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

Recoly

Status and phase

Completed

Phase 2

Conditions

Haemophilia A

Treatments

Drug: Kogenate FS, reconstituted in a suspension of liposomes

Study type

Interventional

Funder types

Industry

Identifiers

NCT00245297

OMRI Lip-FVIII-05-1

Details and patient eligibility

About

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

Full 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.

Enrollment

16 patients

Sex

Male

Ages

18 to 60 years old

Volunteers

No Healthy Volunteers

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