Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

71 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-11-30

Study Completion Date

2013-12-31

Brief Summary

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The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc consumption for prevention and treatment of bleeding episodes.

Detailed Description

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Previously treated pediatric participants will be treated with a prophylactic regimen of rFVIIIFc. PK analysis of pre-study factor VIII (FVIII) and rFVIIIFc will be performed in a sub-group of the study participants prior to commencement of prophylactic treatment. After these PK results are available, remaining participants have the option of proceeding directly to prophylactic treatment. After completing the end of study assessments, eligible participants would be able to continue treatment in Study 8HA01EXT.

Conditions

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Hemophilia A

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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All participants

PK subgroup: After a Washout Period of ≥72 hours, at the Baseline Visit (28±7 days prior to Day 1), participants receive a single IV injection of prestudy FVIII over 5 (±2) minutes at a dose of 50 IU/kg, rounded up to the nearest 250 IU increment, for a PK assessment. Following a second Washout Period of ≥72 hours, participants receive a single IV injection of rFVIIIFc over 5 (±2) minutes at a dose of 50 IU/kg for PK assessment. The first prophylactic dose of rFVIIIFc is administered at a starting dose of 25 IU/kg IV injection on Day 1 and 50 IU/kg on Day 4. Dose increases to a maximum of 80 IU/kg, and frequency of administration to a minimum interval of once every 2 days, are allowed as indicated.

Non-PK subgroup: On Day 1, a first prophylactic dose of rFVIIIFc of 25 IU/kg IV injection is given, followed by a dose of 50 IU/kg on Day 4. Dose increases to a maximum of 80 IU/kg, and frequency of administration to a minimum interval of once every 2 days, are allowed as indicated.

Group Type EXPERIMENTAL

BIIB031 (rFVIIIFc)

Intervention Type DRUG

Vials of rFVIIIFc were combined as needed, based on the actual labeled potency to achieve the participant's calculated dose. Partial vial use was allowed, in order to achieve the calculated dose.

FVIII (PK subgroup only)

Intervention Type DRUG

Baseline prestudy FVIII dosing in participants who enter the PK subgroup. Vials of prestudy FVIII were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose.

Interventions

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BIIB031 (rFVIIIFc)

Vials of rFVIIIFc were combined as needed, based on the actual labeled potency to achieve the participant's calculated dose. Partial vial use was allowed, in order to achieve the calculated dose.

Intervention Type DRUG

FVIII (PK subgroup only)

Baseline prestudy FVIII dosing in participants who enter the PK subgroup. Vials of prestudy FVIII were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose.

Intervention Type DRUG

Other Intervention Names

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ELOCTATE efmoroctocog alfa antihemophilic factor (recombinant), Fc fusion protein recombinant coagulation factor VIII Fc fusion protein

Eligibility Criteria

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Inclusion Criteria

* Severe hemophilia A defined as \<1 IU/dL (\<1%) endogenous FVIII
* Male \<12 years of age and weight ≥13 kg
* History of at least 50 documented prior exposure days to FVIII
* No current, or history of, inhibitor development to FVIII

Exclusion Criteria

* Other coagulation disorders in addition to Hemophilia A
* History of anaphylaxis associated with any FVIII or IV immunoglobulin administration

Maximum Eligible Age

11 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Bioverativ Therapeutics Inc.

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United States Australia Hong Kong Ireland Netherlands Poland South Africa United Kingdom

References

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Raheja P, Kragh N, Bystricka L, Eriksson D, Aroui K, Mezghani M, Barbier S, Linari S. Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes. Ther Adv Hematol. 2024 Jul 30;15:20406207241257917. doi: 10.1177/20406207241257917. eCollection 2024.

Reference Type DERIVED

PMID: 39091324 (View on PubMed)

Katragadda S, Neelakantan S, Diao L, Wong N. Population Pharmacokinetic Analysis of Recombinant Factor VIII Fc Fusion Protein in Subjects With Severe Hemophilia A: Expanded to Include Pediatric Subjects. J Clin Pharmacol. 2021 Jul;61(7):889-900. doi: 10.1002/jcph.1854. Epub 2021 Apr 14.

Reference Type DERIVED

PMID: 33719084 (View on PubMed)

Young G, Mahlangu J, Kulkarni R, Nolan B, Liesner R, Pasi J, Barnes C, Neelakantan S, Gambino G, Cristiano LM, Pierce GF, Allen G. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Haemost. 2015 Jun;13(6):967-77. doi: 10.1111/jth.12911. Epub 2015 Apr 23.

Reference Type DERIVED

PMID: 25912075 (View on PubMed)

Other Identifiers

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2011-003073-28

Identifier Type: -

Identifier Source: secondary_id

8HA02PED

Identifier Type: -

Identifier Source: org_study_id

Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Study Design

Study Groups

All participants

BIIB031 (rFVIIIFc)

FVIII (PK subgroup only)

Interventions

BIIB031 (rFVIIIFc)

FVIII (PK subgroup only)

Other Intervention Names

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Swedish Orphan Biovitrum

Bioverativ Therapeutics Inc.

Responsible Party

Principal Investigators

Medical Director

Locations

Research Site

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Countries

References

Katragadda S, Neelakantan S, Diao L, Wong N. Population Pharmacokinetic Analysis of Recombinant Factor VIII Fc Fusion Protein in Subjects With Severe Hemophilia A: Expanded to Include Pediatric Subjects. J Clin Pharmacol. 2021 Jul;61(7):889-900. doi: 10.1002/jcph.1854. Epub 2021 Apr 14.

Other Identifiers

2011-003073-28

8HA02PED