NCT00623480

Brief Summary

To evaluate the effect of secondary prophylaxis as compared to episodic treatment on bleeding frequency (number of bleeds per year) and on joint damage.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
84

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Mar 2008

Longer than P75 for phase_3

Geographic Reach
4 countries

38 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

First Submitted

Initial submission to the registry

February 4, 2008

Completed
22 days until next milestone

First Posted

Study publicly available on registry

February 26, 2008

Completed
4 days until next milestone

Study Start

First participant enrolled

March 1, 2008

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2011

Completed
1.9 years until next milestone

Results Posted

Study results publicly available

August 7, 2013

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2013

Completed
Last Updated

November 17, 2014

Status Verified

November 1, 2014

Enrollment Period

3.5 years

First QC Date

February 4, 2008

Results QC Date

May 2, 2013

Last Update Submit

November 5, 2014

Conditions

Keywords

Hemophilia A

Outcome Measures

Primary Outcomes (1)

  • Bleeding Frequency (Number of Total Bleeds)

    After the last enrolled patient has been in the study for 1 year. At the cut-off, the median follow-up duration was 616 days (minimum was 111 days and maximum was 1109 days)

Secondary Outcomes (2)

  • Change From Baseline to 3 Years in the MRI (Magnetic Resonance Imaging) Scale.

    Baseline and 3 years

  • Change From Baseline to 3 Years in the Colorado Adult Joint Assessment Scale

    Baseline and 3 years

Other Outcomes (1)

  • Change From Baseline to 3 Years in the Physical Functioning Domain of the Haemo-QoL-A

    Baseline and 3 years

Study Arms (2)

Recombinant Factor VIII prophylaxis treatment

EXPERIMENTAL

Participants received 25 IU/kg of Recombinant Factor VIII (Kogenate FS, BAY14-2222) intravenously (IV), 3 times per week. Dose escalation steps by 5 IU/kg (to 30 IU/kg or 35 IU/kg maximum) for patients exhibiting a bleeding frequency of 12 bleeding episodes per year or greater.

Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Recombinant Factor VIII on-demand treatment

EXPERIMENTAL

Participants received Recombinant Factor VIII (Kogenate FS, BAY14-2222) IV for bleeds in accordance with package insert instructions and study physician recommendations.

Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Interventions

Prophylaxis treatment includes three times per week administration of 25 IU/kg of Kogenate FS. Dose escalation steps by 5 IU/kg (to 30 IU/kg or 35 IU/kg maximum) exhibiting a bleeding frequency of 12 bleeding episodes per year or greater.

Recombinant Factor VIII prophylaxis treatment

Eligibility Criteria

Age12 Years - 50 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Males aged 12 to 50 years (US and Argentina)
  • Males aged 18 to 50 years (other countries)
  • Subjects with at least 150 prior exposure days with any FVIII
  • Subjects who have been on episodic treatment and no known regular prophylaxis treatment for more than 12 consecutive months in the previous 5 years
  • Subjects with 6 to 24 bleeding events and/or treatments in the previous 6 months prior to study entry which are documented and available in the subjects medical records. Documentation can include records from previous physicians, specific home treatment records, emergency room or hospital records, x-ray reports, etc. The investigator can also document with a detailed note the number of bleeds reported by the subject in the last 6 months.
  • Subjects with inhibitor formation surveillance (inhibitor or recovery testing) over the ten years prior to enrollment documented by the investigator and who do not have a history of any of the following:
  • A positive inhibitor titer of 5.0 Bethesda Unit (BU) or greater by either BU assay system at any time since first exposure to exogenous factor VIII
  • A positive inhibitor test result of 1.0 or greater performed by the original BU assay at any time in the past 10 years (A subject can have more than one positive inhibitor test of 0.6 or greater by the original BU assay test but all must be less than 1.0 BU using the original BU assay.)
  • A positive inhibitor test result of 0.6 or greater performed by the Nijmegen method at any time in the past 10 years
  • Subjects with no inhibitor activity by Nijmegen-modified Bethesda assay, either positive (\> 0.6 BU is considered positive) or borderline (\> 0.3 and \< 0.6 BU is considered borderline) as measured in the current study reference laboratory

You may not qualify if:

