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

This study has been completed.
Sponsor:
Collaborators:
Wyeth is now a wholly owned subsidiary of Pfizer
University of North Carolina
Information provided by (Responsible Party):
Margaret Ragni, University of Pittsburgh
ClinicalTrials.gov Identifier:
NCT00151125
First received: September 6, 2005
Last updated: June 5, 2014
Last verified: June 2014
  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:

  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

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: Parallel 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:


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:

  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.

  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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 Ragni, 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: June 5, 2014
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
Oprelvekin
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 22, 2014