Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
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|ClinicalTrials.gov Identifier: NCT01747135|
Recruitment Status : Completed
First Posted : December 11, 2012
Last Update Posted : April 19, 2017
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Information provided by (Responsible Party):
No Study Results Posted on ClinicalTrials.gov for this Study
|Recruitment Status :||Completed|
|Actual Primary Completion Date :||March 2017|
|Actual Study Completion Date :||March 2017|
Aqul A, Liu B, Ramirez CM, Pieper AA, Estill SJ, Burns DK, Liu B, Repa JJ, Turley SD, Dietschy JM. Unesterified cholesterol accumulation in late endosomes/lysosomes causes neurodegeneration and is prevented by driving cholesterol export from this compartment. J Neurosci. 2011 Jun 22;31(25):9404-13. doi: 10.1523/JNEUROSCI.1317-11.2011.
Brewster ME, Loftsson T. Cyclodextrins as pharmaceutical solubilizers. Adv Drug Deliv Rev. 2007 Jul 30;59(7):645-66. Epub 2007 May 29. Review.