Phase II Study of IL-11 (Neumega) in Von Willebrand Disease

Phase 2

Completed

• Conditions: Von Willebrand Disease
• Interventions: Drug: recombinant interleukin-11

• Registration Number: NCT00151125
• Lead Sponsor: University of Pittsburgh

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.

Study Timeline

• Study Start: 2004-07
• Study First Submitted: 2005-09-06
• Study First Submitted QC: 2005-09-06
• Study First Posted: 2005-09-08
• Primary Completion: 2007-12
• Study Completion: 2007-12
• Status Verified: 2016-05
• Last Update Submitted: 2016-05-16
• Last Update Posted: 2016-05-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
B	recombinant interleukin-11	rhIL-11 (interleukin-11, Neumega) 50 mcg/kg subcutaneously daily for 7 days
A	recombinant interleukin-11	rhIL-11 (Interleukin-11, Neumega) 25 mcg/kg subcutaneously daily for 7 days
C	recombinant interleukin-11	rhIL-11 (Interleukin-11, Neumega) 10 mg/kg subcutaneously daily for 7 days

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Hemophilia Center of Western Pennsylvania and General Clinical Research Center; 🇺🇸 Pittsburgh, Pennsylvania, United States