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

This study has been completed.
Sponsor:
Information provided by:
Recoly N.V.
ClinicalTrials.gov Identifier:
NCT00245297
First received: October 26, 2005
Last updated: October 15, 2007
Last verified: October 2007
  Purpose

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 Intervention Phase
Haemophilia A
Drug: Kogenate FS, reconstituted in a suspension of liposomes
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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.

Resource links provided by NLM:


Further study details as provided by Recoly N.V.:

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

Enrollment: 16
Study Start Date: October 2005
Study Completion Date: April 2006
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.

  Eligibility

Ages Eligible for Study:   18 Years to 60 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00245297

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

Publications:
ClinicalTrials.gov Identifier: NCT00245297     History of Changes
Other Study ID Numbers: OMRI Lip-FVIII-05-1
Study First Received: October 26, 2005
Last Updated: October 15, 2007
Health Authority: Russia: Ministry of Health of the Russian Federation

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

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
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 22, 2014