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

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

Study Description

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 Design

• 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

Arms and Interventions

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

Outcome Measures

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)

Eligibility Criteria

• 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

Contacts and Locations

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

More Information

• Responsible Party: LFB USA, Inc.
• ClinicalTrials.gov Identifier: NCT02448680
• Other Study ID Numbers: LFB-FVIIa-007-14
• First Posted: May 19, 2015
• 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