Trial Outcomes & Findings for Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions (NCT NCT02731157)

Last Updated: 2019-01-25

Results Overview

The %HbA decrement is the current pre-treatment HbA - previous post treatment HbA in %. The average %HbA decrement per day was calculated using matched pairs.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

4 participants

Primary outcome timeframe

6 months

Results posted on

2019-01-25

Participant Flow

Participant milestones

Participant milestones
Measure	All Study Participants Each participant in this 4 subject pilot study acted as their own control. Red cell exchanges (RCEs) occurred approximately every month and, over a 6 month period, the sequence of exchanges for each patient was: SRRRSS, where S=standard and R=rejuvenated RCEs.
First Standard RCE STARTED	4
First Standard RCE COMPLETED	4
First Standard RCE NOT COMPLETED	0
First Rejuvenated RCE STARTED	4
First Rejuvenated RCE COMPLETED	3
First Rejuvenated RCE NOT COMPLETED	1
Second Rejuvenated RCE STARTED	3
Second Rejuvenated RCE COMPLETED	3
Second Rejuvenated RCE NOT COMPLETED	0
Third Rejuvenated RCE STARTED	3
Third Rejuvenated RCE COMPLETED	3
Third Rejuvenated RCE NOT COMPLETED	0
Second Standard RCE STARTED	3
Second Standard RCE COMPLETED	3
Second Standard RCE NOT COMPLETED	0
Third Standard RCE STARTED	3
Third Standard RCE COMPLETED	3
Third Standard RCE NOT COMPLETED	0

Reasons for withdrawal

Reasons for withdrawal
Measure	All Study Participants Each participant in this 4 subject pilot study acted as their own control. Red cell exchanges (RCEs) occurred approximately every month and, over a 6 month period, the sequence of exchanges for each patient was: SRRRSS, where S=standard and R=rejuvenated RCEs.
First Rejuvenated RCE Withdrawal by Subject	1

Baseline Characteristics

Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Study Participants n=4 Participants Each participant in this 4 subject pilot study acted as their own control. Red cell exchanges (RCEs) occurred approximately every month and, over a 6 month period, the sequence of exchanges for each patient was: SRRRSS, where S=standard and R=rejuvenated RCEs.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Each participant acted as their own control with a sequence of rejuvenated and standard exchanges over 6 months. One subject ended further study participation after only 1 rejuvenated RBC exchange; limited data available.

The %HbA decrement is the current pre-treatment HbA - previous post treatment HbA in %. The average %HbA decrement per day was calculated using matched pairs.

Outcome measures

Outcome measures
Measure	Transfusion With Rejuvenated Red Blood Cells (RBCs) n=4 Participants Subjects with sickle cell disease will receive RBCs treated with Rejuvesol®. Scheduled red cell exchanges performed with the last 4 units of the exchange having been incubated with Rejuvesol® solution. Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study. Rejuvesol Blood transfusion: Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.	Transfusion With Standard Red Blood Cells n=4 Participants Subjects with sickle cell disease will receive standard RBCs. Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study. Blood transfusion: Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.
Average Percent Hemoglobin (HbA) Decrement Per Day	0.629 %HbA per day Interval 0.261 to 0.846	0.659 %HbA per day Interval 0.261 to 0.846

Adverse Events

Transfusion With Standard RBCs

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Transfusion With Rejuvenated RBCs

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Ian Welsby, M.D.

Duke University

Phone: 919-681-6752

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place