Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR)
Condition: Treatment Resistant Nephrotic Syndrome
MeSH terms: Nephrotic Syndrome; Nephrosis; Syndrome
Intervention: ACTHAR gel
Intervention description: Patients will be treated with ACTHAR gel starting with 40 units given twice weekly subcutaneously for two weeks, then 80 units given twice weekly subcutaneously afterwards for a period of up to six months.
Intervention MeSH terms: Adrenocorticotropic Hormone
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 15.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Treatment Resistance; Nephrotic Syndrome; Focal Segmental Glomerulosclerosis; lipoid nephrosis; Membranous nephropathy; IgA nephropathy; proteinuria

Summary:

      This study will examine the safety and effectiveness of ACTHAR Gel, when used to treat 15
      patients diagnosed with "treatment resistant nephrotic syndrome."

      Nephrotic syndrome is a group of symptoms that includes low levels of protein in the blood,
      swelling of tissue (edema), especially around the eyes, feet and hands; and high plasma
      levels of cholesterol. It is caused by a variety of diseases and underlying disorders that
      damage the kidneys, resulting in excessive excretion of protein in the urine. These diseases
      damage the glomeruli, which are small blood vessels that filter wastes and excess water from
      the blood and pass them into the bladder as urine. As a result of protein loss in the urine,
      the blood is deficient in protein. Normal amounts of blood protein are needed to help
      regulate fluid throughout the body. Protein in the blood normally draws water from the
      tissues and into the bloodstream. When blood protein levels are low, the normal movement of
      water is reversed, and fluid is drawn from the blood and accumulates in the tissues. This
      excess tissue fluid causes the swelling and puffiness (edema) that is a symptom of nephrotic
      syndrome.

      Nephrotic syndrome is described as "treatment resistant" when a patient fails to achieve a
      sustained partial or complete remission after treatment with at least two first line
      therapies.

      The goal of this study is to determine whether injections of ACTHAR Gel (an FDA approved
      treatment for nephrotic syndrome) over a six month period will lead to a correction of
      treatment resistant nephrotic syndrome in these patients.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Adult nephrotic patients, at least 18 years of age

          2. Diagnosis of focal segmental glomerulosclerosis (FSGS), resistant or relapsing minimal
             change disease with failure to achieve a sustained partial or complete remission of
             the nephrotic syndrome after therapy with at least two first line therapies:
             corticosteroids and at least one other immunosuppressive regimen

          3. Diagnosis of membranous nephropathy with failure to achieve a sustained partial or
             complete remission of the nephrotic syndrome after therapy with at least two first
             line therapies: either the "Ponticelli protocol" of alternating months of pulse
             steroids followed by oral steroids and then an alkylating agent with each regimen
             repeated for six full months of therapy or a calcineurin inhibitor, cyclosporine or
             tacrolimus, and at least one other therapy

          4. Diagnosis of immunoglobulin A nephropathy (IgAN) with persistent nephrotic range
             proteinuria in patients on ACE inhibition or angiotension receptor blockers (ARB)
             therapy to reduce proteinuria

          5. Willing and able to sign informed consent

          6. Patients of childbearing age must agree to use birth control

        Exclusion Criteria:

          1. Patients under 18 years of age

          2. Patients unable to sign informed consent

          3. Patients having received rituximab or another monoclonal antibody within 6 months of
             the trial

          4. Patients of childbearing age who refuse to use birth control

          5. Patients with an estimated glomerular filtration rate (eGFR) less than 30
             ml/min/1.73m2

          6. Patients with other renal diseases (e.g. diabetic nephropathy, renal vascular disease)
             that would interfere with interpretation of the study.

          7. Patients with comorbid conditions that would interfere with completion of the trial
             (malignancies, congestive heart failure (CHF), recent myocardial infarction).

          8. Patients with known contraindications to the use of H.P. ACTHAR Gel, including:
             scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent
             surgery, history of or the presence of a peptic ulcer, congestive heart failure,
             uncontrolled hypertension, and allergies to pork or pork products.