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A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)

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ClinicalTrials.gov Identifier: NCT02448680
Recruitment Status : Recruiting
First Posted : May 19, 2015
Last Update Posted : January 25, 2017
Sponsor:
Collaborator:
Laboratoire français de Fractionnement et de Biotechnologies
Information provided by (Responsible Party):
LFB USA, Inc.

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 or IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).

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

Detailed Description:
A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients from birth to <12 years old with Inhibitors to Factor VIII or IX: PerSept 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 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 Pediatric Patients From Birth to <12 Years Old With Inhibitors to Factor VIII or IX: PerSept 2
Study Start Date : December 2015
Estimated Primary Completion Date : June 30, 2017
Estimated Study Completion Date : August 30, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding Hemophilia

Arm Intervention/treatment
Active Comparator: Coagulation Factor VIIa (Recombinant): 75 µg/kg
75 µg/kg treatment regimen for 3 months
Biological: Coagulation FVIIa (Recombinant)
A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/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 or Factor IX

Active Comparator: Coagulation Factor VIIa (Recombinant): 225 µg/kg
225 µg/kg treatment regimen for 3 months
Biological: Coagulation FVIIa (Recombinant)
A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/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 or Factor IX




Primary Outcome Measures :
  1. Bleeding episode treatment success [ Time Frame: 12 hours after first administration of study drug ]
    No additional hemostatic product required after 12 hours of first dose


Secondary Outcome Measures :
  1. Time to bleeding success [ Time Frame: 12 hours ]
    Patients shall rate the treatment of each bleeding episode. If treatment occurs under direct supervision of treating physician, the physician shall rate the response. Ratings based on a four point scale; Excellent, Good, Moderate, None


Other Outcome Measures:
  1. Immunogenicity assessment [ Time Frame: Pre-dose, 3 weeks post dose and then every 6 weeks until end of study ]
    Based on Coagulation Factor VIIa (Recombinant), binding antibody levels

  2. Pharmacokinetic profile assessment [ Time Frame: Half of the patients sampling: at 10±2 minutes, 1 and 4 hours (±10 minutes), and the other half of the patients sampling: at 30±5 minutes and 2 and 8 hours (±10 minutes) relative to the start of infusion of study drug ]
    Based on plasma concentrations of Coagulation Factor VIIa (Recombinant)



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Ages Eligible for Study:   up to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • be male with a diagnosis of congenital hemophilia A or B of any severity
  • have one of the following:
  • a positive inhibitor test BU ≥5, OR
  • a Bethesda Unit (BU) <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR
  • a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes
  • be aged from birth to <12 years old
  • have experienced at least 3 bleeding episodes of any severity in the past 6 months
  • parents or legal guardians must be capable of understanding and be willing to comply with the conditions of the protocol
  • parents or legal guardians must have read, understood, and provided written informed consent

Exclusion Criteria:

  • have any coagulation disorder other than hemophilia A or B
  • be immunosuppressed (i.e., the patient may not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening must be >200/µL)
  • have a known allergy or hypersensitivity to rabbits
  • have platelet count <100,000/mL
  • have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug
  • have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in 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): NCT02448680


Contacts
Contact: Meg Carini, LPN,BS 508-370-5258 meg.carini@lfb-usa.com
Contact: Jeffry Lawrence, MD 508-370-5113 jeff.lawrence@lfb-usa.com

Locations
United States, Colorado
University of Colorado Denver Hemophilia & Thrombosis Center Recruiting
Aurora, Colorado, United States, 80045
Principal Investigator: Michael Wang, MD         
United States, Oklahoma
Jimmy Everest Center for Cancer and Bleeding Disorders Active, not recruiting
Oklahoma City, Oklahoma, United States, 73117
United States, Texas
UT Southwestern Medical Center at Dallas / Children's Medical Center Recruiting
Dallas, Texas, United States, 75390
Contact: Anna Winborn         
Principal Investigator: Janna Journeycake         
Bulgaria
University Multiprofile Hospital for Active Treatment "Sveti Georgi" Active, not recruiting
Plovdiv, Bulgaria
Czech Republic
University Hospital Motol Recruiting
Prague, Czech Republic
Contact: Veronika Cepelakova    +420 (2) 2443 6537    veronika.cepelakova@fnmotol.cz   
Principal Investigator: Vladimir Komrska, MD         
Georgia
Hematology of Department Hemophilia and Thromboses center Recruiting
Tbilisi, Georgia
Contact: Tamila Katselashvili         
Principal Investigator: Tamila Nadiradze         
South Africa
Worthwhile Clinical Trials Recruiting
Benoni, South Africa
Contact: Fadeela Bibi Rahman       fadeela@wwct.co.za   
Principal Investigator: Ismail Haroon Mitha         
Ukraine
National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine) Recruiting
Kyiv, Ukraine
Contact: Kateryna Vilchevska       vilchevskaya@yandex.ru   
Principal Investigator: Kateryna Vilchevska         
Institute of Blood Pathology and Transfusion Medicine Recruiting
Lviv, Ukraine
Contact: Oleksandra Stasyshyn         
Principal Investigator: Oleksandra Stasyshyn         
Sponsors and Collaborators
LFB USA, Inc.
Laboratoire français de Fractionnement et de Biotechnologies
Investigators
Principal Investigator: Michael Wang, MD University of Colorado, Denver

Responsible Party: LFB USA, Inc.
ClinicalTrials.gov Identifier: NCT02448680     History of Changes
Other Study ID Numbers: LFB-FVIIa-007-14
First Posted: May 19, 2015    Key Record Dates
Last Update Posted: January 25, 2017
Last Verified: January 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
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
Factor VIII
Coagulants