Sickle Cell Disease
Conditions
Brief summary
The purpose of this study is to evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease.
Detailed description
Evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease. Open label design.
Interventions
DEVICERed blood cell exchange in sickle cell
One Red Blood Cell Exchange using Spectra Optia Apheresis System per enrolled patient
Sponsors
Terumo BCT
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* At least 12 years old * Enrolled in a program of regular red blood cell exchange (RBCx) to prevent symptoms/complications of sickle cell disease (SCD) or Initiating a program of regular RBCx or Receiving RBCx as a pre-surgical procedure. * Medically stable * Previous documentation of diagnosis by hemoglobin electrophoresis of a type of sickle cell disorder requiring RBCx. * Sufficient vascular access to accommodate the RBCx procedure as determined by the apheresis technician performing the procedure or phlebotomist responsible for obtaining intravenous access. * Availability of sickle trait negative, leukoreduced, ABO blood group, Rhesus factor D (Rh (D)) compatible, unexpired replacement blood. See Glossary for definition of replacement blood. * Able to commit to the study follow-up schedule. * Agree to report adverse events (AEs) during the required reporting period.
Exclusion criteria
* Inability to obtain informed consent/assent from patient, or permission from parent or guardian. * Pregnancy (negative serum pregnancy test required for females of childbearing potential). * Life expectancy is fewer than 30 days from time of procedure. * Incarcerated or a ward of the court. * Refusal of blood products. * Failure to comply with site standard requirements for cessation of medications (e.g., angiotensin converting enzyme (ACE) inhibitors) that interfere with or increase risk of RBCx procedures. * History of drug or alcohol abuse that, in the opinion of the investigator, could affect the ability of the patient to comply with the study requirements Inability to comply with the protocol in the opinion of the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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) | Length of the procedure | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Procedural Success of the Spectra Optia System in the Evaluable Population | Length of the procedure | 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). |
| Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population | Length of the procedure | 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. |
| Device-related Serious Adverse Events (SAE) in the Full Analysis Set | upon signing consent to 24 hours post-procedure | Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients). |
Countries
United States
Participant flow
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).
Participants by arm
| Arm | Count |
|---|---|
| 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. | 72 |
| Total | 72 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | insufficient vascular access | 1 |
Baseline characteristics
| Characteristic | Red Blood Cell Exchange for Patients With Sickle Cell Disease |
|---|---|
| Age, Categorical <=18 years | 26 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 46 Participants |
| Age, Continuous | 23.0 years STANDARD_DEVIATION 10.32 |
| Region of Enrollment United States | 72 participants |
| Sex: Female, Male Female | 29 Participants |
| Sex: Female, Male Male | 43 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 10 / 72 |
| serious Total, serious adverse events | 0 / 72 |
Outcome results
Primary
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)
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
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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Red Blood Cell Exchange for Patients With Sickle Cell Disease | 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 |
Secondary
Device-related Serious Adverse Events (SAE) in the Full Analysis Set
Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).
Time frame: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Red Blood Cell Exchange for Patients With Sickle Cell Disease | Device-related Serious Adverse Events (SAE) in the Full Analysis Set | 0 participants |
Secondary
Procedural Success of the Spectra Optia System in the Evaluable Population
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
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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Red Blood Cell Exchange for Patients With Sickle Cell Disease | Procedural Success of the Spectra Optia System in the Evaluable Population | 100 percentage of participants |
Secondary
Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population
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
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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Red Blood Cell Exchange for Patients With Sickle Cell Disease | Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population | 1.03 ratio |