Adrenocorticotropic Hormone in Membranous Nephropathy

Mount Sinai Health System

Status and phase: Completed; Phase 4

Conditions: Membranous Nephropathy

Treatments: Drug: ACTHar

Study type: Interventional

Funder types: Other

Identifiers

NCT03025828

GCO 16-2402

Details and patient eligibility

About

The purpose of this study is to evaluate the effect of adrenocorticotropic hormone (ACTH, Acthar) on the loss of proteins in the urine (proteinuria) in patients with membranous nephropathy. Acthar is a hormone that stimulates steroid production from small glands above the kidneys. It has direct protective effects on the kidney and is currently approved by the FDA to treat kidney disorders associated with proteins in the urine, but the mechanisms of action are not entirely understood and will be studied in the present trial.

Full description

Patients with membranous nephropathy and nephrotic syndrome will be treated with ACTH for 6 months. Proteinuria remission at 12 months will be the primary endpoint. Different biomarkers including anti-PLA2R autoantibodies, circulating regulatory T cells, and autoreactive memory B cells will be serially measured to identify predictors of response to therapy.

Enrollment: 5 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age 18 to 70 years
• Free of immunosuppression for at least 3 months
• Capability of understanding the purpose of the study
• Written informed consent

Exclusion criteria

• Epidermal growth factor receptor (eGFR) < 30ml/min/1.73m2
• Kidney Transplant
• Secondary MN (defined on the basis of clinical criteria)
• Type 1 or Type 2 diabetes mellitus (prior diagnosis of gestational diabetes mellitus is not an exclusion)
• History of previous use of Acthar for treatment of nephrotic syndrome
• Prior sensitivity to Acthar or other porcine protein products
• Contraindication to Acthar per Prescribing Information
• Planned treatment with live or live attenuated vaccines once enrolled in the study
• More than three previous treatment regiments
• Participation to other clinical trials over the previous 12 months
• History of cancer, except carcinoma in situ and treated basal and squamous cell carcinomas
• Pregnancy
• Lactation
• Current substance abuse
• Any clinically relevant condition that might affect study participation and/or study results

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Acthar

Experimental group

Description:

Acthar will be administered subcutaneously (SC) 80 units for the first week and then 80 units twice weekly

Treatment:

Drug: ACTHar