Clinical trial NCT02750501: Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Official title: Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study in Patients With Cystic Fibrosis Receiving Enteral Feeding
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cystic Fibrosis
Keywords: CF, EPI, diabetes, PERT, LCPUFA, DHA, EPA, Omega-3 index
Lead sponsor: Alcresta Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2016-04-25
Enrollment: 49
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change From Baseline of Erythrocyte Omega-3 Index % (DHA+EPA)
timeFrame: Day 0 to Day 90
Secondary outcomes:
measure: Unanticipated Adverse Device Effects (UADE)
timeFrame: RELiZORB Treatment Period (Day 0-Day 90): 90 days with additional 30 days of follow up.
measure: Changes in Plasma Concentration Total DHA+EPA
timeFrame: RELiZORB Treatment Period (Day 0-Day 90): 90 days
measure: Erythrocyte Composition (%) of DHA
timeFrame: RELiZORB Treatment Period (Day 0-Day 90): 90 days
measure: Erythrocyte Composition (%) of EPA
timeFrame: RELiZORB Treatment Period (Day 0-Day 90): 90 days
measure: Erythrocyte Composition (%) Ratio of n6/n3 Fatty Acids
timeFrame: RELiZORB Treatment Period (Day 0-Day 90): 90 days
measure: Plasma Composition (%) Ratio of n6/n3 Fatty Acids.
timeFrame: RELiZORB Treatment Period (Day 0-Day 90): 90 days
Other outcomes:
measure: GI Symptoms
timeFrame: Observation, Baseline and RELiZORB Treatment periods (Day -14 to Day 90): 104 days with additional 30 days of follow up.
Locations:
city: Hollywood
country: United States
facility: Joe DiMaggio Children's Hospital / Memorial Healthcare System
state: Florida
city: Boise
country: United States
facility: St. Luke's CF Center of Idaho
state: Idaho
city: Indianapolis
country: United States
facility: Riley Hospital for Children at Indiana University Health
state: Indiana
city: Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital CF Care Center
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Cardinal Glennon Children's Hospital / Saint Louis University
state: Missouri
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Dayton
country: United States
facility: Dayton Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
Study officials:
affiliation: Alcresta Therapeutics, Inc.
name: Madhumalli Sarkar, MD, PhD
role: STUDY_DIRECTOR
References: