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

• 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: Terumo BCT
• Information provided by (Responsible Party): Terumo BCT

Tracking Information

• First Submitted Date: November 27, 2012
• First Posted Date: November 29, 2012
• Results First Submitted Date: August 22, 2013
• Results First Posted Date: July 14, 2014
• Last Update Posted Date: July 14, 2014
• Study Start Date: November 2012
• Actual Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 10, 2014)

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) [ Time Frame: 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.

Original Primary Outcome Measures (submitted: November 28, 2012)

Evaluate the mean FCRa and FCRp [ Time Frame: Six months ]

The primary endpoint will evaluate 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), in the Evaluable subject population.

Current Secondary Outcome Measures (submitted: July 10, 2014)

• Procedural Success of the Spectra Optia System in the Evaluable Population [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: upon signing consent to 24 hours post-procedure ]
  Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).

Original Secondary Outcome Measures (submitted: November 28, 2012)

• Procedural Success of the Spectra Optia System in the Evaluable Population [ Time Frame: Six months ]
• Spectra Optia System's Ability to Achieve the Desired Final Hematocrit in the Evaluable Population [ Time Frame: Six months ]
• Device-related Serious Adverse Events (SAE) in the Full Analysis Set [ Time Frame: Six months ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients
• Official Title: Evaluation of the Spectra Optia Apheresis Red Blood Cell Exchange Protocol in Patients With Sickle Cell Disease.
• 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.
• Study Type: Interventional
• Study Phase: Not Applicable
• 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

One Red Blood Cell Exchange using Spectra Optia Apheresis System per enrolled patient

Other Name: Specta Optia Apheresis System

Study Arms

Experimental: Red cell exchange in sickle cell

Open arm; Red cell blood exchange for patients with sickle cell disease

Intervention: Device: Red blood cell exchange in sickle cell

• Publications *: Quirolo K, Bertolone S, Hassell K, Howard T, King KE, Rhodes DK, Bill J. The evaluation of a new apheresis device for automated red blood cell exchange procedures in patients with sickle cell disease. Transfusion. 2015 Apr;55(4):775-81. doi: 10.1111/trf.12891. Epub 2014 Oct 21.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: July 22, 2013): 73
• Original Estimated Enrollment (submitted: November 28, 2012): 60
• Actual Study Completion Date: June 2013
• Actual Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01736657
• Other Study ID Numbers: CTS-5001
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Terumo BCT
• Study Sponsor: Terumo BCT
• Collaborators: Not Provided

Investigators

• Principal Investigator: Keith Quirolo, MD; Children's Hospital and Research Center at Oakland

• PRS Account: Terumo BCT
• Verification Date: January 2014