Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study

Study Overview
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Brief Summary
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Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB use, in patients with cystic fibrosis and exocrine pancreatic insufficiency.

Detailed Description
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Study Entry (Day -14): Baseline blood samples collected for plasma and erythrocyte concentrations of docosahexaenoic acid (DHA) and eicosapentaenoic (EPA). Baseline characteristics collected included BMI and cystic fibrosis related diabetes. Observation Period (Day -14 to Day -8): Subjects followed their usual enteral nutrition regimen with pancreatic enzyme replacement therapy (PERT). Run-in Period (Day -7 to Day -1): Subjects used Peptamen 1.5 enteral formula at their normal volume of administration from 500 mL to 1,000 mL per feeding with usual PERT regimen. Treatment Period (Day 0 to Day 90): Subjects used Impact Peptide 1.5 up to a maximum volume of 1,000 mL per feeding with RELiZORB for the 90 day treatment period. Blood screening measurements were repeated at start of treatment period (Day 0), Day 30, Day 60 and Day 90. PERT use with enteral feedings was prohibited. Safety and tolerability were assessed with GI symptom diaries and systematic assessments of adverse events and unanticipated adverse device effects.

Official Title
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Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study in Patients With Cystic Fibrosis Receiving Enteral Feeding

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Device: RELiZORB (immobilized lipase) cartridge
* Other: Impact Peptide 1.5


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed diagnosis of cystic fibrosis Documented history of exocrine pancreatic insufficiency Enteral formula use a minimum of 4x/week, using PERT, consuming an unrestricted fat diet, and willing to use Peptamen 1.5 and Impact Peptide 1.5 Written informed consent or assent. Exclusion Criteria: Uncontrolled diabetes mellitus Signs and symptoms of liver cirrhosis or portal hypertension Lung or liver transplant Active cancer currently receiving cancer treatment Crohn's or celiac disease, infectious gastroenteritis, sprue, lactose intolerance, inflammatory bowel disease

Ages Eligible for Study
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Minimum Age: 4 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: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Single Arm: Open label RELiZORB Cartridge & Impact Peptide 1.5<br>RELiZORB (immobilized lipase) cartridge and Impact Peptide 1.5 with enteral feedings ranging from 500 mL to 1,000 mL per feeding for a period of 90 days. | Device: RELiZORB (immobilized lipase) cartridge<br>* Hydrolyzing fats from enteral formula, ex vivo, with in-line enteral feed RELiZORB (immobilized lipase) cartridge<br>Other: Impact Peptide 1.5<br>* Impact Peptide 1.5 at a volume of administration from 500 mL to 1,000 mL per enteral feeding<br>* Other names: Enteral Formula;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline of Erythrocyte Omega-3 Index % (DHA+EPA) | Change from baseline Day 0 to Day 90 of erythrocyte tissue composition % of the omega-3 index | Day 0 to Day 90 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Unanticipated Adverse Device Effects (UADE) | A UADE is analogous to a serious adverse event (SAE), defined as an AE, occurring at any exposure to the therapeutic agent, that results in any of the following outcomes: death, life-threatening AE, inpatient hospitalization or prolonged existing hospitalization, a persistent or significant disability or incapacity or a congenital anomaly/birth defect. | RELiZORB Treatment Period (Day 0-Day 90): 90 days with additional 30 days of follow up. | 
| Changes in Plasma Concentration Total DHA+EPA | Changes in plasma concentration total DHA+EPA from baseline (Day 0 to Day 90). | RELiZORB Treatment Period (Day 0-Day 90): 90 days | 
| Erythrocyte Composition (%) of DHA | Changes over time in erythrocyte composition (%) for total DHA in ITT population (n=39) | RELiZORB Treatment Period (Day 0-Day 90): 90 days | 
| Erythrocyte Composition (%) of EPA | Changes over time in erythrocyte composition (%) for EPA in ITT population | RELiZORB Treatment Period (Day 0-Day 90): 90 days | 
| Erythrocyte Composition (%) Ratio of n6/n3 Fatty Acids | Change from baseline to Day 90 in n6/n3 ratio in erythrocytes | RELiZORB Treatment Period (Day 0-Day 90): 90 days | 
| Plasma Composition (%) Ratio of n6/n3 Fatty Acids. | Change over time in n6/n3 ratio in plasma in the ITT population | RELiZORB Treatment Period (Day 0-Day 90): 90 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CF, EPI, diabetes, PERT, LCPUFA, DHA, EPA, Omega-3 index