This study is limited to patients with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, but has the potential to significantly improve the lives of patients with other forms of adrenal insufficiency. Each of these diseases requires life-long treatment and this novel approach to drug delivery has the potential to significantly improve patients lives. This pilot study is fully enrolled and is in follow-up only status and data analysis is underway. In addition, this study will provide information on the safety and tolerability of using a pump to deliver hydrocortisone, and will generate data that will be used in the design of future studies.

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Support Year
5
Fiscal Year
2017
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Indirect Cost
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U.S. National Inst/Child Hlth/Human Dev
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Mallappa, Ashwini; Nella, Aikaterini A; Sinaii, Ninet et al. (2018) Long-term use of continuous subcutaneous hydrocortisone infusion therapy in patients with congenital adrenal hyperplasia. Clin Endocrinol (Oxf) 89:399-407
Mallappa, Ashwini; Nella, Aikaterini A; Kumar, Parag et al. (2017) Alterations in Hydrocortisone Pharmacokinetics in a Patient With Congenital Adrenal Hyperplasia Following Bariatric Surgery. J Endocr Soc 1:994-1001
Nella, Aikaterini A; Mallappa, Ashwini; Perritt, Ashley F et al. (2016) A Phase 2 Study of Continuous Subcutaneous Hydrocortisone Infusion in Adults with Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab :jc20161916
El-Maouche, Diala; Collier, Suzanne; Prasad, Mala et al. (2015) Cortical bone mineral density in patients with congenital adrenal hyperplasia due to 21-hydroxylase deficiency. Clin Endocrinol (Oxf) 82:330-7
Merke, Deborah P; Chen, Wuyan; Morissette, Rachel et al. (2013) Tenascin-X haploinsufficiency associated with Ehlers-Danlos syndrome in patients with congenital adrenal hyperplasia. J Clin Endocrinol Metab 98:E379-87