Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome

Overview

  • Study type

    Interventional
  • Study phase

    III
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 14-000177
    NCT ID: NCT02132195
    Sponsor Protocol Number: 14-000177

About this study

In childhood nephrotic syndrome, the kidneys leak protein, causing body swelling and a variety of possible complications such as infection, blood clots, and kidney failure. The first-line treatment for nephrotic syndrome is corticosteroids. Many children respond to prednisone treatment, but the disease comes back (relapses) when the prednisone is stopped or the dose is reduced. Children with frequently relapsing or steroid dependent nephrotic syndrome are at risk for toxicity from frequent exposure to corticosteroids. Currently, the standard treatment for frequently relapsing and steroid dependent nephrotic syndrome involves a variety of medications that suppress the immune system, which can produce serious side effects. We propose a study to examine the effects of a different medication, ACTH, on nephrotic syndrome. ACTH is a hormone naturally found in the body. Recently, in adult studies, ACTH has been shown to be effective for the treatment of nephrotic syndrome. It has also been shown to have mild and reversible side effects. ACTH is potentially an attractive therapeutic alternative for the treatment of frequently relapsing and steroid dependent nephrotic syndrome in children. Our study will randomly assign patients with frequently relapsing or steroid dependent nephrotic syndrome to either ACTH treatment or no treatment. This will allow us to study the effects of ACTH on this disease and its side effects, by comparing how patients do on ACTH treatment versus no treatment. We hypothesize that ACTH gel is superior to no treatment in maintaining remission in children with frequently relapsing or steroid dependent nephrotic syndrome.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  1. Age >1 year at onset of nephrotic syndrome
  2. Age 2-20 years at time of randomization
  3. Estimated glomerular filtration rate (GFR) > 50 ml/min/1.73 m2 at most recent measure prior to randomization (Schwartz formula)
  4. Steroid responsive nephrotic syndrome throughout clinical course (never required a second agent to attain remission of a relapse of nephrotic syndrome)
  5. 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).
  6. 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:

  1. Prior treatment with ACTH.
  2. Cyclophosphamide or rituximab within the last 4 months.
  3. Lactation, pregnancy, or refusal of birth control in females with child-bearing potential
  4. Planned treatment with live or live-attenuated vaccines once enrolled in the study.
  5. Participation in another therapeutic trial concurrently or 30 days prior to randomization
  6. Active/serious infection (including, but not limited to Hepatitis B or C, HIV)
  7. Malignancy concurrently or within the last 2 years.
  8. Blood pressure >95% for age/height while receiving maximal doses of 3 or more medications.
  9. Prior diagnosis of diabetes mellitus (Type I or II) or fasting glucose >200mg/dL
  10. Organ transplantation
  11. 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
  12. Secondary cause of nephrotic syndrome (e.g., SLE)
  13. Biopsy demonstrating a diagnosis other than minimal change, focal segmental glomerulosclerosis (FSGS) or a variant (mesangial proliferation, Immunoglobulin M nephropathy)
  14. Inability to consent/assent -

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Cheryl Tran, M.D.

Open for enrollment

Contact information:

Heather LaBrec

(507)538-3823

LaBrec.Heather@mayo.edu

.
CLS-20142980

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