The clinical stage drug and development company, Dynacure, is conducting a multi-centre clinical trial called Unite-CNM (DYN101-C101) for patients from the age of 16 years with centronuclear myopathy (CNM) due to mutations in DNM2 or MTM1.
The trial is currently ongoing, but there is still some availability for interested patients/families, in particular for patients with x-linked Myotubular Myopathy (XLMTM, or with the MTM1 mutation).
Trial centres are currently looking for patients 16 years or older, including female carriers, that are symptomatic, though preferably able to walk at least a few steps.
What will patients need to do?
The trial will require weekly visits to the hospital, travel would be organised and expenses covered by Dynacure.
There is a comprehensive FAQ that you may review on the Dynacure website, and more background on the study can also be found here.
Important: Patients are ultimately selected for eligibility by the clinicians at the participating clinical trial sites, not by Dynacure or by any patient advocacy organisation.
What to do if I am interested in joining this trial?
If you or your family member is interested, the most appropriate next step is to have their treating consultant or other medical professional involved in your care, contact a clinical trial site or you can contact the clinical trial site directly.
In the UK the clinical trial sites are:-
MRC centre for Neuromuscular Disease, National Hospital for Neurology and Neurosurgery
London, United Kingdom
Contact: Professor Rosaline Quinlivan, Professor 0044 7527 442 063 r.quinlivan@ucl.ac.uk
Royal Victoria Infirmary
Newcastle Upon Tyne, United Kingdom
Contact: Dr Sam McDonald 0044 191 241 8649 sam.mcdonald@newcastle.ac.uk
What is DYN101?
DYN101 is the name given to the drug (or ‘compound’) being tested – which is an antisense oligonucleotide (ASO). DYN101 is designed to decrease the expression of a protein named dynamin-2, which is elevated in patients with MTM1 mutations and thought to be overly active in patients with DNM2 mutations (ADCNM).
Preclinical studies of DYN101 in mouse models of these two forms of CNM have indicated that this may be a promising treatment approach, which is why we are developing the product candidate in patients with these mutations.
There are patients with other CNM mutations that are not included in our clinical development program at this time.
What is ASO technology?
Antisense technology aims to bind a synthetic drug to a specific messenger RNA that is involved in a particular disease and to stop unwanted proteins from being produced. For X-linked myotubular myopathy (XLCNM) or autosomal dominant CNM (ADCNM), the ASO is created to decrease the amount of dynamin-2 protein which is too high or too active. Antisense oligonucleotides are short chemically modified strands of nucleotides (parts of DNA). They bind to parts of the messenger RNA that produce proteins, that lead to a particular disease. In many cases, when the ASO or antisense drug binds to the specific mRNA, it results in degradation of the mRNA, which means the targeted or unwanted protein cannot be produced. Therefore, the overall amount of the targeted protein in the cell will be reduced.
You can read more here about the science behind this ASO technology.
