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) as of July 2011
Compound |
Aim |
Phase |
Status |
Clinicaltrial.gov
|
| ACE-031 | Increase muscle mass | II | Terminated | NCT01099761 |
| Ataluren (PTC124) |
Stop mutation read through | IIb | Completed | NCT00592553 |
| AVI-4658 | Exon 51 skipping | I/II | Completed | NCT00159250 |
| Carvedilol | Improve heart function | IV | Recruiting | 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 | Recruiting | NCT01183767 |
| Flavocoxid | Improve muscle function and strength | I | Recruiting | NCT01335295 |
| Gentamicin | Stop mutation read through | I | Completed | NCT00451074 |
| GSK2402968 | Exon 51 skipping | III | Recruiting | NCT01254019 |
| Idebenone | Improve respiratory and heart function | III | Recruiting | NCT01027884 |
| IGF1 | Improve muscle quality | I/II | Recruiting | NCT01207908 |
| L-glutamine | Improve muscle mass | II | Completed | NCT00296621 |
| Lisinopril vs Coenzyme Q10 |
Improve heart function | II | Not yet recruiting | NCT01126697 |
| Mini-dystrophin | Test safety of gene therapy | I | Unknown | NCT00428935 |
| Oxatomide (tinset) |
Improve muscle strength | II | Completed | NCT00033813 |
| Pentoxifylline | Improve muscle function | I/II | Completed | NCT00102453 |
| PRO044 | Exon 44 skipping | I/II | Active (not recruiting) | NCT01037309 |
| Ramipril vs carvedilol |
Improve heart function | IV | Recruiting | NCT00819845 |
| Revatio (Sildenafil) |
Improve heart function | II | Recruiting | NCT01168908 |
| Tadalafil vs Sildenafil |
Improve blood flow in muscle | ? | Recruiting | NCT01359670 |
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.

