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

NCT02448680 | Completed Phase 3 Results DMC

• 1 other identifier: LFB-FVIIa-007-14
• Study Type: interventional
• Target: 25
• Locations: 6 countries
• Sites: 9

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).

Conditions

Hemophilia A With Inhibitors; Hemophilia B With Inhibitors

Outcome Measures

Primary Outcomes (2)

• Proportion of Successfully Treated Mild/Moderate Bleeding Episodes Per FDA Requirement.

  For the primary efficacy endpoint, successful treatment of mild/moderate bleeding episode was defined as meeting all of the following: * "Good" or "excellent" response noted by the patient/parent/legal guardian or other caregiver, depending on patient's age and maturity * Study drug treatment: No further treatment with LR769 beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted * No other hemostatic treatment needed for this bleeding episode * No administration of blood products that would indicate continuation of bleeding beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted * No increase of pain beyond timepoint where a "good" or "excellent" response for this bleeding episode was noted that could not be explained other than as continuation of bleeding

  12 hours after first administration of study drug

• Proportion of Successfully Treated Bleeding Episodes (Mild/Moderate/Severe) Per EMA Definition

  * "Good" or "excellent" response noted by the patient/caregiver for mild/moderate bleeding episodes; * "Good" or "excellent" response noted by the physician for severe bleeding episodes.

  12 hours after first administration of study drug

Secondary Outcomes (4)

• Patient-Reported "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes

  12 hour after first administration of study drug

• Time to Patient Assessment of a "Good" or "Excellent" Response for Mild/Moderate Bleeding Episodes

  Within 24 hours of Bleeding Episode

• Number of Administrations of Study Drug Per Mild/Moderate Bleeding Episode

  Within 24 hours of Bleeding Episode

• Total Amount of Study Drug Administered Per Mild/Moderate Bleeding Episode

  Within 24 hours of Bleeding Episode

Other Outcomes (1)

• Mild/Moderate Bleeding Episodes With Successful Pain Relief

  12 hour after first administration of study drug

Study Arms (2)

Coagulation Factor VIIa (Recombinant): 75 µg/kg

ACTIVE COMPARATOR

75 µg/kg treatment regimen for 3 months

Biological: Coagulation FVIIa (Recombinant)

Coagulation Factor VIIa (Recombinant): 225 µg/kg

ACTIVE COMPARATOR

225 µg/kg treatment regimen for 3 months

Biological: Coagulation FVIIa (Recombinant)

Interventions

Coagulation FVIIa (Recombinant) 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; Coagulation Factor VIIa (Recombinant): 75 µg/kg

Eligibility Criteria

• Age: Up to 11 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Laboratoire français de Fractionnement et de Biotechnologies: lead
• LFB USA, Inc.: collaborator

Study Sites (9)

University of Colorado Denver Hemophilia & Thrombosis Center

Aurora, Colorado, 80045, United States

Location

Jimmy Everest Center for Cancer and Bleeding Disorders

Oklahoma City, Oklahoma, 73117, United States

Location

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

Dallas, Texas, 75390, United States

Location

University Multiprofile Hospital for Active Treatment "Sveti Georgi"

Plovdiv, Bulgaria

Location

University Hospital Motol

Prague, Czechia

Location

Hematology of Department Hemophilia and Thromboses center

Tbilisi, Georgia

Location

Worthwhile Clinical Trials

Benoni, South Africa

Location

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

Kyiv, Ukraine

Location

Institute of Blood Pathology and Transfusion Medicine

Lviv, Ukraine

Location

Related Publications (2)

• 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.

  PMID: 40765904 DERIVED

• 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.

  PMID: 40674256 DERIVED

MeSH Terms

Interventions

recombinant FVIIa

Results Point of Contact

• Title: Kerry Biron, Director US Clinical Operations
• Organization: LFB USA, Inc.

kerry.biron@lfb-usa.com

508-370-5166

Study Officials

• Michael Wang, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2015
• First Posted: May 19, 2015
• Study Start: December 7, 2015
• Primary Completion: June 30, 2017
• Study Completion: August 30, 2017
• Last Updated: February 25, 2022
• Results First Posted: August 28, 2020

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will not share

Locations

GE (1); UA (2); US (3); ZA (1); BU (1); CZ (1)