Kidney Disease
Conditions
Keywords
AfricanAmericans, Hypertension, Proteinuria, ApoL1 gene
Brief 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.
Interventions
DRUGActhar
FDA approved drug being used in this study for sub-nephrotic proteinuria. Given Investigational New Drug (IND) exemption by FDA.
Sponsors
Wake Forest University Health Sciences
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
21 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in proteinuria with H.C. Acthar gel | End of treatment with H.C. Acthar gel (end of 6 months or 1 year of treatment) | Complete remission (CR) (UPCR \<0.2g/g) or partial remission (PR) (50% drop in UPCR from baseline) of proteinuria at the end of Treatment period in patients with baseline nephrotic proteinuria |
| Change in eGFR with H.C. Acthar gel | End of treatment with H.C. Acthar gel (end of 6 months or 1 year of treatment) | Percent change in Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) eGFR at the end of Treatment period |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in CKD-EPI eGFR | 1 year and 2 years of study follow-up after treatment completion | Change in eGFR over time, based on baseline proteinuria (nephrotic vs. sub-nephrotic), baseline eGFR (eGFR 30-45 vs. eGFR 45-59), and Acthar dose |
| Duration of remission after H.C. Acthar gel treatment | 1 year and 2 years of study follow-up after treatment completion | Proportion of patients with baseline nephrotic proteinuria who sustained CR or PR at 1 and 2 years of study follow-up |
| Percent change in proteinuria | 1 year and 2 years of study follow-up after treatment completion | Percent change in proteinuria after 1 year and 2 years of follow-up |
| Cholesterol and lipoprotein profile before and after treatment with H.C. Acthar gel | End of treatment with H.C. Acthar gel (end of 6 months or 1 year of treatment) | Changes in cholesterol and lipoprotein levels compared with baseline profiles |
| Changes in kidney fibrosis after H.C. Acthar gel treatment | End of treatment with H.C. Acthar gel (end of 6 months or 1 year of treatment) | Modifications in kidney histopathology on second post-treatment kidney biopsy (% glomerulosclerosis, % tubulointerstitial fibrosis, restoration of podocyte markers \[e.g.,podocin, synaptopodin, Wilms tumor 1\]) compared with baseline biopsy |
| Percent change in CKD-EPI eGFR | 1 year and 2 years of study follow-up after treatment completion | Percent change in CKD-EPI eGFR at 1 and 2 years of study follow-up |
Countries
United States
Outcome results
None listed