
The Institute of Myology is recruiting patients and healthy subjects to participate in the ULENAP clinical trial.
Mohamed Benali, one of two physiotherapists responsible for carrying out the tests, explains the procedure of a visit.
To begin, can you briefly recall what this study involves*?
It involves studying the relevance of tools for measuring muscle strength of upper limbs in non-ambulatory (not able to walk more than 10 meters) patients with neuromuscular diseases. To verify the reproducibility of measurements, tests are repeated over four visits: V1 and V2 (at least 30 days apart), V3 at 6 months and V4 12 months later. These measurement tools will be used to evaluate the efficacy of future therapies.
There is indeed no such test for these patients. Yet, evaluation tools should be ready when the trials start.
To verify the reproducibility and reliability of measurements, the full battery of tests is repeated over four visits: V1 and V2 at least 30 days apart (it is possible to make these two visits in the same day), V3 six months later then V4 after 12 months.
What happens specifically during a visit?
Each participant tests 1-5 tools (depending on the capacity of each). First, one has to tighten the Handgrip handle, then pinch with the thumb and forefinger with the Pinch, and then we measure the strength of the wrist with Myowrist. We will then ask the participant to alternately touch as quickly as possible with their fingers the two Moviplate cylinders, a tool specially designed at the Institute of Myology for non-ambulatory NM patients (everyone can try to beat his previous score). Finally, we conclude with Taping, which involves typing as many times as possible with the index finger on the sensor.
In addition, during the first visit, participating patients respond to a questionnaire relating to the use of the hand and perform functional exercises (MFM). For patients, the battery of tests lasts between 1-1 ½ hours, according to the visits; for healthy subjects who only test the tools, the session lasts 45 minutes.
How many people do you plan to include?The tests will be conducted in 100 patients (50 DMD, 30 SMA and 20 other NMD) and 60 control subjects aged 8-30 years.
So far, 30 patients and 12 healthy subjects have participated and we have already observed a very good reproducibility of results of the different measurement tests between the first and second visit. It is very encouraging!
We aim to terminate the ULENAP in late 2011 in order to be prepared for a gene therapy clinical trial (exon skipping in Duchenne muscular dystrophy), which could begin in 2012. Therefore, we must have recruited all participants before the end of 2010.
To continue the study, we are still looking for patients (DMD, SMA or other neuromuscular disorders) as well as healthy boys from 8-30 years. As we will be present at the JDF, 25 and 26 June, come and visit us at the booth of the Institute of Myology to ask questions and register! Contact: Mohamed Benali - 01 42 16 66 43 – ulenap(at)institut-myologie.org
In short - the ULENAP team of the Institute
Principal Investigator: Laurent Servais
Laboratory Director: Jean-Yves Hogrel
Physiotherapist evaluators: Mohamed Benali and Aurélie Canal *A more detailed description is available
here.
June 2010 Interview by Anne Berthomier Translation by Racquel N. Cooper