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

Not Applicable

Completed

• Conditions: Cystic Fibrosis
• Interventions: Device: RELiZORB (immobilized lipase) cartridge; Other: Impact Peptide 1.5

• Registration Number: NCT02750501
• Lead Sponsor: Alcresta Therapeutics, Inc.

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2016-04-20
• Study First Submitted QC: 2016-04-21
• Study First Posted: 2016-04-25
• Study Start: 2016-07-20
• Primary Completion: 2017-03-30
• Study Completion: 2017-03-30
• Results First Submitted: 2018-03-30
• Status Verified: 2018-06
• Results First Submitted QC: 2018-06-18
• Last Update Submitted: 2018-07-17
• Results First Posted: 2018-07-18
• Last Update Posted: 2018-08-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 49

Inclusion Criteria

1. Confirmed diagnosis of cystic fibrosis
2. Documented history of exocrine pancreatic insufficiency
3. 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
4. Written informed consent or assent.

Exclusion Criteria

1. Uncontrolled diabetes mellitus
2. Signs and symptoms of liver cirrhosis or portal hypertension
3. Lung or liver transplant
4. Active cancer currently receiving cancer treatment
5. Crohn's or celiac disease, infectious gastroenteritis, sprue, lactose intolerance, inflammatory bowel disease

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Single Arm: Open label RELiZORB Cartridge & Impact Peptide 1.5	RELiZORB (immobilized lipase) cartridge	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.
Single Arm: Open label RELiZORB Cartridge & Impact Peptide 1.5	Impact Peptide 1.5	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Unanticipated Adverse Device Effects (UADE)	RELiZORB Treatment Period (Day 0-Day 90): 90 days with additional 30 days of follow up.	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.
Changes in Plasma Concentration Total DHA+EPA	RELiZORB Treatment Period (Day 0-Day 90): 90 days	Changes in plasma concentration total DHA+EPA from baseline (Day 0 to Day 90).
Erythrocyte Composition (%) of DHA	RELiZORB Treatment Period (Day 0-Day 90): 90 days	Changes over time in erythrocyte composition (%) for total DHA in ITT population (n=39)
Erythrocyte Composition (%) of EPA	RELiZORB Treatment Period (Day 0-Day 90): 90 days	Changes over time in erythrocyte composition (%) for EPA in ITT population
Erythrocyte Composition (%) Ratio of n6/n3 Fatty Acids	RELiZORB Treatment Period (Day 0-Day 90): 90 days	Change from baseline to Day 90 in n6/n3 ratio in erythrocytes
Plasma Composition (%) Ratio of n6/n3 Fatty Acids.	RELiZORB Treatment Period (Day 0-Day 90): 90 days	Change over time in n6/n3 ratio in plasma in the ITT population

Trial Locations

Locations (10)

Joe DiMaggio Children's Hospital / Memorial Healthcare System; 🇺🇸 Hollywood, Florida, United States

St. Luke's CF Center of Idaho; 🇺🇸 Boise, Idaho, United States

Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Riley Hospital for Children at Indiana University Health; 🇺🇸 Indianapolis, Indiana, United States

Cardinal Glennon Children's Hospital / Saint Louis University; 🇺🇸 Saint Louis, Missouri, United States

Helen DeVos Children's Hospital CF Care Center; 🇺🇸 Grand Rapids, Michigan, United States

Dayton Children's Hospital; 🇺🇸 Dayton, Ohio, United States

Maine Medical Center; 🇺🇸 Portland, Maine, United States

Children's Hospital of Pittsburgh of UPMC; 🇺🇸 Pittsburgh, Pennsylvania, United States

Children's Mercy Hospital; 🇺🇸 Kansas City, Missouri, United States