Evaluation of the AMICUS RBCx System in Sickle Cell Patients

July 7, 2022 updated by: Fenwal, Inc.

Evaluation of the AMICUS Red Blood Cell Exchange (RBCx) System in Sickle Cell Patients

The purpose of this study is to evaluate the performance of the AMICUS Red Blood Cell Exchange (RBCx) System (Exchange and Depletion/Exchange procedures) in patients with sickle cell disease.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The goal of Red Blood Cell Exchange is to remove a patient's red blood cells (RBCs) and replace the blood volume removed with either healthy donor RBCs and/or colloid/crystalloid solutions. Depending on the RF used, the procedure can be considered an RBC Exchange or RBC Depletion/Exchange procedure. The RBC Depletion/Exchange procedure is a modification of the RBC Exchange procedure. The AMICUS RBCx protocol provides the ability to use various RFs including healthy donor RBCs or a combination of fluids, such as saline followed by RBCs. In RBC Exchange and the exchange portion of the RBC Depletion/Exchange procedures, RBC units are used as the RF, while colloid and/or crystalloid solutions are used as the RF in the depletion portion of the RBC Depletion/Exchange procedures. A new operating protocol on the AMICUS Separator that enables the device to perform Red Blood Cell Exchange (RBCx) procedures in accordance with the replacement fluid(s) (RF) prescribed by a physician for patients with sickle cell disease

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
83

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Barbara Ann Karmanos Cancer Institute
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Texas
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern Medical Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • BloodCenter of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Subjects with documented diagnosis of a type of sickle cell disorder who require RBC Exchange or RBC Depletion/Exchange treatment.
  • Medically stable subjects who have been previously treated for sickle cell disease with RBC Exchange or RBC Depletion/Exchange.
  • Subjects, or subject's legal representative, who have provided signed informed consent, and assent when applicable, prior to participation.
  • Adequate availability of sickle trait negative, leukoreduced, Blood type (ABO) blood group, Rhesus factor D (Rh (D)) compatible, unexpired replacement RBC products.
  • Subjects with sufficient vascular access to accommodate the RBCx procedure as determined by the medical staff responsible for obtaining intravenous access.
  • Subjects who are able and agree to report adverse events (AEs) during the required reporting period.

Exclusion Criteria:

  • Procedures that occur during acute hospitalization.
  • Procedures prescribed within one week of discharge of a hospitalization.
  • Subjects with altered mental status that would prohibit the giving and understanding of informed consent, and assent when applicable, who do not have a legally authorized representative.
  • Drug abuse, alcohol abuse, or other factors that in the opinion of the investigator could affect the ability of the subject to comply with the requirements of the protocol.
  • Subjects who have experienced a serious adverse event associated with an RBCx procedure in the past.
  • In the opinion of the investigator, subjects who have a life expectancy fewer than 30 days.
  • Subjects who refuse blood products.
  • Subjects who are pregnant.
  • Subjects who fail to comply with site requirements for cessation of medication that interfere or increase procedure risk.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Amicus Red Cell Exchange in SCD patients
Open arm. Patients with sickle cell disease (SCD) treated using one Amicus Red Cell Exchange procedure.
Device: Amicus Red Cell Exchange in SCD patients
Each patient will be treated with one RBCx procedure using the AMICUS RBCx System
Other Names:
  • AMICUS RBCx System

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Actual Fraction of Original Red Blood Cells Remaining to the Target Fraction of Cells Remaining (FCR).
Time Frame: Actual FCR will be assessed by taking a pre-procedure sample from the patient on the day of the procedure and a post-procedure sample approximately 10-15 min after procedure completion.
The primary endpoint will evaluate the mean ratio of the Actual FCR (FCRa) to Target FCR (FCRt) as measured by the patient's Hb S pre-procedure and post-procedure. The predefined mean ratio of the FCRa to FCRt is 0.75 to 1.25.
Actual FCR will be assessed by taking a pre-procedure sample from the patient on the day of the procedure and a post-procedure sample approximately 10-15 min after procedure completion.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Accuracy of Patient's End Hematocrit as Measured by the Patient's Post-procedure Hematocrit.
Time Frame: Actual End Hematocrit will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
Evaluate the accuracy of subject hematocrit post-procedure (End Hematocrit) (as measured by the patient's hematocrit at the end of the procedure) compared to the target End Hematocrit.
Actual End Hematocrit will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
Subject Cell Loss Post-Procedure (WBC)
Time Frame: Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
WBC loss post-procedure as measured with a complete blood count
Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
Serious Adverse Events
Time Frame: During the procedure up to 24 hours post-procedure.
Evaluate the number of device related serious adverse events during the procedure and approximately 18-24 hours post-procedure.
During the procedure up to 24 hours post-procedure.
Subject Cell Loss Post-Procedure (Platelets)
Time Frame: Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
Platelet loss post-procedure as measured with a complete blood count
Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Fenwal, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Children's Hospital of Philadelphia
Washington University School of Medicine
University of Texas
Barbara Ann Karmanos Cancer Institute
Phoenix Children's Hospital
Versiti

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Chair: Paul Swerdlow, MD, Barbara Ann Karmanos Cancer Institute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 7, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 19, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 6, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 20, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 26, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 29, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 7, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AMIC-003-CMD

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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