Early Stage Amyotrophic Lateral Sclerosis Phrenic Stimulation (RespiStimALS)
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|ClinicalTrials.gov Identifier: NCT01583088|
Recruitment Status : Terminated (in the absence of benefits and because of a statistically significant excess mortality in the group of patients receiving active stimulation.)
First Posted : April 23, 2012
Last Update Posted : December 22, 2015
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis Respiratory Insufficiency||Device: phenique nerve stimulation NeurX™ (Synapse Biomedical) Device: sham phrenic nerve stimulation||Phase 3|
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
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||74 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Care Provider)|
|Official Title:||Can Diaphragm Pacing Delay Non Invasive Ventilation in Amyotrophic Lateral Sclerosis ? a Randomized Controlled Study|
|Study Start Date :||September 2012|
|Actual Primary Completion Date :||November 2015|
|Actual Study Completion Date :||November 2015|
Experimental: phenic nerve stimulation
effective phenic nerve stimulation NeurX™ (Synapse Biomedical)
Device: phenique nerve stimulation NeurX™ (Synapse Biomedical)
phenique nerve stimulation NeurX™ (Synapse Biomedical)
Sham Comparator: sham
sham phenic nerve stimulation
Device: sham phrenic nerve stimulation
sham phenic nerve stimulation
Other Name: sham phenic nerve stimulation
- Survival without NIV 2 years after the phrenic nerve implantation. [ Time Frame: 2 years ]
- global survival from onset disease [ Time Frame: 2 years ]
- effects on sleep [ Time Frame: 24 months ]
- Quality of life and daily activities [ Time Frame: 24 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01583088
|APHP, GH Pitié Salpêtrière|
|Paris, France, 75013|
|Principal Investigator:||Gonzalez-Bermejo Jesus, Md, PhD||APHP|