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)
Compound |
Aim |
Phase |
Status |
Clinicaltrial.gov
|
| ACE-031 | Increase muscle mass | II | Terminated | NCT01099761 |
| Ataluren (PTC124) |
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 glutamine |
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 recruiting |
NCT01521546 | |
| Eteplirsen | Safety study of exon 51 skipping compound in patients with advanced stage DMD |
II | Ongoing, not recruiting |
|
| Eteplirsen | Safety study of exon 51 skipping compound in patients with early stage DMD |
II | Recruiting patients at sites in US |
NCT02420379 |
| Eteplirsen | Open-label study of exon 51 skipping in patients with DMD |
IIb | Ongoing, not recruiting |
NCT01540409 |
| Eteplirsen | Confirmatory study of exon 51 skipping compound in patients with DMD |
III | Recruiting patients at sites in US |
NCT02255552 |
| 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 |
| Oxatomide (tinset) |
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 carvedilol |
Improve heart function | IV | Recruiting patients at sites in Italy | NCT00819845 |
| Revatio (Sildenafil) |
Improve heart function | II | Suspended recruitment | NCT01168908 |
| SRP-4053 | Two part study of exon 53 skipping compound in patients with DMD |
I/II |
Recruiting patients at sites in Europe |
NCT02310906 |
| Tadalafil vs Sildenafil |
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 |
|
SRP-4045 |
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.