  • Subjects with any other bleeding disease besides hemophilia A (i.e. von Willebrand disease)
  • Subjects with thrombocytopenia (platelets \< 100,000/mm3)
  • Subjects with abnormal renal function (Cockcroft-Gault Creatinine Clearance value of 60 mL/min or lower)
  • Subjects with active hepatic disease (Aspartate aminotransferase \[AST\] or Alanine aminotransferase \[ALT\] \> 5xUpper Limit of Normal (ULN))
  • Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study (the following drugs are however allowed: interferon-a treatment for Hepatitis C virus (HCV), Highly active anti-retroviral therapy (HAART) therapy for human immunodeficiency virus (HIV) and/or a total of two courses of pulse treatment with steroids for a maximum of 7 days at 1mg/kg or less)
  • Subjects with an absolute CD4 lymphocyte cell count \< 200 cells/mm3 (due to HIV, HCV or another suspected medical condition)
  • Subjects with known hypersensitivity to rFVIII, mouse or hamster proteins
  • Subjects who are receiving or had received other experimental drugs within 1 month prior to study entry
  • Subjects who require any pre-medication to tolerate FVIII injections (e.g. anti-histamines)
  • Subjects who are unwilling to comply with study visits or either of the possible treatment regimens
  • Subjects who have a planned orthopedic intervention to be performed during the study that may substantially affect bleeding (e.g. surgical or chemical or radiological synovectomy)
  • Subjects who are not suitable for participation in this study for any reason, according to the Investigator
  • Subjects who have poor joint status as defined by routine need for a wheelchair or unable to ambulate without the assistance of a brace, cane or crutches
  • Three or more joints that are already fused or "frozen" also called ankylosis

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (38)

Unknown Facility

Tucson, Arizona, 85724, United States

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Little Rock, Arkansas, 72202, United States

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Orange, California, 92868, United States

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Sacramento, California, 95817, United States

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Aurora, Colorado, 80045, United States

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Washington D.C., District of Columbia, 20007-2197, United States

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Unknown Facility

Orlando, Florida, 32801, United States

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Unknown Facility

Atlanta, Georgia, 30322, United States

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Chicago, Illinois, 60611, United States

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Chicago, Illinois, 60612, United States

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Indianapolis, Indiana, 46260, United States

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Unknown Facility

Iowa City, Iowa, 52242-1089, United States

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Unknown Facility

Louisville, Kentucky, 40202, United States

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Boston, Massachusetts, 02115, United States

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Detroit, Michigan, 48201-2196, United States

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Minneapolis, Minnesota, 55455, United States

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Kansas City, Missouri, 64108, United States

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Las Vegas, Nevada, 89109-2803, United States

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Newark, New Jersey, 07112, United States

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New York, New York, 10029, United States

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New York, New York, 10065, United States

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Cleveland, Ohio, 44106, United States

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Hershey, Pennsylvania, 17033-0850, United States

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Pittsburgh, Pennsylvania, 15213, United States

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Knoxville, Tennessee, 37920, United States

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Houston, Texas, 77030, United States

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Salt Lake City, Utah, 84132, United States

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Milwaukee, Wisconsin, 53226, United States

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Buenos Aires, Ciudad Auton. de Buenos Aires, C1221 ADC, Argentina

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Unknown Facility

Rosario, Santa Fe Province, S2000CKF, Argentina

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Plovdiv, 4002, Bulgaria

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Unknown Facility

Sofia, 1756, Bulgaria

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Varna, 9010, Bulgaria

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Unknown Facility

Timișoara, Timiș County, 300011, Romania

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Unknown Facility

Brasov, 50035, Romania

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Unknown Facility

Bucharest, 022328, Romania

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Unknown Facility

Bucharest, 11026, Romania

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Unknown Facility

Constanța, 900591, Romania

Location

Related Publications (2)

  • Manco-Johnson MJ, Kempton CL, Reding MT, Lissitchkov T, Goranov S, Gercheva L, Rusen L, Ghinea M, Uscatescu V, Rescia V, Hong W. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost. 2013 Jun;11(6):1119-27. doi: 10.1111/jth.12202.

  • Manco-Johnson MJ, Lundin B, Funk S, Peterfy C, Raunig D, Werk M, Kempton CL, Reding MT, Goranov S, Gercheva L, Rusen L, Uscatescu V, Pierdominici M, Engelen S, Pocoski J, Walker D, Hong W. Effect of late prophylaxis in hemophilia on joint status: a randomized trial. J Thromb Haemost. 2017 Nov;15(11):2115-2124. doi: 10.1111/jth.13811. Epub 2017 Oct 10.

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIIIF8 protein, humanBAY 14-2222

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Results Point of Contact

Title
Therapeutic Area Head
Organization
BAYER

Study Officials

  • Bayer Study Director

    Bayer

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

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

Study Record Dates

First Submitted

February 4, 2008

First Posted

February 26, 2008

Study Start

March 1, 2008

Primary Completion

September 1, 2011

Study Completion

November 1, 2013

Last Updated

November 17, 2014

Results First Posted

August 7, 2013

Record last verified: 2014-11

Locations