Evaluation of the AMICUS RBCx System in Sickle Cell Patients

NCT02372877 | Completed Results DMC

• 1 other identifier: AMIC-003-CMD
• Study Type: interventional
• Target: 83
• Locations: 1 country
• Sites: 6

Brief Summary

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.

Conditions

Sickle Cell Anemia

Keywords

Sickle Cell Disease; Hemoglobin (Hb) S Disease; Hb S Disease; Congenital Hemolytic Anemia

Outcome Measures

Primary Outcomes (1)

• Actual Fraction of Original Red Blood Cells Remaining to the Target Fraction of Cells Remaining (FCR).

  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 Outcomes (4)

• Accuracy of Patient's End Hematocrit as Measured by the Patient's Post-procedure 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)

  Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.

• Serious Adverse Events

  During the procedure up to 24 hours post-procedure.

• Subject Cell Loss Post-Procedure (Platelets)

  Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.

Study Arms (1)

Amicus Red Cell Exchange in SCD patients

EXPERIMENTAL

Open arm. Patients with sickle cell disease (SCD) treated using one Amicus Red Cell Exchange procedure.

Device: Amicus Red Cell Exchange in SCD patients

Interventions

Amicus Red Cell Exchange in SCD patients DEVICE

Each patient will be treated with one RBCx procedure using the AMICUS RBCx System

Also known as: AMICUS RBCx System

Amicus Red Cell Exchange in SCD patients

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Fenwal, Inc.: lead
• Versiti Blood Health: collaborator
• Children's Hospital of Philadelphia: collaborator
• Barbara Ann Karmanos Cancer Institute: collaborator
• University of Texas: collaborator
• Washington University School of Medicine: collaborator
• Phoenix Children's Hospital: collaborator

Study Sites (6)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

BloodCenter of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Anemia, Hemolytic, Congenital

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

No limitations or caveats apply to this study.

Results Point of Contact

• Title: Jason Friedmann
• Organization: Fresenius Kabi

jason.friedmann@fresenius-kabi.com

847-550-2365

Study Officials

• Paul Swerdlow, MD

  Barbara Ann Karmanos Cancer Institute

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2015
• First Posted: February 26, 2015
• Study Start: May 1, 2015
• Primary Completion: December 7, 2017
• Study Completion: January 19, 2018
• Last Updated: July 29, 2022
• Results First Posted: July 29, 2022

Record last verified: 2022-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)