Treatment of Resistant Nephrotic Syndrome With ACTH Gel (ACTHAR)
Status:
Completed
Trial end date:
2011-09-01
Target enrollment:
Participant gender:
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.