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Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients (ESSENTIAL)

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ClinicalTrials.gov Identifier: NCT01736657
Recruitment Status : Completed
First Posted : November 29, 2012
Results First Posted : July 14, 2014
Last Update Posted : July 14, 2014
Sponsor:
Information provided by (Responsible Party):
Terumo BCT

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sickle Cell Disease
Intervention Device: Red blood cell exchange in sickle cell
Enrollment 73
Recruitment Details Recruitment period: Nov 2012 through May 2013
Pre-assignment Details 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).
Arm/Group Title Red Blood Cell Exchange for Patients With Sickle Cell Disease
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 73
Completed 72
Not Completed 1
Reason Not Completed
insufficient vascular access             1
Arm/Group Title Red Blood Cell Exchange for Patients With Sickle Cell Disease
Hide Arm/Group Description 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 Number of Baseline Participants 72
Hide Baseline Analysis Population Description
Seventy-two patients were in safety analysis and 60 patients (evaluable popluation) were analyzed in the efficacy analysis. The 12 patients not in the efficacy analysis were "lead-in" patients and the RBCX procedures were conducted as the last phase in Operator training on the device.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 72 participants
<=18 years
26
  36.1%
Between 18 and 65 years
46
  63.9%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 72 participants
23.0  (10.32)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 72 participants
Female
29
  40.3%
Male
43
  59.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 72 participants
72
1.Primary Outcome
Title 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)
Hide Description 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.
Time Frame Length of the procedure
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Red Blood Cell Exchange for Patients With Sickle Cell Disease
Hide Arm/Group Description:
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 Number of Participants Analyzed 60
Mean (95% Confidence Interval)
Unit of Measure: ratio
0.9
(0.855 to 0.941)
2.Secondary Outcome
Title Procedural Success of the Spectra Optia System in the Evaluable Population
Hide Description 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).
Time Frame Length of the procedure
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Red Blood Cell Exchange for Patients With Sickle Cell Disease
Hide Arm/Group Description:
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 Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: percentage of participants
100
3.Secondary Outcome
Title Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population
Hide Description 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.
Time Frame Length of the procedure
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Red Blood Cell Exchange for Patients With Sickle Cell Disease
Hide Arm/Group Description:
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 Number of Participants Analyzed 60
Mean (95% Confidence Interval)
Unit of Measure: ratio
1.03
(1.011 to 1.045)
4.Secondary Outcome
Title Device-related Serious Adverse Events (SAE) in the Full Analysis Set
Hide Description Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).
Time Frame upon signing consent to 24 hours post-procedure
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Red Blood Cell Exchange for Patients With Sickle Cell Disease
Hide Arm/Group Description:
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 Number of Participants Analyzed 72
Measure Type: Number
Unit of Measure: participants
0
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Red Blood Cell Exchange for Patients With Sickle Cell Disease
Hide Arm/Group Description 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.
All-Cause Mortality
Red Blood Cell Exchange for Patients With Sickle Cell Disease
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
Red Blood Cell Exchange for Patients With Sickle Cell Disease
Affected / at Risk (%) # Events
Total   0/72 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Red Blood Cell Exchange for Patients With Sickle Cell Disease
Affected / at Risk (%) # Events
Total   10/72 (13.89%)    
Gastrointestinal disorders   
Nausea  1  4/72 (5.56%)  4
Nervous system disorders   
Dizziness  6/72 (8.33%)  6
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Ray Goodrich, Phd, VP Scientifc and Clinical Affairs
Organization: Terumo BCT, Inc.
Phone: 303-205-2680
Layout table for additonal information
Responsible Party: Terumo BCT
ClinicalTrials.gov Identifier: NCT01736657    
Other Study ID Numbers: CTS-5001
First Submitted: November 27, 2012
First Posted: November 29, 2012
Results First Submitted: August 22, 2013
Results First Posted: July 14, 2014
Last Update Posted: July 14, 2014