Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH

2014-08-27 03:12:30 | BioPortfolio


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.

Study Design

Allocation: Non-Randomized, Control: Uncontrolled, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Kidney Diseases


H.P. Acthar Gel


Stanford University School of Medicine
United States




Stanford University

Results (where available)

View Results


Published on BioPortfolio: 2014-08-27T03:12:30-0400

Clinical Trials [1394 Associated Clinical Trials listed on BioPortfolio]

Effects of ACTHAR on Advanced MRI Surrogate Markers of Disease Activity and on Comprehensive Immune Signature During MS Relapses

ACTHAR is a FDA approved drug for MS relapses. The purpose of the study is to examine the efficacy of this agent in improving relapses as measured by advanced MRI and laboratory techniques...

Efficacy, Radiographic and Laboratory Changes in Refractory Rheumatoid Arthritis Patients Treated With H.P. Acthar Gel

This study will examine the clinical response, cytokine expression and joint imaging after addition of Acthar Gel. The hypothesis is that H.P. Acthar Gel is both safe and effective for tre...

Safety and Efficacy of Acthar Gel in an Outpatient Dialysis Population

This will be a prospective observational study of Acthar Gel in Non-Diabetic Hemodialysis [NDHD] patients receiving dialysis for ≤ 2 years. The project will aimed at providing proof-of-c...

A Multicenter, 2 Part Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in Subjects With Rheumatoid Arthritis

2 part multicenter study to examine the effect of Acthar in adult subjects with rheumatoid arthritis (RA) with persistently active disease. Part 1 is an Open Label Period in which all elig...

Acthar SLE (Systemic Lupus Erythematosus)

This is a randomized study exploring the efficacy, safety and steroid sparing ability of two doses (40 U and 80 U) of Acthar in SLE patients with immune mediated hematologic manifestations...

PubMed Articles [10251 Associated PubMed Articles listed on BioPortfolio]

Apheresis for Kidney Disease.

Plasma exchange or double filtration plasmapheresis for rapidly progressive glomerulonephritis, and low-density lipoprotein (LDL) apheresis or leukocytapheresis for nephritic syndrome are two major ap...

Kidney diseases associated with Waldenström macroglobulinemia.

Waldenström macroglobulinemia (WM) is a rare B-cell lymphoma characterized by lymphoplasmacytic cell infiltration in the bone marrow and other organs and the presence of a monoclonal immunoglobulin M...

Anti-Inflammatory Action of Sitagliptin and Linagliptin in Doxorubicin Nephropathy.

Dipeptidyl peptidase-4 (DPP4) inhibitors are known to have a protective effect on diabetic kidney disease, possibly via reduction of oxidative stress and inflammation in the kidney. However, whether t...

Kidney Tattoos.

Nrf2 activator for the treatment of kidney diseases.

Kidney diseases are highly prevalent worldwide, and significantly reduce the quality of life of patients, creating an urgent need for effective therapeutic modalities. Despite this significant unmet m...

Medical and Biotech [MESH] Definitions

A complication of kidney diseases characterized by cell death involving KIDNEY PAPILLA in the KIDNEY MEDULLA. Damages to this area may hinder the kidney to concentrate urine resulting in POLYURIA. Sloughed off necrotic tissue may block KIDNEY PELVIS or URETER. Necrosis of multiple renal papillae can lead to KIDNEY FAILURE.

Abnormal enlargement or swelling of a KIDNEY due to dilation of the KIDNEY CALICES and the KIDNEY PELVIS. It is often associated with obstruction of the URETER or chronic kidney diseases that prevents normal drainage of urine into the URINARY BLADDER.

Component of the NATIONAL INSTITUTES OF HEALTH. It conducts and supports basic and applied research for a national program in diabetes, endocrinology, and metabolic diseases; digestive diseases and nutrition; and kidney, urologic, and hematologic diseases. It was established in 1948.

A heterogeneous group of hereditary and acquired disorders in which the KIDNEY contains one or more CYSTS unilaterally or bilaterally (KIDNEY, CYSTIC).

Either a single or a single functioning kidney due to NEPHRECTOMY, birth defects or other kidney diseases.

More From BioPortfolio on "Idiopathic Focal Segmental Glomerulosclerosis (FSGS) and Treatment With ACTH"

Quick Search


Relevant Topic

Pharmacy is the science and technique of preparing as well as dispensing drugs and medicines. It is a health profession that links health sciences with chemical sciences and aims to ensure the safe and effective use of pharmaceutical drugs. The scope of...

Searches Linking to this Trial