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National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities

Eight manufacturing facilities participating in the National Institutes of Health–sponsored Clinical Islet Transplantation (CIT) Consortium jointly developed and implemented a harmonized process for the manufacture of allogeneic purified human pancreatic islet (PHPI) product evaluated in a phase 3 t...

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Autores principales: Ricordi, Camillo, Goldstein, Julia S., Balamurugan, A.N., Szot, Gregory L., Kin, Tatsuya, Liu, Chengyang, Czarniecki, Christine W., Barbaro, Barbara, Bridges, Nancy D., Cano, Jose, Clarke, William R., Eggerman, Thomas L., Hunsicker, Lawrence G., Kaufman, Dixon B., Khan, Aisha, Lafontant, David-Erick, Linetsky, Elina, Luo, Xunrong, Markmann, James F., Naji, Ali, Korsgren, Olle, Oberholzer, Jose, Turgeon, Nicole A., Brandhorst, Daniel, Chen, Xiaojuan, Friberg, Andrew S., Lei, Ji, Wang, Ling-jia, Wilhelm, Joshua J., Willits, Jamie, Zhang, Xiaomin, Hering, Bernhard J., Posselt, Andrew M., Stock, Peter G., Shapiro, A.M. James
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Diabetes Association 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5079635/
https://www.ncbi.nlm.nih.gov/pubmed/27465220
http://dx.doi.org/10.2337/db16-0234
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author Ricordi, Camillo
Goldstein, Julia S.
Balamurugan, A.N.
Szot, Gregory L.
Kin, Tatsuya
Liu, Chengyang
Czarniecki, Christine W.
Barbaro, Barbara
Bridges, Nancy D.
Cano, Jose
Clarke, William R.
Eggerman, Thomas L.
Hunsicker, Lawrence G.
Kaufman, Dixon B.
Khan, Aisha
Lafontant, David-Erick
Linetsky, Elina
Luo, Xunrong
Markmann, James F.
Naji, Ali
Korsgren, Olle
Oberholzer, Jose
Turgeon, Nicole A.
Brandhorst, Daniel
Chen, Xiaojuan
Friberg, Andrew S.
Lei, Ji
Wang, Ling-jia
Wilhelm, Joshua J.
Willits, Jamie
Zhang, Xiaomin
Hering, Bernhard J.
Posselt, Andrew M.
Stock, Peter G.
Shapiro, A.M. James
author_facet Ricordi, Camillo
Goldstein, Julia S.
Balamurugan, A.N.
Szot, Gregory L.
Kin, Tatsuya
Liu, Chengyang
Czarniecki, Christine W.
Barbaro, Barbara
Bridges, Nancy D.
Cano, Jose
Clarke, William R.
Eggerman, Thomas L.
Hunsicker, Lawrence G.
Kaufman, Dixon B.
Khan, Aisha
Lafontant, David-Erick
Linetsky, Elina
Luo, Xunrong
Markmann, James F.
Naji, Ali
Korsgren, Olle
Oberholzer, Jose
Turgeon, Nicole A.
Brandhorst, Daniel
Chen, Xiaojuan
Friberg, Andrew S.
Lei, Ji
Wang, Ling-jia
Wilhelm, Joshua J.
Willits, Jamie
Zhang, Xiaomin
Hering, Bernhard J.
Posselt, Andrew M.
Stock, Peter G.
Shapiro, A.M. James
author_sort Ricordi, Camillo
collection PubMed
description Eight manufacturing facilities participating in the National Institutes of Health–sponsored Clinical Islet Transplantation (CIT) Consortium jointly developed and implemented a harmonized process for the manufacture of allogeneic purified human pancreatic islet (PHPI) product evaluated in a phase 3 trial in subjects with type 1 diabetes. Manufacturing was controlled by a common master production batch record, standard operating procedures that included acceptance criteria for deceased donor organ pancreata and critical raw materials, PHPI product specifications, certificate of analysis, and test methods. The process was compliant with Current Good Manufacturing Practices and Current Good Tissue Practices. This report describes the manufacturing process for 75 PHPI clinical lots and summarizes the results, including lot release. The results demonstrate the feasibility of implementing a harmonized process at multiple facilities for the manufacture of a complex cellular product. The quality systems and regulatory and operational strategies developed by the CIT Consortium yielded product lots that met the prespecified characteristics of safety, purity, potency, and identity and were successfully transplanted into 48 subjects. No adverse events attributable to the product and no cases of primary nonfunction were observed.
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spelling pubmed-50796352017-11-01 National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities Ricordi, Camillo Goldstein, Julia S. Balamurugan, A.N. Szot, Gregory L. Kin, Tatsuya Liu, Chengyang Czarniecki, Christine W. Barbaro, Barbara Bridges, Nancy D. Cano, Jose Clarke, William R. Eggerman, Thomas L. Hunsicker, Lawrence G. Kaufman, Dixon B. Khan, Aisha Lafontant, David-Erick Linetsky, Elina Luo, Xunrong Markmann, James F. Naji, Ali Korsgren, Olle Oberholzer, Jose Turgeon, Nicole A. Brandhorst, Daniel Chen, Xiaojuan Friberg, Andrew S. Lei, Ji Wang, Ling-jia Wilhelm, Joshua J. Willits, Jamie Zhang, Xiaomin Hering, Bernhard J. Posselt, Andrew M. Stock, Peter G. Shapiro, A.M. James Diabetes Islet Studies Eight manufacturing facilities participating in the National Institutes of Health–sponsored Clinical Islet Transplantation (CIT) Consortium jointly developed and implemented a harmonized process for the manufacture of allogeneic purified human pancreatic islet (PHPI) product evaluated in a phase 3 trial in subjects with type 1 diabetes. Manufacturing was controlled by a common master production batch record, standard operating procedures that included acceptance criteria for deceased donor organ pancreata and critical raw materials, PHPI product specifications, certificate of analysis, and test methods. The process was compliant with Current Good Manufacturing Practices and Current Good Tissue Practices. This report describes the manufacturing process for 75 PHPI clinical lots and summarizes the results, including lot release. The results demonstrate the feasibility of implementing a harmonized process at multiple facilities for the manufacture of a complex cellular product. The quality systems and regulatory and operational strategies developed by the CIT Consortium yielded product lots that met the prespecified characteristics of safety, purity, potency, and identity and were successfully transplanted into 48 subjects. No adverse events attributable to the product and no cases of primary nonfunction were observed. American Diabetes Association 2016-11 2016-07-27 /pmc/articles/PMC5079635/ /pubmed/27465220 http://dx.doi.org/10.2337/db16-0234 Text en © 2016 by the American Diabetes Association. http://www.diabetesjournals.org/content/licenseReaders may use this article as long as the work is properly cited, the use is educational and not for profit, and the work is not altered. More information is available at http://www.diabetesjournals.org/content/license.
spellingShingle Islet Studies
Ricordi, Camillo
Goldstein, Julia S.
Balamurugan, A.N.
Szot, Gregory L.
Kin, Tatsuya
Liu, Chengyang
Czarniecki, Christine W.
Barbaro, Barbara
Bridges, Nancy D.
Cano, Jose
Clarke, William R.
Eggerman, Thomas L.
Hunsicker, Lawrence G.
Kaufman, Dixon B.
Khan, Aisha
Lafontant, David-Erick
Linetsky, Elina
Luo, Xunrong
Markmann, James F.
Naji, Ali
Korsgren, Olle
Oberholzer, Jose
Turgeon, Nicole A.
Brandhorst, Daniel
Chen, Xiaojuan
Friberg, Andrew S.
Lei, Ji
Wang, Ling-jia
Wilhelm, Joshua J.
Willits, Jamie
Zhang, Xiaomin
Hering, Bernhard J.
Posselt, Andrew M.
Stock, Peter G.
Shapiro, A.M. James
National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities
title National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities
title_full National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities
title_fullStr National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities
title_full_unstemmed National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities
title_short National Institutes of Health–Sponsored Clinical Islet Transplantation Consortium Phase 3 Trial: Manufacture of a Complex Cellular Product at Eight Processing Facilities
title_sort national institutes of health–sponsored clinical islet transplantation consortium phase 3 trial: manufacture of a complex cellular product at eight processing facilities
topic Islet Studies
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5079635/
https://www.ncbi.nlm.nih.gov/pubmed/27465220
http://dx.doi.org/10.2337/db16-0234
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