Focal Segmental Glomerulosclerosis
Conditions
Keywords
pediatric, renal transplantation, recurrence, drug-resistant
Brief summary
Liposorber® LA-15 System is a blood purification therapy that selectively removes malignant lipoproteins including low density lipoprotein from circulating blood flow and rapidly reduces the plasma cholesterol level. The system was originally developed for the treatment of patients with serious dyslipidemia such as familial hypercholesterolemia and then applied to improve the dyslipidemia, a common complication of nephrotic syndrome and found to bring about improvement not only with the dyslipidemic condition but the nephrotic condition (e.g, proteinuria and hypoproteinemia). Although the definitive mechanism by which the system may relieve nephrotic syndrome is unknown, it has been recognized as one of alternative therapies for refractory nephrotic syndrome including focal segmental glomerulosclerosis (FSGS) in Japan and referred in the Guidelines for the Treatment of Nephrotic Syndrome endorsed by The Japanese Society of Nephrology. This study is conducted as a post approval study imposed by Humanitarian Device Exemption (HDE) order to confirm the safety and efficacy of the Liposorber® LA-15 System in the treatment of drug-resistant pediatric primary FSGS.
Interventions
DEVICELIPOSORBER® LA-15 System
LIPOSORBER® LA-15 System is an extracorporeal blood purification system. Approximately 3 to 4 L of plasma is treated in a single treatment session and it takes 2 to 3 hours. Recommended frequency of the treatment is twice weekly for 3 weeks followed by once weekly for 6 weeks, thus it takes 9 weeks for a total of 12 treatment sessions.
Sponsors
Kaneka Medical America LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Patients with focal segmental glomerulosclerosis treated by LIPOSORBER® LA-15 system
Eligibility
Sex/Gender
ALL
Age
No minimum to 21 Years
Healthy volunteers
No
Inclusion criteria
* A pediatric patient is deemed suitable for inclusion in the study if the patient has FSGS with a GFR ≥ 45 ml/min/1.73 m 2 and any of the following: * Refractory nephrotic syndrome in which standard treatment options are unsuccessful (i.e., patient is unresponsive to standard corticosteroid and/or calcineurin inhibitor therapy for at least 8 weeks resulting in failure to achieve complete or partial remission); * Refractory nephrotic syndrome in which standard treatment options are not well tolerated (i.e., patients intolerant to standard therapies due to severe side effects that negatively affect quality of life without providing an acceptable level of clinical benefit); * Refractory or recurrent nephrotic syndrome in which standard therapy is contraindicated. or \- Pediatric post renal transplant patients with nephrotic syndrome associated with primary FSGS.
Exclusion criteria
* General
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The percent of patients who show complete or partial remission | 1 month after the final treatment |
| the rate of device-related and procedure-related serious adverse events | During the period in which the apheresis procedures are administered and up to at the 1-month follow-up visit |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Nephrotic Condition | 1, 3, 6, 12, and 24 months after the final treatment | Nephrotic condition defined as follows: urine protein:creatinine ratio \> 2.0 (g/g) with a first morning void urine sample |
| The percent of patients who obtain complete or partial remission | 3, 6, 12, and 24 months after the final treatment | — |
| Incidence of adverse events | From the initiation of the first apheresis session until the termination of the last (usually 12th) apheresis session, standad period of 9 weeks for a total of 12 aoheresis sessions | The protocol indicates the standard treatment schedule as follows: 2 sessions weekly for the first 3 weeks followed by 1 session weekly for 6 weeks |
| Incidence of adverse events and severe adverse events | From 1 months to 24 months after the final aphresis | — |
| Various laboratory values | 1,3, 6, 12, and 24 months after the final apheresis | Various laboratory values include Urine protein, Urine creatinine, Serum creatinine, Estimated glomerular filtration rate (eGFR), serum total protein, Serum albumin, Serum vitamin E, Hematocrit, Red blood cell, White blood cell, Platelet, Total cholesterol, LDL cholesterol, HDL cholesterol, Triglycerides, Serum soluble urokinase plasminogen activator receptor. |
Countries
United States
Contacts
CONTACTAyaka Kitamura
PRINCIPAL_INVESTIGATORJeffrey I Silberzweig, MD
Weill Medical College of Cornell University
Outcome results
None listed