The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes - Chapter 1: Key ethical requirements and progress toward the definition of an international regulatory framework

Xenotransplantation

Volumn 16, Issue 4, 2009, Pages 203-214

  Cozzi, Emanuele ^a,b,c   Tallacchini, Mariachiara ^d   Flanagan, Enda B ^e   Pierson Iii, Richard N ^f   Sykes, Megan ^g   Vanderpool, Harold Y ^h  

^a UNIVERSITY HOSPITAL OF PADOVA   (Italy)

^b Clinica Chirurgica II   (Italy)

^c Consortium for Research in Organ Transplantation ^*  (Italy)

^d UNIVERSITÀ CATTOLICA DEL SACRO CUORE   (Italy)

^e UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^f UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE   (United States)

^g MASSACHUSETTS GENERAL HOSPITAL   (United States)

^h UNIVERSITY OF TEXAS MEDICAL BRANCH   (United States)

Author keywords

Ethics; Islets of Langerhans; Regulation; Xenotransplantation

Indexed keywords

ANIMAL WELFARE; BREEDING; CLINICAL RESEARCH; CONSENSUS; HUMAN; INSULIN DEPENDENT DIABETES MELLITUS; NEW ZEALAND; NONHUMAN; PANCREAS ISLET TRANSPLANTATION; PRIORITY JOURNAL; RESEARCH ETHICS; REVIEW; RISK BENEFIT ANALYSIS; SWINE; WORLD HEALTH ORGANIZATION; XENOTRANSPLANTATION;

ANIMALS; CLINICAL TRIALS AS TOPIC; EUROPE; HUMANS; INFORMED CONSENT; INTERNATIONAL COOPERATION; ISLETS OF LANGERHANS TRANSPLANTATION; PATIENT SELECTION; SWINE; TRANSPLANTATION, HETEROLOGOUS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 70349597651     PISSN: 0908665X     EISSN: 13993089     Source Type: Journal    
DOI: 10.1111/j.1399-3089.2009.00540.x     Document Type: Review

References (53)

