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

May 16, 2016 updated by: Margaret Ragni, University of Pittsburgh

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • rhIL-11, Neumega
Drug: recombinant interleukin-11
50 mcg/kg subcutaneously daily for 7 days
Other Names:
  • rhIL-11, Neumega
Drug: recombinant interleukin-11
10 mcg/kg subcutaneously daily for 7 days
Other Names:
  • rhIL-11, Neumega
Experimental: B
rhIL-11 (interleukin-11, Neumega) 50 mcg/kg subcutaneously daily for 7 days
Drug: recombinant interleukin-11
25 mcg/kg subcutaneously daily for seven days
Other Names:
  • rhIL-11, Neumega
Drug: recombinant interleukin-11
50 mcg/kg subcutaneously daily for 7 days
Other Names:
  • rhIL-11, Neumega
Drug: recombinant interleukin-11
10 mcg/kg subcutaneously daily for 7 days
Other Names:
  • rhIL-11, Neumega
Experimental: C
rhIL-11 (Interleukin-11, Neumega) 10 mg/kg subcutaneously daily for 7 days
Drug: recombinant interleukin-11
25 mcg/kg subcutaneously daily for seven days
Other Names:
  • rhIL-11, Neumega
Drug: recombinant interleukin-11
50 mcg/kg subcutaneously daily for 7 days
Other Names:
  • rhIL-11, Neumega
Drug: recombinant interleukin-11
10 mcg/kg subcutaneously daily for 7 days
Other Names:
  • rhIL-11, Neumega

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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.
The time frame is up to 14 days per subject.

Secondary Outcome Measures

Outcome Measure
Time Frame
The number and frequency of IL-11 associated adverse events.
Time Frame: The time frame is up to 14 days per subject.
The time frame is up to 14 days per subject.
The mechanism of IL-11 biologic effect by VWFmRNA.
Time Frame: The time frame is within 14 days per subject.
The time frame is within 14 days per subject.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Pittsburgh

Collaborators

Wyeth is now a wholly owned subsidiary of Pfizer
University of North Carolina

Investigators

  • Principal Investigator: Margaret V. Ragni, MD, MPH, University of Pittsburgh

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2004

Primary Completion (Actual)

December 1, 2007

Study Completion (Actual)

December 1, 2007

Study Registration Dates

First Submitted

September 6, 2005

First Submitted That Met QC Criteria

September 6, 2005

First Posted (Estimate)

September 8, 2005

Study Record Updates

Last Update Posted (Estimate)

May 17, 2016

Last Update Submitted That Met QC Criteria

May 16, 2016

Last Verified

May 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 0403006 (Other Identifier: protocol id)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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