ACTH Treatment of APOL1- Associated Nephropathy

Wake Forest University (WFU)

Status

Withdrawn

Conditions

Kidney Disease

Treatments

Drug: Acthar

Study type

Interventional

Funder types

Other

Identifiers

NCT02006849

IRB00024943

Details and patient eligibility

About

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.

Sex

All

Ages

21+ years old

Volunteers

No Healthy Volunteers

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.