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

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ClinicalTrials.gov Identifier: NCT02750501
Recruitment Status : Completed
First Posted : April 25, 2016
Results First Posted : July 18, 2018
Last Update Posted : August 15, 2018
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Device: RELiZORB (immobilized lipase) cartridge Other: Impact Peptide 1.5 Not Applicable

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 49 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study in Patients With Cystic Fibrosis Receiving Enteral Feeding
Actual Study Start Date : July 20, 2016
Actual Primary Completion Date : March 30, 2017
Actual Study Completion Date : March 30, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Single Arm: Open label RELiZORB Cartridge & 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.
Device: RELiZORB (immobilized lipase) cartridge
Hydrolyzing fats from enteral formula, ex vivo, with in-line enteral feed RELiZORB (immobilized lipase) cartridge

Other: Impact Peptide 1.5
Impact Peptide 1.5 at a volume of administration from 500 mL to 1,000 mL per enteral feeding
Other Name: Enteral Formula




Primary Outcome Measures :
  1. Change From Baseline of Erythrocyte Omega-3 Index % (DHA+EPA) [ Time Frame: 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 :
  1. Unanticipated Adverse Device Effects (UADE) [ Time Frame: 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.

  2. Changes in Plasma Concentration Total DHA+EPA [ Time Frame: RELiZORB Treatment Period (Day 0-Day 90): 90 days ]
    Changes in plasma concentration total DHA+EPA from baseline (Day 0 to Day 90).

  3. Erythrocyte Composition (%) of DHA [ Time Frame: RELiZORB Treatment Period (Day 0-Day 90): 90 days ]
    Changes over time in erythrocyte composition (%) for total DHA in ITT population (n=39)

  4. Erythrocyte Composition (%) of EPA [ Time Frame: RELiZORB Treatment Period (Day 0-Day 90): 90 days ]
    Changes over time in erythrocyte composition (%) for EPA in ITT population

  5. Erythrocyte Composition (%) Ratio of n6/n3 Fatty Acids [ Time Frame: RELiZORB Treatment Period (Day 0-Day 90): 90 days ]
    Change from baseline to Day 90 in n6/n3 ratio in erythrocytes

  6. Plasma Composition (%) Ratio of n6/n3 Fatty Acids. [ Time Frame: RELiZORB Treatment Period (Day 0-Day 90): 90 days ]
    Change over time in n6/n3 ratio in plasma in the ITT population


Other Outcome Measures:
  1. GI Symptoms [ Time Frame: Observation, Baseline and RELiZORB Treatment periods (Day -14 to Day 90): 104 days with additional 30 days of follow up. ]
    GI symptoms recorded in GI diaries by subject and/or caregiver.



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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02750501


Locations
United States, Florida
Joe DiMaggio Children's Hospital / Memorial Healthcare System
Hollywood, Florida, United States, 33021
United States, Idaho
St. Luke's CF Center of Idaho
Boise, Idaho, United States, 83712
United States, Indiana
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States, 46202
United States, Maine
Maine Medical Center
Portland, Maine, United States, 04102
United States, Michigan
Helen DeVos Children's Hospital CF Care Center
Grand Rapids, Michigan, United States, 49503
United States, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Cardinal Glennon Children's Hospital / Saint Louis University
Saint Louis, Missouri, United States, 63104
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Dayton Children's Hospital
Dayton, Ohio, United States, 45404-1815
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Sponsors and Collaborators
Alcresta Therapeutics, Inc.
Cystic Fibrosis Foundation
Investigators
Study Director: Madhumalli Sarkar, MD, PhD Alcresta Therapeutics, Inc.
  Study Documents (Full-Text)

Documents provided by Alcresta Therapeutics, Inc.:
Study Protocol  [PDF] March 7, 2016
Statistical Analysis Plan  [PDF] June 29, 2017


Responsible Party: Alcresta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02750501     History of Changes
Other Study ID Numbers: Protocol 0000498-02
First Posted: April 25, 2016    Key Record Dates
Results First Posted: July 18, 2018
Last Update Posted: August 15, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: No
Pediatric Postmarket Surveillance of a Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Alcresta Therapeutics, Inc.:
CF, EPI, diabetes, PERT, LCPUFA, DHA, EPA, Omega-3 index

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases