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

Margaret Ragni7 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

PREVENTION

Masking

NONE

Enrollment

Conditions

Von Willebrand Disease

Interventions

Oprelvekin, Interleukin 11, IL-11DRUG

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

Eligibility

Sex: FEMALEMin age: 18 YearsMax age: 45 Years

Medical Language ↔ Plain English

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

Locations (1)

Hemophilia Center of Western PA

Pittsburgh, Pennsylvania, United States

Outcomes

Primary Outcomes

>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.

Time frame: 6 months

Secondary Outcomes

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.

Time frame: The time frame is up to 7 months per subject.

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

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

Time frame: The time frame is up to 7 months per subject.

Data from ClinicalTrials.gov

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