CLINICAL TRIALS

Our clinical expertise

Two platforms dedicated to paediatric and adult clinical trials in neuromuscular diseases, and a registries and databases division.
A multidisciplinary team serving patients, families and research.

Clinical studies

Clinical studies are central to the work of the Institute of Myology, which has two clinical trial platforms for neuromuscular diseases, I-Motion Pediatric and I-Motion Adults, together with the Registries and Databases division, dedicated to building and monitoring databases, registries, observatories and cohort follow-up. The clinical trial platforms provide clinical care for patients, whether children or adults, evaluate new treatments and run innovative clinical trials.

At present, around sixty research protocols are under way at the Institute of Myology: clinical studies (natural history, care, exercise reconditioning) and also therapeutic trials (pharmacology, cell therapy, gene therapy, gene-targeted therapy, enzyme therapy).

Beyond the trials initiated directly by the teams of the Biotherapies Institute for Rare Diseases created by AFM-Téléthon, the Institute’s scientific and technology platform is recognised internationally by organisations and pharmaceutical companies  (the US National Institutes of Health, Genzyme, PTC Therapeutics, Santhera, Roche ….), which have chosen it as the lead investigator site for some of their trials.

What is a clinical trial?

A clinical trial (or therapeutic trial) is a scientific study in which a potential treatment is tested in humans in order to assess how well it is tolerated and how effective it is.

The purpose of a clinical trial is to confirm, in a significant number of people, that a new treatment is well tolerated and effective in the target disease. It is an essential step before any new treatment can be made available.

Taking part in a clinical trial

Taking part in a clinical trial is a personal choice. It means entering a strictly defined protocol, designed both to ensure the safety of participants and to produce reliable, meaningful results. It also means accepting the constraints of additional hospital visits and tests.

The team

The I-Motion team brings together professionals trained specifically in clinical research and in working with patients and their families: doctors, physiotherapists, clinical research associates, project managers, quality engineers and medical secretaries. All of them work for patients, whether they are taking part in clinical trials or attending a neuromuscular consultation.

Assessment tools

Most neuromuscular diseases are progressive. Assessment tests make it possible both to document a patient’s condition at a given point in time and to track how it changes. They are used in consultations to describe symptoms precisely and to tailor rehabilitation, and as part of clinical trials to build homogeneous patient groups and measure the effect of treatments.

The I-Motion team uses a battery of tools covering motor function, muscle strength, respiratory function and daily activity.

Other tools, more specifically paediatric ones (CHOP INTEND, HFMSE, GMFM, the Bayley scales, the Peabody scales), are presented on the I-Motion Pediatric page.

Motor function scales

  • MFM (Motor Function Measure): an overall quantitative scale that measures motor ability in people with a neuromuscular disease, across three domains (standing and transfers, axial and proximal motor function, distal motor function).
  • NSAA (NorthStar Ambulatory Assessment): developed for ambulant patients with Duchenne muscular dystrophy (DMD) (standing up from the floor, running, jumping and so on).
  • PUL / RULM: assessment of upper limb function (PUL, Performance of Upper Limb, for dystrophinopathies; RULM, Revised Upper Limb Module, validated in children with spinal muscular atrophy).

Timed tests

  • 6-minute walk test (6MWT): measures the distance walked without stopping. A reference test in many clinical studies.
  • 10-metre walk or run test: the time taken to cover 10 m, with or without an assistive device.
  • 4-stair climb: used in particular in DMD.
  • Timed rise from the floor: assesses pelvic girdle weakness.
  • Timed Up and Go (TUG): balance, lower limb strength and walking speed.
  • 30-second sit to stand: the number of stands completed from a seated position in 30 seconds.

Muscle strength assessment

  • MMT (manual muscle testing): analytical muscle assessment using a six-level grading scale (0 = no contraction, 5 = normal strength).
  • Dynamometry (myometer): measurement of the isometric strength of a muscle or muscle group; precise muscle-by-muscle assessment.
  • MyoTools: a set comprising MyoGrip (finger grip strength), MyoPinch (thumb-to-index pinch strength) and MoviPlate (fatigability of the wrist and finger extensors and flexors). Very high sensitivity for patients with limited muscle capacity.

Additional assessments

  • Spirometry and assessment of respiratory function: depending on age, sniff test, cough peak expiratory flow, spirometry, inspiratory and expiratory pressures. This makes it possible to monitor changes in lung capacity and to assess the need for ventilation or airway clearance devices.
  • Goniometry: passive measurement of range of motion, identifying joint limitations and muscle contractures.
  • ActiMyo: a continuous measurement device, developed with Sysnav, which records movements made in everyday life (accelerometer, gyroscope, barometer, magnetometer). Intended for patients aged over 6 years, whether ambulant or not. It provides an outcome measure reflecting the patient’s actual, sustained activity at home.