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

NCT ID: NCT02448680

Last Updated: 2022-02-25

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-12-07

Study Completion Date

2017-08-30

Brief Summary

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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

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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

Conditions

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Hemophilia A With Inhibitors Hemophilia B With Inhibitors

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Coagulation Factor VIIa (Recombinant): 75 µg/kg

75 µg/kg treatment regimen for 3 months

Group Type ACTIVE_COMPARATOR

Coagulation FVIIa (Recombinant)

Intervention Type BIOLOGICAL

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

Coagulation Factor VIIa (Recombinant): 225 µg/kg

225 µg/kg treatment regimen for 3 months

Group Type ACTIVE_COMPARATOR

Coagulation FVIIa (Recombinant)

Intervention Type BIOLOGICAL

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

Interventions

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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

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Maximum Eligible Age

11 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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LFB USA, Inc.

INDUSTRY

Sponsor Role collaborator

Laboratoire français de Fractionnement et de Biotechnologies

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Michael Wang, MD

Role: PRINCIPAL_INVESTIGATOR

University of Colorado, Denver

Locations

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University of Colorado Denver Hemophilia & Thrombosis Center

Aurora, Colorado, United States

Site Status

Jimmy Everest Center for Cancer and Bleeding Disorders

Oklahoma City, Oklahoma, United States

Site Status

UT Southwestern Medical Center at Dallas / Children's Medical Center

Dallas, Texas, United States

Site Status

University Multiprofile Hospital for Active Treatment "Sveti Georgi"

Plovdiv, , Bulgaria

Site Status

University Hospital Motol

Prague, , Czechia

Site Status

Hematology of Department Hemophilia and Thromboses center

Tbilisi, , Georgia

Site Status

Worthwhile Clinical Trials

Benoni, , South Africa

Site Status

National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine)

Kyiv, , Ukraine

Site Status

Institute of Blood Pathology and Transfusion Medicine

Lviv, , Ukraine

Site Status

Countries

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United States Bulgaria Czechia Georgia South Africa Ukraine

References

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Young G, Mahlangu J, Boggio LN, Carcao M, Dargaud Y, Escobar M, Giermasz A, Hermans C, Kuriakose P, Miesbach W, Nance D, Rafique A, Sidonio RF Jr, Vilchevska KV, Wang M, Pipe SW. Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials. Blood Vessel Thromb Hemost. 2025 Mar 27;2(3):100069. doi: 10.1016/j.bvth.2025.100069. eCollection 2025 Aug.

Reference Type DERIVED
PMID: 40765904 (View on PubMed)

Carcao M, Hermans C, Giermasz A, Kessler C, Miesbach W, Quon D, Windyga J, Mahlangu J. Safety and Use of Eptacog Beta 225 microg/kg in Patients With Haemophilia A or B With Inhibitors. Haemophilia. 2025 Sep;31(5):957-965. doi: 10.1111/hae.70083. Epub 2025 Jul 17.

Reference Type DERIVED
PMID: 40674256 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan: Final Version 2.0

View Document

Document Type: Statistical Analysis Plan: Statistical Analysis Plan (v2.0) Addendum

View Document

Other Identifiers

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LFB-FVIIa-007-14

Identifier Type: -

Identifier Source: org_study_id

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