Phase II Study of IL-11 (Neumega) in Von Willebrand Disease
Status and phase
Conditions
Treatments
Details and patient eligibility
About
This study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate:
- if rhIL-11 corrects VWF (Von Willebrand Factor) levels to normal
- if rhIL-11 and DDAVP together will boost VWF levels even higher
- the onset, peak, and duration of rhIL-11 effect
- if rhIL-11 is safe in individuals with Von Willebrand Disease
Full 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:
- age >= 18 years of age
- 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:
- to compare the hemostatic efficacy of three escalating doses of rhIL-11
- to determine the biologic effects of rhIL-11
- to determine whether DDAVP, when given after the seventh daily dose of rhIL-11, enhances hemostatic efficacy or rhIL-11
- 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.
Enrollment
Sex
Ages
Volunteers
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
Trial design
Primary purpose
Allocation
Interventional model
Masking
12 participants in 3 patient groups
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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