Sickle Cell Anemia
Conditions
Keywords
Sickle Cell Disease, Hemoglobin (Hb) S Disease, Hb S Disease, Congenital Hemolytic Anemia
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.
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
Interventions
DEVICEAmicus Red Cell Exchange in SCD patients
Each patient will be treated with one RBCx procedure using the AMICUS RBCx System
Sponsors
Versiti Blood Health
Children's Hospital of Philadelphia
Barbara Ann Karmanos Cancer Institute
University of Texas
Washington University School of Medicine
Phoenix Children's Hospital
Fenwal, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Actual Fraction of Original Red Blood Cells Remaining to the Target Fraction of Cells Remaining (FCR). | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. | 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. |
| 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. | WBC loss post-procedure as measured with a complete blood count |
| Serious Adverse Events | 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. |
| 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. | Platelet loss post-procedure as measured with a complete blood count |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Amicus Red Cell Exchange in SCD Patients Open arm. Patients with sickle cell disease (SCD) treated using one Amicus Red Cell Exchange procedure.
Amicus Red Cell Exchange in SCD patients: Each patient will be treated with one RBCx procedure using the AMICUS RBCx System | 83 |
| Total | 83 |
Baseline characteristics
| Characteristic | Amicus Red Cell Exchange in SCD Patients | — |
|---|---|---|
| Age at Procedure | 26.8 years STANDARD_DEVIATION 10.26 | — |
| Age, Categorical <=18 years | 20 Participants | — |
| Age, Categorical >=65 years | 0 Participants | — |
| Age, Categorical Between 18 and 65 years | 63 Participants | — |
| Age, Continuous | 27.0 years STANDARD_DEVIATION 10.59 | — |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | — |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 81 Participants | — |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | — |
| Race (NIH/OMB) Asian | 0 Participants | — |
| Race (NIH/OMB) Black or African American | 80 Participants | — |
| Race (NIH/OMB) More than one race | 0 Participants | — |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | — |
| Race (NIH/OMB) Unknown or Not Reported | 3 Participants | — |
| Race (NIH/OMB) White | 0 Participants | — |
| Region of Enrollment United States | 83 Participants | — |
| Sex: Female, Male Female | 41 Participants | — |
| Sex: Female, Male Male | 42 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 83 |
| other Total, other adverse events | 12 / 83 |
| serious Total, serious adverse events | 2 / 83 |
Outcome results
Primary
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.
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Amicus Red Cell Exchange in SCD Patients | Actual Fraction of Original Red Blood Cells Remaining to the Target Fraction of Cells Remaining (FCR). | 0.978 Ratio | Standard Deviation 0.1933 |
Secondary
Accuracy of Patient's End Hematocrit as Measured by the Patient's Post-procedure Hematocrit.
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.
Time frame: Actual End Hematocrit will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Amicus Red Cell Exchange in SCD Patients | Accuracy of Patient's End Hematocrit as Measured by the Patient's Post-procedure Hematocrit. | 1.19 Ratio | Standard Deviation 0.817 |
Secondary
Serious Adverse Events
Evaluate the number of device related serious adverse events during the procedure and approximately 18-24 hours post-procedure.
Time frame: During the procedure up to 24 hours post-procedure.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Amicus Red Cell Exchange in SCD Patients | Serious Adverse Events | 0 Device Related Serious Adverse Events |
Secondary
Subject Cell Loss Post-Procedure (Platelets)
Platelet loss post-procedure as measured with a complete blood count
Time frame: Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Amicus Red Cell Exchange in SCD Patients | Subject Cell Loss Post-Procedure (Platelets) | 48.967 Percentage of cells | Standard Deviation 12.2895 |
Secondary
Subject Cell Loss Post-Procedure (WBC)
WBC loss post-procedure as measured with a complete blood count
Time frame: Patient's blood cells will be measured by taking a sample from the patient approximately 10-15 min after procedure completion.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Amicus Red Cell Exchange in SCD Patients | Subject Cell Loss Post-Procedure (WBC) | 27.414 Percentage of cells | Standard Deviation 21.6834 |