Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome
Nephrotic Syndrome
About this trial
This is an interventional treatment trial for Nephrotic Syndrome
Eligibility Criteria
Inclusion Criteria:
- Age >1 year at onset of nephrotic syndrome
- Age 2-20 years at time of randomization
- Estimated glomerular filtration rate (GFR) > 50 ml/min/1.73 m2 at most recent measure prior to randomization (Schwartz formula)
- Steroid responsive nephrotic syndrome throughout clinical course (never required a second agent to attain remission of a relapse of nephrotic syndrome)
- History of frequently relapsing or steroid dependent nephrotic syndrome (defined as 2 or more relapses within 6 months after initial therapy or 4 or more relapses in any 12 month period OR relapse during taper or within 2 weeks of discontinuing prednisone).
- Patient is currently in relapse of nephrotic syndrome or had a relapse within the last 4 months (defined as an increase in the first morning urine protein to creatinine ratio ≥2 or Albustix reading of ≥2 for 3 or 5 consecutive days).
Exclusion Criteria:
- Prior treatment with ACTH.
- Cyclophosphamide or rituximab within the last 4 months.
- Lactation, pregnancy, or refusal of birth control in females with child-bearing potential
- Planned treatment with live or live-attenuated vaccines once enrolled in the study.
- Participation in another therapeutic trial concurrently or 30 days prior to randomization
- Active/serious infection (including, but not limited to Hepatitis B or C, HIV)
- Malignancy concurrently or within the last 2 years.
- Blood pressure >95% for age/height while receiving maximal doses of 3 or more medications.
- Prior diagnosis of diabetes mellitus (Type I or II) or fasting glucose >200mg/dL
- Organ transplantation
- Contraindications to Acthar: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction
- Secondary cause of nephrotic syndrome (e.g., SLE)
- Biopsy demonstrating a diagnosis other than minimal change, focal segmental glomerulosclerosis (FSGS) or a variant (mesangial proliferation, Immunoglobulin M nephropathy)
- Inability to consent/assent -
Sites / Locations
- Pediatric Nephrology of Alabama, PC.
- University of California, Los Angeles
- Nemours/AI duPont Hospital for Children
- Nemours Children's Hospital
- Emory University
- Riley Hospital for Children
- Boston Children's Hopsital
- Helen DeVos Children's Hospital at Spectrum Health
- Mayo Clinic
- Children's Mercy
- Children's Hospital at Montefiore
- Duke Children's Health Center
- East Carolina University
- Driscoll Children's Hospital
- Texas Children's Hospital
- Children's Hospital of Richmond at VCU
Arms of the Study
Arm 1
Arm 2
Arm 3
Active Comparator
No Intervention
Active Comparator
Adrenocorticotropic hormone (ACTH)
No treatment
Rescue therapy
Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2 The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).
Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study.
There is an option for the patient in no-treatment arm to elect to be placed in the active treatment arm of the trial (rescue therapy).