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

Von Willebrand Disease Clinical Trial

Official title:

Phase II Comparison Study of Hemostatic Efficacy of Escalating Doses of Interleukin-11 (rhIL-11, Neumega) in Subjects With Type 1 Von Willebrand Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00151125
• Other study ID #: 0403006
• Secondary ID: 0403006
• Status: Completed
• Phase: Phase 2
• First received: September 6, 2005
• Last updated: May 16, 2016
• Start date: July 2004
• Est. completion date: December 2007

Study information

• Verified date: May 2016
• Source: University of Pittsburgh
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: December 2007
• Est. primary completion date: December 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Intervention

Drug:
• recombinant interleukin-11
25 mcg/kg subcutaneously daily for seven days
• recombinant interleukin-11
50 mcg/kg subcutaneously daily for 7 days
• recombinant interleukin-11
10 mcg/kg subcutaneously daily for 7 days

Locations

Country	Name	City	State
United States	Hemophilia Center of Western Pennsylvania and General Clinical Research Center	Pittsburgh	Pennsylvania

Sponsors (3)

Lead Sponsor	Collaborator
University of Pittsburgh	University of North Carolina, Wyeth is now a wholly owned subsidiary of Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	No
Secondary	The number and frequency of IL-11 associated adverse events.		The time frame is up to 14 days per subject.	Yes
Secondary	The mechanism of IL-11 biologic effect by VWFmRNA.		The time frame is within 14 days per subject.	No

External Links

•   Bleeding Disorders, Von Willebrand Disease