NCT01027377

Brief Summary

The study is to investigate the safety, tolerability, and pharmacokinetics (the determination of the concentration of the administered drug in blood over time) of recombinant Factor VIII Fc fusion protein (rFVIIIFc) in previously-treated subjects with severe hemophilia A.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Dec 2009

Geographic Reach
3 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2009

Completed
3 days until next milestone

First Submitted

Initial submission to the registry

December 4, 2009

Completed
3 days until next milestone

First Posted

Study publicly available on registry

December 7, 2009

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2010

Completed
10 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2011

Completed
Last Updated

December 19, 2020

Status Verified

August 1, 2018

Enrollment Period

7 months

First QC Date

December 4, 2009

Last Update Submit

December 16, 2020

Conditions

Severe Hemophilia A

Outcome Measures

Primary Outcomes (1)

  • Safety will be assessed by physical examination, vital signs, laboratory changes overtime, adverse events, and assessment of inhibitor (Bethesda) and antibody development against rFVIIIFc.

    78 weeks

Secondary Outcomes (3)

  • Pharmacokinetic parameters will be measured such as, but not limited to Tmax, Cmax, t1/2, CL, Vd, AUC, MRT, and incremental recovery.

    78 weeks

  • To determine the pharmacokinetics parameters after single administration of 25 and 65 IU/kg of rFVIIFc compared to advate

    78 weeks

  • To determine the pharmacodynamic activity of FVIII over time for both doses of rFVIIIFc

    78 weeks

Study Arms (2)

A

EXPERIMENTAL

Cohort to receive a single low dose intravenous injection of commercially available rFVIII with safety and PK assessments followed by a single low dose intravenous injection of rFVIIIFc with safety and PK assessments

Drug: rFVIIIFc

B

EXPERIMENTAL

Cohort to receive a single high dose intravenous injection of commercially available rFVIII with safety and PK assessments followed by a single high dose intravenous injection of rFVIIIFc with safety and PK assessments

Drug: rFVIIIFc

Interventions

rFVIIIFcDRUG

Single dose

Also known as: Recombinant Factor VIII fusion protein
A

Eligibility Criteria

Age12 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male and 12 years of age and older and weigh at least 40 kg
  • Diagnosed with severe hemophilia A (baseline Factor VIII level less than 1%)
  • History of at least 100 exposure days to any Factor VIII product

You may not qualify if:

  • History of Factor VIII inhibitors
  • Kidney or liver dysfunction
  • Diagnosed with another coagulation defect other than hemophilia A

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Research Site

Los Angeles, California, United States

Location

Research Site

Sacramento, California, United States

Location

Research Site

Waltham, Massachusetts, United States

Location

Research Site

Pittsburgh, Pennsylvania, United States

Location

Research Site

Seattle, Washington, United States

Location

Research Site

Hong Kong, Hong Kong

Location

Research Site

Tel Aviv, Israel

Location

Related Publications (2)

  • 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.

    PMID: 33719084DERIVED

  • Powell JS, Josephson NC, Quon D, Ragni MV, Cheng G, Li E, Jiang H, Li L, Dumont JA, Goyal J, Zhang X, Sommer J, McCue J, Barbetti M, Luk A, Pierce GF. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood. 2012 Mar 29;119(13):3031-7. doi: 10.1182/blood-2011-09-382846. Epub 2012 Jan 5.

    PMID: 22223821DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

factor VIII-Fc fusion protein

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 4, 2009

First Posted

December 7, 2009

Study Start

December 1, 2009

Primary Completion

July 1, 2010

Study Completion

May 1, 2011

Last Updated

December 19, 2020

Record last verified: 2018-08

Locations

US(5)IL(1)HK(1)