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

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

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

Brief Summary: ACTHAR Gel has activity in sarcoidosis associated hypercalciuria and calcium dysregulation.

Detailed Summary:

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 struc
Sponsor: Albany Medical College

Current Primary Outcome: Reduction of 24 hour urine calcium [ Time Frame: Between week 0 and week 12. ]

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


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in serum calcium during 12 week ACTHAR GEL treatment [ Time Frame: Baseline compared to 12 weeks. ]
  • Change in 1,25 di-hydroxy Vitamin D during 12 week ACTHAR GEL treatment [ Time Frame: Baseline compared to 12 weeks. ]
  • Change in patient global VAS during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 weeks ]
  • Change in physician global VAS during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in urinary symptoms during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in Short Form-36 during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in Sarcoidosis Health Questionnaire during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in eCOST during 12 week ACTHAR gel treatment [ Time Frame: Baseline to 12 Weeks ]
  • 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 ]


Original Secondary Outcome:

  • Change in serum calcium during 12 week ACTHAR GEL treatment [ Time Frame: Baseline compared to 12 weeks. ]
  • Change in 1,25 di-OH Vitamin D during 12 week ACTHAR GEL treatment [ Time Frame: Baseline compared to 12 weeks. ]
  • Change in patient global VAS during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 weeks ]
  • Change in physician global VAS during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in urinary symptoms during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in SF-36 during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in SHQ during 12 week ACTHAR GEL treatment [ Time Frame: Baseline to 12 Weeks ]
  • Change in eCOST during 12 week ACTHAR gel treatment [ Time Frame: Baseline to 12 Weeks ]
  • 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 ]


Information By: Albany Medical College

Dates:
Date Received: May 30, 2014
Date Started: February 2015
Date Completion: November 2015
Last Updated: January 23, 2015
Last Verified: January 2015