Clinical trial NCT02418546: Electronic-health Application To Measure Outcomes REmotely Clinical Trial
Official title: Electronic-health Application To Measure Outcomes REmotely Clinical Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Neurodegenerative Disease
Weight Loss
Amyotrophic Lateral Sclerosis
Parkinson's Disease
Huntington's Disease
Cachexia
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: ALS Association
First posted: 2015-04-16
Enrollment: 78
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: This is a phase II feasibility, safety, tolerability and preliminary efficacy study of an e-Health application versus in-person nutritional counseling to maintain or increase weight in patients with neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), Parkinson's Disease (PD) and Huntington's disease (HD). Primary Objectives include the feasibility, safety, tolerability and efficacy of an e-Health application to maintain or increase body weight compared to in-person nutritional counseling. Secondary Objectives are to measure the number of calories required to maintain or increase body weight in neurodegenerative diseases at all stages of the disease. Tertiary Objectives are to test the effects of an e-Health application compared to in-person nutritional counseling on disease progression using the ALSFRS-R, UHDRS or UDysRS, on survival, and on quality of life using the PROMIS SF v1.1 scale.

Detailed description: This is a single center, randomized, open-label, placebo controlled, parallel group study. The study treatment period is approximately 6 months and observation period is approximately 7 months. There are three parallel treatment groups: in-person nutritional counseling, nutritional counseling using an e-Health App, and standard care. Subjects will be randomly assigned in a 1:1:1 ratio using a computer-generated randomization scheme. Subjects in the two intervention arms will be given caloric recommendations designed to cause weight stability or modest weight gain, depending on their current BMI and weight loss history. All participants in the intervention arms will receive counseling either in-person or remotely through the App by registered dietitians (RD) working in the Bionutrition department of the Clinical Research Center at MGH. At MGH, approximately 150 ALS subjects (50 per treatment group) will be enrolled. Approximately 75-150 PD and HD subjects will enroll.

Eligibility criteria: Inclusion Criteria:

1. Adults with neurodegenerative diseases such as ALS, PD or HD with or without a history of unintentional weight loss.
2. Male or female subjects aged 18 years or older.
3. Participants must be capable of providing informed consent and complying with trial procedures.
4. Participants must have an MGH swallowing screening tool score\>5 at the time of the screening visit
5. Participants or a designated caregiver must be able to obtain home weights and communicate to their RD

Exclusion Criteria:

1. Clinical evidence of unstable medical or psychiatric illness, in the investigator's judgment, which would prevent the participant from completing their assessments.
2. BMI \> 35 combined with a history of cardiovascular disease; or a history of diabetes regardless of BMI.

Primary outcomes:
measure: Estimated Mean Change in Weight From Baseline to 6 Months
timeFrame: Change over time from Baseline to 6 months
Secondary outcomes:
measure: Change in Calorie Intake Over Time
timeFrame: Change from baseline over 6 months
measure: Safety: Frequency of Adverse Events
timeFrame: From baseline to month 7 (one month after 6 month end of study visit)
measure: Tolerability: The Number of Participants Who Complete the Study While Complying With at Least 80% of the Counseling Sessions
timeFrame: Baseline, 3 months and 6 months
Other outcomes:
measure: Exploratory Efficacy Measure: Survival
timeFrame: baseline to 18 months
measure: Exploratory Efficacy Measure: Disease Progression in ALS Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: Change over time from Baseline to 6 months
measure: Exploratory Efficacy Measure: Quality of Life
timeFrame: 18 months
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Anne-Marie Wills, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Wills AM, Garry J, Hubbard J, Mezoian T, Breen CT, Ortiz-Miller C, Nalipinski P, Sullivan S, Berry JD, Cudkowicz M, Paganoni S, Chan J, Macklin EA. Nutritional counseling with or without mobile health technology: a randomized open-label standard-of-care-controlled trial in ALS. BMC Neurol. 2019 May 29;19(1):104. doi: 10.1186/s12883-019-1330-6.