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IL-11 in Women With Von Willebrand Disease and Refractory Menorrhagia

Primary Purpose

Von Willebrand Disease

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Oprelvekin, Interleukin 11, IL-11

About this trial

This is an interventional prevention trial for Von Willebrand Disease focused on measuring IL-11, von Willebrand disease, von Willebrand factor, VWFmRNA, menorrhagia

Eligibility Criteria

18 Years - 45 Years (Adult)FemaleDoes not accept healthy volunteers

Inclusion Criteria:

Females 18-45 years of age
Confirmed type 1 VWD, as defined by low VWF:RCoF or low VWF:Ag and qualitatively normal VWF multimers
Menorrhagia refractory to estrogens, hormones, hemostatic agents
Willingness to have blood drawn

Exclusion Criteria:

Use of immunomodulatory or experimental drugs, or diuretics
Pregnant or lactating women or those unwilling to use contraception during study
Previous 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 substantial quantities of FVIII and/or VWF within five days of study

Sites / Locations

Hemophilia Center of Western PA

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Oprelvekin, Interleukin 11, IL-11

Arm Description

25 micrograms/kg by subcutaneous injection once daily for four days, then once daily on day 1-7 during each of six consecutive menstrual cycles

Outcomes

Primary Outcome Measures

>50% Reduction in PBAC (Pictorial Blood Assessment Chart) at 6 Months

Subjective estimate of blood loss was measured by using a Pictorial blood assessment chart (PBAC) . The PBAC measures scores on a scale where 0 to 100 is normal and greater than 100-200 are abnormal.

Secondary Outcome Measures

No. of Subjects With Detectable VWF mRNA (Von Willebrand Factor Messenger RNA).

No. of subjects with detectable VWFmRNA, a measure of IL-11 (interleukin-11) function, specifically increasing VWF synthesis.

No. of Subjects With IL-11 Associated Adverse Events.

The number of subjects with IL-11 associated adverse events.

Full Information

NCT ID

NCT00524342

First Posted

August 31, 2007

Last Updated

January 5, 2018

Sponsor

Margaret Ragni

Collaborators

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

1. Study Identification

Unique Protocol Identification Number

NCT00524342

Brief Title

IL-11 in Women With Von Willebrand Disease and Refractory Menorrhagia

Official Title

Phase II Clinical Efficacy Trial of Recombinant Interleukin-11 (rhIL-11, Neumega) in Women With Type 1 Von Willebrand Disease and Refractory Menorrhagia

Study Type

Interventional

2. Study Status

Record Verification Date

January 2018

Overall Recruitment Status

Completed

Study Start Date

January 2008 (undefined)

Primary Completion Date

December 2010 (Actual)

Study Completion Date

December 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Margaret Ragni

Collaborators

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

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purpose of this study is to determine the efficacy and safety of rhIL-11, when given subcutaneously for six consecutive months, in reducing menstrual blood loss in women with type 1 von Willebrand disease and refractory menorrhagia. Efficacy will be measured by subjective bleeding severity scale and pictorial bleeding chart. Safety will be measured by the frequency of adverse events, including fever, headache, fatigue, myalgias, arthralgias, fluid retention, or edema.

Detailed Description

This study is a prospective, single-center Phase II trial of Neumega (rhIL-11) in women with type 1 VWD and menorrhagia refractory to estrogen, hormones, or hemostatic agents. It is anticipated that 10 subjects who meet eligibility criteria will enroll and complete this study. All aspects of this study, including the rhIL-11 injections and the screening, hemostatic and safety monitoring, and coagulation testing, are considered experimental. The specific objectives are to determine the efficacy and safety of rhIL-11 in reducing menstrual blood loss in adult women with type 1 VWD during six consecutive menstrual cycles, and to determine the mechanism of the hemostatic response of rhIL-11.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Von Willebrand Disease

Keywords

IL-11, von Willebrand disease, von Willebrand factor, VWFmRNA, menorrhagia

7. Study Design

Primary Purpose

Prevention

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

7 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Oprelvekin, Interleukin 11, IL-11

Arm Type

Experimental

Arm Description

25 micrograms/kg by subcutaneous injection once daily for four days, then once daily on day 1-7 during each of six consecutive menstrual cycles

Intervention Type

Drug

Intervention Name(s)

Oprelvekin, Interleukin 11, IL-11

Other Intervention Name(s)

Neumega (Oprelvekin, Interleukin 11, IL-11)

Intervention Description

25 micrograms/kg by subcutaneous injection once daily for four days, then once daily on day 1-7 during each of six consecutive menstrual cycles

Primary Outcome Measure Information:

Title

>50% Reduction in PBAC (Pictorial Blood Assessment Chart) at 6 Months

Description

Subjective estimate of blood loss was measured by using a Pictorial blood assessment chart (PBAC) . The PBAC measures scores on a scale where 0 to 100 is normal and greater than 100-200 are abnormal.

Time Frame

6 months

Secondary Outcome Measure Information:

Title

No. of Subjects With Detectable VWF mRNA (Von Willebrand Factor Messenger RNA).

Description

No. of subjects with detectable VWFmRNA, a measure of IL-11 (interleukin-11) function, specifically increasing VWF synthesis.

Time Frame

The time frame is up to 7 months per subject.

Title

No. of Subjects With IL-11 Associated Adverse Events.

Description

The number of subjects with IL-11 associated adverse events.

Time Frame

The time frame is up to 7 months per subject.

10. Eligibility

Sex

Female

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Females 18-45 years of age Confirmed type 1 VWD, as defined by low VWF:RCoF or low VWF:Ag and qualitatively normal VWF multimers Menorrhagia refractory to estrogens, hormones, hemostatic agents Willingness to have blood drawn Exclusion Criteria: Use of immunomodulatory or experimental drugs, or diuretics Pregnant or lactating women or those unwilling to use contraception during study Previous 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 substantial quantities of FVIII and/or VWF within five days of study

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Margaret V. Ragni, MD, MPH

Organizational Affiliation

University of Pittsburgh Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Hemophilia Center of Western PA

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15213-4306

Country

United States

12. IPD Sharing Statement

Citations:

Citation

Jankowitz RC, Nichols TC, Ragni MV. Recombinant IL-11 (Neumega, rhIL-11) increases plasma Von Willebrand Factor in Type 1 Von Willebrand Disease. Blood 108: 1003, 2006 (abstract).

Results Reference

background

PubMed Identifier

18680527

Citation

Ragni MV, Jankowitz RC, Chapman HL, Merricks EP, Kloos MT, Dillow AM, Nichols TC. A phase II prospective open-label escalating dose trial of recombinant interleukin-11 in mild von Willebrand disease. Haemophilia. 2008 Sep;14(5):968-77. doi: 10.1111/j.1365-2516.2008.01827.x. Epub 2008 Aug 1.

Results Reference

background

PubMed Identifier

21833452

Citation

Ragni MV, Jankowitz RC, Jaworski K, Merricks EP, Kloos MT, Nichols TC. Phase II prospective open-label trial of recombinant interleukin-11 in women with mild von Willebrand disease and refractory menorrhagia. Thromb Haemost. 2011 Oct;106(4):641-5. doi: 10.1160/TH11-04-0274. Epub 2011 Aug 11.

Results Reference

result

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IL-11 in Women With Von Willebrand Disease and Refractory Menorrhagia

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