ClinicalTrials.gov
ClinicalTrials.gov Menu

Remote Pulmonary Function Testing in Amyotrophic Lateral Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03214224
Recruitment Status : Not yet recruiting
First Posted : July 11, 2017
Last Update Posted : August 30, 2017
Sponsor:
Information provided by (Responsible Party):
Zachary Simmons, Milton S. Hershey Medical Center

Brief Summary:

The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program.

The second part is a comparison of respiratory outcomes in patients receiving telemedicine-guided respiratory assessments monthly vs. those receiving standard of care. This is a randomized controlled study which assesses the effects rPFT on respiratory outcomes, quality of life, and survival.


Condition or disease Intervention/treatment Phase
ALS Device: remote pulmonary function testing Device: standard pulmonary function testing Not Applicable

Detailed Description:

The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program. The central hypothesis is that guided home assessment of respiratory function is a valid method for detecting respiratory insufficiency leading to noninvasive ventilation (NIV) recommendation. This study has the potential to transform the current practice of conducting breathing assessments every three months, resulting in timelier detection of respiratory insufficiency, thereby staining quality of life and lengthening survival. This protocol has the potential to demonstrate telemanagement exceeding the standards of ALS care.

The study has two parts, which patients can participate in either or both. The first part is a self-controlled study which will enroll 40 patients from the ALS clinic. On the day of their clinical visit, study participants will perform both a standard PFT as well as a simulated rPFT, both generating three valid repetitions of forced vital capacity (FVC) and maximal inspiratory pressure (MIP) procedures. The simulated rPFT will mimic the practice of home telemonitoring by having patients be instructed by a respiratory therapist over the telemanagement portal while in a research room within the ALS clinic. The primary hypothesis is that there is no difference in the results of PFT and rPFT for respiratory assessment of FVC and MIP.

The second part is a comparison of respiratory outcomes in patients receiving telemedicine-guided respiratory assessments monthly vs. those receiving standard of care. This is a randomized controlled study which assesses the effects rPFT on respiratory outcomes, quality of life, and survival. Forty patients with ALS will be randomized into two arms, both receiving standard respiratory assessments for pulmonary function every three months, and the experimental group additionally receiving rPFTs in the interim months over the course of a year. The primary hypothesis is that home respiratory assessment enables thresholds for NIV recommendation to be met in the experimental group significantly closer to the initiation criteria, at a time when quality of life is higher, compared to the control group.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 83 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

This study has two parts

  1. A self-controlled validation study of experimental remote PFT assessment vs standard of care
  2. A longitudinal parallel group study of repeated measures of remote PFTs and their impact on care decisions.
Masking: None (Open Label)
Primary Purpose: Health Services Research
Official Title: Remote Pulmonary Function Testing in Amyotrophic Lateral Sclerosis (ALS)
Estimated Study Start Date : September 2017
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : October 2019


Arm Intervention/treatment
Active Comparator: standard PFT
Those randomized to the control group will undergo standard pulmonary function testing as part of standard clinical procedure. In addition, they will complete monthly surveys pertaining to respiratory function.
Device: standard pulmonary function testing
Standard clinical delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)

Experimental: remote PFT (rPFT)
Those randomized to the experimental group will undergo standard pulmonary function testing as part of standard clinical procedure. They will also undergo regular interim remote pulmonary function testing using the telemedicine interface and study equipment. In addition, they will complete monthly surveys pertaining to respiratory function.
Device: remote pulmonary function testing
Telemedicine delivery of pulmonary function testing in ALS, including Forced Vital Capacity (FVC) and Maximal Inspiratory Pressure (MIP)




Primary Outcome Measures :
  1. (Part 1) standard PFT - Forced vital capacity [ Time Frame: One administration - 10 minutes ]
    Respiratory therapist will administer three valid maneuvers of forced vital capacity (FVC) The best FVC value is the outcome.

  2. (Part 1) standard PFT - Maximal inspiratory pressure [ Time Frame: One administration - 10 minutes ]
    Respiratory therapist will administer three valid maneuvers of maximal inspiratory pressure (MIP). The best MIP value is the outcome.

  3. (Part 1) remote PFT - Forced vital capacity [ Time Frame: One administration - 10 minutes ]
    Respiratory therapist will use the telehealth interface to guide the patient and caregiver to self-administer three valid FVC maneuvers. The best FVC value is the outcome.

  4. (Part 1) remote PFT - Maximal inspiratory pressure [ Time Frame: One administration - 10 minutes ]
    Respiratory therapist will use the telehealth interface to guide the patient and caregiver to self-administer three valid MIP maneuvers. The best MIP value is the outcome.

  5. (Part 2) Longitudinal standard PFT - FVC [ Time Frame: 10 minute assessment done approximately every 3 months for one year ]
    The result of standard FVC testing during clinical visits

  6. (Part 2) Longitudinal standard PFT - MIP [ Time Frame: 10 minute assessment done approximately every 3 months for one year ]
    The result of standard MIP testing during clinical visits

  7. (Part 2) Longitudinal remote PFT - FVC [ Time Frame: 15 minute assessment done at monthly intervals (except months when standard PFTs are done) for one year ]
    The results of remote FVC testing during in between clinical visits.

  8. (Part 2) Longitudinal remote PFT - MIP [ Time Frame: 15 minute assessment done at monthly intervals (except months when standard PFTs are done) for one year ]
    The results of remote MIP testing during in between clinical visits.


Secondary Outcome Measures :
  1. (Part 1) Patient and caregiver reported outcomes [ Time Frame: 10 minute survey administered following completion of standard and remote PFT of Part 1 ]
    Survey responses from the patient/caregiver pair. Likert-type scales are used to generate aggregate scores pertaining to: quality of communication over telehealth, confidence in procedures, and overall impressions.

  2. (Part 1) Therapist reported outcomes [ Time Frame: 10 minute survey administered following completion of standard and remote PFT of Part 1 ]
    Survey responses from the respiratory therapist. Likert-type scales are used to generate aggregate scores pertaining to: quality of communication over telehealth, confidence in procedures, and overall impressions.

  3. (Part 2) Patient-reported outcome - Short Form 36 (SF-36) [ Time Frame: 10 minute survey done at monthly intervals for one year ]
    Patient and caregiver complete SF-36, a questionnaire of general health.

  4. (Part 2) Patient-reported outcome - Sleep Apnea Quality of Life Index (SAQLI) [ Time Frame: 5 minute survey done at monthly intervals for one year ]
    Patient and caregiver complete SAQLI, documentation of symptoms and severity associated with sleep apnea.

  5. (Part 2) Patient-reported outcome - Motor neuron disease dyspnoea rating scale (MDRS-D) [ Time Frame: 5 minute survey done at monthly intervals for one year ]
    Patient and caregiver complete MDRS-D, an ALS-specific questionnaire concerning breathlessness during activities of daily living.

  6. (Part 2) Survival Data [ Time Frame: During and one year following primary data collection period. ]
    Study participants will be followed after completion of study procedures and date of death, if available,will be recorded. Survival rates between the experimental and control groups will be compared.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Part 1

Patients:

  1. Possess a diagnosis of definite, probable, probable laboratory-supported, or possible ALS by revised El Escorial research criteria [Brooks2000].
  2. Be 18 years of age or older.
  3. Have a caregiver available to participate in the study

Caregivers:

  1. Be 18 years of age or older, of either gender.
  2. Be able and willing to provide informed consent.

Respiratory Therapist

  1. Be a member of the Hershey Medical Center ALS multidisciplinary care team.
  2. Be able and willing to provide verbal informed consent after receiving a summary explanation of research (SER).

Part 2 imposes additional inclusion criteria for patients only.

Patients:

4) Symptom onset within the last three years. 5) Intent to attend the Penn State Hershey ALS clinic every three months for the next year.

6) Have home wireless internet service sufficient for engaging in telemedicine sessions.

Exclusion Criteria:

  • Exclusion criteria are the same for both parts of the study.

Patients:

  1. Use of NIV or diaphragm pacer at time of obtaining informed consent.
  2. FVC ≤50% predicted or MIP > -60 cm H2O.
  3. ALS Functional Rating Scale (ALSFRS-R) [Cedarbaum1999] score on day of screening of ≥2 on items for speech, swallowing, and salivation. These items are indicators of bulbar dysfunction, which limits the reliability of PFT administration.
  4. Cognitive impairment, as judged by the ALS clinic neurologist, that prevents participation in the study.

Caregivers: None

Respiratory Therapists: None


Information from the National Library of Medicine

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): NCT03214224


Contacts
Contact: Andrew Geronimo, PhD 717-531-0003 ext 282576 geronimo@psu.edu
Contact: Anne Morris, MPH 717-531-0003 ext 289123 amorris2@pennstatehealth.psu.edu

Sponsors and Collaborators
Milton S. Hershey Medical Center

Responsible Party: Zachary Simmons, Professor of Neurology and Humanities, Milton S. Hershey Medical Center
ClinicalTrials.gov Identifier: NCT03214224     History of Changes
Other Study ID Numbers: STUDY00006924
First Posted: July 11, 2017    Key Record Dates
Last Update Posted: August 30, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: No plan at this time to share data with other researchers

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: No
Pediatric Postmarket Surveillance of a Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Zachary Simmons, Milton S. Hershey Medical Center:
Telemedicine
Pulmonary Function Testing
Telehealth

Additional relevant MeSH terms:
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases