LDL-Apheresis for FSGS CardioRenal Outcomes

NCT04088799 | Completed

• 1 other identifier: CIN001 - LDL Outcomes
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

Focal segmental glomerulosclerosis (FSGS) is the most common cause of end-stage renal disease (ESRD) in adolescents. The refractory nature of FSGS and a more than 30% recurrence rate after kidney transplantation renders treatment of FSGS one of the most difficult challenges in pediatric nephrology. A significant knowledge gap in understanding the mechanism of FSGS treatment resistance and progression hampers development of successful treatment strategies. Beneficial effect of removal of low-density lipoproteins by LDL-apheresis indicates that lipids contribute to progression in FSGS. The investigators will test the hypothesis that removal of Lp-PLA2 and lipid metabolites by LDL-apheresis ameliorates proteinuria and cardiovascular comorbidities. Patients with FSGS and FSGS recurrence after kidney transplantation receiving LDL-apheresis as part of standard of care will be enrolled to the study. Pre-and post serum and effluent concentrations of LPC, free FA, Lp-PLA2, oxidized LDL, fasting lipid profile, interleukin (IL)-6, tumor necrosis factor (TNF)-α, and IL-1β will be monitored in patients undergoing LDL-apheresis. Investigators will also study the impact of LDL-apheresis on cardiovascular and clinical comorbidities by monitoring degree of proteinuria, blood pressures and arterial stiffness index.

Conditions

Focal Segmental Glomerulosclerosis

Outcome Measures

Primary Outcomes (1)

• Severity of Proteinuria

  Standard of care proteinuria will be monitored for expected improvement in severity

  9 weeks

Secondary Outcomes (1)

• Cardiovascular comorbidities

  9 weeks

Study Arms (1)

FSGS requiring LDL-apheresis

Pediatric patients with FSGS requiring LDL-apheresis with the Liposorber

Other: No intervention

Interventions

No intervention OTHER

No intervention

FSGS requiring LDL-apheresis

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Pediatric patients with focal segmental glomerulosclerosis (FSGS) in need of LDL-apheresis per standard of care

You may qualify if:

• FSGS and a glomerular filtration rate (GFR) ≥ 60 ml/min/1.73m2 AND
• 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), OR
• Refractory nephrotic syndrome in which standard treatment options are not well tolerated (i.e., patients intolerant to standard therapies due to severe side effects without providing an acceptable level of clinical benefit), OR
• Refractory or recurrent nephrotic syndrome in which standard therapy is contraindicated
• Post renal transplant with nephrotic syndrome associated with primary FSGS

You may not qualify if:

• Greater than 21 years of age
• Parent or patient unwilling or unable to signed and date the informed consent
• Pregnant, lactating, or planning to become pregnant prior to completing the study
• Unable or unwilling to comply with the follow-up schedule
• Simultaneously participating in another investigational drug or device study (except for LDL-apheresis associated trials)
• Body weight less than 21 kilograms (46 pounds)
• Currently being administered angiotensin converting enzyme (ACE) inhibitors that cannot be withheld for at least 24 hours prior to each apheresis treatment
• Currently being administered antihypertensive drugs other than ACE inhibitors than cannot be withheld on the day of LDL-apheresis until after the procedure
• Medical condition or disorder that would limit life expectancy to less than the primary clinical study endpoint or that may cause noncompliance with the study plan or confound the data analysis
• Hypersensitivity to dextran sulfate, heparin, or ethylene oxide
• Inability to achieve adequate anticoagulation
• Inability to tolerate extracorporeal circulation therapy with Liposorber® LA-15
• Cardiac impairments such as uncontrolled arrhythmia, unstable angina, decompensated congestive heart failure, or valvular disease
• Thyroid disease or liver abnormalities
• Unresolved systemic or local infection that could affect the clinical study outcomes

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Kaneka Medical America LLC: collaborator

Study Sites (2)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum samples from before and after LDL apheresis treatments

MeSH Terms

Conditions

Glomerulosclerosis, Focal Segmental

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 12, 2019
• First Posted: September 13, 2019
• Study Start: January 1, 2019
• Primary Completion: June 1, 2023
• Study Completion: June 1, 2023
• Last Updated: December 21, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)