Study of LYM-X-SORB™ to Improve Fatty Acid and Choline Status in Children With CF and PI

Study Overview
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Brief Summary
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The purpose of this phase II research study is to evaluate the effectiveness of the next generation LYM-X-SORB™ in improving the essential fatty acid (EFA) and choline status for children and adolescents with Cystic Fibrosis (CF) and pancreatic insufficiency (PI).

Detailed Description
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Fat malabsorption is common in individuals with cystic fibrosis (CF) and pancreatic insufficiency (PI). This places them at risk for caloric, essential fatty acid, and choline deficiency, which may in turn, lead to growth failure and a poorer clinical course. The purpose of this research study is to find whether or not taking LYM-X-SORB™ over an 18-month period, every day, will correct the problem people with CF and PI have with absorbing fat and choline. Participation will help CF doctors and other healthcare professionals learn more about the potential benefits of LYM-X-SORB™ to children and adolescents with CF and PI. These benefits may include better absorption of fat, better choline status, better growth in height, weight, muscle and bone, better lungs, and improvement of health status. The study will enroll a total of 78 participants from Children's Hospital of Philadelphia (CHOP) and from several other Cystic Fibrosis Centers. One half of the participants will be randomly picked to receive the active powder with the LYM-X-SORB™ supplement and one half will receive a placebo (with no active supplement).

Official Title
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Phase II Study: LYM-X-SORB™, an Organized Lipid Matrix: Fatty Acids and Choline in CF

Conditions
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Cystic Fibrosis, Pancreatic Insufficiency

Intervention / Treatment
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* Dietary Supplement: Lym-X-Sorb powder
* Dietary Supplement: Placebo powder


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed as having cystic fibrosis (CF) with pancreatic insufficiency (PI) Subjects aged 6 to 17 years of age In usual state of good health Family and subject commitment to the 18-month study protocol Fecal elastase < 15µg/g stool Exclusion Criteria: Forced expiratory volume at one second, % predicted (FEV1) < 40% predicted Other chronic health conditions that may affect GI absorption, growth, dietary intake, nutritional status Liver disease, lung transplant, celiac disease, allergy/intolerance to wheat/gluten, pregnant Participation in another CF nutrition-related intervention study Regular intake of fatty acids (i.e., fish oils) or choline nutritional supplements Home parenteral lipid administration (i.e., intralipids)

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 1<br> | Dietary Supplement: Lym-X-Sorb powder<br>* Lym-X-Sorb is an organized matrix of lyso phosphatidylcholine (LPC), free fatty acid (FFA) and monoglyceride formulated at 20wt% with flour and sugar. A dosage contains 32 grams of powder and is consumed with food twice a day (6 to 11.9 years old) or three times per day (12 to 17.9 years old) for 18 months.<br>|
| Placebo Comparator: 2<br> | Dietary Supplement: Placebo powder<br>* The placebo is composed of soybean oil, sunflower oil, fully hydrogenated cottonseed oil and flax seed oil dispersed at 16wt% on flour and sugar. The placebo is >99% triglycerides with no trans fatty acids. The fatty acid composition and caloric content is designed to match the active supplement (Lym-X-Sorb). A dosage contains 32 grams of powder and is consumed with food twice a day (6 to 11.9 years old) or three times per day (12 to 17.9 years old) for 18 months.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Improvements in essential fatty acid (EFA) and choline status over a 12-month period. |  | 12-month period | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Improvements in growth, nutritional, and pulmonary status over an 18-month period. |  | 18-month period | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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LXS, Cystic Fibrosis, Pancreatic Insufficiency, LYM-X-SORB™