Treatment Resistant Nephrotic Syndrome Clinical Trial
Official title:
The Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR)
| Verified date | March 2014 |
| Source | Columbia University |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | United States: Institutional Review Board |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 15 |
| Est. completion date | September 2011 |
| Est. primary completion date | September 2011 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 18 Years and older |
| Eligibility |
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. |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| United States | Columbia University Medical Center | New York | New York |
| Lead Sponsor | Collaborator |
|---|---|
| Columbia University | Mallinckrodt |
United States,
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. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | No |
| Secondary | Sustained Remissions up to 1 Year After Discontinuing Therapy | Complete remission defined as stable or improved renal function, serum creatinine < or = 125% baseline, with proteinuria falling <500 mg/day at last follow-up visit (6 to 12 months post treatment). Partial remission 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 last follow-up visit (6 to 12 months post treatment). | Up to 1 year after treatment | No |