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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Terumo BCT
ClinicalTrials.gov Identifier:
NCT01736657
First received: November 27, 2012
Last updated: July 10, 2014
Last verified: January 2014
  Purpose

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.


Condition Intervention
Sickle Cell Disease
Device: Red blood cell exchange in sickle cell

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluation of the Spectra Optia Apheresis Red Blood Cell Exchange Protocol in Patients With Sickle Cell Disease.

Resource links provided by NLM:


Further study details as provided by Terumo BCT:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Procedural Success of the Spectra Optia System in the Evaluable Population [ Time Frame: Length of the procedure ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Device-related serious adverse events (SAE) in the Full Analysis Set (72 patients).


Enrollment: 73
Study Start Date: November 2012
Study Completion Date: June 2013
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: Specta Optia Apheresis System

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.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01736657

Locations
United States, Alabama
Children's of Alabama
Birmingham, Alabama, United States, 35233
United States, California
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
United States, Colorado
University of Colorado at Denver
Aurora, Colorado, United States, 80045
United States, Kentucky
Kosair Children's Hospital
Louisville, Kentucky, United States, 40202
United States, Maryland
Johns Hopkins Medical
Baltimore, Maryland, United States, 21205
Sponsors and Collaborators
Terumo BCT
Investigators
Principal Investigator: Keith Quirolo, MD Children's Hospital and Research Center at Oakland
  More Information

No publications provided

Responsible Party: Terumo BCT
ClinicalTrials.gov Identifier: NCT01736657     History of Changes
Other Study ID Numbers: CTS-5001
Study First Received: November 27, 2012
Results First Received: August 22, 2013
Last Updated: July 10, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 24, 2014