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ACTHAR GEL for Sarcoidosis-Associated Calcium Dysregulation: An Open-label Pilot Study

Primary Purpose

Sarcoidosis, Hypercalcemia Due to Sarcoidosis

Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ACTHAR Gel (adrenocorticotropic hormone)
Sponsored by
Albany Medical College
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sarcoidosis focused on measuring Sarcoidosis-Associated Calcium Dysregulation, Sarcoidosis-Associated hypercalciuria, hypercalcemia, hypercalciuria, nephrocalcinosis, nephrolithiasis, interstitial nephritis, glomerulonephritis, acute kidney disease, chronic kidney disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Age greater than 18 years old.
  2. Able to understand English to the point of comprehending the informed consent form.
  3. Biopsy proven sarcoidosis.
  4. Documented hypercalciuria (urinary excretion of > 4mg/kg of calcium/day) or hypercalcemia within 4 weeks of study entry.
  5. Historical evidence that the patient's hypercalciuria/hypercalcemia is related to sarcoidosis. This should include a serum parathyroid hormone (PTH) level which is not elevated.

Exclusion Criteria:

  1. A change in anti-sarcoidosis medications within 3 months of study entry.
  2. A history of hyperparathyroidism or another non-sarcoidosis cause of hypercalcemia/hypercalciuria
  3. A history of Cushing's disease.
  4. Have a diagnosis of a medical disorder other than sarcoidosis that in the opinion of the investigator would complicate the evaluation of response treatment.
  5. Have used any investigational drug within 1 month prior to screening or within 5 half-lives of the investigational agent, whichever is longer.
  6. Use of loop or thiazide diuretics for hypertension or other disorders.
  7. Chronic use of antacids.

Sites / Locations

  • Albany Medical College

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Sarcoidosis related Calcium Dysregulation

Arm Description

Subjects with Sarcoidosis associated calcium dysregulation will be administered 80 units of Acthar Gel (adrenocorticotropic hormone) twice a week for 12 weeks. Clinical visits will be scheduled for -30 days, day of 1st dose and 4,8,12 and 16 week after 1st dose to monitor the health of subjects.

Outcomes

Primary Outcome Measures

Reduction of 24 hour urine calcium
Reduction of 24 hour urine calcium in patients with sarcoidosis associated hypercalciuria (primary endpoint) between week 0 and week 12.

Secondary Outcome Measures

Change in serum calcium during 12 week ACTHAR GEL treatment
Change in 1,25 di-hydroxy Vitamin D during 12 week ACTHAR GEL treatment
Change in patient global VAS during 12 week ACTHAR GEL treatment
Change in physician global VAS during 12 week ACTHAR GEL treatment
Change in urinary symptoms during 12 week ACTHAR GEL treatment
Change in Short Form-36 during 12 week ACTHAR GEL treatment
Change in Sarcoidosis Health Questionnaire during 12 week ACTHAR GEL treatment
Change in eCOST during 12 week ACTHAR gel treatment
Change in adjusted eCOST (eCOSTadj) calculated as the eCOST/# organs with an eCOST score > 022 during 12 week ACTHAR gel treatment

Full Information

First Posted
May 30, 2014
Last Updated
January 23, 2015
Sponsor
Albany Medical College
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1. Study Identification

Unique Protocol Identification Number
NCT02155803
Brief Title
ACTHAR GEL for Sarcoidosis-Associated Calcium Dysregulation: An Open-label Pilot Study
Official Title
ACTHAR GEL for Sarcoidosis-Associated Calcium Dysregulation: An Open-label Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
January 2015
Overall Recruitment Status
Unknown status
Study Start Date
February 2015 (undefined)
Primary Completion Date
June 2015 (Anticipated)
Study Completion Date
November 2015 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Albany Medical College

