ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children

• Status: Withdrawn
• Trial end date: 2021-07-01
• Target enrollment: 0
• Participant gender: All

Summary

We propose to study the use of purified porcine Acthar Gel (ACTHAR, Mallinckrodt Pharmaceuticals) for treatment of steroid resistance nephrotic syndrome (SRNS) in a prospective pilot study. We plan to enroll 25 children between the ages of 2 to 21 years. Children fulfilling strict inclusion criteria, whose parents agree to written informed consent after institutional IRB approval for the study, will be enrolled. Purified porcine Acthar Gel will be administered SQ to all children using a defined treatment protocol for a period of six months. Renal function, urine protein excretion, serum albumin levels, blood pressure and growth parameters will be monitored closely on all patients. Baseline urine protein excretion will be compared to end of treatment levels to determine successful response to therapy. There will be an 18 month enrollment period, 6 month treatment period and a 12 month follow-up period.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Rhode Island Hospital
• Collaborators: Mallinckrodt; Medalytics; University of Minnesota; University of Minnesota, MN
• Treatments: Adrenocorticotropic Hormone; Pharmaceutical Solutions

Criteria

Inclusion Criteria:

1. Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL

2. Calculated GFR (eGFR) using modified Schwartz formula of > 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL)

3. A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study.

4. Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy

Exclusion Criteria:

1. Patients with an inherited or genetic disorder presenting with nephrotic syndrome (eg:

NPHS 1 & 2 defects, WT-1 mutations, α actinin 4 mutation, TRP-6 mutation).

2. Presence of diabetes or severe (stage 2) uncontrolled hypertension.

3. Any metabolic condition that specifically precludes the use of Acthar Gel for treatment.

4. Pregnancy or unwilling to agree to contraception which may include abstinence.

5. eGFR <50 mls/min/m2