Recruitment

Recruitment Status
Withdrawn
Estimated Enrollment
30

Inclusion Criteria

Strong clinical suspicion of APOL1-associated nephropathy or history of biopsy proven focal segmental glomerulosclerosis (FSGS) or focal global glomerulosclerosis (FGGS)
BMI < 40 kg/m2
Hemoglobin A1c <6.5%
...
Strong clinical suspicion of APOL1-associated nephropathy or history of biopsy proven focal segmental glomerulosclerosis (FSGS) or focal global glomerulosclerosis (FGGS)
BMI < 40 kg/m2
Hemoglobin A1c <6.5%
Age ≥21 years
eGFR ≥30 ml/min/1.73m2
Non-diabetic African-American with two APOL1-risk genotypes
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
Historical urine protein: creatinine ratio ≥ 1.0 g/g

Exclusion Criteria

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
Live or live attenuated vaccine received within 1 month prior to screening, or planned administration once enrolled in the study
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.
...
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
Live or live attenuated vaccine received within 1 month prior to screening, or planned administration once enrolled in the study
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.
Diagnosis of diabetes mellitus and/or on pharmacologic treatment for 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.
Biopsy proven glomerular disease other than FSGS or FGGS
Current malignancy or history of malignancy within 5 years of screening, with the exception of non-melanoma skin cancers and cervical intraepithelial neoplasia
Medical condition that could cause secondary FSGS
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.
Pregnant or breast feeding, or might become pregnant during the study or within 4 weeks after the end of treatment
Uncontrolled hypertension (HTN) (≥ 180/110 mmHg) and frequent admissions (≥1 admission per 6 months interval) for hypertensive urgency or hypertensive emergency
Acute glaucoma diagnosed ≤3 months prior to Screening
History of any organ transplant
History of secondary hypertension (i.e., renal artery stenosis, primary aldosteronism or pheochromocytoma)
Known history of a primary immunodeficiency or an underlying condition such as splenectomy that predisposes the subject to infections
Bipolar disorder, or Major Depressive Disorder characterized by severe depression requiring hospitalization, or history of suicidal ideation/attempts
Subject is expected to initiate dialysis within 6 months
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;
Currently enrolled in another interventional study, or less than 4 weeks since ending another interventional study(s) or receiving investigational agents(s)
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
History of sensitivity to steroids (psychosis, steroid-induced diabetes)
Known diagnosis of Human Immunodeficiency Virus, Hepatitis B, or Hepatitis C
Previous treatment on a drug being investigated for the treatment of FSGS
Systemic hematologic disease (e.g., hematologic malignancy, sickle cell anemia, myelodysplastic syndrome)
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)
Significant comorbidities (e.g., advanced malignancy, advanced liver disease) with a life expectancy of less than 1 year

Summary

Conditions
Kidney Disease
Type
Interventional
Design
  • Allocation: Non-Randomized
  • Intervention Model: Parallel Assignment
  • Masking: None (Open Label)
  • Primary Purpose: Treatment

Participation Requirements

Age
Between 21 years and 125 years
Gender
Both males and females

Inclusion Criteria

Strong clinical suspicion of APOL1-associated nephropathy or history of biopsy proven focal segmental glomerulosclerosis (FSGS) or focal global glomerulosclerosis (FGGS)
BMI < 40 kg/m2
Hemoglobin A1c <6.5%
...
Strong clinical suspicion of APOL1-associated nephropathy or history of biopsy proven focal segmental glomerulosclerosis (FSGS) or focal global glomerulosclerosis (FGGS)
BMI < 40 kg/m2
Hemoglobin A1c <6.5%
Age ≥21 years
eGFR ≥30 ml/min/1.73m2
Non-diabetic African-American with two APOL1-risk genotypes
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
Historical urine protein: creatinine ratio ≥ 1.0 g/g

Exclusion Criteria

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
Live or live attenuated vaccine received within 1 month prior to screening, or planned administration once enrolled in the study
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.
...
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
Live or live attenuated vaccine received within 1 month prior to screening, or planned administration once enrolled in the study
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.
Diagnosis of diabetes mellitus and/or on pharmacologic treatment for 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.
Biopsy proven glomerular disease other than FSGS or FGGS
Current malignancy or history of malignancy within 5 years of screening, with the exception of non-melanoma skin cancers and cervical intraepithelial neoplasia
Medical condition that could cause secondary FSGS
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.
Pregnant or breast feeding, or might become pregnant during the study or within 4 weeks after the end of treatment
Uncontrolled hypertension (HTN) (≥ 180/110 mmHg) and frequent admissions (≥1 admission per 6 months interval) for hypertensive urgency or hypertensive emergency
Acute glaucoma diagnosed ≤3 months prior to Screening
History of any organ transplant
History of secondary hypertension (i.e., renal artery stenosis, primary aldosteronism or pheochromocytoma)
Known history of a primary immunodeficiency or an underlying condition such as splenectomy that predisposes the subject to infections
Bipolar disorder, or Major Depressive Disorder characterized by severe depression requiring hospitalization, or history of suicidal ideation/attempts
Subject is expected to initiate dialysis within 6 months
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;
Currently enrolled in another interventional study, or less than 4 weeks since ending another interventional study(s) or receiving investigational agents(s)
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
History of sensitivity to steroids (psychosis, steroid-induced diabetes)
Known diagnosis of Human Immunodeficiency Virus, Hepatitis B, or Hepatitis C
Previous treatment on a drug being investigated for the treatment of FSGS
Systemic hematologic disease (e.g., hematologic malignancy, sickle cell anemia, myelodysplastic syndrome)
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)
Significant comorbidities (e.g., advanced malignancy, advanced liver disease) with a life expectancy of less than 1 year

Tracking Information

NCT #
NCT02006849
Collaborators
Not Provided
Investigators
  • Principal Investigator: Mariana Murea, MD Wake Forest University Health Sciences
  • Mariana Murea, MD Wake Forest University Health Sciences
About Us
Innovation
Privacy
Terms
Copyright
Get Well Soon © Copyright 2024