1. Hering BJ, Wijkstrom M, Graham ML et al. Prolonged diabetes reversal after intraportal xenotransplantation of wild-type porcine islets in immunosuppressed nonhuman primates. Nat Med 2006 12 : 301 303.
2. Cardona K, Korbutt GS, Milas Z et al. Long-term survival of neonatal porcine islets in nonhuman primates by targeting costimulation pathways. Nat Med 2006 12 : 304 306.
3. Gianello P, Dufrane D. Encapsulation of pig islets by alginate matrix to correct streptozotocin-induced diabetes in primates without immunosuppression. Xenotransplantation 2007 14 : 441.
4. Cryer PE. The barrier of hypoglycemia in diabetes. Diabetes 2008 57 : 3169 3176.
5. Fishman JA, Patience C. Xenotransplantation: infectious risk revisited. Am J Transplant 2004 4 : 1383 1390.
6. Sykes M, d'Apice A, Sandrin M, IXA Ethics Committee. Position paper of the Ethics Committee of the International Xenotransplantation Association. Xenotransplantation 2003 10 : 194 203.
7. Spillman MA, Sade RM. Clinical trials of xenotransplantation: waiver of the right to withdraw from a clinical trial should be required. J Law Med Ethics 2007 35 : 265 272.
8. Vanderpool HY. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes - Chapter 7: informed consent and xenotransplantation clinical trials. Xenotransplantation 2009 16 : 255 262.
9. Eckhoff DE. Selection of candidates for islet xenotransplantation. Xenotransplantation 2008 15 : 102 103.
10. O'Connell PJ. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes - Chapter 6: patient selection for pilot clinical trials of islet xenotransplantation. Xenotransplantation 2009 16 : 249 254.
11. U.S. Food and Drug Administration. Public Health Service Guideline on Infectious Disease Issues in Xenotransplantation. 2001. Available at: http://www.fda.gov/downloads/BiologicsBloodVaccines/ GuidanceComplianceRegulatoryInformation/Guidances/Xenotransplantation/UCM092858. pdf (accessed on 18 July 2009).
12. U.S. Food and Drug Administration. Guidance for Industry: Source Animal, Product, Preclinical, and Clinical Issues Concerning the use of Xenotransplantation Products in Humans. 2003. Available at: http://www.fda.gov/ BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/ Xenotransplantation/ucm074354.htm (accessed on 18 July 2009).
13. U.S. Food and Drug Administration. Guidance for Industry: Public Health Issues Posed by the Use of Non-Human Primate Xenografts in Humans. 1999. 64 FR 16743-16744. Available at: http://www.fda.gov/downloads/BiologicsBloodVaccines/ GuidanceComplianceRegulatoryInformation/Guidances/Xenotransplantation/ucm092866. pdf (accessed on 18 July 2009).
14. Allan JS, Broussard SR, Michaels MG et al. Amplification of simian retroviral sequences from human recipients of baboon liver transplants. AIDS Res Hum Retroviruses 1998 14 : 821 824.
15. Council of Europe. Parliamentary Assembly Recommendation 1399(1999) on Xenotransplantation of 29 January 1999. Available at: http://assembly.coe.int/ Documents/AdoptedText/ta99/erec1399.htm (accessed on 18 July 2009).
16. Council of Europe, Committee of Ministers. Recommendation Rec(2003)10 of the Committee of Ministers to Member States on Xenotransplantation. 2003. Available at: https://wcd.coe.int/ViewDoc.jsp?id=45827&BackColorInternet= 9999CC&BackColorIntranet=FFBB55&BackColorLogged=FFAC75 (accessed on 21 July 2009).
17. Directive 2001/18/EC of the European Parliament and of the Council of 12 March 2001 on the Deliberate Release into the Environment of Genetically Modified Organisms and Repealing Council Directive 90/220/EEC - Commission Declaration. Available at: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do? uri=CELEX:32001L0018:EN:HTML (accessed on 19 August 2009).
18. Directive 2001/20/EC of the European Parliament and of the Council of 4 April 2001 on the Approximation of the Laws, Regulations and Administrative Provisions of the Member States Relating to the Implementation of Good Clinical Practice in the Conduct of Clinical Trials on Medicinal Products for Human use. Available at: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=CELEX: 32001L0020:EN:HTML (accessed on 19 August 2009).
19. Statutory Instrument 2004 No. 1031. The Medicines for Human Use (Clinical Trials) Regulations 2004. Available at: http://www.opsi.gov.uk/si/si2004/ 20041031.htm (accessed on 19 August 2009).
20. Department of Health. Gene Therapy Advisory Committee (GTAC). Available at: http://www.dh.gov.uk/ab/GTAC/index.htm (accessed on 19 August 2009).
21. Medicinal Products Act (The Drug Law) (ARZNEIMITTELGESETZ - AMG) of the Federal Republic of Germany (Non-Official Translation). 2007. Available at: http://www.bmg.bund.de/cln-117/nn-1200354/SharedDocs/Downloads/EN/health/ AMG-pdf,templateId=raw,property=publicationFile.pdf/AMG-pdf.pdf (accessed on 19 August 2009).
22. The European Parliament and the Council of the European Union. Directive 2004/23/EC of the European Parliament and of the Council of 31 March 2004 on Setting Standards of Quality and Safety for the Donation, Procurement, Testing, Processing, Preservation, Storage and Distribution of Human Tissues and Cells. 2004. Available at: http://eurex.europa.eu/smartapi/cgi/sga-doc? smartapi!celexapi!prod!CELEXnumdoc&lg=en&numdoc=32004L0023&model= guichett (accessed on 21 July 2009).
23. The European Parliament and the Council of the European Union. Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on Advanced Therapy Medicinal Products and Amending Directive 2001/83/EC and Regulation (EC) No 726/2004. 2007. Available at: http://ec.europa.eu/ enterprise/pharmaceuticals/eudralex/vol-1/reg-2007-1394/reg-2007-1394-en.pdf (accessed on 19 August 2009).
24. Draft of the Commission Directive Amending Directive 2001/83/EC of the European Parliament and of the Council on the Community Code Relating to Medicinal Products for Human Use as Regards Advanced Therapy Medicinal Products. 2009. Available at: http://ec.europa.eu/enterprise/pharmaceuticals/pharmacos/ docs/doc2009/2009-04/2009-03-10-1-en-act-v7.pdf (accessed on 24 August 2009).
25. European Commission. Implementation of the Advanced Therapies Regulation. Regulation (EC) No. 1394/2007. Public Consultation Paper, Draft Amendments to the Clinical Trial Application Form as Regards Advanced Therapy Medicinal Products, Version 22 July 2008, Deadline for Public Consultation 15 October 2008. 2008. Available at: http://ec.europa.eu/enterprise/pharmaceuticals/ advtherapies/docs/consultation-paper-2008-07-22.pdf (accessed on 19 August 2009).
26. European Commission. Implementation of the 'Advanced Therapies' Regulation (EC) No 1394/2007, Public Consultation Paper Draft Detailed Guideline on Good Clinical Practice Specific to Advanced Therapy Medicinal Products Version: 2 July 2008 Deadline for Public Consultation: 15 October 2008. 2008. Available at: http://ec.europa.eu/enterprise/pharmaceuticals/advtherapies/docs/ 2008-07/Consultation%20paper-NR-2008-07-02.pdf (accessed on 19 August 2009).
27. European Medicines Agency. Committee for Human Medicinal Products. Concept Paper on the Revision of the Points to Consider on Xenogeneic Cell Therapy Medicinal Products (EMEA/CHMP/165085/2007) London. 2007. Available at: http://www.emea.europa.eu/pdfs/human/cpwp/16508507en.pdf (accessed on 21 July 2009).
28. Council of Europe, Committee of Ministers. Recommendation R(97)15 of the Committee of Ministers to Member States on Xenotransplantation. (Adopted by the Committee of Ministers on 30 September 1997 at the 602nd meeting of the Ministers' Deputies). Available at: http://www.coe.int/t/dg3/healthbioethic/ texts-and-documents/Rec(97)15E.pdf (accessed on 19 August 2009).
29. Commission Directive 2005/28/EC of 8 April 2005 Laying Down Principles and Detailed Guidelines for Good Clinical Practice as Regards Investigational Medicinal Products for Human use, as Well as the Requirements for Authorisation of the Manufacturing or Importation of Such Products (Text with EEA Relevance). Available at: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=CELEX: 32005L0028:EN:HTML (accessed on 19 August 2009).
30. European Commission. Implementing Texts for Directive 2001/20/EC. Available at: http://ec.europa.eu/enterprise/pharmaceuticals/pharmacos/ dir200120ec.htm (accessed 19 August 2009).
31. Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community Code Relating to Medicinal Products for Human use (Consolided Version: 30/12/2008 OJ L 311, 28.11.2001, p. 67). Available at: http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-1/dir-2001-83-cons/ dir2001-83-cons-20081230-en.pdf (accessed on 19 August 2009).
32. The Medicines Act (Arzneimittelgesetz) 1983, as Amended in 2004, Implementing the Directive 2001/20/EC. Available at: http:www.ris.bka.gv.at/ Ergebnis.wxe ?Abfrage=Bundesnormen&Kundmachungsorgan=&Index=&Titel= AMG&Gesetzesnummer=&VonArtikel=&BisArtikel=&VonParagraf= &BisParagraf=&VonAnlage=&BisAnlage=&Typ=&Kundmachungsnummer= &Unterzeichnungsdatum=&FassungVom=12.08.2009&ImRisSeit= Undefined&ResultPageSize=100&Suchworte=&WxeFunctionToken= 03917e14-4913-49a0-97a7-5df2a11dce3a (accessed on 24 August 2009).
