Treatment Resistant Nephrotic Syndrome Clinical Trial
Official title:
The Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR)
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.
The nephrotic syndrome is characterized by heavy proteinuria, edema, hyperlipidemia, and a
thrombotic tendency. Membranous nephropathy, focal segmental glomerulosclerosis, resistant
minimal change disease, and Immunoglobulin A (IgA) nephropathy (also known as Berger's
disease) are the common forms of idiopathic nephrotic syndrome in adults. Asymptomatic
patients with idiopathic nephrotic syndrome may be treated with ACE inhibitors and /or
angiotension receptor blockers to reduce proteinuria, with statins to treat hyperlipidemia,
and diuretics to control edema.
Patients with large amounts of proteinuria are refractory to such treatments and often
require immunosuppressive medications to promote a remission of the proteinuria and the
nephrotic syndrome and to prevent progressive renal failure. All such immunosuppressives
have multiple potential serious side effects. Some patients either relapse after remissions
of their proteinuria or are resistant to immunosuppressive therapy.
A synthetic truncated analog of ACTH has been used in patients with the nephrotic syndrome
in both uncontrolled trials and in a randomized controlled trial in membranous nephropathy.
In uncontrolled studies it has led to sustained remissions of the nephrotic syndrome in
multiple forms of idiopathic disease including membranous nephropathy and focal segmental
glomerulosclerosis (FSGS). It has also led to reduction of total cholesterol, LDL
cholesterol, Lpa, and elevations of HDL cholesterol. In a controlled randomized trial in
membranous nephropathy it proved equivalent to an alternating monthly regimen of
corticosteroids and an alkylating agent that is widely regarded as a first line therapy for
this disease.
This synthetic truncated analog of ACTH is not available in the US. ACTHAR gel is a natural,
highly purified, porcine ACTH which is both available in the US and FDA approved for use in
the nephrotic syndrome. Although ACTHAR gel has been used to treat large numbers of patients
with multiple sclerosis and infantile paralysis annually, it has, however, been used in only
small numbers of patients for the indication of the nephrotic syndrome.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment