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

Purpose

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

Condition	Intervention	Phase
Von Willebrand Disease	Drug: recombinant interleukin-11	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
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

Resource links provided by NLM:

Genetics Home Reference related topics: von Willebrand disease

Drug Information available for: Oprelvekin

U.S. FDA Resources

Further study details as provided by University of Pittsburgh:

Primary Outcome Measures:

The number and percent increase of VWD coagulation tests after seven daily doses of rhIL-11, boosted by DDAVP day 7. [ Time Frame: The time frame is up to 14 days per subject. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

The number and frequency of IL-11 associated adverse events. [ Time Frame: The time frame is up to 14 days per subject. ] [ Designated as safety issue: Yes ]
The mechanism of IL-11 biologic effect by VWFmRNA. [ Time Frame: The time frame is within 14 days per subject. ] [ Designated as safety issue: No ]

Enrollment:	12
Study Start Date:	July 2004
Study Completion Date:	December 2007
Primary Completion Date:	December 2007 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: A rhIL-11 (Interleukin-11, Neumega) 25 mcg/kg subcutaneously daily for 7 days	Drug: recombinant interleukin-11 25 mcg/kg subcutaneously daily for seven days Other Name: rhIL-11, Neumega
Experimental: B rhIL-11 (interleukin-11, Neumega) 50 mcg/kg subcutaneously daily for 7 days	Drug: recombinant interleukin-11 50 mcg/kg subcutaneously daily for 7 days Other Name: rhIL-11, Neumega
Experimental: C rhIL-11 (Interleukin-11, Neumega) 10 mg/kg subcutaneously daily for 7 days	Drug: recombinant interleukin-11 10 mcg/kg subcutaneously daily for 7 days Other Name: rhIL-11, Neumega

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:

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.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00151125

Locations

United States, Pennsylvania
Hemophilia Center of Western Pennsylvania and General Clinical Research Center
Pittsburgh, Pennsylvania, United States, 15213-4306

Sponsors and Collaborators

University of Pittsburgh

Wyeth is now a wholly owned subsidiary of Pfizer

University of North Carolina

Investigators

Principal Investigator:

Margaret V. Ragni, MD, MPH

University of Pittsburgh

More Information

No publications provided

Responsible Party:	Margaret V. Ragni, MD, MPH, Principal Investigator, Professor of Medicine, University of Pittsburgh
ClinicalTrials.gov Identifier:	NCT00151125 History of Changes
Other Study ID Numbers:	0403006, (none)
Study First Received:	September 6, 2005
Last Updated:	February 15, 2008
Health Authority:	United States: Food and Drug Administration

Keywords provided by University of Pittsburgh:

Von Willebrand Disease
Bleeding
Coagulation
Hemostatic agent

Additional relevant MeSH terms:

Von Willebrand Diseases
Von Willebrand Disease, Type 1
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders

Genetic Diseases, Inborn
Hemostatics
Oprelvekin
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions
Antineoplastic Agents

ClinicalTrials.gov processed this record on May 22, 2013