Systematic Laryngoscopic Evaluation of Upper Airways in Ventilated ALS Patients in Portugal and in Norway

Systematic Laryngoscopic Evaluation of Upper Airways in Ventilated Amyotrophic Lateral Sclerosis (ALS) Patients in Portugal and in Norway

The study examines if titration of Non-Invasive mechanical ventilation (NIV) settings during ongoing laryngoscopic visualization can improve the compliance of NIV in subjects with Amyotrophic Lateral Sclerosis (ALS). The study is a multicentre study between Norwegian National Advisory Unit on Long-term Mechanical Ventilation at the Thoracic Department, Haukeland University Hospital, Bergen, Norway and Centro Hospital Tras-os-Montes e Alto Douro, Vila Real, Portugal.

Study Overview

• Status: Enrolling by invitation
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Other: Transnasal Fiberoptic Laryngoscopy

Detailed Description

ALS, amyotrophic lateral sclerosis, is a rare and fatal disease causing gradual loss of motor neurons and thus loss of function in all skeletal muscle, eventually also in the breathing muscles, leading to respiratory failure and ultimately death. There is no causal treatment. The treatment provided aims to improve life quality as long as possible. Non-invasive ventilation (NIV) compensates for respiratory failure as breathing deteriorates.

ALS patients with bulbar innervated muscle dysfunction (mouth, tongue, jaw, throat, larynx) have poor compliance to the use of NIV. It is not completely understood why bulbar ALS patients fails NIV, although the upper airways have been a suggested cause also of this treatment failure. The investigators in the Norwegian study group has previously examined laryngeal response patterns (using transnasal fiberoptic laryngoscopy ) during mechanically assisted cough in ALS. The studies revealed that Transnasal fiberoptic laryngoscopy was safely performed with ongoing mechanically assisted cough, and it appeared a feasible tool to visualize the laryngeal responses to mechanical cough therapy.

The further hypothesis is that the use of transnasal fiberoptic laryngoscopy during ongoing NIV titration could improve the compliance of NIV treatment in ALS.

Since ALS is a relatively rare disease and it is difficult to perform large clinical studies, a project group has been established and agreed to a common protocol of a population-based multicenter-study including two hospitals, in Bergen in Norway and Vila Real in Portugal.

Aims of the study are:

To investigate the feasibility of video-recorded Transnasal fiberoptic laryngoscopic evaluation of the upper airways applied during NIV titration, and to describe the upper airway responses to NIV treatment in patients with ALS.

To explore whether the endoscopic view of the upper airways will influence the titration of NIV settings in ALS patients.

To explore whether NIV titration with endoscopic evaluation will influence NIV compliance and ventilation effectiveness in ALS patients.

• Study Type: Interventional
• Enrollment (Anticipated): 60
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Norway
  • Hordaland
    • Bergen, Hordaland, Norway, 5021
      • Norwegian National Advisory Unit on Long-term Mechanical Ventilation at Thoracic Department, Haukeland University Hospital
• Portugal
  • Vila Real, Portugal
    • Centro Hospitalar Tras-os-Montes e Alto Douro

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patients diagnosed Amyotrophic Lateral Sclerosis (ALS) due to El Escorial criteria
• Must have already established Non-invasive ventilation (NIV)

Exclusion Criteria:

• Ages under 18 years
• Unstable ischemic heart disease
• Oncological disease
• Ventilatory support dependency (more than 16h per day)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: NIV settings titration; Transnasal Fiberoptic Laryngoscopy will be used during ongoing NIV setting titration. Aim is to titrate NIV setting to be more optimal for laryngeal responses.	Other: Transnasal Fiberoptic Laryngoscopy; Use of Transnasal Fiberoptic Laryngoscopy during NIV titration to optimalize the NIV settings to laryngeal responses during the NIV treatment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Apnoea-hypopnea index (AHI)	Change in number of apnea and hypopnea events per hour of sleep	Baseline, one and three months after the intervention.
Time of NIV use	Change in median daily and average daily of hours, minutes	Baseline, one and three months after the intervention.
NIV Leaks	Change in median and 95%	Baseline, one and three months after the intervention.
NIV time of use	Change in % of time of use > than 4 hours/day	Baseline, one and three months after the intervention.
NIV compliance	Change in number of interruptions, complains and ventilator adjustments	Baseline, one and three months after the intervention.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Night-time pulseoximeter	Change in oxygen saturation of patients blood	Baseline, one and three months after the intervention.
Night-time capnography	Change in concentration or partial pressure of carbon dioxide in the respiratory gases	Baseline, one and three months after the intervention.

