Abstract

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 newly approved pilot study has enrolled one patient and is actively recruiting. 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 pediatric studies.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Investigator-Initiated Intramural Research Projects (ZIA)
Project #
1ZIAHD008932-02
Application #
8941564
Study Section
Project Start
Project End
Budget Start
Budget End
Support Year
2
Fiscal Year
2014
Total Cost
Indirect Cost

  Institution

Name
U.S. National Inst/Child Hlth/Human Dev
Department
Type
DUNS #
City
State
Country
Zip Code

  Publications

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