Ignite Creation Date:
2024-05-05 @ 9:57 PM
Last Modification Date:
2024-10-26 @ 10:11 AM
Study NCT ID:
NCT00994929
Status:
COMPLETED
Last Update Posted:
2016-03-02
First Post:
2009-10-12
Brief Title:
Efficacy and Safety of IL-11 in DDAVP Unresponsive
Sponsor:
University of Pittsburgh
Organization:
University of Pittsburgh
Study Overview
Official Title:
Phase II Biologic Effects Study of Recombinant Interleukin-11 rhIL-11 Neumega in Subjects With Moderate or Mild Hemophilia A or Von Willebrand Disease Unable to Use DDAVP
Status:
COMPLETED
Status Verified Date:
2016-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
IL-11DDAVP
Brief Summary:
The purpose of this study is to determine the biologic efficacy and safety of rhIL-11 when given subcutaneously in adults with moderate or mild hemophilia A or Von Willebrand disease unresponsive to DDAVP Biologic efficacy will be measured by the number and percent increase of VWD coagulation tests FVIIIC VWF Ag VWF RCo closure time APTT and VWF multimers to the normal range or at least to 15-3 time baseline following dosing of rhIL-11 when given daily for 4 days and boosted by DDAVP infusion on day 4 in those in whom DDAVP is not contraindicated Safety will be measured by the frequency of adverse events including fever headache fatigue myalgias arthralgias fluid retention or edema
Detailed Description:
This is a prospective single center Phase II biologic effects study of recombinant interleukin-11 rhIL-11 Neumega in subjects hemophilia A moderate or mild or with Von Willebrand disease unable to take desmopressin acetate DDAVP because they are unresponsive allergic or DDAVP is contraindicated The purpose of the study is to establish the biologic efficacy and safety of rhIL-11 in those not able to take DDAVP Study subjects will include adults age 18 years with hemophilia A moderate defined as factor VIII 001-004 Uml or mild defined as factor VIII 005 Uml or with VWD defined by low VWFRCo and or low VWFAg past bleeding history andor family history of VWD A total of 10-16 subjects will be enrolled in order to assure that 10 complete the study The specific aims of the study are 1 to determine the biologic effect of rhIL-11 when given 4 consecutive days 2 to determine the safety of rhIL-11 when used in subjects with hemophilia A moderate or mild or with VWD unresponsive or unable to take DDAVP and 3 to determine the mechanism of the hemostatic effects of rhIL-11 The biologic efficacy outcomes will be measured by VWD-related coagulation tests VWFRCo FVIIIC VWFAg closure times before and after rhIL-11 injection Safety outcomes will be measured by the number and frequency of adverse events including fever headache fatigue myalgias arthralgias fluid retention or edema The mechanism of rhIL-11 hemostatic effect will be measured by VWFmRNA before and after rhIL-11 response Response to DDAVP following rhIL-11 will also be assessed in those in whom DDAVP is not contraindicated The study will last up to 1 month per subject and for 24 months for the entire study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None