- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01405742
Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A
R34 Pilot Feasibility Randomized, Noninferiority, Cross-Over Trial of Once-Weekly vs. Thrice-Weekly Prophylaxis With Recombinant Factor VIII in Adults With Severe Hemophilia A
Study Overview
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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District of Columbia
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Washington, District of Columbia, United States, 20057-1168
- Georgetown University
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104-4318
- Children's Hospital of Philadelphia
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Philadelphia, Pennsylvania, United States, 19104-4206
- Hospital of the University of Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15213-4306
- Hemophilia Center of Western Pennsylvania
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Tennessee
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Nashville, Tennessee, United States, 37235-7749
- Vanderbilt University
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Washington
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Seattle, Washington, United States, 98104-1256
- Puget Sound Blood Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Adult males 18 years or older
- Severe hemophilia A (F.VIII < 0.01 U/ml)
- At least 150 exposure days to F.VIII products
- No detectable inhibitor
- No history of allergic reaction
- Platelets at least 150,000/ul
- If HIV(+), CD4 at least 200/ul, HIV-VL <48 copies/ml,and cART compliant
- If HCV(+), no splenomegaly,varices,GI bleed,ascites,edema,encephalopathy
- Willingness to comply with cross-over design, randomization schema
- Willingness to keep a personal diary of bleeding frequency and factor use
- Willingness to make every 3 month visits, coagulation testing at wks 2, 28
Exclusion Criteria:
- Acquired hemophilia
- Any bleeding disorder other than hemophilia A
- Presence of an inhibitor to factor VIII
- Historic platelet count < 100,000
- Use of experimental drugs
- Surgery anticipate in the next 52 weeks
- Symptomatic HCV(splenomegaly,varices,GI bleed,ascites,edema,encephalopathy)
- Symptomatic HIV(CD4<200/ul or HIV VL 48 or more copy/ml,cART noncompliant)
- Life expectancy less than 5 years
- Investigational drug or study within 4 weeks prior to study
- Inability to comply with study requirements
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Arm A
The intervention for Arm A is 40 IU/kg recombinant factor VIII (rFVIII) by once-weekly intravenous injection for 26 weeks. Cross-over will occur at 26 weeks after a 72 hour washout period, after which 40 IU/kg recombinant factor VIII (rFVIII) will be given thrice-weekly by intravenous injection until week 52, with up to two rescue doses per week for bleeds. |
40 IU/kg recombinant factor VIII will be given once-weekly or thrice-weekly by intravenous injection for 26 weeks.
At 26 weeks after a 72 hour washout period, 40 IU/kg recombinant factor VIII will be given thrice-weekly or once-weekly, respectively, by intravenous injection until week 52, with up to two rescue doses per week for bleeds
Other Names:
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Experimental: Arm B
The intervention for Arm B is 40 IU/kg recombinant factor VIII (rFVIII) by thrice-weekly intravenous injection for 26 weeks. Cross-over will occur at 26 weeks after a 72 hour washout period, after which 40 IU/kg recombinant factor VIII (rFVIII) will be given once-weekly by intravenous injection until week 52, with up to two rescue doses per week for bleeds |
40 IU/kg recombinant factor VIII will be given once-weekly or thrice-weekly by intravenous injection for 26 weeks.
At 26 weeks after a 72 hour washout period, 40 IU/kg recombinant factor VIII will be given thrice-weekly or once-weekly, respectively, by intravenous injection until week 52, with up to two rescue doses per week for bleeds
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Bleeds
Time Frame: Weeks 26 (first intervention) and 52 (second intervention)
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The primary outcome was bleed frequency.
The data were total number of events for each Arm, and not per-participant.
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Weeks 26 (first intervention) and 52 (second intervention)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Inter-dose Hypocoagulability by Thrombin Generation
Time Frame: The time frame is 52 weeks per subject.
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Thrombin generation was performed at week 8 and week 34, i.e. 8 weeks after initiation of factor dosing in Weeks 1-26, and 8 weeks after initiation of factor dosing in Weeks 27-52.
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The time frame is 52 weeks per subject.
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F.VIII Activity
Time Frame: The time frame is 52 weeks per subject.
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F.VIII Activity (IU/mL) performed at week 8 and week 34, i.e. 8 weeks after initiation of factor dosing in Weeks 1-26, and 8 weeks after initiation of factor dosing in Weeks 27-52.
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The time frame is 52 weeks per subject.
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Margaret V. Ragni, MD, MPH, University of Pittsburgh
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- PRO10020178
- R34HL105870-01A1 (U.S. NIH Grant/Contract)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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