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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
Sponsor:
Collaborator:
Mallinckrodt
Information provided by (Responsible Party):
Larry Greenbaum, MD, PhD, Emory University

Brief Summary:

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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hormones

Arm Intervention/treatment
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).

Drug: ACTH
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.
Other Names:
  • Acthar
  • Adrenocorticotropic hormone

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).



Primary Outcome Measures :
  1. Relapse of nephrotic syndrome [ Time Frame: 6 months ]
    Relapse of nephrotic syndrome during the initial 6 months of the study.


Secondary Outcome Measures :
  1. 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.

  2. Complications [ Time Frame: 6 months ]
    We will compare side effects during active treatment with ACTH versus no treatment.



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Ages Eligible for Study:   2 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age >1 year at onset of nephrotic syndrome
  2. Age 2-20 years at time of randomization
  3. Estimated glomerular filtration rate (GFR) > 50 ml/min/1.73 m2 at most recent measure prior to randomization (Schwartz formula)
  4. Steroid responsive nephrotic syndrome throughout clinical course (never required a second agent to attain remission of a relapse of nephrotic syndrome)
  5. History of frequently relapsing or steroid dependent nephrotic syndrome (defined as 2 or more relapses within 6 months after initial therapy or 4 or more relapses in any 12 month period OR relapse during taper or within 2 weeks of discontinuing prednisone).
  6. Patient is currently in relapse of nephrotic syndrome or had a relapse within the last 4 months (defined as an increase in the first morning urine protein to creatinine ratio ≥2 or Albustix reading of ≥2 for 3 or 5 consecutive days).

Exclusion Criteria:

  1. Prior treatment with ACTH.
  2. Cyclophosphamide or rituximab within the last 4 months.
  3. Lactation, pregnancy, or refusal of birth control in females with child-bearing potential
  4. Planned treatment with live or live-attenuated vaccines once enrolled in the study.
  5. Participation in another therapeutic trial concurrently or 30 days prior to randomization
  6. Active/serious infection (including, but not limited to Hepatitis B or C, HIV)
  7. Malignancy concurrently or within the last 2 years.
  8. Blood pressure >95% for age/height while receiving maximal doses of 3 or more medications.
  9. Prior diagnosis of diabetes mellitus (Type I or II) or fasting glucose >200mg/dL
  10. Organ transplantation
  11. Contraindications to Acthar: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction
  12. Secondary cause of nephrotic syndrome (e.g., SLE)
  13. Biopsy demonstrating a diagnosis other than minimal change, focal segmental glomerulosclerosis (FSGS) or a variant (mesangial proliferation, Immunoglobulin M nephropathy)
  14. Inability to consent/assent -

Information from the National Library of Medicine

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


Locations
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
Emory University
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
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Missouri
Children's Mercy
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
Sponsors and Collaborators
Emory University
Mallinckrodt
Investigators
Principal Investigator: Larry A Greenbaum, MD, PhD Emory University

Responsible Party: Larry Greenbaum, MD, PhD, Principal Investigator, Emory University
ClinicalTrials.gov Identifier: NCT02132195     History of Changes
Other Study ID Numbers: IRB00068101
EmoryPedNeph-002 ( Other Identifier: Emory Division of Pediatric Nephrology )
First Posted: May 7, 2014    Key Record Dates
Last Update Posted: May 1, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Syndrome
Nephrotic Syndrome
Nephrosis
Disease
Pathologic Processes
Kidney Diseases
Urologic Diseases
Hormones
Adrenocorticotropic Hormone
Melanocyte-Stimulating Hormones
beta-Endorphin
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action