Trial Outcomes & Findings for Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients (NCT NCT01736657)

Results Overview

The primary endpoint evaluated the mean ratio of the Actual Fraction of Cells Remaining (FCRa: as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp: as predicted by the Spectra Optia system FCR algorithm multiplied by the Pre-Procedure % HbS), in the evaluable population (60 pts). The pre-defined range for the mean ratio of the FCRa to the FCRp was 0.75 to 1.25.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

73 participants

Primary outcome timeframe

Length of the procedure

Results posted on

2014-07-14

Participant Flow

Recruitment period: Nov 2012 through May 2013

Evaluable population includes 60 pts. Terumo Optia Trainer was present for Optia Operator support for 12 procedures, called "lead-in" procedures: these were not included in efficacy analysis, but were included in safety analysis (full analysis set).

Participant milestones

Participant milestones
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Overall Study STARTED	73
Overall Study COMPLETED	72
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Overall Study insufficient vascular access	1

Baseline Characteristics

Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease n=72 Participants Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Age, Categorical <=18 years	26 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	46 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Age, Continuous	23.0 years STANDARD_DEVIATION 10.32 • n=99 Participants
Sex: Female, Male Female	29 Participants n=99 Participants
Sex: Female, Male Male	43 Participants n=99 Participants
Region of Enrollment United States	72 participants n=99 Participants

PRIMARY outcome

Timeframe: Length of the procedure

Population: 60 patients analyzed, 73 enrolled:12 patients were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 pts), but were included in the safety analysis (72 patients); 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.

The primary endpoint evaluated the mean ratio of the Actual Fraction of Cells Remaining (FCRa: as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp: as predicted by the Spectra Optia system FCR algorithm multiplied by the Pre-Procedure % HbS), in the evaluable population (60 pts). The pre-defined range for the mean ratio of the FCRa to the FCRp was 0.75 to 1.25.

Outcome measures

Outcome measures
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease n=60 Participants Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Mean Ratio Actual Fraction of Cells Remaining (FCRa; as Measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as Predicted by the Spectra Optia System FCR Algorithm Multiplied by the Pre-Procedure % HbS)	0.9 ratio Interval 0.855 to 0.941

SECONDARY outcome

Timeframe: Length of the procedure

Population: 60 patients analyzed, 73 enrolled:12 patients were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 pts), but were included in the safety analysis (72 patients); 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.

The procedural success of the Spectra Optia System is defined as the ability of the device to complete a red blood cell exchange (RBCx) and to obtain a satisfactory exchange by lowering the patient's hemoglobin S, as determined by the investigator in the evaluable population (60 pts).

Outcome measures

Outcome measures
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease n=60 Participants Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Procedural Success of the Spectra Optia System in the Evaluable Population	100 percentage of participants

SECONDARY outcome

Timeframe: Length of the procedure

Population: 60 patients analyzed, 73 enrolled:12 patients were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 pts), but were included in the safety analysis (72 patients); 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.

Measurement of the patient post-procedure hematocrit compared to the final target hematocrit calculated by the Spectra Optia Apheresis System. Final target hematocrit was calculated by tracking the number of red cells coming into the system versus the number of red cells removed.

Outcome measures

Outcome measures
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease n=60 Participants Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population	1.03 ratio Interval 1.011 to 1.045

SECONDARY outcome

Timeframe: upon signing consent to 24 hours post-procedure

Population: Seventy-three enrolled: 12 were lead-in patients whereby Optia Operators completed their training and data from those procedures were not included in the efficacy analysis (60 patients), but these patients were included in safety analysis as Full Analysis Set; 1 patient was consented but withdrawn prior to procedure due to lack of vascular access.

Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).

Outcome measures

Outcome measures
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease n=72 Participants Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Device-related Serious Adverse Events (SAE) in the Full Analysis Set	0 participants

Adverse Events

Red Blood Cell Exchange for Patients With Sickle Cell Disease

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Red Blood Cell Exchange for Patients With Sickle Cell Disease n=72 participants at risk Red blood cell exchange or depletion/exchange for patients with sickle cell disease : The purpose of this study is to evaluate the performance of the Red Blood Cell Exchange protocol on the Spectra Optia Apheresis System.
Gastrointestinal disorders Nausea	5.6% 4/72 • Number of events 4
Nervous system disorders Dizziness	8.3% 6/72 • Number of events 6

Additional Information

Ray Goodrich, Phd, VP Scientifc and Clinical Affairs

Terumo BCT, Inc.

Phone: 303-205-2680

Results disclosure agreements

Principal investigator is a sponsor employee If Study Site is performing services in a multi-center Study, it agrees not to independently publish, publicly disclose, present or discuss any results of or information pertaining to the Study until a multi-center manuscript is published; provided however, that if a multi-center manuscript is not published within one year after completion of the Study at all Study sites, Study Site may publish the Study data generated by Study Site
Publication restrictions are in place

Restriction type: OTHER