Clinical trial NCT02448680: A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hemophilia A With Inhibitors
Hemophilia B With Inhibitors
Lead sponsor: Laboratoire français de Fractionnement et de Biotechnologies
Sponsor class: INDUSTRY
Collaborators: name: LFB USA, Inc.
First posted: 2015-05-19
Enrollment: 25
Sex: MALE
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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).

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

Eligibility 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

Primary outcomes:
measure: Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement.
timeFrame: 12 hours after first administration of study drug
measure: Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition
timeFrame: 12 hours after first administration of study drug
Secondary outcomes:
measure: Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
timeFrame: 12 hour after first administration of study drug
measure: Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes
timeFrame: Within 24 hours of Bleeding Episode
measure: Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode
timeFrame: Within 24 hours of Bleeding Episode
measure: Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode
timeFrame: Within 24 hours of Bleeding Episode
Other outcomes:
measure: Mild/Moderate Bleeding Episodes With Successful Pain Relief
timeFrame: 12 hour after first administration of study drug
Locations:
city: Aurora
country: United States
facility: University of Colorado Denver Hemophilia & Thrombosis Center
state: Colorado
city: Oklahoma City
country: United States
facility: Jimmy Everest Center for Cancer and Bleeding Disorders
state: Oklahoma
city: Dallas
country: United States
facility: UT Southwestern Medical Center at Dallas / Children's Medical Center
state: Texas
city: Plovdiv
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment "Sveti Georgi"
city: Prague
country: Czechia
facility: University Hospital Motol
city: Tbilisi
country: Georgia
facility: Hematology of Department Hemophilia and Thromboses center
city: Benoni
country: South Africa
facility: Worthwhile Clinical Trials
city: Kyiv
country: Ukraine
facility: National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine)
city: Lviv
country: Ukraine
facility: Institute of Blood Pathology and Transfusion Medicine
Study officials:
affiliation: University of Colorado, Denver
name: Michael Wang, MD
role: PRINCIPAL_INVESTIGATOR
References: