Long-term Follow-up of Response to Treatment and Immune Tolerance Induction (ITI) in Pompe Disease
This study is currently recruiting participants.
Verified March 2013 by Duke University
Information provided by (Responsible Party):
First received: August 13, 2012
Last updated: March 1, 2013
Last verified: March 2013
No Study Results Posted on ClinicalTrials.gov for this Study
|Study Status:||This study is currently recruiting participants.|
|Estimated Study Completion Date:||August 2019|
|Estimated Primary Completion Date:||August 2019 (Final data collection date for primary outcome measure)|
El-Gharbawy AH, Mackey J, DeArmey S, Westby G, Grinnell SG, Malovrh P, Conway R, Kishnani PS. An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions. Mol Genet Metab. 2011 Sep-Oct;104(1-2):118-22. Epub 2011 Jul 13.
Banugaria SG, Prater SN, Ng YK, Kobori JA, Finkel RS, Ladda RL, Chen YT, Rosenberg AS, Kishnani PS. The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease. Genet Med. 2011 Aug;13(8):729-36.
Messinger YH, Mendelsohn NJ, Rhead W, Dimmock D, Hershkovitz E, Champion M, Jones SA, Olson R, White A, Wells C, Bali D, Case LE, Young SP, Rosenberg AS, Kishnani PS. Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease. Genet Med. 2012 Jan;14(1):135-42. doi: 10.1038/gim.2011.4.