A Study of Acthar Gel Alone or With Tacrolimus to Reduce Proteinuria in Fibrillary Glomerulopathy Patients

NephroNet, Inc.34 enrolled

Overview

A Multicenter, Comparative Safety and Efficacy Study of Acthar gel alone or in combination with oral Tacrolimus to reduce urinary proteinuria in patients with idiopathic DNAJB9 Positive Fibrillary glomerulopathy. This study will be a multi-center, prospective, randomized, open-labeled intervention trial of 34 patients randomized to 52 weeks of ACTHar gel alone or Acthar gel plus oral Tacrolimus.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Fibrillary Glomerulonephritis

Interventions

Acthar Gel 80 UNT/ML Injectable SolutionDRUG

Follow up and observation for 12 months off Acthar gel or Acthar gel and Tacrolimus

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male/Female age \> 18 * Biopsy proven Fibrillary glomerulonephritis within 3 years of study randomization * Stable Maximum Renin-Angiotensin-Aldosterone System inhibition times 4 weeks prior to randomization Note: Maximum RAAS inhibition will be left to the discretion of the site Principal Investigator * Estimated Glomerular Filtration Rate \> 25 mls/min calculate by the Chronic Kidney Disease-EPI formula * Protein/creatinine ratio \> 2000 mg/gm 5) Note: If protein/creatinine less than 2000 mg/gm, a formal 24- hour urine collection for total protein can be performed. The total 24-hour protein will need to \>/= 2000mg. * Blood pressure targeted to \< 140/90 at the time of randomization * Patients with Monoclonal Gammopathy without history of myeloma will be eligible. * Patients with monoclonal staining for fibrillary fibers will be excluded * Patients with Type II non-insulin dependent diabetes will be eligible provided the renal biopsy does not show nodular Kimmelstiel Wilson lesions Exclusion Criteria: * Patients with MGUS and history of myeloma will not be eligible * Patients with active viral production of either hepatitis B or C as evidence by historical polymerase chain reaction test positive for active viral shedding * HIV seropositivity * Renal biopsy data with \> 50% Interstitial Fibrosis * Patient with active or a known history lymphoma * Patients with insulin dependent diabetes mellitus will be excluded Note: patients with Type II diabetes mellitus that are well controlled without the need for insulin will be eligible for the study. * Patients with Type II non-insulin dependent diabetes will be eligible provided the renal biopsy does not show nodular Kimmelstiel Wilson lesions. * Patients receiving steroids, mycophenolate mofetil, cyclophosphamide, Azathioprine or other immunosuppressive agent with 4 weeks of study randomization Note: Washout of these medications will be allowed at the screening visit * Patients having received Rituximab or B cell modifying biologic therapy within 6 months of randomization

Locations (5)

Stanford University

Stanford, California, United States

University of Colorado Anschutz Medical Department of Medicine Division of Renal Diseases and Hypertension

Aurora, Colorado, United States

Georgia Nephrology DBA Georgia Nephrology Research Institute

Lawrenceville, Georgia, United States

Columbia University Research Dept of Nephrology

New York, New York, United States

Outcomes

Primary Outcomes

The change in UP/CR ratio with treatment of Acthar gel 80 units subcutaneous 2 times a week alone

The change in protein/creatinine ratio in patients with biopsy proven Fibrillary after 12 months of treatment with treated with Acthar gel (80 units subcutaneous 2 times a week) alone

Time frame: 12 months

The change in UP/CR ratio with treatment of Acthar gel 80 units subcutaneous 2 times a week in combination with oral Tacrolimus

The change in protein/creatinine ratio in patients with biopsy proven Fibrillary after 12 months of treatment with treated with Acthar gel 80 units 2 times a week subcutaneous week in combination with oral Tacrolimus 1.0 mg orally two times a day.

Time frame: 12 months

Secondary Outcomes

The relative change in protein/creatinine ratio at 24 months

The relative change in protein/creatinine at 24 months (12 months after stopping both Acthar and Tacrolimus) in the Acthar gel group and the Acthar gel plus Tacrolimus group.

Time frame: 24 months

Percentage of patients complete or partial response

The percentage of patients in the Acthar gel alone versus Acthar gel + Tacrolimus group that achieves complete, partial or clinical responses after 12 months of therapy

Time frame: 12 months

The change in Estimated Glomerular Filtration Rate

The change in Estimated Glomerular Filtration Rate between the Acthar gel and Acthar Gel plus Tacrolimus groups after 24 months of treatment with Acthar gel alone or in combination with oral Tacrolimus. In addition, we will also compare the relative change in eGFR between those patients receiving Acthar gel alone with those randomized to combination therapy.

Time frame: 24 months

Data from ClinicalTrials.gov

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