Amyotrophic Lateral Sclerosis Clinical Trial
Official title:
Can Diaphragm Pacing Delay Non Invasive Ventilation in Amyotrophic Lateral Sclerosis ? a Randomized Controlled Study
ALS is is characterized by a progressive degeneration of motor neurons, leading to progressive weakness of muscles, including respiratory muscles, the diaphragm. Although specific therapy is lacking, correct respiratory therapy improves quality of life and increases survival. Substituting the failing respiratory muscles by non invasive mechanical ventilatory assistance (NIV) is the current standard of care. Intradiaphragmatic phrenic nerve stimulation is a new treatment and has been the object of a preliminary international proof-of-concept multicenter trial. This trial suggests that the intradiaphragmatic phrenic nerve stimulation slows down the rate of decline of the diaphragm. Our new hypothesis is that phrenic stimulation induces diaphragm conditioning and can delay the need for mechanical ventilation in ALS patients. We will study, during 24 months, 2 groups of 37 patients at the beginning of the respiratory dysfunction, using a intradiaphragmatic phrenic nerve stimulation in one group and a sham stimulation in the other group. Although, all the patients will be implanted, thus, at the end of the study, all the patients will receive effective stimulation.
ALS is is characterized by a progressive degeneration of upper and lower motor neurons,
leading to progressive weakness of bulbar, limb, thoracic and abdominal muscles. Although
specific therapy is lacking, correct respiratory therapy improves quality of life and
increases survival. Substituting the failing respiratory muscles by non invasive mechanical
ventilatory assistance (NIV) is the current standard of care.Intradiaphragmatic phrenic
nerve stimulation, has been the object of a preliminary proof-of-concept multicenter trial
(ClinicalTrials.gov NCT00420719).
Aim of the study : To test the hypothesis that phrenic stimulation induced diaphragm
conditioning can delay the need for mechanical ventilation in ALS patients.
Methods : It is a double blind randomized study. Patients presenting with early signs of
respiratory impairment (VC between 85 and 60%), but with a preserved electromyographic
response of the diaphragm to phrenic nerve stimulation, will be randomized in 2 groups. All
the patients will be treated according to current standards of care. They will all be
implanted with a phrenic stimulator, and then randomized between actual diaphragm
conditioning and sham stimulation.
Respiratory function will be followed up on a trimonthly basis, with polysomnography and
diaphragmatic EMG biannually. NIV (+ stimulation for both groups), will be initiated
according to currently recommended criteria of hypoventilation.
The main outcome of the study will be the number of months between the phrenic nerve
implantation and the introduction of NIV. Currently available data, showing that diaphragm
pacing can increase the number of patients without NIV at 2 years from 2,5% to 15% of the
patients, requires the enrollment of 37 patients in each group. Secondary end-points will
include i.Survival ii. Effects on sleep iii. Quality of life and daily activities
;
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver), Primary Purpose: Treatment
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