Friday 29 June 2012
Phase 3 clinical trial for Ataluren announced
PTC Therapeutics has announced in a press release that they have now started a phase 3 clinical trial for ataluren in the UK, continental Europe, Israel, Canada and Australia. The trial will only be open to boys who previously participated in the phase 2b trial of ataluren.
The 48 week, phase 3 trial being conducted by PTC Therapeutics to test ataluren in boys with Duchenne and Becker muscular dystrophy will be an open-label study open to individuals who previously took part in the phase 2b trial. Although the primary aim is to assess how safe and well tolerated the drug is over a longer time period they will also be carrying out some tests, such as the six minute walk test, to determine if ataluren has a positive effect on muscle function.
Ataluren is an oral drug that has been developed in the US by PTC Therapeutics to overcome a specific change in the DNA called a nonsense mutation. About 10-15% of boys with Duchenne muscular dystrophy have this type of mutation. In very rare cases, nonsense mutations occurring in certain positions in the dystrophin gene can also cause the milder symptoms of Becker muscular dystrophy.
The initial results of the international phase 2b clinical trial of ataluren showed that the lower of the two doses tested was more beneficial than the higher dose and because of this unexpected outcome all ongoing studies had been halted in April 2010. In the US all boys who participated in the phase 2b study have been receiving the lower dose in an extension trial since November 2010 but getting an extension trial started in Europe has been slow.
Further information and links
Read the press release from PTC therapeutics
Find out more about the clinical trial
More information about Duchenne muscular dystrophy
Read about the Duchenne research we fund
If you have a question about this or any other research, please email us: