An interventional study of Medical Food in Amyotrophic Lateral Sclerosis, Gastrointestinal Intolerance and Enteral Nutrition Therapy, sponsored by Andrea Charvet. Recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-04.
Sponsored by Andrea Charvet · Not applicable, Interventional, and Prevention
The main objective of the proposed study is to evaluate if oral intake of EN formula preceding Gtube placement will impact tolerance upon placement and feeding via Gtube in pALS. This single arm intervention study all participants will receive the intervention and researchers will utilize validated indicators combined with clinical expertise to assess gastrointestinal symptoms of feeding intolerance before and after the intervention.
The main questions this study aims to answer are:
This proposed study consists of three stages, as follows:
Brief Background:
Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disease. Over 82% of patients with ALS (pALS), develop dysphagia as disease progresses. Dysphagia leads to a decrease in oral intake associated with the fear of choking and long duration of meals, which results in weight loss and consequently malnutrition. Malnutrition is associated with a shorter survival time in pALS. Feeding via a gastrostomy tube (Gtube) bypasses oral intake and helps individuals maintain adequate nutrition. Research shows that Gtube usage significantly increases survival time in pALS. Despite a low risk for major complications with a Gtube placement, enteral nutrition (EN) intolerance is reported in 27-38% of hospitalized and critically ill patients. Intolerance, frequently defined by the presence of gastrointestinal (GI) symptoms, contributes to poor adherence with nutritional recommendations, further weight loss and greater risk of malnutrition. Currently, there is no data of the prevalence of EN intolerance among pALS or evidence on the impact of oral intake of EN formula on intolerance. Data from the Cathy J. Husman ALS Center at NSU Health show that 28.6% of the patients who had a Gtube tube placed changed EN formula due to intolerance.
Objectives:
The primary aim of this single-arm study is to evaluate whether oral intake of EN formula preceding Gtube placement will impact tolerance upon placement and feeding via Gtube in pALS. GI symptoms and dietary intake will be assessed before and after the intervention in a proposed sample of 22 pALS. This study involves a multidisciplinary collaboration of professionals in our clinic. It was designed with the teams expertise and includes pertinent indicators to assess EN intolerance in a population with great needs and insufficient research. Potential therapeutic strategies learned from this study can be incorporated into clinical practice guidelines much needed in the management of Gtube usage to minimize malnutrition, increase survival time, and improve quality of life in pALS.
Specific Aims:
Aim 1: Analyze dietary intake before and after placement and feeding via Gtube and determine its relationship with disease progression and GI symptoms of feeding intolerance.
Aim 2: Evaluate GI symptoms of feeding intolerance at baseline, during oral intake of EN formula, and after placement and feeding via Gtube.
Study Design:
In this single arm intervention study participants will be recruited during their routine multidisciplinary visit at the NSU Health ALS Center. The intervention will take place as part of the daily feeding routine of the patients, at their desired location. This proposed study consists of three stages: pre-intervention, phase I, and phase II. Assessments will be completed by the patients and/or their caregivers at home and collected weekly by research personnel via phone calls and email. The primary outcome measure will be gastrointestinal symptoms of EN intolerance. Secondary outcome measures include dietary intake, body mass index (BMI), and ALS Functional Rating Scale (ALSFRS-R). Treatment fidelity will be assessed at the end of the study.
Experimental Procedures and Outcome Measures
This proposed study consists of three stages, as follows:
Demographics:
At baseline, the following demographic and clinical information will be obtained: age, biological sex, race, ethnicity, ALS disease onset type and duration (months), height, weight, body mass index (BMI), malnutrition risk (change in BMI from first appointment to current), overall disease severity as indexed by the validated the ALS Functional Rating Scale (ALSFRS) score, and disease progression rate (change in ALSFRS from first appointment to current).
Dietary Intake:
To determine dietary intake of each participant, a food diary will be completed by the participant, with or without the aid of a caregiver, for the duration of the study. Patients and caregivers will be provided instructions on how to complete the food diary and will be directed to record intake at the time of consumption. For each week, the food diary must be completed for a minimum of 3 days and a maximum of 7 days. The weekly intake of selected nutrients will be averaged. A diet analysis software will be utilized to analyze the dietary intake information obtained. Nutritionist Pro, by Axxya Systems, is a software application for nutrition technology and data that uses the Nutrition Raw Database of Foods and Beverages to calculate nutrition values, a system compliant with USA requirements.
Gastrointestinal Symptoms:
Gastrointestinal symptoms of feeding intolerance will be monitored weekly using a clinician-administered survey. The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item scale that combines each item into five symptom clusters including reflux, abdominal pain, indigestion, diarrhea, and constipation. Patients rate each item on a 7-point Likert scale (from no discomfort at all to very severe discomfort). The recall period is seven days, and it is estimated to take about 3 to 5 minutes to complete. The clinical team will also be monitoring study participants for any significant gastrointestinal event that requires medical attention at any point in time during the study. These will be recorded when the patient contacts the clinic for medical attention, or patient-reported if medical attention is sought elsewhere.
Data Analysis Descriptive statistics will be used to explore dietary intake and the prevalence of GI symptoms of feeding intolerance in pALS. Dietary intake and prevalence of GI symptoms of feeding intolerance before and after placement and feeding via Gtube will be compared within subjects using paired t-test. To assess the relationships between the percentage of estimated nutritional needs met at baseline and disease progression indicators, as well as the incidence of GI symptoms of feeding intolerance after placement and feeding via Gtube, Pearson\'s correlation will be used (Aim 1). To determine the change in GI symptoms of feeding intolerance at baseline, during oral intake of enteral nutrition formula, and after placement and feeding via Gtube, a one-way repeated measures analysis of variance (ANOVA) will be conducted (Aim2). Statistical analysis will be conducted using SPSS (SPSS Statistics V29, IBM) with statistical significance at p-value less than 0.05.
981 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 283 are open to participants now.
This study's planned enrollment of 22 is below the median of 36 across 667 interventional studies indexed under Amyotrophic Lateral Sclerosis.
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Exclusion Criteria:
This is a single arm study in which all participants receive the intervention.
Other: Medical Food
Enteral nutrition formula will be used both by mouth and via feeding tube. This product is considered a medical food.
Gastrointestinal Symptoms
Gastrointestinal symptoms of feeding intolerance will be monitored weekly using a clinician-administered survey in-person or via phone interview. The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item scale that combines each item into five symptom clusters including reflux, abdominal pain, indigestion, diarrhea, and constipation (Svedlund et al., 1988). Patients rate each item on a 7-point Likert scale (from no discomfort at all to very severe discomfort).
Time frame: 5 weeks
Dietary Intake
To determine dietary intake of each participant, a food diary will be completed by the participant, with or without the aid of a caregiver, for the duration of the study. Patients and caregivers will be provided instructions on how to complete the food diary and will be directed to record intake at the time of consumption. For each week, the food diary must be completed for a minimum of 3 days and a maximum of 7 days. The weekly intake of selected nutrients will be averaged. A diet analysis software will be utilized to analyze the dietary intake information obtained. Nutritionist Pro, by Axxya Systems, is a software application for nutrition technology and data that uses the Nutrition Raw Database of Foods and Beverages to calculate nutrition values, a system compliant with USA requirements.
Time frame: 5 weeks
Plan to share: No — The study protocol does not include a plan to share IPD.
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