ACTH Treatment of APOL1- Associated Nephropathy

• Status: Withdrawn
• Trial end date: 2019-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Wake Forest University Health Sciences
• Treatments: Adrenocorticotropic Hormone

Criteria

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.