Apixaban in Patients With Sickle Cell Disease
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|ClinicalTrials.gov Identifier: NCT02179177|
Recruitment Status : Terminated (funding has been exhausted)
First Posted : July 1, 2014
Results First Posted : March 11, 2020
Last Update Posted : March 11, 2020
|Condition or disease||Intervention/treatment||Phase|
|Vaso-occlusive Crisis Reduction in Hospitalizations Sickle Cell Disease||Drug: Apixaban Drug: Placebo||Phase 3|
There is not only significant morbidity associated with patients with SCD, but also costs associated with the numerous hospitalizations. Small studies have been unable to show clear benefit of the use of low dose anticoagulation in SCD due to limited sample size or the inclusion of very specific populations. However, studies have shown a decrease in the level of elevated prothrombotic markers with anticoagulation, and one study using full dose anticoagulation in patients with a generally milder form of SCD (with high protective hemoglobin) showed more rapid decrease in clinical pain with use of anticoagulation, suggesting a possible benefit of such therapy. Due to the paucity of data to support therapeutic dose LMWH in the more severe forms of SCD seen in the United States, we have chosen prophylactic dose anticoagulation. This study proposal attempts to critically avoid admissions by reducing daily pain scores and pain crisis as an outpatient by use of a novel oral anticoagulant.
The development of novel anticoagulants such as oral direct factor Xa (FXa) inhibitors allows the realistic use of daily prophylactic dosing as an outpatient. Past studies as detailed earlier have been limited by attempts to use subcutaneous injections or frequent, close monitoring for acenocoumarol treatment, both which are not ideal for chronic daily use. Furthermore, the use of global assays such calibrated automated thrombography (CAT) have shown further details about thrombin generation in a population which is hypercoagulable at baseline.
This is a double blind, parallel group, placebo controlled feasibility study with an enrollment target of 25 patients (12 per arm). All subjects that meet inclusion criteria as an outpatient, following a 1 month observation, will be randomized to receive an oral prophylactic dose factor Xa inhibitor (Apixaban 2.5mg po bid) or placebo for 6 months. Subjects will return for a 30 day (+/- 5 days) follow-up visit after the End of Treatment (EOT) visit. Initial randomization will occur by computerized randomization technique by the investigational drug services (IDS) at Duke University Medical Center.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||16 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||Impact of Daily Prophylaxis Dose Anticoagulation With a Factor Xa Inhibitor (Apixaban) in Patients With Sickle Cell Disease|
|Study Start Date :||January 2015|
|Actual Primary Completion Date :||September 3, 2017|
|Actual Study Completion Date :||September 3, 2017|
Active Comparator: Apixaban
Active drug Apixaban 2.5mg taken by mouth twice a day
Drug is taken by mouth twice a day for 6 months
Placebo Comparator: Placebo
Sugar pills that look like Apixaban that will be taken by mouth twice a day
- Change in Pain as Measured by Visual Analog Scale (VAS) [ Time Frame: Month 1 to Month 8 ]The primary pain assessment tool will be a 10-cm horizontal visual analog scale (VAS), with "0" corresponding to no pain at one end and "10" indicating the worst pain at the other.
- Change in Thrombin Generation Using D-dimer Measurement as a Surrogate [ Time Frame: Enrollment to 2 months ]
- Daily Pain Scores While Hospitalized as Measured by VAS [ Time Frame: up to 8 months ]The primary pain assessment tool will be a 10-cm horizontal visual analog scale (VAS), with "0" corresponding to no pain at one end and "10" indicating the worst pain at the other. Secondary analysis will be performed to evaluate differences when patients are hospitalized and on study drug versus placebo.
- Number of Hospitalizations During Treatment [ Time Frame: up to 8 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02179177
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27710|
|Principal Investigator:||Nirmish Shah, MD||Duke University|