Details Regarding Study DMD114117 for Duchenne Muscular Dystrophy

Details regarding several of the investigator sites for the following study have been posted to www.orpha.net (the portal for rare diseases and orphan drugs) today:  

Study DMD114117: A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy

Additional study details, including inclusion/exclusion criteria and study endpoints are posted on www.clinicaltrials.gov.  Investigator site details for this study, taking place in Europe, Turkey and Australia, will also appear on www.clinicaltrials.gov in the near future.

Sincerely,

 John E. Kraus, MD, PhD                                Padraig Wright, MD, PhD

john.e.kraus@gsk.com                                 padraig.x.wright@gsk.com

GSK Anticipates Delay in 48 week Phase 3 DMD Study

ON BEHALF OF JOHN KRAUS, MD, PHD and PADRAIG WRIGHT, MD, PHD

(GLAXOSMITHKLINE)

Today, GSK informed a number of potential study investigators that we anticipate a delay in the planned 48 week phase 3 study (Study DMD114044) of GSK2402968, commencing in Canada. 

Health Canada has requested that GSK provides additional data to inform their decision regarding approval of initiation of Study DMD114044.  This study is planned to investigate the safety and efficacy of GSK2402968 in ambulant boys with DMD, who have a dystrophin gene mutation amenable to an exon 51 skip. 

GSK is working with Health Canada to provide the data they require to initiate the study in Canada as soon as possible. 

In the meantime, GSK is working with other regulators and progressing with plans to begin the study outside of North America.  GSK will alert you when study details, including study site locations, are posted to www.clinicaltrials.gov. 

 John E. Kraus, MD, PhD                                  Padraig Wright, MD, PhD                                          
john.e.kraus@gsk.com                                    padraig.x.wright@gsk.com

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Glaxo Smith Kline Announces New Exon Skipping Study

Glaxo Smith Kline announced a new Exon Skipping study. The study, officially titled, “A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects (DEMAND I)”, will assess the pharmacokinetics, safety and tolerability of GSK2402968 after a single administration, at escalating dose levels, in non-ambulatory boys with DMD who have a dystrophin gene mutation amenable to an exon 51 skip.

Read more about the study here:

Glaxo Smith Kline Announces New Exon Skipping Study