Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01155141
Recruitment Status : Completed
First Posted : July 1, 2010
Results First Posted : September 1, 2014
Last Update Posted : September 1, 2014
Information provided by (Responsible Party):
Stanford University

June 29, 2010
July 1, 2010
August 1, 2014
September 1, 2014
September 1, 2014
September 2009
January 2014   (Final data collection date for primary outcome measure)
  • 24 Hour Proteinuria [ Time Frame: Baseline - Month 6 ]
  • Serum Creatinine [ Time Frame: Baseline - Month 6 ]
  • Protein/Creatinine Ratio [ Time Frame: Baseline - Month 6 ]
Reduction of proteinuria. [ Time Frame: 6 months. ]
Complete list of historical versions of study NCT01155141 on Archive Site
  • eGFR [ Time Frame: Baseline - Month 6 ]
  • Weight [ Time Frame: Baseline - Month 6 ]
  • Systolic Blood Pressure [ Time Frame: Baseline - Month 6 ]
  • Diastolic Blood Pressure [ Time Frame: Baseline - Month 6 ]
  • Glucose [ Time Frame: Baseline - Month 6 ]
  • Total Cholesterol [ Time Frame: Baseline - Month 6 ]
Changes in kidney function (eGFR), glucose, blood pressure, and immune biomarkers. [ Time Frame: 6 months ]
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Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH
Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH
FSGS is an immunologic disorder wherein circulating immune proteins cause damage to the kidneys and progressive injury and scarring. Corticosteroid therapy is occasionally, but not nearly universally, successful in reducing proteinuria, and when patients respond, they have a favorable prognosis. The investigators believe that ACTH therapy (H.P. Acthar Gel) can provide a more rapid, well tolerated reduction in glomerular injury.

EXPERIMENTAL TREATMENT: Patients with biopsy proven FSGS will be treated with ACTH in an open-label pilot study of tolerability and efficacy. The investigators will recruit 18-20 patients to complete this study, over 2 years.

ACTH therapy: Patients will receive H.P. Acthar gel IM or SQ, initially at 40 IU SQ every week for 16 weeks of therapy. All patients will be treated to goal BP of <140/90mmHg with all patients treated with ACEi or ARB therapy as tolerated. H2 receptor blockade or proton pump inhibitor therapy will also be offered all patients. Dose will be titrated up to 160 IU SQ every week, if at 1 month the patient has had no substantial reduction in proteinuria, no deterioration of blood pressure and no development of hyperglycemia as well as no serious adverse events felt to be related to the medication.

CLINICAL OUTCOME: Patients will be followed for the primary outcome of remission of proteinuria. This will be defined as partial remission when the proteinuria is reduced below 50% of the initial, pre treatment value and as complete remission when the proteinuria is reduced to < 0.5 g/g or <500 mg/day on a 24 hour urine sample. Secondary outcomes will include effects on eGFR, effects on glucose levels, effects on blood pressure (total doses of antihypertensive medications and absolute changes in blood pressure) and on immune status. Outcomes will be determined by looking at 3 month and 6 month values of urine protein and eGFR following initiation of treatment.

IMMUNOLOGIC TESTING: In order to further assess the role of immunologic perturations on FSGS and the effect of ACTH on the immune system, all patients will bank blood and urine before the start of the study for cytokine analysis, RNA, DNA, protein and protoarray testing.

MONITORING AND SAFETY: All patients will undergo full informed consent per the Stanford Institutional Review Board. Contact numbers will be provided and study staff will be available at all times in case of any medical emergencies. All patients will continue with routine health monitoring with a minimal of monthly assessments. A comprehensive interview will be undertaken to assess for side effects, complete physical exams will be accomplished including vital signs, CBC, clinical chemistries (including electrolytes, creatinine, glucose and liver function tests), urine for protein and creatinine and fasting lipid profiles every 3 months. Also at screening and baseline.

PATIENT WITHDRAWAL/TERMINATION OF STUDY: Patients will be closely monitored, as detailed above. Patients may voluntarily leave the study at any time, although every effort will be made to follow their clinical course and monitor for safety issues and possible benefits of therapy. Patient will be monitored for adverse events. Patients with severe adverse events will be evaluated for the relatedness of their event to the study medication. If the event is considered severe and possibly or probably related to the study medication, the medication will be discontinued and the patient will continue to be monitored. In the case the adverse event is possibly related, the medication may be restarted, if the investigator and subject agree. For patients with probably related severe adverse events, study treatment will be discontinued however, the investigators will still follow the patient to see if the course of their underlying disease was modified by treatment. As this is an open label, pilot study, no data safety monitoring board is felt to be necessary. If drug related SAEs develop in more than 20% of patients, the study will be submitted back to the IRB to determine if it should continue.

Phase 4
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Kidney Diseases
Drug: H.P. Acthar Gel
Patients were treated with 40 units subcutaneously (SC) weekly for 2 weeks, then dose increased to 80 units SC weekly for 2 weeks followed by 80 units SC twice weekly to complete 16 weeks of therapy.
Experimental: No arms
There are no arms to this study. All patients receive drug (H.P. Acthar Gel)
Intervention: Drug: H.P. Acthar Gel
Hogan J, Bomback AS, Mehta K, Canetta PA, Rao MK, Appel GB, Radhakrishnan J, Lafayette RA. Treatment of idiopathic FSGS with adrenocorticotropic hormone gel. Clin J Am Soc Nephrol. 2013 Dec;8(12):2072-81. doi: 10.2215/CJN.02840313. Epub 2013 Sep 5.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
January 2014
January 2014   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • 1. Biopsy proven FSGS
  • 2. Ability to consent and complete study evaluations
  • 3. Greater than 2g/day of proteinuria
  • 4. No contraindications to treatment with corticosteroids or ACTH
  • 5. Women of childbearing potential will agree to use effective forms of birth control throughout this study

Exclusion Criteria:

  • 1. Known secondary cause of FSGS
  • 2. Receiving active immune therapy (within 90 days)
  • 3. Pregnancy
  • 4. Creatinine >2.5 mg/dl
  • 5. Uncontrolled HTN (>180/100mm Hg)
  • 6. Diabetes
  • 7. Acute or chronic infection
  • 8. Severe comorbidity (active coronary, cerebrovascular disease, cancer, psychiatric disease)
  • 9. Age < 16, >65 years
  • 10. Evidence of untreated tuberculosis (+PPD or Ellispot Gold testing)
  • 11. A known contraindication to ACTH. Corticotropin is considered contraindicated in patients with scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of a peptic ulcer, congestive heart failure, uncontrolled hypertension, or sensitivity to proteins of porcine origin.
Sexes Eligible for Study: All
16 Years to 65 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
United States
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Stanford University
Stanford University
Principal Investigator: Richard Lafayette Stanford University
Stanford University
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP