Primary objective: To assess the feasibility and adherence of home-based monitoring of multiple aspects of disease progression in patients with ALS in order to develop a widely supported set of reliable digital parameters to capture data, collected…
ID
Source
Brief title
Condition
- Neuromuscular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Patient adherence defined as the number of completed measurements divided by
the total number of assessments. Adherence will be assessed per parameter.
Secondary outcome
Overall survival, defined as time to death from any cause or respiratory
insufficiency (RI; defined as tracheostomy or the use of non-invasive
ventilation for >22 h per day for >=10 consecutive days). Daily functioning,
defined as mean change from baseline in ALSFRS-R total score and PRO-ALS score.
Respiratory function, defined as mean change from baseline in VC (% predicted
of normal according to the GLI-2012 reference standard) as measured by
spirometry, and MND-DS. Physical function, defined as mean change from baseline
in vertical variation as assessed by accelerometry. Bulbar function, defined as
mean change from baseline in acoustic measures (e.g., rate, duration, voicing)
and visual measures (e.g., higher order statistics of the jaw and lip) as
assessed by audio-visual speech measures. Gastrostomy placement, defined as
time-to-gastrostomy since enrollment. Non-invasive ventilation use, defined as
time-to-non-invasive ventilation initiation since enrollment. Body weight,
defined as mean change from baseline in kilograms. Clinical disease stage,
defined as mean time spent in each stage of the King*s and ALS Milano-Torino
staging systems. Patient burden and user-experience as assessed by custom-made
questionnaire. Patient reported outcomes measures as assessed by global
assessment ratings in a custom-made questionnaire.
Background summary
Amyotrophic lateral sclerosis (ALS) is a debilitating disease, making trial
participation particular challenging. Implementing a remote, home-based
monitoring infrastructure to assess trial outcomes and drug safety could make
in-clinic visits superfluous, significantly lower the trial burden, and pave
the way to a patient-centric clinical trial model.
Study objective
Primary objective:
To assess the feasibility and adherence of home-based monitoring of multiple
aspects of disease progression in patients with ALS in order to develop a
widely supported set of reliable digital parameters to capture data, collected
under free-living conditions, that reflect patient-centric measures, and can be
used to develop effective therapies.
Key secondary objectives:
To assess the discriminative value of remote digital endpoints in
distinguishing different subtypes of motor neuron disease (i.e. fast versus
slow progressing patients)
To assess the predictive value of remote digital endpoints for clinically
relevant outcomes (e.g. death, wheelchair dependency or respiratory
insufficiency)
To assess the validity of unsupervised, home-based vital capacity assessment
versus supervised in-clinic assessment.
Estimate the Minimal Important Difference (MID) of each digital parameter
Study design
Longitudinal cohort study conducted in the UMC Utrecht with a 12-month
follow-up period.
Study burden and risks
All procedures in this study are non-invasive and there is no direct
physiological discomfort associated with participation. Patients are
followed-up at monthly intervals and evaluated digitally at home for body
weight, spirometry, accelerometery, audio-visual speech measures, and clinical
progression. At baseline visit and at month 3 (at the patients* home or
in-clinic) a healthcare professional will supervise the patients* spirometry
testing. After completion or premature ending of the study, a close-out visit
(at the patients' home or in-clinic) will be scheduled. We estimated that study
participation takes approximately 13 hours distributed over a 12-month time
period. Development of reliable validated technological parameters is paramount
to lower trial burden, and may advance the search for effective treatment.
Given the low risk and burden of this study, we believe that the benefits
outweigh the risks.
Heidelberglaan 100
Utrecht 3584 CX
NL
Heidelberglaan 100
Utrecht 3584 CX
NL
Listed location countries
Age
Inclusion criteria
1. Diagnosis with ALS according to the Gold Coast criteria
2. ENCALS risk profile ranging between -6 and -2
3. 18 years or older
4. Able to provide informed consent
Exclusion criteria
Fulfilling the criteria for respiratory insufficiency (non-invasive ventilation
use >22 hours per day for 10 consecutive days or having a tracheostomy)
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL79677.041.22 |