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Note of the 57th GTAC meeting held on 15 February 2006 in London Present: Professor Norman Nevin (Chair), Professor Terry Hamblin, Mrs Fiona Sandford, Professor David Harrison, Mrs Debbie Beirne, Professor Martin Gore, Dr Adrian Lepper, Professor Nick Lemoine, Dr Michael Waterhouse, Dr Richard Ashcroft, Professor Andrew Baker, Professor Mary Collins, Bishop Dr Lee Rayfield, Dr Peter Harris and Professor Andrew Lever
1: Welcome The Chair welcomed the Committee to its 57th meeting. The Chair thanked the Human Genetics Commission for use of the venue. Congratulations were given to Prof Martin Gore who has recently been appointed the new chair of GTAC from July 2006 after Prof Nevin steps down and to Prof David Harrison who has been appointed vice-chair from July 2006. The Chair went on to remind members of the hand-over meeting to be held in Belfast on 23 June. 2: Minutes of the 56th meeting of GTAC The minutes were agreed as an accurate and true record of the 56th meeting. 3: Matters Arising A number of letters had been sent out and received, concerning correspondence arising from the last meeting. None of which gave rise to any discussion 4: Chairman's Actions Eight letters were sent out under Chairman's Actions, none of which gave rise to any discussion. 5: Substantial Amendment GTAC 073: This issue was discussed at the previous meeting, when GTAC declined the proposal. The GOSH team were asked to send an amended protocol specifying the precise inclusion and exclusion criteria for enrolment of ADA patients. In line with GTAC's request, the secretariat then sent the protocol amendment for external review. GTAC discussed this amendment in some detail, given the comments from the expert reviewers. After some discussion, the decision was to grant approval on the understanding that it would be reviewed in the light of any new data. The committee also spoke about the current process of giving approval on a case-by-case basis for all ADA-SCID patients. It was decided that notification of new patients would be more appropriate.
This is a proposal for a trial in adult patients with Haemophilia B, a sex-linked bleeding disorder caused by a deficient clotting factor IX (FIX). It affects some 1:30,000-50,000 males and can, on occasion, affect females. The study aims to develop a treatment for patients with severe Haemophilia B (less than 1-2% FIX activity). Such patients experience spontaneous bleeding (mainly into knee, elbow and ankle joints) which can cause permanent arthritis and disability if not promptly treated. This is controlled by maintaining weekly dosing with recombinant factor IX. The study proposes to administer a modified adeno-associated virus (serotype 8) vector containing the gene for FIX. Unlike the more common AAV2, AAV8 rarely infects humans. Therefore, it is anticipated that the AAV8 derived vector may result in a reduced immune response when compared to the more common AAV2 vector. The vector contains a liver specific promoter to drive FIX expression. This application produced a large amount of discussion, much of it surrounding the fundamental question 'does this patient group need this therapy?' There was some discussion about the 'quality of life' of the current treatment with some patients having to inject themselves 2-3 times per week. The committee felt that this inconvenience should not be trivialised and that there were issues in terms of compliance with weekly injections in some patient groups such as children and teenagers. No consensus emerged from the discussion. It was suggested therefore that the secretariat organise an extraordinary GTAC meeting inviting prominent clinicians, patient groups and any other interested parties by the end of March. This meeting should be able to answer many of the questions that GTAC raised as well as giving GTAC first hand view of the patient voice. 7: Substantial amendment GTAC 29C: This concerns an application for approval of a substantial amendment to GTAC 029C, a myeloma study. The investigators have proposed to explore the technique of electroporation exploiting the hypothesis is that using DNA priming and boosting with DNA and electroporation may yield better immune responses. This amendment was declined in its current form but a number of suggestions were made on how this proposal can be taken forward. 8: Substantial amendment GTAC 090: This concerns a submission for approval of a substantial amendment to the GTAC 090 study. The proposers wish to make several amendments to the end-point, the statistical analysis and the inclusion criteria. The amendment was approved. 9: EMEA Consultation on Gene Transfer Medicinal Products The EMEA is currently consulting on a draft note for guidance on the quality, preclinical and clinical aspects of gene transfer medicinal products. GTAC members were asked for any comments regarding either of the draft papers. 10: SUSAR Update There have been seven new SUSARs since the previous meeting. The committee discussed some in detail but decided that they were not related to the trial product. 11: Any other Business EMEA proposal on Advanced Therapies This was a paper which contained details about a draft regulation for future approvals for market authorisation of "advanced therapy medicinal products" by the European Medicines Evaluation Agency (EMEA). Named Patient Exemption Members were asked for approval of a named patient exemption from GTAC 084, "A phase I study of Immunotherapy for patients with metastatic melanoma using dendritic cells transfected with a plasmid encoding two melanoma antigens". The committee declined the application.
GTAC Secretariat
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