Phase 2
N=9
Efficacy and Safety of IL-11 in DDAVP Unresponsive
Hemophilia A · Von Willebrand Disease
Bottom Line
View on ClinicalTrials.gov: NCT00994929 ↗Enrolled (actual)
9
Serious AEs
0.0%
Results posted
Mar 2016
Primary outcome: Primary: Biologic Effects by Coagulation Tests — 34.75; 143.4 percentage of normal
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Neumega (Oprelvekin, Interleukin 11, IL-11) (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- University of Pittsburgh
- Primary completion
- Apr 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Biologic Effects by Coagulation Tests |
34.75; 143.4 | — |
| SECONDARY The Frequency of Adverse Events |
6 | — |
| SECONDARY The Mechanism of Study Drug Effect by VWF mRNA. |
0.81 | — |
Summary
The purpose of this study is to determine the biologic efficacy and safety of rhIL-11 when given subcutaneously in adults with moderate or mild hemophilia A or Von Willebrand disease unresponsive to DDAVP. Biologic efficacy will be measured by the number and percent increase of VWD coagulation tests (FVIII:C, VWF: Ag, VWF: RCo, closure time, APTT, and VWF multimers) to the normal range, or at least to 1.5-3 time baseline, following dosing of rhIL-11 when given daily for 4 days, and boosted by DDAVP infusion on day 4, in those in whom DDAVP is not contraindicated. Safety will be measured by the frequency of adverse events, including fever, headache, fatigue, myalgias, arthralgias, fluid retention, or edema.
Eligibility Criteria
Inclusion Criteria
- Males or females >= 18 years of age.
- A diagnosis of hemophilia A, moderate (FVIII:C 0.01-0.04 U/ml) or mild (FVIII:C >= 0.05 U/ml); or a diagnosis of VWD, defined by a low VWF:RCo and past bleeding history.
- For those with VWD, an inability to use DDAVP due to i) unresponsiveness defined as VWF:RCo or FVIII:C level lower than 50 IU/dl or less than a 3-fold increase after 0.3 microgram/kg DDAVP; ii) allergic reactions or seizures; or iii) a contraindication to DDAVP.
- Willingness to have blood drawn.
- Willingness to sign informed consent.
Exclusion Criteria
- Presence of other bleeding disorders, acquired Von Willebrand disease, primary thrombocytopenia.
- Use of immunomodulatory or experimental drugs, or diuretics.
- Pregnant or lactating women.
- Previous cardiac disease, congestive failure, arrhythmia (e.g. atrial fibrillation, atrial flutter), hypertension, MI, stroke, or thrombosis.
- Past allergic reaction to Neumega.
- 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 substantial quantities of FVIII and/or VWF within five days of study.
- Baseline safety and/or hematology lab values outside the normal limits and/or an EKG indicating an arrhythmia.
Data sourced from ClinicalTrials.gov (NCT00994929). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.