ACTH Treatment of APOL1- Associated Nephropathy

ACTH Treatment of APOL1- Associated Nephropathy

The purpose of this research study is to determine if the study drug H.C. Acthar gel slows the progression of your kidney disease. This drug is a steroid-based medicine with fewer side effects than other steroids used for treatment of kidney diseases similar to APOL1 nephropathy.

Inclusion Criteria:

• Non-diabetic African-American with two APOL1-risk genotypes
• Age ≥21 years
• BMI < 40 kg/m2
• Hemoglobin A1c <6.5%
• eGFR ≥30 ml/min/1.73m2
• Historical urine protein: creatinine ratio ≥ 1.0 g/g
• Strong clinical suspicion of APOL1-associated nephropathy or history of biopsy proven focal segmental glomerulosclerosis (FSGS) or focal global glomerulosclerosis (FGGS)
• Women of childbearing potential: negative serum pregnancy test at Screening and agreement to follow a medically acceptable form of contraception for the duration of Acthar administration and 4 weeks thereafter

Exclusion Criteria:

• Diagnosis of diabetes mellitus and/or on pharmacologic treatment for diabetes
• Medical condition that could cause secondary FSGS
• History of sensitivity to steroids (psychosis, steroid-induced diabetes)
• Chronic systemic corticosteroid use (Prednisone or equivalent systemic steroid taken for more than 4 consecutive weeks within 6 months prior to screening). Intra-articular, inhaled, and topical steroids are not exclusion criteria.
• Contraindication to Acthar per package insert: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery (within previous 6 months), history of or the presence of peptic ulcer (within 6 months prior to Screening), adrenal insufficiency or hyperfunction.
• Acute glaucoma diagnosed ≤3 months prior to Screening
• Biopsy proven glomerular disease other than FSGS or FGGS
• Live or live attenuated vaccine received within 1 month prior to screening, or planned administration once enrolled in the study
• Uncontrolled hypertension (HTN) (≥ 180/110 mmHg) and frequent admissions (≥1 admission per 6 months interval) for hypertensive urgency or hypertensive emergency
• Unstable cardiovascular disease: history of congestive heart failure (NYHA Functional Class III-IV); history of dilated cardiomyopathy with ejection fraction < 40%; any of the following events within 3 months of screening: unstable angina, myocardial infarction, coronary artery bypass graft or percutaneous transluminal coronary angioplasty, transient ischemic attack or cerebrovascular accident, unstable arrhythmia
• Uncontrolled volume overload: history of moderate or severe peripheral edema; on loop diuretics ≥ 120 mg daily of furosemide or ≥ 3.0 mg daily of bumetanide or ≥ 150 mg daily of ethacrynic acid or ≥ 60 mg daily of torsemide;
• History of secondary hypertension (i.e., renal artery stenosis, primary aldosteronism or pheochromocytoma)
• Significant comorbidities (e.g., advanced malignancy, advanced liver disease) with a life expectancy of less than 1 year
• Subject is expected to initiate dialysis within 6 months
• Previous treatment on a drug being investigated for the treatment of FSGS
• Known diagnosis of Human Immunodeficiency Virus, Hepatitis B, or Hepatitis C
• Known history of a primary immunodeficiency or an underlying condition such as splenectomy that predisposes the subject to infections
• Systemic hematologic disease (e.g., hematologic malignancy, sickle cell anemia, myelodysplastic syndrome)
• Current malignancy or history of malignancy within 5 years of screening, with the exception of non-melanoma skin cancers and cervical intraepithelial neoplasia
• Treatment for any malignancy (e.g., radiation, chemotherapy, hormone therapy, or biologics) within 5 years of screening, with the exception of locally excised non-melanoma skin cancer or cervical intraepithelial neoplasia
• Pregnant or breast feeding, or might become pregnant during the study or within 4 weeks after the end of treatment
• Female of reproductive potential not willing to use highly effective methods of birth control during treatment and for 4 weeks after the end of treatment
• Currently receiving systemic antibiotics for treatment of an active infection; or history of frequent infections (more than one event per 6 months)
• History of any organ transplant
• Bipolar disorder, or Major Depressive Disorder characterized by severe depression requiring hospitalization, or history of suicidal ideation/attempts
• Currently enrolled in another interventional study, or less than 4 weeks since ending another interventional study(s) or receiving investigational agents(s)
• Subject has a disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or comply with all required study procedures.