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January 2008 MDEX Newsletter
Honours
The chair of the Scientific Advisory Board for the MDEX Consortium Professor Kay Davies has been made a Dame of the British Empire (DBE) in the New Years honours list. Congratulations.
Clinical Trials
AVI 4658
This trial is ACTIVELY recruiting. The first patient who had his IM injection of the trial drug AVI 4658 at St Mary’s Hospital in December 2007 had EDB muscle biopsy on 8 January 2008. A clinical and laboratory report was submitted to the trial Safety Monitoring Committee (SMC) on 18 January 2008. The SMC approved the report and agreed for the trial to proceed to the next patient.
The second patient who consented in December was injected with the trial drug on 29 January 2008 and all seems well.
Pre assessment studies on a third patient were done in mid-January 2008.
There is ongoing discussion with AVI BioPharma on how to structure the planned systemic delivery trial.
PTC124
A Phase 2b efficacy and safety study of PTC124 in subjects with nonsense-mutation-mediated Duchenne and Becker Muscular Dystrophy is planned for multiple centres in Europe. Subject to funding, this may include the MDEX consortium centres in Newcastle and the Institute of Child Health, London. This study is planned to start recruitment in early 2008.
Prosensa systemic 20ME trial for exon 51.
Following the positive outcome of the intramuscular 2OME antisense oligonucleotide study in Holland, Prosensa is planning a systemic trial of the same antisense across several European countries, and TREAT-NMD will be involved in facilitating this study.
The Move
The move of the Neuromuscular Unit (NMU) from Imperial College London to the Institute of Child Health (ICH), UCL commenced on the 1st December 2007 with Francesco’s appointment with ICH. The first batch of NMU laboratory and associated staff (~seven) will move on or about the 14th February 2008.
Francesco’s new contact details are as follows:
Address: Dubowitz Neuromuscular Unit, 1st Floor Institute of Child Health, 30 Guildford Street, London WC1N 1EH.
Tel: 020 7905 2608 Fax: 020 7905 2832 email: f.muntoni@ic.ac.uk
Your comments would be welcome and any news that you would like to include in the February issue should be sent, latest a few days before the end of the month. k.ganeshaguru@imperial.ac.uk
1 Comments
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- Mark Silverman
- February 04th 2008
- 22:06pm
Do you know when the team of clinicians, including Dr Adnan Manzur, will be moving across to the new site? We have an appointment at the end of February and want to make sure we attend the right hospital.