Von Willebrand Disease
Conditions
Keywords
Von Willebrand Disease, Bleeding, Coagulation, Hemostatic agent
Brief summary
This study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate: 1. if rhIL-11 corrects VWF (Von Willebrand Factor) levels to normal 2. if rhIL-11 and DDAVP together will boost VWF levels even higher 3. the onset, peak, and duration of rhIL-11 effect 4. if rhIL-11 is safe in individuals with Von Willebrand Disease
Detailed description
This is a prospective, single center, open-label, escalating dose Phase II comparison study of interleukin-11 (rhIL-11, Neumega) in subjects with type 1 Von Willebrand Disease (VWD). The purpose is to establish the clinical safety and hemostatic efficacy of rhIL-11 in individuals with type 1 Von Willebrand disease. Study subjects will include the following subjects: 1. age \>= 18 years of age 2. diagnosis of VWD confirmed by: 2a) at least 2 of 4 abnormal vWD-related coagulation tests; 2b) a past bleeding history A total of 10-16 subjects are anticipated to be enrolled and complete the study. The specific aims of the study are: 1. to compare the hemostatic efficacy of three escalating doses of rhIL-11 2. to determine the biologic effects of rhIL-11 3. to determine whether DDAVP, when given after the seventh daily dose of rhIL-11, enhances hemostatic efficacy or rhIL-11 4. to compare the safety of three escalating doses of rhIL-11 Efficacy will be based on the number and percent increase of VWD-related coagulation tests into the normal range, or at least to 2-3 times baseline. Safety will be based on the number and frequency of adverse reactions, including fever, headache, fatigue, arthralgias, myalgias, fluid retention, and edema. The study will last up to 4 weeks per subject, and for 24 months for the entire study.
Interventions
25 mcg/kg subcutaneously daily for seven days
Sponsors
Wyeth is now a wholly owned subsidiary of Pfizer
University of North Carolina
University of Pittsburgh
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Males and females 18 years of age or older * Confirmed VWD by 2 of 4 VWD coagulation tests abnormal * A past bleeding history * No hormone, oral contraceptive, estrogen use in past 8 weeks * Willingness to have blood drawn * Willingness to sign informed consent
Exclusion criteria
* Presence of other bleeding disorder, e.g. acquired VWD, thrombocytopenia * Use of estrogens, hormones, oral contraceptives in past 8 weeks * Use of immunomodulatory or experimental drugs or diuretics * Pregnant or lactating women * Past cardiac disease, congestive failure, arrhythmia (e.g. atrial fibrillation, atrial flutter), hypertension, MI, stroke, or thrombosis * Past allergic reaction to Neumega or DDAVP * Surgery within the past 8 weeks * Inability to comply with study protocol requirements * Concomitant use of antiplatelet drugs, anticoagulants, dextran, aspirin, or NSAIDs * Treatment with DDAVP, cryoprecipitate, whole blood, plasma, and plasma derivatives containing FVIII, VWF within 5 days of study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The number and percent increase of VWD coagulation tests after seven daily doses of rhIL-11, boosted by DDAVP day 7. | The time frame is up to 14 days per subject. |
Secondary
| Measure | Time frame |
|---|---|
| The number and frequency of IL-11 associated adverse events. | The time frame is up to 14 days per subject. |
| The mechanism of IL-11 biologic effect by VWFmRNA. | The time frame is within 14 days per subject. |
Countries
United States
Outcome results
None listed