Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome
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|ClinicalTrials.gov Identifier: NCT02132195|
Recruitment Status : Active, not recruiting
First Posted : May 7, 2014
Last Update Posted : May 1, 2018
In childhood nephrotic syndrome, the kidneys leak protein, causing body swelling and a variety of possible complications such as infection, blood clots, and kidney failure. The first-line treatment for nephrotic syndrome is corticosteroids. Many children respond to prednisone treatment, but the disease comes back (relapses) when the prednisone is stopped or the dose is reduced. Children with frequently relapsing or steroid dependent nephrotic syndrome are at risk for toxicity from frequent exposure to corticosteroids.
Currently, the standard treatment for frequently relapsing and steroid dependent nephrotic syndrome involves a variety of medications that suppress the immune system, which can produce serious side effects. We propose a study to examine the effects of a different medication, ACTH, on nephrotic syndrome. ACTH is a hormone naturally found in the body. Recently, in adult studies, ACTH has been shown to be effective for the treatment of nephrotic syndrome. It has also been shown to have mild and reversible side effects. ACTH is potentially an attractive therapeutic alternative for the treatment of frequently relapsing and steroid dependent nephrotic syndrome in children. Our study will randomly assign patients with frequently relapsing or steroid dependent nephrotic syndrome to either ACTH treatment or no treatment. This will allow us to study the effects of ACTH on this disease and its side effects, by comparing how patients do on ACTH treatment versus no treatment. We hypothesize that ACTH gel is superior to no treatment in maintaining remission in children with frequently relapsing or steroid dependent nephrotic syndrome.
|Condition or disease||Intervention/treatment||Phase|
|Nephrotic Syndrome||Drug: ACTH||Phase 3|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||32 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome|
|Study Start Date :||May 2014|
|Estimated Primary Completion Date :||October 2018|
|Estimated Study Completion Date :||October 2018|
Active Comparator: Adrenocorticotropic hormone (ACTH)
Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2
The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).
Patients will receive ACTH twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.
No Intervention: No treatment
Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study. There is an option for the patient to elect to be placed in the active treatment arm of the trial (rescue therapy).
- Relapse of nephrotic syndrome [ Time Frame: 6 months ]Relapse of nephrotic syndrome during the initial 6 months of the study.
- Relapses after dose reduction of ACTH [ Time Frame: 6 months ]The dose of ACTH will be reduced by 50% after 6 months and the rate of relapse during this period will be evaluated.
- Complications [ Time Frame: 6 months ]We will compare side effects during active treatment with ACTH versus no treatment.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02132195
|United States, Alabama|
|Pediatric Nephrology of Alabama, PC.|
|Birmingham, Alabama, United States, 35205|
|United States, California|
|University of California, Los Angeles|
|Los Angeles, California, United States, 90095|
|United States, Delaware|
|Nemours/AI duPont Hospital for Children|
|Wilmington, Delaware, United States, 19803|
|United States, Florida|
|Nemours Children's Hospital|
|Orlando, Florida, United States, 32827|
|United States, Georgia|
|Atlanta, Georgia, United States, 30322|
|United States, Indiana|
|Riley Hospital for Children|
|Indianapolis, Indiana, United States, 46202|
|United States, Massachusetts|
|Boston Children's Hopsital|
|Boston, Massachusetts, United States, 02115|
|United States, Michigan|
|Helen DeVos Children's Hospital at Spectrum Health|
|Grand Rapids, Michigan, United States, 49503|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|United States, Missouri|
|Kansas City, Missouri, United States, 64109|
|United States, New York|
|Children's Hospital at Montefiore|
|Bronx, New York, United States, 10467|
|United States, North Carolina|
|Duke Children's Health Center|
|Durham, North Carolina, United States, 27704|
|East Carolina University|
|Greenville, North Carolina, United States, 27834|
|United States, Texas|
|Driscoll Children's Hospital|
|Corpus Christi, Texas, United States, 78411|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|United States, Virginia|
|Children's Hospital of Richmond at VCU|
|Richmond, Virginia, United States, 23298|
|Principal Investigator:||Larry A Greenbaum, MD, PhD||Emory University|