Abstract

Cystic Fibrosis (CF) is a fatal disease caused by a defective gene that encodes for CF transmembrane conductance regulator (CFTR), a glycoprotein important in chloride transport. Approximately 75% of CF patients carry a deletion of phenylalanine from position 508 of the coding sequence for CFTR. This mutant CFTR is incompletely processed within the endoplasmic reticulum with limited transport of nonfunctinal CFTR to the airway epithelial surface, thereby causing defective chloride secretion. """"""""Protein assist"""""""" therapy, such as CPX is a new approach to activate chloride secretion via the mutant protein channels. CPX binds with high specificity and affinity to the first nucleotide binding domain of CFTR and restores function to mutant CFTR molecules in vitro. This Phase II study will evaluate the safety and efficacy of multiple ascending oral doses of oral CPX in adults with CF.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000037-41
Application #
6416884
Study Section
Project Start
2000-12-01
Project End
2001-11-30
Budget Start
Budget End
Support Year
41
Fiscal Year
2001
Total Cost
Indirect Cost

  Institution

Name
University of Washington
Department
Type
DUNS #
135646524
City
Seattle
State
WA
Country
United States
Zip Code
98195

  Publications

Courcoulas, Anita P; King, Wendy C; Belle, Steven H et al. (2018) Seven-Year Weight Trajectories and Health Outcomes in the Longitudinal Assessment of Bariatric Surgery (LABS) Study. JAMA Surg 153:427-434
Field, Alison E; Inge, Thomas H; Belle, Steven H et al. (2018) Association of Obesity Subtypes in the Longitudinal Assessment of Bariatric Surgery Study and 3-Year Postoperative Weight Change. Obesity (Silver Spring) 26:1931-1937
O'Rourke, Robert W; Johnson, Geoffrey S; Purnell, Jonathan Q et al. (2018) Serum biomarkers of inflammation and adiposity in the LABS cohort: associations with metabolic disease and surgical outcomes. Int J Obes (Lond) :
Cherrier, M M; Cross, D J; Higano, C S et al. (2018) Changes in cerebral metabolic activity in men undergoing androgen deprivation therapy for non-metastatic prostate cancer. Prostate Cancer Prostatic Dis 21:394-402
Duggan, Catherine; Baumgartner, Richard N; Baumgartner, Kathy B et al. (2018) Genetic variation in TNF?, PPAR?, and IRS-1 genes, and their association with breast-cancer survival in the HEAL cohort. Breast Cancer Res Treat 168:567-576
Han, Seung Jin; Boyko, Edward J; Kim, Soo Kyung et al. (2018) Association of Thigh Muscle Mass with Insulin Resistance and Incident Type 2 Diabetes Mellitus in Japanese Americans. Diabetes Metab J 42:488-495
Wander, Pandora L; Hayashi, Tomoshige; Sato, Kyoko Kogawa et al. (2018) Design and validation of a novel estimator of visceral adipose tissue area and comparison to existing adiposity surrogates. J Diabetes Complications 32:1062-1067
Purnell, Jonathan Q; Johnson, Geoffrey S; Wahed, Abdus S et al. (2018) Prospective evaluation of insulin and incretin dynamics in obese adults with and without diabetes for 2 years after Roux-en-Y gastric bypass. Diabetologia 61:1142-1154
King, Wendy C; Hinerman, Amanda S; Belle, Steven H et al. (2018) Comparison of the Performance of Common Measures of Weight Regain After Bariatric Surgery for Association With Clinical Outcomes. JAMA 320:1560-1569
Han, Seung Jin; Fujimoto, Wilfred Y; Kahn, Steven E et al. (2018) Change in visceral adiposity is an independent predictor of future arterial pulse pressure. J Hypertens 36:299-305

Showing the most recent 10 out of 563 publications