Phase 2 Completed N=14 Randomized Quadruple-blind Treatment

The Effect of Rivaroxaban in Sickle Cell Disease

Sickle-Cell Anemia · Sickle Cell-Beta0-Thalassemia

Source: ClinicalTrials.gov NCT02072668 ↗

Enrolled (actual)

Serious AEs

7.4%

Results posted

Apr 2020

Primary outcomePrimary: Change From Baseline to 4 Weeks in Soluble Vascular Cell Adhesion Molecule-1 (VCAM-1) — 40.9; 10.7 pg/mL — p=0.6281

Summary

The primary study hypothesis is that inhibition of factor Xa with rivaroxaban will reduce inflammation, coagulation and endothelial cell activation, and improve microvascular blood flow in patients with sickle cell disease (SCD) during the non-crisis, steady state. To test this hypothesis, this study will evaluate the effects of rivaroxaban on: * plasma markers of inflammation; * plasma markers of endothelial activation; * plasma markers of thrombin generation; and * microvascular blood flow assessed using laser Doppler velocimetry (LDV) of post-occlusive reactive hyperemia (PORH). In a cross-over design, subjects will receive rivaroxaban 20 mg/day and placebo for 4 weeks each, separated by a 2-week washout phase.

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to 4 Weeks in Soluble Vascular Cell Adhesion Molecule-1 (VCAM-1)	40.9; 10.7	0.6281
PRIMARY Change From Baseline to 4 Weeks in Interleukin-6 (IL-6)	-1.1; -0.54	0.7973
SECONDARY Change From Baseline to Week 4 in the Plasma Marker of Inflammation IL-2	-1.14; 0.36	0.4442
SECONDARY Change From Baseline to Week 4 in the Plasma Marker of Inflammation IL-8	0.95; -4.08	0.2545
SECONDARY Change From Baseline to Week 4 in Plasma Marker of Inflammation hsCRP	—	—
SECONDARY Change From Baseline to Week 4 in Plasma Marker of Inflammation MPO	—	—
SECONDARY Change From Baseline to Week 4 in Plasma Marker of Inflammation TNF-a	—	—
SECONDARY Change From Baseline to Week 4 in Plasma Marker of Inflammation sPLA2	—	—
SECONDARY Change From Baseline to Week 4 in Marker of Endothelial Cell (EC) Activation sICAM	—	—
SECONDARY Change From Baseline to Week 4 in TH1	0.84; -0.51	0.4374
SECONDARY Change From Baseline to Week 4 in TM	-0.97; -2.01	0.3470
SECONDARY Change From Baseline to Week 4 in AH	128; -1189	0.0755
SECONDARY Change in Ratio From Baseline to Week 4 in AH/AO	0.05; -0.81	—
SECONDARY Change From Baseline to Week 4 in PF	3.14; -12.62	0.0708
SECONDARY Change From Baseline to Week 4 in RF	0.29; -0.62	0.4501
SECONDARY Change From Baseline to Week 4 in TAT	-34.44; 0.35	0.0767
SECONDARY Change From Baseline to Week 4 in D-Dimer	-471; -1035	0.7250

Eligibility Criteria

Inclusion Criteria

18 to 65 years of age; sickle cell anemia (HbSS) or sickle-beta0 (HbSβ0) thalassemia;
serum creatinine ≤ 1.0 mg/dL men) or 1.2 mg/dL (women);
ALT 3 times upper limit of normal;
on chronic anticoagulant, non-steroidal anti-inflammatory (NSAID) or statin therapy;
history of metastatic cancer;
current alcohol abuse;
on a chronic transfusion program or any blood transfusion in the 3 months prior to enrollment;
ingested any investigational drugs within the past 4 weeks;
use of CYP3A4/P-glycoprotein inducers such as carbamazepine, phenytoin, rifampin, and St John's wort;
use of CYP3A4/P- glycoprotein inhibitors such as ketoconazole, indinavir/ritonavir, itraconazole, lopinavir/ritonavir, ritonavir, and conivaptan.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02072668). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.