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| Name | Class |
|---|---|
| ALS Association | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| In-Person Nutritional Counseling | Active Comparator | Participants in the in-person nutritional counseling arm will meet with a Registered Dietitian at every clinic visit and will also receive regular phone calls to monitor weight and food intake. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. |
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| E-Health App for Nutritional Counseling | Experimental | Participants randomized to the e-Health App arm with receive nutritional counseling using the e-Health Application. Participants will enter weights at home and complete electronic food records using the App. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. Participants will receive these calorie recommendations through the Application. |
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| Standard Care | No Intervention | Participants are allowed to receive all usual treatments and medications. Participation in other research studies is allowed. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| In-Person Nutritional Counseling by a Registered Dietitian | Behavioral |
| ||
| Nutritional counseling using an e-Health Application |
| Measure | Description | Time Frame |
|---|---|---|
| Estimated Mean Change in Weight From Baseline to 6 Months | The Primary Aim is to study the feasibility and efficacy to maintain or increase body weight of an e-Health application and in-person nutritional counseling compared to standard of care and to each other. Weights were measured in the clinic every 3 months. | Change over time from Baseline to 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Calorie Intake Over Time | Secondary aims include measuring the number of calories required to maintain or increase body weight in patients with neurodegenerative diseases. Total daily energy intake was calculated using 4 day food records at baseline, 3 months and 6 months | Change from baseline over 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Exploratory Efficacy Measure: Survival | Vital status will be measured until the last subject last visit. | baseline to 18 months |
| Exploratory Efficacy Measure: Disease Progression in ALS Functional Rating Scale-Revised (ALSFRS-R) |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Anne-Marie Wills, MD | Massachusetts General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31142272 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | In-Person Nutritional Counseling | Participants in the in-person nutritional counseling arm will meet with a Registered Dietitian at every clinic visit and will also receive regular phone calls to monitor weight and food intake. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. In-Person Nutritional Counseling by a Registered Dietitian |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 10, 2016 | Aug 7, 2019 |
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| Behavioral |
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| Safety: Frequency of Adverse Events |
To study the safety of an e-Health application and in-person nutritional counseling compared to standard of care and to each other. |
| From baseline to month 7 (one month after 6 month end of study visit) |
| Tolerability: The Number of Participants Who Complete the Study While Complying With at Least 80% of the Counseling Sessions | Baseline, 3 months and 6 months |
Disease progression will be measured using disease-specific outcome measures (the ALSFRS-R scale). The range of the ALSFRS-R is 0-40 with higher scores indicating better function. Change in ALSFRS-R is reported as units/month.
| Change over time from Baseline to 6 months |
| Exploratory Efficacy Measure: Quality of Life | Quality of life will be measured using the PROMIS SF 1.1 in units. | 18 months |
| FG001 | E-Health App for Nutritional Counseling | Participants randomized to the e-Health App arm with receive nutritional counseling using the e-Health Application. Participants will enter weights at home and complete electronic food records using the App. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. Participants will receive these calorie recommendations through the Application. Nutritional counseling using an e-Health Application |
| FG002 | Standard Care | Participants are allowed to receive all usual treatments and medications. Participation in other research studies is allowed. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | In-Person Nutritional Counseling | Participants in the in-person nutritional counseling arm will meet with a Registered Dietitian at every clinic visit and will also receive regular phone calls to monitor weight and food intake. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. In-Person Nutritional Counseling by a Registered Dietitian |
| BG001 | E-Health App for Nutritional Counseling | Participants randomized to the e-Health App arm with receive nutritional counseling using the e-Health Application. Participants will enter weights at home and complete electronic food records using the App. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. Participants will receive these calorie recommendations through the Application. Nutritional counseling using an e-Health Application |
| BG002 | Standard Care | Participants are allowed to receive all usual treatments and medications. Participation in other research studies is allowed. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
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| ALS Functional Rating Scale-Revised | Total score 0-40 with higher scores signifying better function | Mean | Standard Deviation | Total score in points |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Estimated Mean Change in Weight From Baseline to 6 Months | The Primary Aim is to study the feasibility and efficacy to maintain or increase body weight of an e-Health application and in-person nutritional counseling compared to standard of care and to each other. Weights were measured in the clinic every 3 months. | Please note that these are Parameter Estimates rather than "means" | Posted | Mean | 95% Confidence Interval | kg | Change over time from Baseline to 6 months |
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| Secondary | Change in Calorie Intake Over Time | Secondary aims include measuring the number of calories required to maintain or increase body weight in patients with neurodegenerative diseases. Total daily energy intake was calculated using 4 day food records at baseline, 3 months and 6 months | Not Posted | Change from baseline over 6 months | Participants | |||||||||||||||||||||||||||||||||||||
| Secondary | Safety: Frequency of Adverse Events | To study the safety of an e-Health application and in-person nutritional counseling compared to standard of care and to each other. | Not Posted | From baseline to month 7 (one month after 6 month end of study visit) | Participants | |||||||||||||||||||||||||||||||||||||
| Secondary | Tolerability: The Number of Participants Who Complete the Study While Complying With at Least 80% of the Counseling Sessions | Not Posted | Baseline, 3 months and 6 months | Participants | ||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Exploratory Efficacy Measure: Survival | Vital status will be measured until the last subject last visit. | Not Posted | baseline to 18 months | Participants | |||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Exploratory Efficacy Measure: Disease Progression in ALS Functional Rating Scale-Revised (ALSFRS-R) | Disease progression will be measured using disease-specific outcome measures (the ALSFRS-R scale). The range of the ALSFRS-R is 0-40 with higher scores indicating better function. Change in ALSFRS-R is reported as units/month. | Change over time of continuous, longitudinal measures was analyzed using a shared-baseline, mixed effect model with fixed effects for visit and an interaction between post-baseline visit and study arm and with a random slope and intercept for each subject with unstructured covariance. | Posted | Mean | 95% Confidence Interval | units per month | Change over time from Baseline to 6 months |
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| Other Pre-specified | Exploratory Efficacy Measure: Quality of Life | Quality of life will be measured using the PROMIS SF 1.1 in units. | Not Posted | 18 months | Participants |
7 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | In-Person Nutritional Counseling | Participants in the in-person nutritional counseling arm will meet with a Registered Dietitian at every clinic visit and will also receive regular phone calls to monitor weight and food intake. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. In-Person Nutritional Counseling by a Registered Dietitian | 4 | 26 | 4 | 26 | 9 | 26 |
| EG001 | E-Health App for Nutritional Counseling | Participants randomized to the e-Health App arm with receive nutritional counseling using the e-Health Application. Participants will enter weights at home and complete electronic food records using the App. Participants' individual dietary needs will be calculated using baseline calorie consumption, weight history, disease status and current activity level. Participants will receive these calorie recommendations through the Application. Nutritional counseling using an e-Health Application | 0 | 26 | 5 | 26 | 11 | 26 |
| EG002 | Standard Care | Participants are allowed to receive all usual treatments and medications. Participation in other research studies is allowed. | 1 | 26 | 2 | 26 | 12 | 26 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Deep Venous Thrombosis | Blood and lymphatic system disorders | MedDRA (19.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
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| Gastroparesis | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
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| Dysphagia | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
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| Lacunar Stroke | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
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| Ewing's Sarcoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| sialorrhea | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
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| Fatigue | General disorders | MedDRA (19.0) | Systematic Assessment |
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| Falls | Injury, poisoning and procedural complications | MedDRA (19.0) | Systematic Assessment |
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| Choking | Gastrointestinal disorders | MedDRA (19.0) | Systematic Assessment |
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| Dyspnea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Anne-Marie Wills | Massachusetts General Hospital | 6177265532 | awills@mgh.harvard.edu |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 11, 2018 | Feb 24, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D019636 | Neurodegenerative Diseases |
| D015431 | Weight Loss |
| D000690 | Amyotrophic Lateral Sclerosis |
| D010300 | Parkinson Disease |
| D006816 | Huntington Disease |
| D002100 | Cachexia |
| ID | Term |
|---|---|
| D009422 | Nervous System Diseases |
| D001836 | Body Weight Changes |
| D001835 | Body Weight |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D003704 | Dementia |
| D002819 | Chorea |
| D020820 | Dyskinesias |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| D013851 | Thinness |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| OG002 | Standard Care | Participants are allowed to receive all usual treatments and medications. Participation in other research studies is allowed. |
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