Von Willebrand DiseasePhase 2NCT00151125
Phase II Study of IL-11 (Neumega) in Von Willebrand Disease
A Phase 2 study testing recombinant interleukin-11 in people with Von Willebrand Disease, run by University of Pittsburgh at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- recombinant interleukin-11
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2004to Dec 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Hemophilia Center of Western Pennsylvania and General Clinical Research Center
Pittsburgh, Pennsylvania, 15213-4306
About this study
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
This study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate:
- if rhIL-11 corrects VWF (Von Willebrand Factor) levels to normal
- if rhIL-11 and DDAVP together will boost VWF levels even higher
- the onset, peak, and duration of rhIL-11 effect
- if rhIL-11 is safe in individuals with Von Willebrand Disease
Read the 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:
- age >= 18 years of age
- 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:
- to compare the hemostatic efficacy of three escalating doses of rhIL-11
- to determine the biologic effects of rhIL-11
- to determine whether DDAVP, when given after the seventh daily dose of rhIL-11, enhances hemostatic efficacy or rhIL-11
- 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.
- Conditions
- Von Willebrand Disease
- Being tested
- recombinant interleukin-11
- Keywords
- Von Willebrand Disease, Bleeding, Coagulation, Hemostatic agent
- Medical subject headings
- von Willebrand Diseases, Hemorrhage, Thrombosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant interleukin-11Drug
Also called rhIL-11, Neumega
25 mcg/kg subcutaneously daily for seven days
recombinant interleukin-11Drug
Also called rhIL-11, Neumega
50 mcg/kg subcutaneously daily for 7 days
recombinant interleukin-11Drug
Also called rhIL-11, Neumega
10 mcg/kg subcutaneously daily for 7 days
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | rhIL-11 (Interleukin-11, Neumega) 25 mcg/kg subcutaneously daily for 7 days Receives: Drug: recombinant interleukin-11 |
| B | Experimental | rhIL-11 (interleukin-11, Neumega) 50 mcg/kg subcutaneously daily for 7 days Receives: Drug: recombinant interleukin-11 |
| C | Experimental | rhIL-11 (Interleukin-11, Neumega) 10 mg/kg subcutaneously daily for 7 days Receives: Drug: recombinant interleukin-11 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number and percent increase of VWD coagulation tests after seven daily doses of rhIL-11, boosted by DDAVP day 7.
Measured: The time frame is up to 14 days per subject.
Secondary outcomes (2)
The number and frequency of IL-11 associated adverse events.
Measured: The time frame is up to 14 days per subject.
The mechanism of IL-11 biologic effect by VWFmRNA.
Measured: The time frame is within 14 days per subject.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2005 (estimated)
- Study startJul 2004
- Primary completionDec 2007
- Study completionDec 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand DiseaseStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe sponsor's other registered studies.
- 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Collaborators
- Wyeth is now a wholly owned subsidiary of Pfizer, University of North Carolina
- Responsible party
- Principal investigator, Margaret Ragni, University of Pittsburgh
- Investigators
- Margaret V. Ragni, MD, MPH, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00151125
- Sponsor's protocol ID
- 0403006
- Other IDs
- 0403006
- Status last verified
- May 2016
- First submitted
- 6 Sep 2005
- Last updated
- 17 May 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00151125
- Acronym / sponsor ID
- 0403006
- Conditions
- Von Willebrand Disease
- Keywords
- Von Willebrand Disease, Bleeding, Coagulation, Hemostatic agent
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Collaborators
- Wyeth is now a wholly owned subsidiary of Pfizer, University of North Carolina
- Study start
- Jul 2004
- Primary completion
- Dec 2007 (actual)
- Study completion
- Dec 2007 (actual)
- First posted
- 8 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is testing the use of rhIL-11 (recombinant interleukin 11, Neumega) in individuals with Von Willebrand disease. The purpose is to evaluate:
- if rhIL-11 corrects VWF (Von Willebrand Factor) levels to normal
- if rhIL-11 and DDAVP together will boost VWF levels even higher
- the onset, peak, and duration of rhIL-11 effect
- if rhIL-11 is safe in individuals with Von Willebrand Disease
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:
- age >= 18 years of age
- 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:
- to compare the hemostatic efficacy of three escalating doses of rhIL-11
- to determine the biologic effects of rhIL-11
- to determine whether DDAVP, when given after the seventh daily dose of rhIL-11, enhances hemostatic efficacy or rhIL-11
- 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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: recombinant interleukin-11rhIL-11 (Interleukin-11, Neumega) 25 mcg/kg subcutaneously daily for 7 days |
| B | Experimental | Drug: recombinant interleukin-11rhIL-11 (interleukin-11, Neumega) 50 mcg/kg subcutaneously daily for 7 days |
| C | Experimental | Drug: recombinant interleukin-11rhIL-11 (Interleukin-11, Neumega) 10 mg/kg subcutaneously daily for 7 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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
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 |
|---|---|---|
| recombinant interleukin-11Also: rhIL-11, Neumega25 mcg/kg subcutaneously daily for seven days | Drug | A |
| recombinant interleukin-11Also: rhIL-11, Neumega50 mcg/kg subcutaneously daily for 7 days | Drug | B |
| recombinant interleukin-11Also: rhIL-11, Neumega10 mcg/kg subcutaneously daily for 7 days | Drug | C |
Primary outcome
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.
2 secondary outcomes
The number and frequency of IL-11 associated adverse events.
Time frame: 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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2016.
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HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
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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
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 recombinant interleukin-11 in people with Von Willebrand Disease, run by University of Pittsburgh 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 6 inclusion and 10 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 17 May 2016. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 May 2016; AzTrials last refreshed its copy on 8 Nov 2026.