Trial Outcomes & Findings for Evaluation of the AMICUS RBCx System in Sickle Cell Patients (NCT NCT02372877)
NCT ID: NCT02372877
Last Updated: 2022-07-29
Results Overview
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.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
83 participants
Primary outcome timeframe
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.
Results posted on
2022-07-29
Participant Flow
Participant milestones
| Measure |
Amicus Red Cell Exchange or Depletion/Exchange in SCD Patients
Single-arm, open label study in patients with sickle cell disease (SCD) . SCD patients meeting the eligibility requirements will be treated with one RBCx procedure (Exchange or Depletion/Exchange) using the AMICUS RBCx System.
The RBC Exchange or RBC Depletion/Exchange will be completed based on the physician's prescription.
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|---|---|
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Overall Study
STARTED
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83
|
|
Overall Study
COMPLETED
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59
|
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Overall Study
NOT COMPLETED
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24
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=83 Participants
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
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|---|---|
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Age, Categorical
<=18 years
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20 Participants
n=83 Participants
|
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Age, Categorical
Between 18 and 65 years
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63 Participants
n=83 Participants
|
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Age, Categorical
>=65 years
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0 Participants
n=83 Participants
|
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Age, Continuous
|
27.0 years
STANDARD_DEVIATION 10.59 • n=83 Participants
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Sex: Female, Male
Female
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41 Participants
n=83 Participants
|
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Sex: Female, Male
Male
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42 Participants
n=83 Participants
|
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Ethnicity (NIH/OMB)
Hispanic or Latino
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2 Participants
n=83 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
81 Participants
n=83 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=83 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=83 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=83 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=83 Participants
|
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Race (NIH/OMB)
Black or African American
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80 Participants
n=83 Participants
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Race (NIH/OMB)
White
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0 Participants
n=83 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=83 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=83 Participants
|
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Region of Enrollment
United States
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83 Participants
n=83 Participants
|
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Age at Procedure
|
26.8 years
STANDARD_DEVIATION 10.26 • n=83 Participants
|
PRIMARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=59 Participants
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
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|---|---|
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Actual Fraction of Original Red Blood Cells Remaining to the Target Fraction of Cells Remaining (FCR).
|
0.978 Ratio
Standard Deviation .1933
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SECONDARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=59 Participants
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
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|---|---|
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Accuracy of Patient's End Hematocrit as Measured by the Patient's Post-procedure Hematocrit.
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1.19 Ratio
Standard Deviation 0.817
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SECONDARY outcome
Timeframe: 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
Outcome measures
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=59 Participants
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
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|---|---|
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Subject Cell Loss Post-Procedure (WBC)
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27.414 Percentage of cells
Standard Deviation 21.6834
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SECONDARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=83 Participants
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
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|---|---|
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Serious Adverse Events
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0 Device Related Serious Adverse Events
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SECONDARY outcome
Timeframe: 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
Outcome measures
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=59 Participants
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
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|---|---|
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Subject Cell Loss Post-Procedure (Platelets)
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48.967 Percentage of cells
Standard Deviation 12.2895
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Adverse Events
Amicus Red Cell Exchange in SCD Patients
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=83 participants at risk
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
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|---|---|
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General disorders
Post Procedure - chest pain
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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General disorders
Pain
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Other adverse events
| Measure |
Amicus Red Cell Exchange in SCD Patients
n=83 participants at risk
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
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|---|---|
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Nervous system disorders
During Procedure - Dizziness
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3.6%
3/83 • Number of events 3 • Adverse events were collected during the procedure and 24 hours post-procedure.
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General disorders
During Procedure - Device occlusion
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2.4%
2/83 • Number of events 2 • Adverse events were collected during the procedure and 24 hours post-procedure.
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General disorders
During Procedure - Chest Pain
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Eye disorders
During Procedure - Vision blurred
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Gastrointestinal disorders
During Procedure - Hypoaesthesia oral
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Gastrointestinal disorders
During Procedure - Nausea
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Gastrointestinal disorders
During Procedure - Paraesthesia oral
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Immune system disorders
During Procedure - Hypersensitivity
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Investigations
During Procedure - Blood pressure decreased
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Musculoskeletal and connective tissue disorders
During Procedure - Back Pain
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
|
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Surgical and medical procedures
During Procedure - Catheterisation venous
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
|
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Nervous system disorders
Post-Procedure - dizziness
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6.0%
5/83 • Number of events 5 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Nervous system disorders
Post-Procedure - Headache
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Nervous system disorders
Post-Procedure - Restless Legs Syndrome
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Gastrointestinal disorders
Post-Procedure - Nausea
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2.4%
2/83 • Number of events 2 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Gastrointestinal disorders
Post-Procedure - Abdominal pain upper
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Gastrointestinal disorders
Post-Procedure - Vomiting
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Musculoskeletal and connective tissue disorders
Post-Procedure - Pain in extremity
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2.4%
2/83 • Number of events 2 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Musculoskeletal and connective tissue disorders
Post-Procedure - back pain
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
|
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General disorders
Post-Procedure - fatigue
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Injury, poisoning and procedural complications
Post-Procedure - Infusion site haemorrhage
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Skin and subcutaneous tissue disorders
Post-Procedure - Cold Sweat
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1.2%
1/83 • Number of events 1 • Adverse events were collected during the procedure and 24 hours post-procedure.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place