Trial Outcomes & Findings for Apixaban in Patients With Sickle Cell Disease (NCT NCT02179177)

NCT ID: NCT02179177

Last Updated: 2020-03-11

Results Overview

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.

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 16 participants
• Primary outcome timeframe: Month 1 to Month 8
• Results posted on: 2020-03-11

Participant Flow

Patients with sickle cell disease were enrolled as an outpatient in clinic while at baseline pain from January 2015 to September 2017.

Participant milestones

Measure	Apixaban Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo
Overall Study STARTED	8	8
Overall Study COMPLETED	6	6
Overall Study NOT COMPLETED	2	2

Reasons for withdrawal

Measure	Apixaban Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo
Overall Study Lost to Follow-up	1	1
Overall Study Withdrawal by Subject	1	1

Baseline Characteristics

Apixaban in Patients With Sickle Cell Disease

Baseline characteristics by cohort

Measure	Apixaban n=8 Participants Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo n=8 Participants Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo	Total n=16 Participants Total of all reporting groups
Age, Continuous	30 years STANDARD_DEVIATION 6.2 • n=5 Participants	33 years STANDARD_DEVIATION 3.9 • n=7 Participants	31.5 years STANDARD_DEVIATION 7.8 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants

PRIMARY outcome

Timeframe: Month 1 to Month 8

Population: Two participants in each group (Apixaban and Placebo) did not return for Month 8 visit.

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.

Outcome measures

Measure	Apixaban n=6 Participants Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo n=6 Participants Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo
Change in Pain as Measured by Visual Analog Scale (VAS)	-1 score on a scale Standard Deviation 0.5	0 score on a scale Standard Deviation 0.4

SECONDARY outcome

Timeframe: Enrollment to 2 months

Population: Data not collected.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 8 months

Population: Two participants from each group (Apixaban and Placebo) did not return for Month 8 visit.

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.

Outcome measures

Measure	Apixaban n=6 Participants Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo n=6 Participants Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo
Daily Pain Scores While Hospitalized as Measured by VAS	6.4 score on a scale Standard Deviation 3.4	6.6 score on a scale Standard Deviation 3.8

SECONDARY outcome

Timeframe: up to 8 months

Population: Two participants from each group (Apixaban and Placebo) did not return for 8 Month visit.

Outcome measures

Measure	Apixaban n=6 Participants Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo n=6 Participants Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo
Number of Hospitalizations During Treatment	3 hospitalizations Standard Deviation 3.3	1.5 hospitalizations Standard Deviation 1.5

Adverse Events

Apixaban

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Apixaban n=8 participants at risk Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo n=8 participants at risk Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo
Pregnancy, puerperium and perinatal conditions Pregnancy	12.5% 1/8 • 8 months	0.00% 0/8 • 8 months
Surgical and medical procedures Hospitalization	12.5% 1/8 • 8 months	0.00% 0/8 • 8 months

Other adverse events

Measure	Apixaban n=8 participants at risk Active drug Apixaban 2.5mg taken by mouth twice a day Apixaban: Drug is taken by mouth twice a day for 6 months	Placebo n=8 participants at risk Sugar pills that look like Apixaban that will be taken by mouth twice a day Placebo
Cardiac disorders Sickle Cell Crisis	12.5% 1/8 • 8 months	0.00% 0/8 • 8 months
Musculoskeletal and connective tissue disorders Sickle Cell Crisis	12.5% 1/8 • 8 months	0.00% 0/8 • 8 months

Additional Information

Nirmish Shah, MD

Duke University

Phone: 919-668-5178

Email: nirmish.shah@duke.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place