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SUMMARY OF ATALUREN RESULTS ISSUEDLow dose of ataluren appears to show better results in 6 minute walk test APRIL 20, 2010
Additional results, presented this week at the American Academy of Neurology in Toronto, show that trial participants who took the lower dose of ataluren did better on a six-minute walking test than did participants who took the placebo or higher dose. The findings were presented by Dr Brenda Wong, study investigator, based at Cincinnati Children's Hospital.
Read More...
GENZYME UPDATE 25/03/10
Update for patients and families on ataluren trial results in nonsense mutation Duchenne/Becker muscular dystrophy
PTC Therapeutics and Genzyme Corporation are continuing to analyze results from the Phase 2b clinical trial of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy (nmDBMD). While the initial examination of the data showed that the primary endpoint of change in 6-minute walk distance did not reach statistical significance within the 48 week duration of the study, additional statistical analyses are necessary to fully understand the results. This will help determine the clinical and regulatory path forward for nmDMBD patients and for ataluren.
This landmark Phase 2b trial, which enrolled 174 boys and young men ages 5 to 20, showed that ataluren was well tolerated. No clinical trial patients discontinued treatment due to an adverse event. Thanks to the diligence of the investigators, patients and families, the trial was well conducted: Compliance with the drug regimen and study procedures was exceptionally high.
Now our work must continue. PTC and Genzyme are performing multiple additional analyses of data to better understand variations in trial results and, in conjunction with regulatory authorities, to identify the appropriate next steps for the ataluren program in nmDBMD. We understand that data from this study are important for ataluren clinical trial patients and the DBMD community as a whole. As we continue to collaborate with investigators, regulatory authorities, and patient advocates, we will provide updates on results.
We recommend that patients and families who participated in the Phase 2b and other ataluren clinical trials follow up with their investigator to discuss their individual results. We have provided treatment assignments to investigators that indicate whether a patient participating in the Phase 2b trial received placebo, ataluren low dose, or ataluren high dose. In addition, we have informed investigators that they may share trial-related information contained in local medical records with their patients.
We would like to extend our warm thanks and gratitude to everyone involved in this trial and in the Phase 2a trial and in particular the boys and young men who participated. Through their contributions, we have advanced our understanding of Duchenne/Becker Muscular Dystrophy for the benefit of the whole community. The trial has provided a wealth of data that will inform the design of future trials and the development of new treatment options.
If you have any questions regarding the ataluren clinical program you are encouraged to contact Genzyme Corporation at:eumedinfo@genzyme.com
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Ataluren Update with PTC Therapeutics
Compiled by PPMD Senior Director of Research and Advocacy,
Sharon Hesterlee, Ph.D.
March 5, 2010
In response to the press release that went out on March 3, 2010 from PTC Therapeutics regarding the results of the ataluren trials in Duchenne, PPMD thought it essential to put together, immediately, an open conference call for parents and families to have their questions answered by PTC’s senior staff members. On Friday, March 5, 150 people joined this call. PPMD President Pat Furlong and Senior Director of Research and Advocacy Sharon Hesterlee moderated the call.
Read the Transcript...
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PTC THERAPEUTICS AND GENZYME CORPORATION ANNOUNCE PRELIMINARY RESULTS FROM THE PHASE 2B CLINICAL TRIAL OF ATALUREN FOR NONSENSE MUTATION DUCHENNE/BECKER MUSCULAR DYSTROPHY
SOUTH PLAINFIELD, NJ and CAMBRIDGE, MA – March 03, 2010 – PTC Therapeutics, Inc. and Genzyme Corporation (Nasdaq: GENZ) today announced preliminary results from the Phase 2b clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation Duchenne/Becker Muscular Dystrophy (nmDBMD). The primary endpoint of change in 6-minute walk distance did not reach statistical significance within the 48 week duration of the study. Study results showed that ataluren was well tolerated and no clinical trial patients discontinued treatment due to an adverse event. Additional efficacy analyses are underway in patient subgroups. Read The Press Release...
Read PPMD Response from PAT FURLONG
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PTC Therapeutics Announces Data showing Six-Minute Walk Test Consistently
Measures Ambulatory Function in Patients With Duchenne Muscular Dystrophy
Findings Suggest Clinically Meaningful Primary Endpoint for Clinical Trials;
Data Presented at the 13th International World Muscle Society Congress -
South Plainfield, NJ – September 30, 2008 – PTC Therapeutics, Inc. (PTC) today announced promising findings in patients with Duchenne/Becker Muscular Dystrophy (DMD/BMD) at the 13th International World Muscle Society (WMS) Congress. Results from an observational study assessing the utility of the 6-minute walk test (6MWT) as a primary outcome measure in trials of treatment for DMD/BMD showed that the 6MWT clearly differentiates boys with DMD from healthy boys, especially when adjusted for age. The findings also demonstrate that the 6MWT provides a reliable assessment of ambulatory function in boys as young as five years of age, offering a practical and clinically meaningful outcome measure for use in DMD/BMD registration-directed clinical trials. Results from the study were presented today by Craig McDonald, M.D., principal investigator and director of the Rehabilitation Research and Training Center in Neuromuscular Diseases, University of California Davis. <Read more>
Latest PTC News
Update on Aussie Clinical Trial Centres (July 29, 2008)
Information about the Melbourne and Sydney Phase 2b trial sites is available on PTC's website, www.ptcbio.com (search clinical trials, then the map) and www.clinicaltrials.gov (search on PTC124)
This Frequently Asked Questions document explains the basic trial design and inclusion/exclusion criteria.
In addition to information from the American Academy of Neurology meeting last year, read an update in the form of an announcement of this trial.
CLINICAL TRIALS TO START IN AUSTRALIA (April 11, 2008)
A multicentre international phase 2B study of PTC124 is about to start up. The Royal Children’s Melbourne and Children’s Hospital at Westmead, Sydney will be the 2 Australian study sites. Both are open to enrolling children from around Australia, and both are funded to cover travel and accommodation costs for families going to their centres.
In brief the inclusion criteria are:
1. Male, aged >5yrs with DMD or BMD (this includes adults with BMD).
2. Ambulant for >75m unassisted in 6 minutes or less
3. Documented nonsense (point) mutation in the dystrophin gene as determined by gene sequencing from an accredited laboratory.
Exclusion:
1. Treatment with a systemic aminoglycoside within 3m of starting treatment
2. Initiation of corticosteroid treatment within 6m prior or change in steroid dose within 3m
3. Any change in treatment for CHF within 3m of initiation of treatment.
4. Expectation of major surgery (including scoliosis surgery) within 12months of starting treatment.
5. Requirement for daytime ventilator assistance
It is a 12month study with a fairly demanding schedule ie
- lab visits every 3w for the first 24 weeks (local or at study centre)
- Muscle biopsy pre and post study even in those who have undergone previous muscle biopsy
- The study will involve 12 2-day visits to the study centre in a 12m period
If you believe your child has a point mutation, please contact Dr Monique Ryan, [monique.ryan@rch.org.au] or Kristi (KristiJ@chw.edu.au). Queensland parents with any questions may contact Dr Sinclair [Kate_Sinclair@health.qld.gov.au] too as the Brisbane neurouscular clinic is actively seeking candidates to be involved.
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(Click on the orange link to download the .pdf file at the top of the resources list.)
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