Amyotrophic Lateral SclerosisALS AT HOMENCT03016897
ALS Testing Through Home-based Outcome Measures
An observational study of Amyotrophic Lateral Sclerosis, run by Jeremy Shefner at 1 site in United States.
Trial summary
ALS AT HOME is a single-center study of up to 150 participants being done to determine the extent to which frequent sampling can improve the qualities of outcome measures collected at home by study participants. Read more about this study
- Who is it for?
- Ages 18 to 85 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
- How many people
- 144 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Feb 2017to 30 Sep 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: ALS patient volunteers will be invited to participate in this study as well as healthy subjects for the control group.
Who may be able to join
- male or female, age 18 to 85 years old,
- ownership of a Smart Device (phone, tablet, etc) with Bluetooth capabilities,
- continuous internet access at home,
- willing and able to provide informed consent in compliance with the regulatory requirements through a web based interface,
- definite, Probable, or Possible ALS by modified El Escorial criteria, as documented by medical records, with duration from diagnosis 60 months or less, and
- for the 50 participants in Group 2, participation in the Answer ALS study.
Who cannot join
- diagnosed and actively undergoing treatment for cancer, heart failure, end stage renal disease, or another significant medical condition deemed by the PI to likely affect the participant's ability to comply with the protocol,
- unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the participant's ability to comply with the protocol,
- any other reasons that, in the opinion of the PI, the candidate is determined to be unsuitable for entry into the study.
- Healthy volunteers who have family members with ALS
Trial locations and contacts
1 site in 1 country.
United States1 site
Barrow Neurological Institute
Phoenix, Arizona, 85013
About this study
Official title: ALS Testing Through Home-based Outcome Measures (ALS AT HOME)
ALS AT HOME is a single-center study of up to 150 participants being done to determine the extent to which frequent sampling can improve the qualities of outcome measures collected at home by study participants.
Read the detailed description
ALS AT HOME is a single-center study of up to 150 participants being done to determine the extent to which frequent sampling can improve the qualities of outcome measures collected at home by study participants.
The objectives of this study are to
- assess the extent to which frequent sampling can reduce variability for the following outcome measures in ALS patients: ALSFRS-R, quantitative hand grip, pulmonary function, EIM in 4 extremities, and actigraphy and voice/speech tracking;
- assess the compliance of ALS patients in obtained outcome measures at home over the course of 9 months;
- directly compare outcome measures collected by patients with measurements obtained at study sites at less frequent intervals by co-enrolling 50 patients participating in the Answer ALS study; and
- provide information to Answer ALS to more fully characterize patients in that study.
The study proposed here will assess the extent to which reducing variability of measurement through frequent sampling can reduce both sample size and duration of clinical trials, provide a first-of-its-kind detailed assessment of disease progression, and evaluate an entirely new paradigm for patient participation in such trials without the need to be geographically yoked to a clinical study center. An important component of this proposal is to jointly recruit 50 patients who also participate in the Answer ALS study that intends to generate 1000 IP cell lines from patients who receive ongoing assessment at 5 clinical sites. The 100 participants that will be co-enrolled in Answer ALS and ALS AT HOME will perform outcome measures at home in addition to having outcome measures performed at clinic sites through Answer ALS participation. Therefore, the investigators will be able to directly assess how the measures obtained by patients at home relate to those obtained by a trained evaluator at a study center using standard metrics. The investigators will also provide all data generated at home to the Answer ALS investigators, further increasing the phenotypic data available for that project.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
- Keywords
- ALS, neurodegenerative
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeterOther
Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Participants enrolled solely in ALS AT HOME Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter Receives: Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter | |
| Group 2 | Current participants of the Answer ALS study Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter Receives: Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter | |
| Group 3 | Participants without neurological disease (controls) Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter Receives: Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Pulmonary Function
Respirometer
Measured: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Change in Quantitative Hand Grip
Digital Handgrip Meter
Measured: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Change in Electrical Impedance Myography (EIM) Measurements
Skulpt Chisel
Measured: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Change in Actigraphy
Activity Band
Measured: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
ALSFRS-R
Questionnaire
Measured: Weekly up to 9 months
Change in Patient-reported Experience measures (PREMs)
Questionnaire
Measured: At Week 1, and then at 3, 6, and 9 months
Adverse Events
Questionnaire
Measured: Monthly up to 9 months
Change in Voice/Speech Tracking
Smartphone application
Measured: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2017 (estimated)
- Study start22 Feb 2017
- Primary completion28 Jun 2019
- Study completion30 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 144 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jeremy Shefner
- Sponsor type
- University, hospital or other
- Collaborators
- Beth Israel Deaconess Medical Center
- Responsible party
- Sponsor-investigator, Jeremy Shefner, Barrow Neurological Institute
- Investigators
- Jeremy Shefner, MD, Principal investigator, Barrow Neurological Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03016897
- Sponsor's protocol ID
- BNI_ALS_001
- Acronym
- ALS AT HOME
- Status last verified
- Feb 2023
- First submitted
- 22 Dec 2016
- Last updated
- 8 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: IPD may be shared with researchers conducting the Answer ALS study. It will be available through the GUID.
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ALS Testing Through Home-based Outcome Measures (ALS AT HOME)
- ClinicalTrials.gov ID
- NCT03016897
- Acronym / sponsor ID
- ALS AT HOME / BNI_ALS_001
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, neurodegenerative
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 144 participants (actual)
- Lead sponsor
- Jeremy ShefnerUniversity, hospital or other
- Collaborators
- Beth Israel Deaconess Medical Center
- Study start
- 22 Feb 2017 (actual)
- Primary completion
- 28 Jun 2019 (actual)
- Study completion
- 30 Sep 2019 (actual)
- First posted
- 11 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
ALS AT HOME is a single-center study of up to 150 participants being done to determine the extent to which frequent sampling can improve the qualities of outcome measures collected at home by study participants.
Detailed description
ALS AT HOME is a single-center study of up to 150 participants being done to determine the extent to which frequent sampling can improve the qualities of outcome measures collected at home by study participants.
The objectives of this study are to
- assess the extent to which frequent sampling can reduce variability for the following outcome measures in ALS patients: ALSFRS-R, quantitative hand grip, pulmonary function, EIM in 4 extremities, and actigraphy and voice/speech tracking;
- assess the compliance of ALS patients in obtained outcome measures at home over the course of 9 months;
- directly compare outcome measures collected by patients with measurements obtained at study sites at less frequent intervals by co-enrolling 50 patients participating in the Answer ALS study; and
- provide information to Answer ALS to more fully characterize patients in that study.
The study proposed here will assess the extent to which reducing variability of measurement through frequent sampling can reduce both sample size and duration of clinical trials, provide a first-of-its-kind detailed assessment of disease progression, and evaluate an entirely new paradigm for patient participation in such trials without the need to be geographically yoked to a clinical study center. An important component of this proposal is to jointly recruit 50 patients who also participate in the Answer ALS study that intends to generate 1000 IP cell lines from patients who receive ongoing assessment at 5 clinical sites. The 100 participants that will be co-enrolled in Answer ALS and ALS AT HOME will perform outcome measures at home in addition to having outcome measures performed at clinic sites through Answer ALS participation. Therefore, the investigators will be able to directly assess how the measures obtained by patients at home relate to those obtained by a trained evaluator at a study center using standard metrics. The investigators will also provide all data generated at home to the Answer ALS investigators, further increasing the phenotypic data available for that project.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeterParticipants enrolled solely in ALS AT HOME Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter |
| Group 2 | – | Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeterCurrent participants of the Answer ALS study Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter |
| Group 3 | – | Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeterParticipants without neurological disease (controls) Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- ALS patient volunteers will be invited to participate in this study as well as healthy subjects for the control group.
Key inclusion criteria (6 listed)
- male or female, age 18 to 85 years old,
- ownership of a Smart Device (phone, tablet, etc) with Bluetooth capabilities,
- continuous internet access at home,
- willing and able to provide informed consent in compliance with the regulatory requirements through a web based interface,
- definite, Probable, or Possible ALS by modified El Escorial criteria, as documented by medical records, with duration from diagnosis 60 months or less, and
Key exclusion criteria (4 listed)
- diagnosed and actively undergoing treatment for cancer, heart failure, end stage renal disease, or another significant medical condition deemed by the PI to likely affect the participant's ability to comply with the protocol,
- unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the participant's ability to comply with the protocol,
- any other reasons that, in the opinion of the PI, the candidate is determined to be unsuitable for entry into the study.
- Healthy volunteers who have family members with ALS
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeterRespirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter | Other | Group 1, Group 2, Group 3 |
Primary outcome
Change in Pulmonary Function
Time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Respirometer
Change in Quantitative Hand Grip
Time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Digital Handgrip Meter
Change in Electrical Impedance Myography (EIM) Measurements
Time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Skulpt Chisel
Change in Actigraphy
Time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Activity Band
ALSFRS-R
Time frame: Weekly up to 9 months
Questionnaire
Change in Patient-reported Experience measures (PREMs)
Time frame: At Week 1, and then at 3, 6, and 9 months
Questionnaire
Adverse Events
Time frame: Monthly up to 9 months
Questionnaire
Change in Voice/Speech Tracking
Time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Smartphone application
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2023.
- Individual participant dataSharing planned: Yes. IPD may be shared with researchers conducting the Answer ALS study. It will be available through the GUID.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeremy Shefner, MD principal investigator
Barrow Neurological Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis, run by Jeremy Shefner at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Pulmonary Function (time frame: Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Feb 2023.