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
ACTHAR Gel has activity in sarcoidosis associated hypercalciuria and calcium dysregulation.
Detailed Description
Sarcoidosis is a multisystem granulomatous disease of unknown cause. Although sarcoidosis most commonly affects the lung, it may affect any organ. Although corticosteroids are recognized as the drug of choice for sarcoidosis. ACTH(adrenocorticotropic hormone) is the only drug that is FDA-approved for this disorder. However, there is limited data on the efficacy of ACTH for this condition. Calcium metabolism is disregulated in active sarcoidosis. The primary abnormality in calcium metabolism stems from an increased 1-α hydroxylase activity in sarcoid alveolar macrophages that converts 25-hydroxyvitamin D to 1, 25-dihydroxyvitamin D, the active form of the vitamin. This can result in hypercalcemia, hypercalciuria, nephrocalcinosis, nephrolithiasis, interstitial nephritis, glomerulonephritis, acute and chronic kidney disease. Importantly, almost of the renal manifestations stem from disordered calcium metabolism. Unlike other organ manifestations of sarcoidosis, the disorder of calcium metabolism is more common in whites compared to african americans.Compared to hypercalcemia, hypercalciuria is three times more common in sarcoidosis, nevertheless, it has largely been ignored. In general, the patient with hypercalcemia should be advised to avoid sunlight, curtail intake of major sources of dietary calcium and vitamin D, and drink ample fluids.If the patient is symptomatic, serum calcium is greater than 11 mg/dl, the serum creatinine is elevated, or the patient has nephrolithiasis, drug therapy is usually required. The drug of choice is prednisone at an initial daily dose of 20 - 40 mg/day.Unfortunately, prolonged corticosteroid therapy may result in unacceptable side effects including osteoporosis. This is particularly important as elevated calcitriol observed in patients with sarcoidosis can further jeopardize bone structure by resorption. Alternative medications that have shown benefit for sarcoidosis associated calcium dysregulation have included chloroquine,hydroxychloroquine, ketoconazole. Not only may ACTHER GEL have obvious anti-inflammatory effects by resulting in corticosteroid production, but it may also activate melanocortin receptors. The melanocortin system has powerful anti-inflammatory properties that may be beneficial in the treatment of sarcoidosis. We believe that there are several specific advantages of assessing the effectiveness of anti-sarcoidosis therapy by examining sarcoidosis-associated disorders of calcium metabolism. The measures of granulomatous activity (serum calcium, urinary calcium, serum 25-hydroxyvitamin D, and serum 1, 25-dihydroxyvitamin D levels) are directly related to the granulomatous inflammation of sarcoidosis. These parameters can be accurately and objectively quantified. This is an important issue in sarcoidosis as the endpoint for involvement of the lungs, skin, and eyes is problematic because it is either inexact and/or not unidimensional. These constituents can be easily used to clinically monitor sarcoidosis. This is not the case for other forms of sarcoidosis including involvement of the lung and skin. Although hypercalciuria and disordered calcium metabolism is not as common a manifestation of sarcoidosis as lung involvement, there is little evidence that the anti-granulomatous response to this disease is organ specific. In a randomized double-blind placebo control trial of infliximab for pulmonary sarcoidosis, extrapulmonary sarcoidosis also responded to this therapy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sarcoidosis, Hypercalcemia Due to Sarcoidosis
Keywords
Sarcoidosis-Associated Calcium Dysregulation, Sarcoidosis-Associated hypercalciuria, hypercalcemia, hypercalciuria, nephrocalcinosis, nephrolithiasis, interstitial nephritis, glomerulonephritis, acute kidney disease, chronic kidney disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Sarcoidosis related Calcium Dysregulation
Arm Type
Experimental
Arm Description
Subjects with Sarcoidosis associated calcium dysregulation will be administered 80 units of Acthar Gel (adrenocorticotropic hormone) twice a week for 12 weeks. Clinical visits will be scheduled for -30 days, day of 1st dose and 4,8,12 and 16 week after 1st dose to monitor the health of subjects.
Intervention Type
Drug
Intervention Name(s)
ACTHAR Gel (adrenocorticotropic hormone)
Intervention Description
ACTHAR GEL (adrenocorticotropic hormone) 80 units subcutaneously twice weekly for 12 weeks
Primary Outcome Measure Information:
Title
Reduction of 24 hour urine calcium
Description
Reduction of 24 hour urine calcium in patients with sarcoidosis associated hypercalciuria (primary endpoint) between week 0 and week 12.
Time Frame
Between week 0 and week 12.
Secondary Outcome Measure Information:
Title
Change in serum calcium during 12 week ACTHAR GEL treatment
Time Frame
Baseline compared to 12 weeks.
Title
Change in 1,25 di-hydroxy Vitamin D during 12 week ACTHAR GEL treatment
Time Frame
Baseline compared to 12 weeks.
Title
Change in patient global VAS during 12 week ACTHAR GEL treatment
Time Frame
Baseline to 12 weeks
Title
Change in physician global VAS during 12 week ACTHAR GEL treatment
Time Frame
Baseline to 12 Weeks
Title
Change in urinary symptoms during 12 week ACTHAR GEL treatment
Time Frame
Baseline to 12 Weeks
Title
Change in Short Form-36 during 12 week ACTHAR GEL treatment
Time Frame
Baseline to 12 Weeks
Title
Change in Sarcoidosis Health Questionnaire during 12 week ACTHAR GEL treatment
Time Frame
Baseline to 12 Weeks
Title
Change in eCOST during 12 week ACTHAR gel treatment
Time Frame
Baseline to 12 Weeks
Title
Change in adjusted eCOST (eCOSTadj) calculated as the eCOST/# organs with an eCOST score > 022 during 12 week ACTHAR gel treatment
Time Frame
Baseline to 12 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age greater than 18 years old. Able to understand English to the point of comprehending the informed consent form. Biopsy proven sarcoidosis. Documented hypercalciuria (urinary excretion of > 4mg/kg of calcium/day) or hypercalcemia within 4 weeks of study entry. Historical evidence that the patient's hypercalciuria/hypercalcemia is related to sarcoidosis. This should include a serum parathyroid hormone (PTH) level which is not elevated. Exclusion Criteria: A change in anti-sarcoidosis medications within 3 months of study entry. A history of hyperparathyroidism or another non-sarcoidosis cause of hypercalcemia/hypercalciuria A history of Cushing's disease. Have a diagnosis of a medical disorder other than sarcoidosis that in the opinion of the investigator would complicate the evaluation of response treatment. Have used any investigational drug within 1 month prior to screening or within 5 half-lives of the investigational agent, whichever is longer. Use of loop or thiazide diuretics for hypertension or other disorders. Chronic use of antacids.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Haroon Chaudhry, MBBS
Phone
518-262-1542
Email
chaudhh@mail.amc.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Marc A. Judson, MD
Phone
518-262-5196
Email
judsonm@mail.amc.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Marc A Judson, MD
Organizational Affiliation
Albany Medical College
Official's Role
Principal Investigator
Facility Information:
Facility Name
Albany Medical College
City
Albany
State/Province
New York
ZIP/Postal Code
12208
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Haroon Chaudhry, MBBS
Phone
518-262-1542
Email
chaudhh@mail.amc.edu
First Name & Middle Initial & Last Name & Degree
Marc A. Judson, MD

12. IPD Sharing Statement

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ACTHAR GEL for Sarcoidosis-Associated Calcium Dysregulation: An Open-label Pilot Study

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