Trial Outcomes & Findings for Efficacy and Safety of IL-11 in DDAVP Unresponsive (NCT NCT00994929)
NCT ID: NCT00994929
Last Updated: 2016-03-02
Results Overview
VWF activity was measured by ristocetin-induced platelet agglutination using a Chronolog aggregometer11-14 and VWF:Ag by "sandwich" ELISA, using anti-VWF antibodies (DakoA082, Carpintera CA). Results were expressed in percent, with normal human plasma pool designated 100%, and severe type 3 VWD plasma used as the negative control
COMPLETED
PHASE2
9 participants
within 4 days of study drug.
2016-03-02
Participant Flow
Nine (9) subjects 18 years or older, including five with mild or moderate hemophilia A (HA) and four with mild or moderate von Willebrand disease (VWD) unresponsive or allergic to DDAVP were enrolled in and completed the study between January 2010 and February 2012.
Following enrollment, subjects initiated study drug.
Participant milestones
| Measure |
Neumega (Oprelveken, Interleukin 11)
25 µg/kilogram of body weight of rhIL-11 subcutaneously administered on days 1 to 4. On day 4 following rhIL-11 injection DDAVP was subsequently given at 0.3 mcg/kg intravenously over 30 minutes. For any subject with past allergic reactions, hypersensitivity, or seizures with DDAVP, or in whom DDAVP is contraindicated, DDAVP was not given: only rhIL-11 was given on Day 4.
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|---|---|
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Overall Study
STARTED
|
9
|
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Overall Study
COMPLETED
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9
|
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Overall Study
NOT COMPLETED
|
0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy and Safety of IL-11 in DDAVP Unresponsive
Baseline characteristics by cohort
| Measure |
Neumega (Oprelveken, Interleukin 11)
n=9 Participants
The VWD subjects included two with type 1 VWD and two with type 2 VWD, including one with type 2B, with increased platelet aggregation at low strength ristocetin and absent high molecular weight multimers, and one with type 2M disease, with reduced VWF:RCoF/ VWF:Ag ratio \<0.50, per NHLBI criteria (Table 2).5 All subjects had positive past bleeding histories. Pregnancy, lactation, heart disease, uncontrolled hypertension, arrhythmia, thrombosis, recent surgery or receipt of blood products were exclusions. A total of nine subjects were enrolled and completed study, including five with hemophilia A and four with VWD. The median age was 26 years, range 22-51 years
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
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Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
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Age, Categorical
>=65 years
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0 Participants
n=5 Participants
|
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Age, Continuous
|
26 years
n=5 Participants
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Sex: Female, Male
Female
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4 Participants
n=5 Participants
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Sex: Female, Male
Male
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5 Participants
n=5 Participants
|
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Region of Enrollment
United States
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9 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: within 4 days of study drug.Population: Four subjects had VWD and five subjects had mild hemophilia A
VWF activity was measured by ristocetin-induced platelet agglutination using a Chronolog aggregometer11-14 and VWF:Ag by "sandwich" ELISA, using anti-VWF antibodies (DakoA082, Carpintera CA). Results were expressed in percent, with normal human plasma pool designated 100%, and severe type 3 VWD plasma used as the negative control
Outcome measures
| Measure |
Neumega (Oprelveken, Interleukin 11)
n=9 Participants
Neumega (Oprelvekin, Interleukin 11, IL-11): 25 microgram/kg IL-11 by subcutaneous injection once daily for four days, followed by DDAVP 0.3 microgram/kg by intravenous infusion over 30 minutes on day 4, 30 minutes after IL-11.
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|---|---|
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Biologic Effects by Coagulation Tests
VWD patients
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34.75 percentage of normal
Standard Error 19.2
|
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Biologic Effects by Coagulation Tests
Mild hemophilia A patients
|
143.4 percentage of normal
Standard Error 18.6
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SECONDARY outcome
Timeframe: within 11 days of study drugOutcome measures
| Measure |
Neumega (Oprelveken, Interleukin 11)
n=9 Participants
Neumega (Oprelvekin, Interleukin 11, IL-11): 25 microgram/kg IL-11 by subcutaneous injection once daily for four days, followed by DDAVP 0.3 microgram/kg by intravenous infusion over 30 minutes on day 4, 30 minutes after IL-11.
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|---|---|
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The Frequency of Adverse Events
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6 participants
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SECONDARY outcome
Timeframe: within 11 days of study drug.Outcome measures
| Measure |
Neumega (Oprelveken, Interleukin 11)
n=9 Participants
Neumega (Oprelvekin, Interleukin 11, IL-11): 25 microgram/kg IL-11 by subcutaneous injection once daily for four days, followed by DDAVP 0.3 microgram/kg by intravenous infusion over 30 minutes on day 4, 30 minutes after IL-11.
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|---|---|
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The Mechanism of Study Drug Effect by VWF mRNA.
|
0.81 fold increase
Interval -0.07 to 2.65
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Adverse Events
Neumega (Oprelveken, Interleukin 11)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Neumega (Oprelveken, Interleukin 11)
n=9 participants at risk
Neumega (Oprelvekin, Interleukin 11, IL-11): 25 microgram/kg IL-11 by subcutaneous injection once daily for four days, followed on day 4 by DDAVP 0.3 microgram/kg by intravenous infusion.
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|---|---|
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Eye disorders
Conjunctival Infection
|
66.7%
6/9 • Number of events 6 • January 2010 to February 2012
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Gastrointestinal disorders
Dyspepsia
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33.3%
3/9 • Number of events 3 • January 2010 to February 2012
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General disorders
Fluid Retention
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22.2%
2/9 • Number of events 2 • January 2010 to February 2012
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General disorders
Flu-Like Symptoms
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22.2%
2/9 • Number of events 2 • January 2010 to February 2012
|
|
General disorders
Headache
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22.2%
2/9 • Number of events 2 • January 2010 to February 2012
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General disorders
Injection Site Erythema
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22.2%
2/9 • Number of events 2 • January 2010 to February 2012
|
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General disorders
Lightheadedness
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11.1%
1/9 • Number of events 1 • January 2010 to February 2012
|
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General disorders
Flushing
|
11.1%
1/9 • Number of events 1 • January 2010 to February 2012
|
|
General disorders
Insomnia
|
11.1%
1/9 • Number of events 1 • January 2010 to February 2012
|
|
General disorders
Drowsiness
|
11.1%
1/9 • Number of events 1 • January 2010 to February 2012
|
|
General disorders
Hyponatremia
|
11.1%
1/9 • Number of events 1 • January 2010 to February 2012
|
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General disorders
Thrombocytopenia
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22.2%
2/9 • Number of events 2 • January 2010 to February 2012
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Additional Information
Margaret V. Ragni, MD, MPH
The University of Pittsburgh
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place