Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00245297
Recruitment Status : Completed
First Posted : October 27, 2005
Last Update Posted : October 16, 2007
Information provided by:
Recoly N.V.

Brief Summary:

Primary efficacy endpoint: To study whether there is a difference in the length of the bleeding free periods between infusion of FVIII that is reconstituted with 22.1, 12.6, or 4.2 mg of pegylated liposomes per kg bwt compared with standard formulation.

Safety endpoint: To study the safety and tolerability of Kogenate FS reconstituted with Pegylated liposomes

Condition or disease Intervention/treatment Phase
Haemophilia A Drug: Kogenate FS, reconstituted in a suspension of liposomes Phase 2

Detailed Description:
Each subject will receive treatment for three bleeding episodes ("wash-in", on demand injections) followed by a minimum 4 day wash-out. Following this each subject will be randomized to a specific treatment order. Each order consists of four identical blocks. In each block the following three injections will be given: 1 One prophylactic treatment (randomized solution for dissolution) 2 and 3. Two standard on-demand treatments for a spontaneous bleeding episode (standard Kogenate FS). Each block will be followed by a four day wash-out. In total each subject should receive 3+4x3=15 injections. It is estimated that each subject will be in the study for about 4 months. A treatment for a spontaneous bleeding episode may require more than one infusion.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: A Patient-Blinded, Randomized, Comparative, Multicenter, Crossover, (Liposome) Dose Finding Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
Study Start Date : October 2005
Actual Study Completion Date : April 2006

Primary Outcome Measures :
  1. The primary efficacy parameter is the number of bleeding free days (without a bleeding/treatment) between a randomized prophylactic infusion and following on demand injection. [ Time Frame: Bleeding free time following four single doses will be studies ]

Secondary Outcome Measures :
  1. In vivo recovery (IVR). [ Time Frame: IVR will be performed at the first and last dose. ]

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Between 18 and 60 years of age
  • Severe haemophilia A (≤ 1% of baseline Factor VIII activity)
  • At least 250 treatment cumulative exposure-days (CEDs) to previous products
  • At least 25 cumulative exposure-days (CEDs) to previous products during one year prior to study start
  • If HIV positive, CD4 lymphocytes ≥ 400/µl
  • Subjects on demand treatment, and with a minimum bleeding/treatment pattern of a four episodes per month, evenly distributed within each month, during the three month preceding study start
  • Subjects who have given their written informed consent.

Exclusion Criteria:

  • Inhibitors or history of inhibitors
  • History of adverse reactions related to Factor VIII
  • Platelet count <90,000 /µl
  • Subjects on prophylaxis treatment
  • Subjects with concomitant debilitating disease (e.g. cancer, non-controlled diabetes, heart insufficiency, renal failure)
  • Subjects with known sensitivity to blood products
  • Subjects who have participated in another Ethical Committee approved Clinical Trial (including medical device studies) within the past 30 days
  • Subjects with a weight over 86 kg or below 50 kg
  • Subjects who do not understand or are not willing to comply with the requirement of the study protocol
  • Subjects who cannot differentiate a bleeding episode from other causes of joint pain

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00245297

Russian Federation
Center for Hematological Research, Department of Reconstructive Orthopedic Surgery
Moscow, Russian Federation, 125167
The Russian Academy of Medical Sciences, Haemophilia Center
Moscow, Russian Federation, 125167
Republic Haemophilia Center.
St. Petersburg, Russian Federation, 191186
Sponsors and Collaborators
Recoly N.V.
Study Director: Jack Spira, MD. PhD. Recoly C/o InSpira Medical AB, Nasbyv 38, 13553 Tyreso Sweden

Publications: Identifier: NCT00245297     History of Changes
Other Study ID Numbers: OMRI Lip-FVIII-05-1
First Posted: October 27, 2005    Key Record Dates
Last Update Posted: October 16, 2007
Last Verified: October 2007

Keywords provided by Recoly N.V.:
Bleeding disorder
severe Haemophilia A

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII