Trial Outcomes & Findings for Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study (NCT NCT02750501)
NCT ID: NCT02750501
Last Updated: 2018-08-15
Results Overview
Change from baseline Day 0 to Day 90 of erythrocyte tissue composition % of the omega-3 index
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
49 participants
Primary outcome timeframe
Day 0 to Day 90
Results posted on
2018-08-15
Participant Flow
Recruitment was conducted through Cystic Fibrosis Care Centers.
7-day Observation Period: Maintain usual enteral feeding (EF) regimen. 7-day Run-in period: Maintain usual EF volume up to a max of 1000 mL using standard formula for at least 5 days. 49 subjects signed consent; 5 screen failed; 44 started the observation period; 39 completed observation and run-in periods entering the treatment period.
Participant milestones
| Measure |
Single Arm: Open Label
49 subjects signed consent and were screened for the study. 44 subjects were eligible at time of screening. 39 subjects entered the treatment period.
|
|---|---|
|
Overall Study
STARTED
|
44
|
|
Overall Study
Enrolled
|
44
|
|
Overall Study
Treated
|
39
|
|
Overall Study
COMPLETED
|
36
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Single Arm: Open Label
49 subjects signed consent and were screened for the study. 44 subjects were eligible at time of screening. 39 subjects entered the treatment period.
|
|---|---|
|
Overall Study
Adverse Event
|
4
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Protocol Violation
|
1
|
|
Overall Study
G-tube came out
|
1
|
|
Overall Study
Non-compliant use of PERT
|
1
|
Baseline Characteristics
ITT was 39, missing sample data due to one subject discontinuing prior Visit 3 therefore analysis population: N=38 Erythrocyte omega-3 index % measures erythrocyte membrane composition (%) of DHA+EPA.
Baseline characteristics by cohort
| Measure |
Single Arm: Open Label
n=39 Participants
RELiZORB cartridge and Impact Peptide 1.5 with enteral feeding 500 mL to 1,000 mL per enteral feeding for a period of 90 days.
RELiZORB: Novel enteral feeding in-line digestive enzyme cartridge
Impact Peptide 1.5: Impact Peptide 1.5 at a volume of administration from 500 mL to 1,000 mL per enteral feeding
|
|---|---|
|
Age, Categorical
<=18 years
|
34 Participants
n=39 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=39 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=39 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=39 Participants
|
|
Race (NIH/OMB)
White
|
37 Participants
n=39 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
|
Erythrocyte omega-3 index %
|
4.43 % of omega-3 index of RBC membrane
STANDARD_DEVIATION 2.05 • n=38 Participants • ITT was 39, missing sample data due to one subject discontinuing prior Visit 3 therefore analysis population: N=38 Erythrocyte omega-3 index % measures erythrocyte membrane composition (%) of DHA+EPA.
|
|
BMI
|
17.7 weight (kg) / [height (m)]2
STANDARD_DEVIATION 1.8 • n=39 Participants
|
|
Cystic fibrosis related diabetes
Cystic fibrosis related diabetes
|
9 Participants
n=39 Participants
|
|
Cystic fibrosis related diabetes
Non-cystic fibrosis related diabetes
|
30 Participants
n=39 Participants
|
PRIMARY outcome
Timeframe: Day 0 to Day 90Population: Intent to treat population = 39 subjects who received at least one exposure to RELiZORB. Analysis group is 38 due to one subject discontinuing prior to Visit 3.
Change from baseline Day 0 to Day 90 of erythrocyte tissue composition % of the omega-3 index
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=38 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
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Change From Baseline of Erythrocyte Omega-3 Index % (DHA+EPA)
|
5.01 percentage of omega-3 composition
Standard Error 0.40
|
SECONDARY outcome
Timeframe: RELiZORB Treatment Period (Day 0-Day 90): 90 days with additional 30 days of follow up.Population: Total ITT population (n=39)
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.
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=39 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
|
Unanticipated Adverse Device Effects (UADE)
Patients with at least one UADE
|
10 participants
|
|
Unanticipated Adverse Device Effects (UADE)
Infections and Infestation
|
2 participants
|
|
Unanticipated Adverse Device Effects (UADE)
Respiratory, Thoracic, and Mediastinal
|
8 participants
|
SECONDARY outcome
Timeframe: RELiZORB Treatment Period (Day 0-Day 90): 90 daysPopulation: All subjects who entered the RELiZORB Treatment period, received at least one treatment with the study device and completed the study.
Changes in plasma concentration total DHA+EPA from baseline (Day 0 to Day 90).
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=36 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
|
Changes in Plasma Concentration Total DHA+EPA
|
93.73 percentage of total plasma concentration
Interval 69.5 to 117.96
|
SECONDARY outcome
Timeframe: RELiZORB Treatment Period (Day 0-Day 90): 90 daysPopulation: ITTT
Changes over time in erythrocyte composition (%) for total DHA in ITT population (n=39)
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=39 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
|
Erythrocyte Composition (%) of DHA
|
3.28 percentage of RBC composition
Interval 2.74 to 3.82
|
SECONDARY outcome
Timeframe: RELiZORB Treatment Period (Day 0-Day 90): 90 daysChanges over time in erythrocyte composition (%) for EPA in ITT population
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=39 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
|
Erythrocyte Composition (%) of EPA
|
1.73 percentage of RBC composition
Interval 1.32 to 2.14
|
SECONDARY outcome
Timeframe: RELiZORB Treatment Period (Day 0-Day 90): 90 daysChange from baseline to Day 90 in n6/n3 ratio in erythrocytes
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=39 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
|
Erythrocyte Composition (%) Ratio of n6/n3 Fatty Acids
|
-2.51 percentage of RBC composition
Interval -2.94 to -2.09
|
SECONDARY outcome
Timeframe: RELiZORB Treatment Period (Day 0-Day 90): 90 daysChange over time in n6/n3 ratio in plasma in the ITT population
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=39 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
|
Plasma Composition (%) Ratio of n6/n3 Fatty Acids.
|
-6.29 percentage of total plasma concentration
Interval -7.85 to -4.73
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OTHER_PRE_SPECIFIED outcome
Timeframe: Observation, Baseline and RELiZORB Treatment periods (Day -14 to Day 90): 104 days with additional 30 days of follow up.Population: All subjects who entered the RELiZORB Treatment Period and received at least one treatment with RELiZORB.
GI symptoms recorded in GI diaries by subject and/or caregiver.
Outcome measures
| Measure |
RELiZORB Cartridge With Standard Enteral Formula
n=39 Participants
Patients with exocrine pancreatic insufficiency, age 5 and older, all sexes who have an enteral feeding a minimum of 4 times per week.
|
|---|---|
|
GI Symptoms
End of Study
|
12 Participants
|
|
GI Symptoms
Study Entry
|
23 Participants
|
Adverse Events
Observation and Run-in Period (Day -14 to Day -1)
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
RELiZORB Treatment Period (Day 0 - Day 90)
Serious events: 0 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Observation and Run-in Period (Day -14 to Day -1)
n=44 participants at risk
This 14 day period was comprised of 7 day Observation period followed by 7 day Run-in period. 44 subjects were enrolled of which 39 proceeded to treatment period of EF with RELiZORB.
|
RELiZORB Treatment Period (Day 0 - Day 90)
n=39 participants at risk
Safety population is defined as all subjects who entered the RELiZORB Treatment period and received at least one treatment with the study device. 39 subjects entered treatment period of EF with RELiZORB.
|
|---|---|---|
|
Infections and infestations
Infectious colitis
|
2.3%
1/44 • Number of events 1 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
0.00%
0/39 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
2.3%
1/44 • Number of events 1 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
0.00%
0/39 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
Other adverse events
| Measure |
Observation and Run-in Period (Day -14 to Day -1)
n=44 participants at risk
This 14 day period was comprised of 7 day Observation period followed by 7 day Run-in period. 44 subjects were enrolled of which 39 proceeded to treatment period of EF with RELiZORB.
|
RELiZORB Treatment Period (Day 0 - Day 90)
n=39 participants at risk
Safety population is defined as all subjects who entered the RELiZORB Treatment period and received at least one treatment with the study device. 39 subjects entered treatment period of EF with RELiZORB.
|
|---|---|---|
|
Gastrointestinal disorders
Vomiting
|
9.1%
4/44 • Number of events 6 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
0.00%
0/39 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Investigations
Pulmonary function decreased
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
7.7%
3/39 • Number of events 3 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
20.5%
8/39 • Number of events 9 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Respiratory, thoracic and mediastinal disorders
Lung disorder
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
25.6%
10/39 • Number of events 12 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
General disorders
Pyrexia
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
5.1%
2/39 • Number of events 2 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Infections and infestations
Ear infection
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
5.1%
2/39 • Number of events 2 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Investigations
Forced expiratory volume decreased
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
5.1%
2/39 • Number of events 2 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Investigations
Vitamin D decrease
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
5.1%
2/39 • Number of events 2 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
5.1%
2/39 • Number of events 2 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/44 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
5.1%
2/39 • Number of events 2 • Adverse event data collected and reported for 14 days during the Observation and Run-in periods. Adverse event data collected and reported for 120 days for the RELiZORB period.
Adverse event data were collected from study entry at day -14 to end of study (Day -14 through 90). Follow up of events present at end of study/termination continued for an additional 30 days after completion of study.
|
Additional Information
Madhumalli ("Molly") Sarkar, MD, PhD, Head of Clinical Development
Alcresta Therapeutics, Inc.
Phone: 617 431 8866
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Publication by the PI of any data from this study is strictly prohibited without consent from the sponsor.
- Publication restrictions are in place
Restriction type: OTHER