Clinical Trial: Compassionate Use of SOM230 for Hyperinsulinemic/Hypoglycemia

Study Status: Available
Recruit Status: Available
Study Type: Expanded Access

Official Title: Compassionate Use of SOM230 for Individual Patient (NS, 14-Jan-1986) With Hyperinsulinemic/Hypoglycemia

Brief Summary: Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, and such individuals may respond to a new somatostatin analog, pasireotide. This is a compassionate use study of the effects of pasireotide on individuals with suboptimally treated congenital hyperinsulinism.

Detailed Summary:

Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, with diazoxide as the mainstay of treatment. Individuals who are not adequately treated with diazoxide may undergo pancreatectomy. Octreotide has been used with some success in congenital hyperinsulinism but there is limited data on pasireotide, the newest somatostatin receptor agonist, which, compared to octreotide, has 30-40 times greater affinity for somatostatin receptors 1 (SSTR1) and 5 (SSTR5), 5 times greater for somatostatin receptor 3 (SSTR3) and a comparable affinity for somatostatin receptor 2 (SSTR2).

In human islets, SSTR2 & SSTR5 are present in beta cells. Therefore, there is reason to believe that pasireotide may have greater effectiveness than octreotide in preventing hypoglycemia due to hyperinsulinism. Indeed hyperglycemia is a known effect of pasireotide when used for treatment of Cushing's disease and Acromegaly. The current study is of compassionate use for prevention of hypoglycemia in individuals with congenital hyperinsulinemic hypoglycemia who are not adequately treated with diazoxide.

Sponsor: Montefiore Medical Center

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Information By: Montefiore Medical Center

Dates:
Date Received: July 13, 2016
Date Started:
Date Completion:
Last Updated: July 13, 2016
Last Verified: May 2016