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ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children (ADRENL)

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ClinicalTrials.gov Identifier: NCT03408405
Recruitment Status : Not yet recruiting
First Posted : January 24, 2018
Last Update Posted : January 25, 2018
Sponsor:
Collaborators:
Mallinckrodt
University of Minnesota, MN
Medalytics
Information provided by (Responsible Party):
Mohammed Khurram Faizan, Rhode Island Hospital

Brief Summary:
We propose to study the use of purified porcine Acthar Gel (ACTHAR, Mallinckrodt Pharmaceuticals) for treatment of steroid resistance nephrotic syndrome (SRNS) in a prospective pilot study. We plan to enroll 25 children between the ages of 2 to 21 years. Children fulfilling strict inclusion criteria, whose parents agree to written informed consent after institutional IRB approval for the study, will be enrolled. Purified porcine Acthar Gel will be administered SQ to all children using a defined treatment protocol for a period of six months. Renal function, urine protein excretion, serum albumin levels, blood pressure and growth parameters will be monitored closely on all patients. Baseline urine protein excretion will be compared to end of treatment levels to determine successful response to therapy. There will be an 18 month enrollment period, 6 month treatment period and a 12 month follow-up period.

Condition or disease Intervention/treatment Phase
Steroid-Resistant Nephrotic Syndrome Drug: Acthar Gel 80 UNT/ML Injectable Solution Phase 4

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Sequential Assignment
Intervention Model Description:

Inclusion Criteria:

  1. Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL
  2. Calculated GFR (eGFR) using modified Schwartz formula of > 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL)
  3. A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study.
  4. Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: ADRENL - ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children, Pilot Study
Estimated Study Start Date : January 2018
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : July 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Acthar Gel treatment group
Participants will be treated with 'Acthar Gel 80 UNT/ML Injectable Solution'. Initial dose for week 1 will be 50% of 80 units, injected twice per week. Week 2 is 75% of 80 units, week 3 and throughout treatment period (6 months in total) will be 80 units/ml twice per week.
Drug: Acthar Gel 80 UNT/ML Injectable Solution
Participant will self inject 'Acthar Gel 80 UNT/ML Injectable Solution' 2 x per week for six months.
Other Name: H.P. Acthar gel




Primary Outcome Measures :
  1. Remission of proteinuria [ Time Frame: 18 months ]

    Partial remission: Greater than or equal to 50% reduction in proteinuria when compared to baseline.

    Complete remission: Greater than or equal to 90% reduction in proteinuria when compared to baseline or Urine Protein Creatinine ratio less than 0.5.



Secondary Outcome Measures :
  1. Blood pressure [ Time Frame: 18 months ]
    Blood pressure during study visit as measured in mm/Hg

  2. Serum Creatinine [ Time Frame: 18 months ]
    Serum value in mg/dL

  3. BMI [ Time Frame: 18 months ]
    Weight (kgs) and height (cms) will be combined to report BMI in kg/m^2

  4. Serum Glucose [ Time Frame: 18 months ]
    Serum value in mg/dL

  5. Serum Lipids [ Time Frame: 18 months ]
    Serum Value in mg/dL

  6. Quality of life measures obtained via survey [ Time Frame: 18 months ]
    PedsQL 3.0 will be used as the standardized survey tool



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Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL
  2. Calculated GFR (eGFR) using modified Schwartz formula of > 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL)
  3. A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study.
  4. Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy

Exclusion Criteria:

  1. Patients with an inherited or genetic disorder presenting with nephrotic syndrome (eg: NPHS 1 & 2 defects, WT-1 mutations, α actinin 4 mutation, TRP-6 mutation).
  2. Presence of diabetes or severe (stage 2) uncontrolled hypertension.
  3. Any metabolic condition that specifically precludes the use of Acthar Gel for treatment.
  4. Pregnancy or unwilling to agree to contraception which may include abstinence.
  5. eGFR <50 mls/min/m2

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): NCT03408405


Contacts
Contact: Mohammed K Faizan, MD 401-444-5672 mfaizan@lifespan.org
Contact: Evelyn Tolbert, MS 401-444-4566 ADRENLstudyinfo@Lifespan.org

Sponsors and Collaborators
Rhode Island Hospital
Mallinckrodt
University of Minnesota, MN
Medalytics
Investigators
Principal Investigator: Mohammed K Faizan, MD Rhode Island Hospital

Publications:

Responsible Party: Mohammed Khurram Faizan, Principal Investigator, Rhode Island Hospital
ClinicalTrials.gov Identifier: NCT03408405     History of Changes
Other Study ID Numbers: 1050945-5
First Posted: January 24, 2018    Key Record Dates
Last Update Posted: January 25, 2018
Last Verified: January 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
Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:
Syndrome
Nephrotic Syndrome
Nephrosis
Disease
Pathologic Processes
Kidney Diseases
Urologic Diseases
Pharmaceutical Solutions
Adrenocorticotropic Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs