Characterisation of cysteamine prodrugs for the treatment of cystinosis and evaluation of liquid fill technology.

McCormick, John James

Abstract

Cystinosis is a rare, autosomal, recessive disease characterised by raised levels of the amino acid cystine in the cells of most organs in the body which can cause organ damage. The treatment involves the oral administration of the aminothiol, cysteamine (Cystagon(TM)), but this has an offensive taste and smell, can cause vomiting and its metabolites are excreted from the body via breath (halitosis) and sweat, which can cause poor patient compliance. The main research aim was to characterise two cysteamine prodrugs for the treatment of cystinosis and evaluate the suitability of liquid fill technology using prodrugs, or alternative drug models, to determine excipient usage using solubility, stability and capsule shell compatibility data to potentially develop prototype formulations based on liquid fill technology.

Citation

MCCORMICK, J.J. 2016. Characterisation of cysteamine prodrugs for the treatment of cystinosis and evaluation of liquid fill technology. Robert Gordon University, MRes thesis.

• Thesis Type: Thesis
• Deposit Date: Feb 1, 2017
• Publicly Available Date: Feb 1, 2017
• Keywords: Cystinosis; Formulation; Capsules; Encapsulation; Modified release; Gelucire; Liquid fill technology
• Public URL: http://hdl.handle.net/10059/2138
• Award Date: May 31, 2016