33. Austrian Federal Office for Safety in Health Care. Austrian Medicines and Medical Devices Agency. Clinical Trials (CT). Application for the Conduct of a Clinical Trial According to § 40 AMG (Arzneimittelgesetz = Austrian Medicinal Product Act, as Amended). Available at: http://www.basg.at/uploads/ media/L-W05-Guidance-CT-submission.pdf (accessed on 19 August 2009).
34. Loi no. 2004-800 du 6 août 2004 relative à la bioéthique. Available at: http://www.droit.org/jo/20040807/SANX0100053L. html (accessed on 19 August 2009).
35. Act XCV of 2005 on Medicinal Products for Human Use and on the Amendment of Other Regulations Related to Medicinal Products. Available at: http://net.jogtar.hu/jr/gen/getdoc.cgi?docid=a0500095.tv&dbnum=62 (accessed on 19 August 2009).
36. Ley 29/2006, de 26 de julio, de garantías y uso racional de los medicamentos y productos sanitarios. Available at: http://www.060.es/te- ayudamos-a/legislacion/disposiciones/35257-LEG-ides-idimp.html (accessed on 19 August 2009).
37. Law of 16 May 2002 (Stb. 263) Amending the Law on Special Medical Procedures [Stb. 1997, 515, as Last Amended by the Law of 5 July 2000, Stb. 359), with Respect to the Introduction of a Prohibition on Xenotransplantation. (Staatsblad van het Koninkrijk der Nederlanden, 2002, 2 pp.). Available at: http://apps.who.int/idhl-rils/results.cfm?language=english&type= ByTopic&strTopicCode=IVC&strRefCode=Neth (accessed on 19 August 2009).
38. Regulations on Medical Research Involving Human Subjects (Medical Research [Human Subjects] Act), March 1, 2006 (Implementing the European Clinical Trials Directive 2001/20/EC). Available at: http://ccmo-online.nl/main. asp?pid=21&thid=3 (accessed on 19 August 2009).
39. Department of Health. United Kingdom Xenotransplantation Interim Regulatory Authority (UKXIRA, Discontinued in 2006). Available at: http://www.dh.gov.uk/ab/Archive/UKXIRA/index.htm (accessed on 19 August 2009).
40. Department of Health. Xenotransplantation Guidance. 2006. Available at: (accessed on 19 August 2009).
41. The Federal Assembly of the Swiss Confederation. Federal Act on the Transplantation of Organs, Tissues and Cells (Transplantation Act) of 8 October 2004 (Status as at 1 July 2007), 810.21. Available at: http://www.admin.ch/ch/e/ rs/8/810.21.en.pdf (accessed on 19 August 2009).
42. er Juillet 2007). Available at: http://www.admin.ch/ ch/f/rs/8/810.213.fr.pdf (accessed on 19 August 2009).
43. Patience C, Takeuchi Y, Weiss RA. Infection of human cells by an endogenous retrovirus of pigs. Nat Med 1997 3 : 282 286.
44. Ministry for the Environment. Report of the Royal Commission on Genetic Modification. 2002. Available at: http://www.mfe.govt.nz/publications/organisms/ royal-commission-gm/ (accessed on 21 July 2009).
45. Restriction on Specified Biotechnical Procedures. 2002. Available at: http://www.legislation.govt.nz/act/public/1981/0118/latest/DLM56503.html?search= ts-act-medicines+act+1981-resel (accessed on 21 July 2009).
46. HRC Gene Technology Advisory Committee, GTAC Report on LCT Application with Cover Letter 030407. 2007. Available at: http://www.hrc.govt.nz/assets/ pdfs/GTAC%20report%20and%20cover%20letter%20030407.pdf (accessed on 21 July 2009).
47. National Health Committee's Advice on Living Cell Technologies Application for Xenotransplantation Clinical Trials in New Zealand. 2008. Available at: http://www.nhc.health.govt.nz/moh.nsf/pagescm/7547/$File/ nhc-living-cell-technologies-report-oct082.pdf (accessed on 21 July 2009).
48. Authorisation of a specified biotechnical procedure. N Z Gaz. 2008 162 : 4250 4251. Available at: http://www.dia.govt.nz/pubforms.nsf/NZGZT/ NZGazette162Oct08.pdf/$file/NZGazette162Oct08.pdf (accessed on 21 July 2009).
49. Living Cell Technologies Limited. Company Announcement: Living Cell Technologies' Diabetes Clinical Trial Authorized by New Zealand Government. 2009. Available at: http://www.lctglobal.com/downloads/cms-latest-news/2009-06- 23-LCT%20NZ%20Trial%20%2024%20Jun09%20Final%20.pdf (accessed on 21 July 2009).
50. First WHO Global Consultation on Regulatory Requirements for Xenotransplantation Clinical Trials, Changsha, China, The Changsha Communiqué, November 19-21 2008. 2008. Available at: http://www.who.int/ transplantation/xeno/ChangshaCommunique.pdf (accessed on 21 July 2009).
51. ® in Diabetes Clinical Trial. 2009. Available at: http://www.lctglobal.com/downloads/cms-latest-news/2009-05-12- Clinical%20Announcement%20FINAL%20%205May09.pdf (accessed on 21 July 2009).
52. Hughes JM. The impressive and rapidly expanding knowledge base on SARS. Emerg Infect Dis 2004 10 : 171 172.
53. Fifty-Seventh World Health Assembly, Agenda Item 12.14, May 22, 2004. Resolution 57.18. 5-22-2004. Available at: http://www.who.int/gb/ebwha/pdf- files/WHA57/A57-R18-en.pdf (accessed on 21 July 2009).