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

Evaluation of the Spectra Optia Apheresis Red Blood Cell Exchange Protocol in Patients With Sickle Cell Disease.

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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Device: Red blood cell exchange in sickle cell

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
• Enrollment (Actual): 73
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Children's of Alabama
  • California
    • Oakland, California, United States, 94609
      • Children's Hospital and Research Center at Oakland
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado at Denver
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Kosair Children's Hospital
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins Medical

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Red cell exchange in sickle cell; Open arm; Red cell blood exchange for patients with sickle cell disease	Device: Red blood cell exchange in sickle cell; One Red Blood Cell Exchange using Spectra Optia Apheresis System per enrolled patient; Other Names: Specta Optia Apheresis System

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Length of the procedure

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	Length of the procedure
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.	Length of the procedure
Device-related Serious Adverse Events (SAE) in the Full Analysis Set	Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).	upon signing consent to 24 hours post-procedure

Collaborators and Investigators

• Sponsor: Terumo BCT
• Investigators: Principal Investigator: Keith Quirolo, MD, Children's Hospital and Research Center at Oakland

Publications and helpful links

General 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.

Study record dates

Study Major Dates

• Study Start: November 1, 2012
• Primary Completion (Actual): June 1, 2013
• Study Completion (Actual): June 1, 2013

Study Registration Dates

• First Submitted: November 27, 2012
• First Submitted That Met QC Criteria: November 28, 2012
• First Posted (Estimate): November 29, 2012

Study Record Updates

• Last Update Posted (Estimate): July 14, 2014
• Last Update Submitted That Met QC Criteria: July 10, 2014
• Last Verified: January 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: CTS-5001