Von Willebrand DiseasePhase 2NCT00524342
IL-11 in Women With Von Willebrand Disease and Refractory Menorrhagia
A Phase 2 study testing Oprelvekin, Interleukin 11, IL-11 in people with Von Willebrand Disease, run by Margaret Ragni at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years, women only
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- Oprelvekin, Interleukin 11, IL-11
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2008to Dec 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Hemophilia Center of Western PA
Pittsburgh, Pennsylvania, 15213-4306
About this study
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
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.
Read the 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.
- Conditions
- Von Willebrand Disease
- Being tested
- Oprelvekin, Interleukin 11, IL-11
- Keywords
- IL-11, von Willebrand disease, von Willebrand factor, VWFmRNA, menorrhagia
- Medical subject headings
- von Willebrand Diseases, Menorrhagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oprelvekin, Interleukin 11, IL-11Drug
Also called Neumega (Oprelvekin, Interleukin 11, IL-11)
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
| Study group | Role | What they receive |
|---|---|---|
| Oprelvekin, Interleukin 11, IL-11 | Experimental | 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 Receives: Drug: Oprelvekin, Interleukin 11, IL-11 |
What is being measured
The primary outcome is the main question the study is designed to answer.
>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.
Measured: 6 months
Secondary outcomes (2)
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.
Measured: 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.
Measured: The time frame is up to 7 months per subject.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2007 (estimated)
- Study startJan 2008
- Primary completionDec 2010
- Study completionDec 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Von Willebrand DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Margaret Ragni
- Sponsor type
- University, hospital or other
- Collaborators
- University of North Carolina, Wyeth is now a wholly owned subsidiary of Pfizer
- Responsible party
- Sponsor-investigator, Margaret Ragni, University of Pittsburgh
- Investigators
- Margaret V. Ragni, MD, MPH, Principal investigator, University of Pittsburgh Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00524342
- Sponsor's protocol ID
- PRO07040157
- Other IDs
- Wyeth 102344
- Status last verified
- Jan 2018
- First submitted
- 31 Aug 2007
- Last updated
- 31 Jan 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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).
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.
PubMed 18680527Ragni 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.
PubMed 21833452
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00524342
- Acronym / sponsor ID
- PRO07040157
- Conditions
- Von Willebrand Disease
- Keywords
- IL-11, von Willebrand disease, von Willebrand factor, VWFmRNA, menorrhagia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Margaret RagniUniversity, hospital or other
- Collaborators
- University of North Carolina, Wyeth is now a wholly owned subsidiary of Pfizer
- Study start
- Jan 2008
- Primary completion
- Dec 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 3 Sep 2007 (estimated)
- Results
- Posted 7 Nov 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Oprelvekin, Interleukin 11, IL-11 | Experimental | Drug: Oprelvekin, Interleukin 11, IL-1125 micrograms/kg by subcutaneous injection once daily for four days, then once daily on day 1-7 during each of six consecutive menstrual cycles |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Oprelvekin, Interleukin 11, IL-11Also: Neumega (Oprelvekin, Interleukin 11, IL-11)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 | Drug | Oprelvekin, Interleukin 11, IL-11 |
Primary outcome
>50% Reduction in PBAC (Pictorial Blood Assessment Chart) at 6 Months
Time frame: 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.
2 secondary outcomes
No. of Subjects With Detectable VWF mRNA (Von Willebrand Factor Messenger RNA).
Time frame: The time frame is up to 7 months per subject.
No. of Subjects With IL-11 Associated Adverse Events.
Time frame: The time frame is up to 7 months per subject.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- BackgroundJankowitz 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).
- Background: PubMed 18680527Ragni 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 publication: PubMed 21833452Ragni 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.
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Von Willebrand Diseases
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Margaret V. Ragni, MD, MPH principal investigator
University of Pittsburgh Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Oprelvekin, Interleukin 11, IL-11 in people with Von Willebrand Disease, run by Margaret Ragni at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Nov 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
>50% Reduction in PBAC (Pictorial Blood Assessment Chart) at 6 Months (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00524342, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2018.