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX

Condition or disease Intervention/treatment Phase
Hemophilia A With Inhibitors Hemophilia B With Inhibitors Biological: Coagulation Factor VIIa (Recombinant) Phase 3

Detailed Description:

This was a global, multicenter, Phase III, prospective, open-label, randomized, crossover study. After obtaining informed consent and performance of screening procedures, patients who met all inclusion and exclusion criteria were randomized to one of two treatment regimens as follows:

  • 75 µg/kg treatment regimen
  • 225 µg/kg treatment regimen

For each treatment regimen there were two phases:

  • Phase A (Initial phase)
  • Phase B (Treatment phase)

The assigned treatment regimen was the dose administered in Phase A and was the starting dose in Phase B.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX
Study Start Date : April 2014
Actual Primary Completion Date : July 2015
Actual Study Completion Date : August 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding Hemophilia

Arm Intervention/treatment
Experimental: FVIIa: 75 µg/kg first, then 225 µg/kg
Coagulation Factor VIIa (Recombinant): First Intervention (3 months), Second Intervention (3 months), repeat cycle until study completion.
Biological: Coagulation Factor VIIa (Recombinant)
A cross over design to assess the efficacy of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX

Experimental: FVIIa: 225 µg/kg first, then 75 µg/kg
Coagulation Factor VIIa (Recombinant): First Intervention (3 months), Second Intervention (3 months), repeat cycle until study completion.
Biological: Coagulation Factor VIIa (Recombinant)
A cross over design to assess the efficacy of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII/IX




Primary Outcome Measures :
  1. Proportion of Successfully Treated Mild/Moderate Bleeding Episodes [ Time Frame: 12 hours after first administration of study drug ]

    For the primary efficacy endpoint, successful treatment of a bleeding episode was defined as a combination of the following:

    • "Good" or "Excellent" response noted by the patient
    • Study drug treatment: No further treatment with study drug beyond timepoint for this bleeding episode
    • No other hemostatic treatment needed for this bleeding episode
    • No administration of blood products that would indicate continuation of bleeding beyond timepoint
    • No increase of pain beyond timepoint that could not otherwise be explained


Secondary Outcome Measures :
  1. Proportion of Mild/Moderate Bleeding Episodes With Patient (Pt)-Reported "Good" or "Excellent" Responses at 12 Hours [ Time Frame: at 12 hours ]

    Based on Patient-Reported "Good" or "Excellent" responses as per the below descriptions:

    Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.

    Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required.


  2. Time to Assessment of a "Good" or "Excellent" Response of Mild/Moderate Bleeding Episodes by the Patient [ Time Frame: Within 24 hours of Bleeding Episode ]

    Categories of Response to Treatment are Described as Follows:

    None: No noticeable effect of the treatment on the bleed or worsening of patient's condition. Continuation of treatment with the study drug was needed.

    Moderate: Some effect of the treatment on the bleed was noticed, e.g., pain decreased or bleeding signs improved, but bleed continued and required continued treatment with the study drug. Good: Symptoms of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage) had largely been reduced by the treatment, but had not completely disappeared. Symptoms had improved enough to not require more infusions of the study drug.

    Excellent: Full relief of pain and cessation of objective signs of bleed (e.g., swelling, tenderness, and decreased range of motion in the case of musculoskeletal haemorrhage). No additional infusion of study drug was required.


  3. Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode [ Time Frame: Within 24 hours of Bleeding Episode ]
  4. Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode [ Time Frame: Through study completion ]


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.


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Ages Eligible for Study:   12 Years to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • be male with a diagnosis of congenital hemophilia A and/or B of any severity
  • have one of the following:
  • a positive inhibitor test Bethesda Unit (BU) ≥ 5 (as confirmed at screening by the institutional lab), OR
  • a BU<5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of Factor VIII or IX products to treat bleedings, OR
  • a BU<5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the subject's medical history, precluding the use of Factor VIII or IX products to treat bleedings
  • be 12 years or older, up to and including 75 years of age (NOTE: different age restrictions may apply per local regulation and/or ethical considerations)
  • have at least 3 bleeding episodes of any severity in the past 6 months be capable of understanding and willing to comply with the conditions of the protocol
  • have read, understood and provided written informed consent (patient and/or parent(s)/legal guardian(s) if <18 years of age)

Exclusion Criteria:

  • have any coagulation disorder other than hemophilia A or B
  • be immuno-suppressed (i.e., the patient should not be receiving systemic immunosuppressive medication, cluster of differentiation 4 (CD4) counts at screening should be >200/µl)
  • have a known allergy or hypersensitivity to rabbits
  • have platelet count <100,000/mL
  • have had within one month prior to first administration of the study drug in this study a major surgical procedure (e.g. orthopedic, abdominal)
  • have received an investigational drug within 30 days of the first study drug administration, or is expected to receive such drug during participation in this study
  • have a clinically relevant hepatic (AST and/or alanine aminotransferase (ALT) >3 times the upper limit of normal) and/or renal impairment (creatinine >2 times the upper limit of normal)
  • have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or pulmonary embolism) within 2 years prior to first dose of study drug, or current New York Heart Association (NYHA) functional classification score of stage II -IV
  • have an active malignancy (those with non-melanoma skin cancer are allowed)
  • have any life-threatening disease or other disease or condition which, according to the investigator's judgment, could imply a potential hazard to the patient, interfere with the trial participation or trial outcome (e.g., a history of non-responsiveness to bypassing products).

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): NCT02020369


Locations
Show Show 17 study locations
Sponsors and Collaborators
rEVO Biologics
Laboratoire français de Fractionnement et de Biotechnologies
Investigators
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Principal Investigator: Jean Francois Schved, MD Saint Eloi Hospital

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Responsible Party: rEVO Biologics
ClinicalTrials.gov Identifier: NCT02020369    
Other Study ID Numbers: RB-FVIIa-006-13
First Posted: December 24, 2013    Key Record Dates
Results First Posted: June 14, 2017
Last Update Posted: June 14, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked