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| General |
| Study Status |
Progress Adequate |
| Application Number |
H120005 / PAS001 |
| Date Current Protocol Accepted |
10/31/2017
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| Study Name |
New Enroll. PAS for Liposorber LA-15 Sys
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| General Study Protocol Parameters |
| Study Design |
Prospective Cohort Study
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| Data Source |
New Data Collection
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| Comparison Group |
Device Subjects Serve as Own Control
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| Analysis Type |
Descriptive
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| Study Population |
Child: 2-12 yrs,
Adolescent: 13-18 yrs,
Transit. Adolescent A (distinctively) : 18-21 yrs
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| Detailed Study Protocol Parameters |
| Study Design Description |
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 Description |
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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| Data Collection |
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 Safety Information |
Safety:
Pediatric patients only reported two adverse events not related to the treatment with the device.
Adult patients reported 25 adverse events. All of them were resolved. Only one that was mild was related to the treatment with the device.
Effectiveness:
Pediatric patients
¿ At 1 month of follow-up, there were 2/14 complete remissions, 4/14 partial remissions, 6/14 nephrotic syndrome, and 2/14 data are not available.
¿ At 3 months of follow-up, there were 3/10 complete remissions, 3/10 partial remissions, 4/10 nephrotic syndrome, and 4 patients were withdrawn from the study.
¿ At 6 months of follow-up, there were 3/9 complete remissions, 4/9 partial remissions, 2/9 nephrotic syndrome, and 5 patients were withdrawn from the study.
¿ At 12 months of follow-up, there were 2/9 complete remissions, 3/9 partial remissions, 2/9 nephrotic syndrome, and 5 patients were withdrawn from the study.
¿ At 24 months of follow-up, there were 2/8 complete remissions, 2/8 partial remissions, 1/8 nephrotic syndrome, and 6 patients were withdrawn from the study.
Adult patients
¿ At 1 month of follow-up, there were 0/6 complete remissions, 1/6 partial remission, 4/6 nephrotic syndrome, and 1/6 data are not available.
¿ At 3 months of follow-up, there were 0/4 complete
remissions, 0/4 partial remissions, 2/4 nephrotic syndrome, and 2/4 data are not available.
¿ At 6 months of follow-up, there was 1/1 patients with data not available.
¿ No patient has been followed for 12 months.
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| Actual Number of Patients Enrolled |
10 adults and 25 pediatric patients enrolled
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| Actual Number of Sites Enrolled |
18 participating sites: 10 sites for pediatrics and 8 sites for adults
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| Patient Follow-up Rate |
Pediatric: 1-month (14/14), 3-months (10/14), 6-months (9/14), 12-months (7/13), 24-months (5/12)
Adults: 1-month (6/6), 3-months (4/4). It is early for the follow-up at 6, 12, and 24 months.
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| Study Strengths & Weaknesses |
Weaknesses: Case series study, no comparison group, small sample size, patient enrollment is challenging
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