Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR)

NCT01129284 | Completed Phase 4 Results

• 1 other identifier: AAAD9539
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Treatment Resistant Nephrotic Syndrome

Keywords

Treatment Resistance; Nephrotic Syndrome; Focal Segmental Glomerulosclerosis; lipoid nephrosis; Membranous nephropathy; IgA nephropathy; proteinuria

Outcome Measures

Primary Outcomes (1)

• Significant Reduction in Proteinuria to Remission Levels During the Treatment Period (Includes Complete and Partial Remission, as Defined in the Outcome Measure Description Below)

  Complete remission is defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with proteinuria falling \<500 mg/day at month 6. Partial remission is defined as stable or improved renal function, serum creatinine \< or = 125% baseline, with 50% reduction in proteinuria and final proteinuria 500-3500 mg/day at month 6.

  6 months

Secondary Outcomes (1)

• Sustained Remissions up to 1 Year After Discontinuing Therapy

  Up to 1 year after treatment

Study Arms (1)

Acthar gel

EXPERIMENTAL

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.

Drug: ACTHAR gel

Interventions

ACTHAR gel DRUG

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.

Also known as: H.P. Acthar gel

Acthar gel

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult nephrotic patients, at least 18 years of age
• 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
• 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
• 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
• Willing and able to sign informed consent
• Patients of childbearing age must agree to use birth control

You may not qualify if:

• Patients under 18 years of age
• Patients unable to sign informed consent
• Patients having received rituximab or another monoclonal antibody within 6 months of the trial
• Patients of childbearing age who refuse to use birth control
• Patients with an estimated glomerular filtration rate (eGFR) less than 30 ml/min/1.73m2
• Patients with other renal diseases (e.g. diabetic nephropathy, renal vascular disease) that would interfere with interpretation of the study.
• Patients with comorbid conditions that would interfere with completion of the trial (malignancies, congestive heart failure (CHF), recent myocardial infarction).
• 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.

Sponsors & Collaborators

• Columbia University: lead
• Mallinckrodt: collaborator

Study Sites (1)

Columbia University Medical Center

New York, New York, 10032, United States

Location

Related Publications (2)

• Bomback AS, Canetta PA, Beck LH Jr, Ayalon R, Radhakrishnan J, Appel GB. Treatment of resistant glomerular diseases with adrenocorticotropic hormone gel: a prospective trial. Am J Nephrol. 2012;36(1):58-67. doi: 10.1159/000339287. Epub 2012 Jun 19.

  PMID: 22722778 RESULT

• 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.

  PMID: 24009220 DERIVED

MeSH Terms

Conditions

Nephrotic Syndrome; Glomerulosclerosis, Focal Segmental; Nephrosis, Lipoid; Glomerulonephritis, Membranous; Glomerulonephritis, IGA; Proteinuria

Condition Hierarchy (Ancestors)

Nephrosis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Glomerulonephritis; Nephritis; Autoimmune Diseases; Immune System Diseases; Urination Disorders; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Dr. Andrew S. Bomback, MD, MPH
• Organization: Columbia University Medical Center

asb68@columbia.edu

212-305-0320

Study Officials

• Gerald B Appel, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Clinical Medicine, Nephrology

Study Record Dates

• First Submitted: May 21, 2010
• First Posted: May 24, 2010
• Study Start: December 1, 2009
• Primary Completion: September 1, 2011
• Study Completion: September 1, 2011
• Last Updated: April 24, 2014
• Results First Posted: April 24, 2014

Record last verified: 2014-03

Locations

US (1)