Overview of current trials in DMD

TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trials that are currently ongoing.

We aim to update the table below approximately every six months.

Nevertheless, we cannot ourselves keep an up-to-date overview of all the trials that are currently happening across the world. The most comprehensive online listing of trials is at clinicaltrials.gov - companies are obliged to provide details of their trials there. This information is available on the following page which displays information that is pulled through automatically from the clinicaltrials.gov website.

Ongoing trials in Duchenne patients (registered on www.clinicaltrials.gov)






ACE-031 Increase muscle mass II Terminated NCT01099761
Stop mutation read through IIb Completed NCT00592553
Ataluren (Translarna) Long-Term Observational Study of Translarna Safety and Effectiveness
IV Recruiting patients at sites in Germany NCT02369731
AVI-4658 Exon 51 skipping I/II Completed NCT00159250
Carvedilol Improve heart function IV Unknown NCT00606775
Coenzyme Q10 Improve muscle strength II Completed NCT00033189
Creatine vs
Improve muscle strength II/III Completed NCT00016653
EGCg (green
tea extract)
Improve muscle condition II/III Ongoing, not recruiting NCT01183767
Eplerenone for Subclinical Cardiomyopathy in DMD (E-SCAR DMD) Preventing heart muscle damage Ongoing, not
Eteplirsen Safety study of exon 51 skipping compound
in patients with advanced stage DMD
II Ongoing, not



Eteplirsen Safety study of exon 51 skipping compound
in patients with early stage DMD
II Recruiting patients
at sites in US
Eteplirsen Open-label study of exon 51 skipping in
patients with DMD
IIb Ongoing, not
Eteplirsen Confirmatory study of exon 51 skipping
compound in patients with DMD
III Recruiting patients
at sites in US
Flavocoxid Improve muscle function and strength I Completed NCT01335295
Gentamicin Stop mutation read through I Completed NCT00451074
GSK2402968 Exon 51 skipping III Completed NCT01254019
Idebenone Improve respiratory and heart function III Completed NCT01027884
IGF1 Improve muscle quality I/II Unknown NCT01207908
L-glutamine Improve muscle mass II Completed NCT00296621
Lisinopril vs
Coenzyme Q10
Improve heart function II Enrolling by invitation NCT01126697
Mini-dystrophin Test safety of gene therapy I Completed NCT00428935
Improve muscle strength II Completed NCT00033813
Pentoxifylline Improve muscle function I/II Completed NCT00102453
PRO044 Exon 44 skipping I/II Completed NCT01037309
Drisapersen (PRO051) Exon 51 skipping III Enrolling by invitation NCT02636686
Ramipril vs
Improve heart function IV Recruiting patients at sites in Italy NCT00819845
Improve heart function II Suspended recruitment NCT01168908
SRP-4053 Two part study of exon 53 skipping
compound in patients with DMD

Recruiting patients at sites in Europe

Tadalafil vs
Improve blood flow in muscle ? Completed NCT01359670

SRP-4045 & SRP-4053

Exon 45 and exon 53 skipping efficacy vs placebo III Not yet recruiting NCT02500381


Safety and efficacy study of exon 45 skipping in advanced-stage DMD patients I/II Ongoing, not recruiting NCT02530905
FG-3019 Monoclonal antibody to CTFG in non-ambulatory DMD patients II Recruiting patients at sites in US NCT02606136
NPC-14 (Arbekacin Sulfate) Assess safety, tolerability, and efficacy of NPC-14 in ambulant DMD patients with nonsense mutation II Ongoing, not recruiting NCT01918384

More recent developments may not be reflected in this table as we aim to update it about every six months.

Please visit our clinicaltrials.gov web page which draws information directly from clinicaltrials.gov for the latest information.

12 Apr 2017