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| General |
| Study Status |
Delayed |
Application Number /
Requirement Number |
H120005 / PAS001 |
| Date Original Protocol Accepted |
10/10/2013
|
| Date Current Protocol Accepted |
12/05/2022
|
| Study Name |
New Enroll. PAS for Liposorber LA-15 Sys
|
| Device Name |
LIPOSORBER LA-15 SYSTEM
|
| General Study Protocol Parameters |
| Study Design |
Prospective Cohort Study
|
| Data Source |
New Data Collection
|
| Comparison Group |
No Control
|
| Analysis Type |
Descriptive
|
| Study Population |
Infant: 29 days-2 yrs,
Child: 2-12 yrs,
Adolescent: 13-18 yrs,
Transit. Adolescent A (distinctively) : 18-21 yrs,
Transit. Adolescent B (as adults) : 18-21 yrs,
Adult: >21
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| Detailed Study Protocol Parameters |
| Study Objectives |
This is a prospective, multicenter, single arm study with a total of 35 newly enrolled patients, treated at 3 to 10 clinical centers in the United States. The primary objectives of this study are to evaluate the safety and probable benefit of the Liposorber LA-15 System in relieving nephrotic syndrome associated with refractory pediatric primary FSGS at 1 month after the final apheresis treatment.
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| Study Population |
Pediatric patients with nephrotic syndrome associated with primary focal segmental glomerulosclerosis, when the standard treatment options, including corticosteroid and/or calcineurin inhibitors treatments, have been unsuccessful or not well tolerated, and the patient has a GFR greater than or equal to 60 ml/min/1.73m2, or the patient is post renal transplantation.
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| Sample Size |
A total of 35 patients will be included.
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| Key Study Endpoints |
The primary probable benefit endpoint is the percent of patients who show complete or partial remission at 1 month after the final apheresis treatment. The primary probable benefit endpoint will be assessed by calculating the percent reduction in Up/c values at screening and at 1 month after the final apheresis procedure by using urine protein and urine creatinine values as reported in laboratory results.
The primary safety endpoint is the rate of device-related and procedure-related serious adverse events (SAEs) occurring during the period in which the apheresis procedures are administered and up to at the 1-month follow-up visit. The primary safety endpoint will be assessed by calculating the incidence of device-related and procedure- related SAEs reported by the clinical sites and occurring during the apheresis treatment period. The clinical site will be asked to provide a determination of the relatedness of the SAE to the study device and procedure for each SAE reported.
The secondary endpoints will be to measure the following:
- Nephrotic condition (complete remission, partial remission, and nephrotic state) at 1, 3, 6, 12, and 24 months after the final apheresis treatment, including the percentage of patients who obtain complete or partial remission at 3, 6, 12, and 24 months
- Incidence of adverse events encountered during the period in which apheresis treatments are given
- Incidence of all AEs and SAEs occurring within 3, 6, 12, and 24 months after the final apheresis treatment
- Laboratory values, including eGFR, at baseline, after the last treatment, and at 1, 3, 6, 12, and 24 months after the final apheresis treatment, including percent change from baseline and percentage of patients showing an increase or decrease in each value
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| Follow-up Visits and Length of Follow-up |
Patients will be followed for 24 months after they complete the apheresis procedures.
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| Interim or Final Data Summary |
| Interim Results |
Safety:
Among pediatric patients, there were 8 adverse events including 4 serious adverse events reported. Among adult patients, 17
adverse events including 1 serious adverse event were reported. All events were deemed not related to the treatment.
Effectiveness:
The proportion of pediatric patients in complete or partial remission ranged from 53.3% (8/15) at 1 month follow-up to 80% (4/5) at 24
months follow-up. The proportion of adult patients in complete or partial remission ranged from 27% (3/11) at 1 month follow-up to
0% (0/2) at 24 months. However, the small number of pediatric and adult patients, particularly at the later follow-up visits, make it
difficult to get a stable estimate of device effectiveness at this time.
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| Actual Number of Patients Enrolled |
27 pediatric and 19 adult enrollments
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| Actual Number of Sites Enrolled |
16 sites
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| Patient Follow-up Rate |
Pediatric: 88% (15/17) at 1 month, 71% (12/17) at 3 months, 59% (10/17) at 6 months, 59% (10/17) at 12 months, and 29% (5/17) at
24 months.
Adults: 92% (11/12) at 1 month, 67% (8/12) at 3 months, 58% (7/12) at 6 months, 33% (4/12) at 12 months, and 17% (2/12) at 24
months.
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