Back to resultsStudy of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
Primary Purpose
Haemophilia A
Status
Completed
Phase
Phase 2
Locations
Russian Federation
Study Type
Interventional
Intervention
Kogenate FS, reconstituted in a suspension of liposomes
Sponsored by
About this trial
This is an interventional prevention trial for Haemophilia A focused on measuring Bleeding disorder, severe Haemophilia A, Prophylaxis
Eligibility 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
Sites / Locations
- Center for Hematological Research, Department of Reconstructive Orthopedic Surgery
- The Russian Academy of Medical Sciences, Haemophilia Center
- Republic Haemophilia Center.
Outcomes
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.
Secondary Outcome Measures
In vivo recovery (IVR).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00245297
Brief Title
Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
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 Type
Interventional
2. Study Status
Record Verification Date
October 2007
Overall Recruitment Status
Completed
Study Start Date
October 2005 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
April 2006 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Recoly N.V.
4. Oversight
Data Monitoring Committee
No
5. Study Description
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
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Haemophilia A
Keywords
Bleeding disorder, severe Haemophilia A, Prophylaxis
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Kogenate FS, reconstituted in a suspension of liposomes
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
In vivo recovery (IVR).
Time Frame
IVR will be performed at the first and last dose.
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jack Spira, MD. PhD.
Organizational Affiliation
Recoly C/o InSpira Medical AB, Nasbyv 38, 13553 Tyreso Sweden
Official's Role
Study Director
Facility Information:
Facility Name
Center for Hematological Research, Department of Reconstructive Orthopedic Surgery
City
Moscow
ZIP/Postal Code
125167
Country
Russian Federation
Facility Name
The Russian Academy of Medical Sciences, Haemophilia Center
City
Moscow
ZIP/Postal Code
125167
Country
Russian Federation
Facility Name
Republic Haemophilia Center.
City
St. Petersburg
ZIP/Postal Code
191186
Country
Russian Federation
12. IPD Sharing Statement
Citations:
PubMed Identifier
15968389
Citation
Baru M, Carmel-Goren L, Barenholz Y, Dayan I, Ostropolets S, Slepoy I, Gvirtzer N, Fukson V, Spira J. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy. Thromb Haemost. 2005 Jun;93(6):1061-8. doi: 10.1160/TH04-08-0485.
Results Reference
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Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
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