Collaborators and Investigators

• Sponsor: Tiina Maarit Andersen
• Collaborators: Haukeland University Hospital; Universidade do Porto; University of Trás-os-Montes and Alto Douro
• Investigators: Principal Investigator: Tiina M Andersen, PhD, Haukeland University Hospital; Principal Investigator: Bebiana Conde, PhD fellow, Centro Hospitalar Tras-os-Montes e Alto Douro

Publications and helpful links

General Publications

• Andersen TM, Sandnes A, Fondenes O, Clemm H, Halvorsen T, Nilsen RM, Tysnes OB, Heimdal JH, Vollsaeter M, Roksund OD. Laryngoscopy Can Be a Valuable Tool for Unexpected Therapeutic Response in Noninvasive Respiratory Interventions. Respir Care. 2018 Nov;63(11):1459-1461. doi: 10.4187/respcare.06674. No abstract available.
• Andersen T, Sandnes A, Brekka AK, Hilland M, Clemm H, Fondenes O, Tysnes OB, Heimdal JH, Halvorsen T, Vollsaeter M, Roksund OD. Laryngeal response patterns influence the efficacy of mechanical assisted cough in amyotrophic lateral sclerosis. Thorax. 2017 Mar;72(3):221-229. doi: 10.1136/thoraxjnl-2015-207555. Epub 2016 May 12.
• Andersen TM, Sandnes A, Fondenes O, Nilsen RM, Tysnes OB, Heimdal JH, Clemm HH, Halvorsen T, Vollsaeter M, Roksund OD. Laryngeal Responses to Mechanically Assisted Cough in Progressing Amyotrophic Lateral Sclerosis. Respir Care. 2018 May;63(5):538-549. doi: 10.4187/respcare.05924. Epub 2018 Apr 17.
• Conde B, Martins N, Brandao M, Pimenta AC, Winck JC. Upper Airway Video Endoscopy: Assessment of the response to positive pressure ventilation and mechanical in-exsufflation. Pulmonology. 2019 Sep-Oct;25(5):299-304. doi: 10.1016/j.pulmoe.2019.02.008. Epub 2019 Apr 16.
• Andersen TM, Halvorsen T, Fondenes O, Heimdal JH, Roksund OD, Vollsaeter M, Roksund OD. Larynx: The Complex Gateway to the Lungs. Respir Care. 2019 Jul;64(7):866-869. doi: 10.4187/respcare.06989. No abstract available.

Study record dates

Study Major Dates

• Study Start (Actual): March 21, 2019
• Primary Completion (Anticipated): December 31, 2021
• Study Completion (Anticipated): December 31, 2022

Study Registration Dates

• First Submitted: March 15, 2019
• First Submitted That Met QC Criteria: March 21, 2019
• First Posted (Actual): March 22, 2019

Study Record Updates

• Last Update Posted (Actual): March 2, 2021
• Last Update Submitted That Met QC Criteria: March 1, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Keywords: Noninvasive Ventilation; Laryngoscopy
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 2018/1306; 39/2016 (Centro Hospitalar Trás-os-Montes e Alto Douro, Vila Real)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: Since the study examines patients with a rare disease in the defined geographical areas, we need to be careful not to share data that can be identifiable. A study protocol, informed consent forms and data analysis plan will be available within 6 months of study completion.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No