TREAT-NMD involvement in clinical trials

In one form or another, TREAT‐NMD is assisting many companies currently planning or running neuromuscular trials. It is also involved in investigator-led research, and many TREAT-NMD partners are also experienced trial sites taking part in neuromuscular trials. An overview of the support provided to companies and an idea of the timelines involved in some of the ongoing and upcoming trials is provided in the table below.

Note that this table is not a comprehensive list of all neuromuscular-related trials, but is restricted to those with TREAT-NMD involvement. Details of other trials can be found at the clinicaltrials.gov website, where you can search by disease, company name or a number of other criteria to see the trials currently ongoing and their recruitment status.
 

Trial/company Drug/disease	How TREAT‐NMD is involved		Trial Status Information
ACE‐031 DMD, FSHD	SAB setup for DMD program TREAT‐NMD partners on advisory board		Current trial status
AVI‐4658 DMD	TREAT‐NMD partners on advisory board TREAT‐NMD partners as trial sites Patient registries enquiry (patient numbers; frequency of top 5 exon skippable mutations)		Current trial status
BMN‐195	SAB setup for DMD trial under negotiation
GSK2402968			Current trial status
GSK2402968 PRO‐051 DMD	TREAT‐NMD partners on advisory board Patient registries enquiry (DMD patients meeting trial inclusion criteria) CTSR enquiry and provision of site contact details for pivotal trial site selection		Current trial status
Ataluren DMD	TREAT‐NMD partners on advisory board TREAT‐NMD partners as trial sites TREAT‐NMD conducted European arm of clinical evaluator training Patient registries enquiry (patients with nonsense mutations)		Current trial status Extension study status Non-ambulant study status
Catena/ Sovrima DMD Omigapil CMD	TREAT‐NMD partner TREAT‐NMD partners on advisory board TREAT‐NMD will assist with European arm of physiotherapist training TREAT‐NMD assisting with application for FP7 funding for CMD trial		Current trial status
TRO19622 SMA	TREAT‐NMD Clinical Trials Coordination Centre to perform site selection and protocol development for phase II efficacy study through collaboration with Trophos and CRO LabConsult Feasibility information		Current trial status
FOR-DMD NIH‐funded trial to assess steroid dosing regimes Prednisone/ Deflazacort	Collaboration with US Muscle Study Group TREAT‐NMD to coordinate European trial site recruitment (CTSR sites will be asked to apply) and regulatory submissions TREAT‐NMD to conduct European arm of clinical evaluator training