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| Name | Class |
|---|---|
| Cystic Fibrosis Foundation | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single Arm: Open label RELiZORB Cartridge & Impact Peptide 1.5 | Other | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RELiZORB (immobilized lipase) cartridge | Device | Hydrolyzing fats from enteral formula, ex vivo, with in-line enteral feed RELiZORB (immobilized lipase) cartridge |
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| 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 |
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| GI Symptoms | GI symptoms recorded in GI diaries by subject and/or caregiver. | Observation, Baseline and RELiZORB Treatment periods (Day -14 to Day 90): 104 days with additional 30 days of follow up. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Madhumalli Sarkar, MD, PhD | Alcresta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Joe DiMaggio Children's Hospital / Memorial Healthcare System | Hollywood | Florida | 33021 | United States | ||
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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.
Recruitment was conducted through Cystic Fibrosis Care Centers.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 7, 2016 | May 10, 2018 |
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| Impact Peptide 1.5 | Other | Impact Peptide 1.5 at a volume of administration from 500 mL to 1,000 mL per enteral feeding |
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| 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 |
| St. Luke's CF Center of Idaho |
| Boise |
| Idaho |
| 83712 |
| United States |
| Riley Hospital for Children at Indiana University Health | Indianapolis | Indiana | 46202 | United States |
| Maine Medical Center | Portland | Maine | 04102 | United States |
| Helen DeVos Children's Hospital CF Care Center | Grand Rapids | Michigan | 49503 | United States |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Cardinal Glennon Children's Hospital / Saint Louis University | St Louis | Missouri | 63104 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Dayton Children's Hospital | Dayton | Ohio | 45404-1815 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Enrolled |
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| Treated |
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| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Single Arm: Open Label | 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 |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Erythrocyte omega-3 index % | 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. | Mean | Standard Deviation | % of omega-3 index of RBC membrane |
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| BMI | BMI formula: weight (kg) / [height (m)]2 | Mean | Standard Deviation | weight (kg) / [height (m)]2 |
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| Cystic fibrosis related diabetes | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | 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. | Posted | Least Squares Mean | Standard Error | percentage of omega-3 composition | Day 0 to Day 90 |
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| Secondary | 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. | Total ITT population (n=39) | Posted | Number | participants | RELiZORB Treatment Period (Day 0-Day 90): 90 days with additional 30 days of follow up. |
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| Secondary | Changes in Plasma Concentration Total DHA+EPA | Changes in plasma concentration total DHA+EPA from baseline (Day 0 to Day 90). | All subjects who entered the RELiZORB Treatment period, received at least one treatment with the study device and completed the study. | Posted | Least Squares Mean | 95% Confidence Interval | percentage of total plasma concentration | RELiZORB Treatment Period (Day 0-Day 90): 90 days |
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| Secondary | Erythrocyte Composition (%) of DHA | Changes over time in erythrocyte composition (%) for total DHA in ITT population (n=39) | ITTT | Posted | Least Squares Mean | 95% Confidence Interval | percentage of RBC composition | RELiZORB Treatment Period (Day 0-Day 90): 90 days |
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| Secondary | Erythrocyte Composition (%) of EPA | Changes over time in erythrocyte composition (%) for EPA in ITT population | Posted | Least Squares Mean | 95% Confidence Interval | percentage of RBC composition | RELiZORB Treatment Period (Day 0-Day 90): 90 days |
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| Secondary | Erythrocyte Composition (%) Ratio of n6/n3 Fatty Acids | Change from baseline to Day 90 in n6/n3 ratio in erythrocytes | Posted | Least Squares Mean | 95% Confidence Interval | percentage of RBC composition | RELiZORB Treatment Period (Day 0-Day 90): 90 days |
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| Secondary | Plasma Composition (%) Ratio of n6/n3 Fatty Acids. | Change over time in n6/n3 ratio in plasma in the ITT population | Posted | Least Squares Mean | 95% Confidence Interval | percentage of total plasma concentration | RELiZORB Treatment Period (Day 0-Day 90): 90 days |
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| Other Pre-specified | GI Symptoms | GI symptoms recorded in GI diaries by subject and/or caregiver. | All subjects who entered the RELiZORB Treatment Period and received at least one treatment with RELiZORB. | Posted | Count of Participants | Participants | Observation, Baseline and RELiZORB Treatment periods (Day -14 to Day 90): 104 days with additional 30 days of follow up. |
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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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Observation and Run-in Period (Day -14 to Day -1) | 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. | 0 | 44 | 2 | 44 | 4 | 44 |
| EG001 | RELiZORB Treatment Period (Day 0 - Day 90) | 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. | 0 | 39 | 0 | 39 | 23 | 39 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infectious colitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Pulmonary function decreased | Investigations | MedDRA (18.0) | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (18.0) | Systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Forced expiratory volume decreased | Investigations | MedDRA (18.0) | Systematic Assessment |
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| Vitamin D decrease | Investigations | MedDRA (18.0) | Systematic Assessment |
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| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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Patient GI symptom diary: recall method using unvalidated tool. Patients were allowed to eat normal diet during the day, hence the amount of LCPUFA that was measured in the plasma is not the sole reflection of the device use only.
Publication by the PI of any data from this study is strictly prohibited without consent from the sponsor.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Madhumalli ("Molly") Sarkar, MD, PhD, Head of Clinical Development | Alcresta Therapeutics, Inc. | 617 431 8866 | msarkar@alcresta.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 29, 2017 | May 10, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D010188 | Exocrine Pancreatic Insufficiency |
| D003920 | Diabetes Mellitus |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D004700 | Endocrine System Diseases |
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| >=65 years |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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