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

NCT05546047 | Unknown Phase 4 FDA Drug

• 1 other identifier: NN-002
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Fibrillary Glomerulonephritis

Outcome Measures

Primary Outcomes (2)

• 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

  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.

  12 months

Secondary Outcomes (4)

• The relative change in protein/creatinine ratio at 24 months

  24 months

• Percentage of patients complete or partial response

  12 months

• The change in Estimated Glomerular Filtration Rate

  24 months

• To compare the change in urinary biomarkers of Urinary VEGF 121, 165 189, and206, Urinary MCP-1, Urinary Synaptopodin, Urinary TGF-beta, Urinary Podocalyxin, and Urinary Nephrin

  12 months

Other Outcomes (1)

• To determine if patients with concurrent Type II diabetes mellitus resulted in hyperglycemia, increased proteinuria, loss of renal function or led to immunosuppressive therapies

  24 months

Study Arms (2)

Treatment with Acthar gel

ACTIVE COMPARATOR

Group 1 - (17 patients) Acthar gel 80 units 2 times a week alone for 12 months of therapy.

Drug: Acthar Gel 80 UNT/ML Injectable Solution

Treatment with combination Acthar gel and Tacrolimus therapy

ACTIVE COMPARATOR

Group 2 - (17 patients) Acthar get 80 units 2 times a week plus oral Tacrolimus 1.0 mg two times a day titrated to trough Tacrolimus levels between 4-6 ng/ml

Drug: Acthar Gel 80 UNT/ML Injectable Solution

Interventions

Acthar Gel 80 UNT/ML Injectable Solution DRUG

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

Also known as: Tacrolimus 1.0 mg

Treatment with Acthar gel; Treatment with combination Acthar gel and Tacrolimus therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• NephroNet, Inc.: lead
• Mallinckrodt: collaborator

Study Sites (5)

Stanford University

Stanford, California, 94305, United States

Location

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

Aurora, Colorado, 80045, United States

Location

Georgia Nephrology DBA Georgia Nephrology Research Institute

Lawrenceville, Georgia, 30046, United States

Location

Columbia University Research Dept of Nephrology

New York, New York, 10032, United States

Location

Northeast Clinical Research Center

Bethlehem, Pennsylvania, 18017, United States

Location

MeSH Terms

Interventions

Tacrolimus

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Jeremy Whitson, BS

  NephroNet, Inc.

  STUDY DIRECTOR

• James Tumlin, MD

  NephroNet, Inc.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: A 12-month treatment with combination Acthar gel and Tacrolimus therapy will be superior to Acthar gel therapy alone in lowering urinary protein to creatinine ratios in patients with Fibrillary glomerulonephritis

Study Record Dates

• First Submitted: August 23, 2022
• First Posted: September 19, 2022
• Study Start: March 14, 2019
• Primary Completion: October 27, 2023
• Study Completion: October 27, 2024
• Last Updated: October 31, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)