Apixaban in Patients With Sickle Cell Disease

NCT02179177 | Terminated Phase 3 Results DMC

• 1 other identifier: Pro00048953
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

In patients with SCD, the use of low dose anticoagulation as an outpatient may lead to a significant decrease in morbidity and as a result, decrease healthcare utilization and costs. This study attempts to critically avoid admissions by reducing daily pain scores and pain crisis as an outpatient by use of a novel oral anticoagulant.

Conditions

Vaso-occlusive Crisis; Reduction in Hospitalizations; Sickle Cell Disease

Keywords

Vaso-occlusive crisis; Reduction in hospitalizations; Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Change in Pain as Measured by Visual Analog Scale (VAS)

  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.

  Month 1 to Month 8

Secondary Outcomes (3)

• Change in Thrombin Generation Using D-dimer Measurement as a Surrogate

  Enrollment to 2 months

• Daily Pain Scores While Hospitalized as Measured by VAS

  up to 8 months

• Number of Hospitalizations During Treatment

  up to 8 months

Study Arms (2)

Apixaban

ACTIVE COMPARATOR

Active drug Apixaban 2.5mg taken by mouth twice a day

Drug: Apixaban

Placebo

PLACEBO COMPARATOR

Sugar pills that look like Apixaban that will be taken by mouth twice a day

Drug: Placebo

Interventions

Apixaban DRUG

Drug is taken by mouth twice a day for 6 months

Apixaban

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• documented HgbSS, SC or HgbS-beta0 thalassemia,
• age ≥18 years old and ≤80,
• seen in outpatient clinic ≥2 times in past year
• seen for an acute care visit (hospitalization, emergency department, or day hospital visit) for pain \>2 times in the past year.

You may not qualify if:

• Hospitalization or day hospital visit for pain crisis within the past 2 weeks
• Patients with ≥10 acute care visits within the past year will be excluded
• Creatinine \>3.0 mg/dL
• creatinine ≥1.5 mg/dL AND weight ≤60 kg
• chronic use of antiplatelet or anticoagulation medication
• Patients with known vasculopathy or Moya-Moya
• platelet count \<100 X 109/L
• AST or ALT \>3 times normal
• chronic red blood cell transfusions (scheduled transfusions)
• packed red blood cell transfusion within the past 2 months
• Use of CYP3A4 and P-gp inhibitor medications

Sponsors & Collaborators

• Nirmish Shah: lead
• Bristol-Myers Squibb: collaborator

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

Vaso-Occlusive Crises; Anemia, Sickle Cell

Interventions

apixaban

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Nirmish Shah, MD
• Organization: Duke University

nirmish.shah@duke.edu

919-668-5178

Study Officials

• Nirmish Shah, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: June 27, 2014
• First Posted: July 1, 2014
• Study Start: January 1, 2015
• Primary Completion: September 3, 2017
• Study Completion: September 3, 2017
• Last Updated: March 11, 2020
• Results First Posted: March 11, 2020

Record last verified: 2020-02

Locations

US (1)