Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Protocol ALCT-0000497 is a multicenter safety, tolerability and fat absorption study that anticipates enrolling 35 male and female subjects (pediatric and adult) with cystic fibrosis. Subjects with confirmed exocrine pancreatic insufficiency will use a novel enteral feeding in-line digestive enzyme cartridge (RELiZORB) connected to enteral pump sets.
Protocol ALCT-0000497 consists of three distinct study periods as follows:
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RELiZORB | Experimental | Treatment (RELiZORB) |
|
| Control | Placebo Comparator | Placebo control |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RELiZORB | Device | Peptamen 1.5 received Period A and Period B (washout only). Impact Peptide 1.5 received Period B (Days 1 and 9 only) and Period C. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients With Adverse Events and Unanticipated Adverse Device Effects | 1) Frequency and severity of adverse events; 2) Patients with at least one unanticipated adverse device effects (UADE) | 27 days |
| Long Chain Polyunsaturated Fatty Acid Plasma Concentration (Intent to Treat Population) | AUC analysis of plasma fatty acid concentration for DHA + EPA baseline adjusted over 24-hours | Day 1 first intervention and Day 9 second intervention. |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Ease of Use of RELiZORB (Per-Protocol Population) | Effect of enteral nutrition on select activities of daily living. Patients judged the size of breakfast after overnight enteral tube feeding with the following choices: No breakfast; Small breakfast; Normal breakfast; Big breakfast; Other. | Period C: Single assessment on Day 19 or 20 |
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Russell G. Clayton, Sr., DO | Chief Medical Officer, Alcresta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | United States | ||
| St. Luke's Cystic Fibrosis Center of Idaho |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28471913 | Derived | Freedman S, Orenstein D, Black P, Brown P, McCoy K, Stevens J, Grujic D, Clayton R. Increased Fat Absorption From Enteral Formula Through an In-line Digestive Cartridge in Patients With Cystic Fibrosis. J Pediatr Gastroenterol Nutr. 2017 Jul;65(1):97-101. doi: 10.1097/MPG.0000000000001617. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
33 eligible patients according to the inclusion/exclusion criteria were randomized in Period B (double-blind crossover) to the study.
34 patients enrolled; 33 patients completed the study from 11 U.S. sites.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Period A: Clinical Treatment Practice (Days -7 to -1) | Period A was a 7-day baseline period. Patients received Peptamen 1.5 at their normal volume of enteral formula administration up to a maximum of 1000 mL per feeding. Current treatment practice was followed with normal use of oral pancreatic enzyme replacement therapy (PERT) during daily meals and nightly enteral feedings. A gastrointestinal symptom diary was completed for 7 consecutive days. Baseline Day -7 required a clinic visit followed by Days -6 to -1 at home. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Period A: Days -7 to -1 |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Device | Sham device |
|
| 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 |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Cardinal Glennon Children's Medical Center | St Louis | Missouri | 63104 | United States |
| Rainbow Babies and Children's Hospital | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Monroe Carell Junior Children's Hospital at Vanderbilt | Nashville | Tennessee | 37332 | United States |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| FG001 | Crossover Period B: Placebo Then RELiZORB | Eligible subjects were randomized to Placebo then RELiZORB. |
| FG002 | Crossover Period B: RELiZORB Then Placebo | Eligible subjects were randomized to RELiZORB then Placebo. |
| FG003 | Period C: Clinical Treatment Practice + RELiZORB (Days 12-20) | Period C was the open label clinical treatment period with RELiZORB. All patients used RELiZORB with Impact Peptide 1.5 at their normal volume as in Period A up to a maximum of 1000 mL per feeding. Patients were instructed to use the same daily dose and schedule of oral PERT taken in Period C as in Period A. There were no dietary restrictions. A gastrointestinal symptom diary was completed for 7 consecutive days. Patients received enteral feeding at home from Days 12 to 18 and returned to clinic for their end of study Day 19. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Period B: Days 1 to 11 |
|
| Period C: Days 12 to 20 |
|
Safety Population
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Clinical Treatment Practice: Period A: Days -7 to -1 | Period A was a 7-day baseline period. Patients received Peptamen 1.5 at their normal volume of enteral formula administration up to a maximum of 1000 mL per feeding. Current treatment practice was followed with normal use of oral pancreatic enzyme replacement therapy (PERT) during daily meals and nightly enteral feedings. A gastrointestinal symptom diary was completed for 7 consecutive days. Baseline Day -7 required a clinic visit followed by Days -6 to -1 at home. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Region of Enrollment | 11 United States clinical sites. | Count of Participants | Participants |
| |||||||||||||||||||
| Baseline Weight | Baseline weight obtained in Period A. | Mean | Standard Deviation | kilograms |
| ||||||||||||||||||
| Baseline Height | Safety Population: Baseline Height | Baseline height obtained in Period A. | Mean | Standard Deviation | centimeters |
| |||||||||||||||||
| Baseline Body Mass Index | Baseline Body Mass Index obtained in Period A. | Mean | Standard Deviation | kg/m^2 |
|
| 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 | Number of Patients With Adverse Events and Unanticipated Adverse Device Effects | 1) Frequency and severity of adverse events; 2) Patients with at least one unanticipated adverse device effects (UADE) | Safety Population | Posted | Count of Participants | Participants | 27 days |
|
|
| |||||||||||||||||||||||||||||||||||
| Primary | Long Chain Polyunsaturated Fatty Acid Plasma Concentration (Intent to Treat Population) | AUC analysis of plasma fatty acid concentration for DHA + EPA baseline adjusted over 24-hours | Posted | Mean | Standard Deviation | ug*h/mL | Day 1 first intervention and Day 9 second intervention. |
|
| ||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Ease of Use of RELiZORB (Per-Protocol Population) | Effect of enteral nutrition on select activities of daily living. Patients judged the size of breakfast after overnight enteral tube feeding with the following choices: No breakfast; Small breakfast; Normal breakfast; Big breakfast; Other. | Per Protocol Population. | Posted | Count of Participants | Participants | Period C: Single assessment on Day 19 or 20 |
|
|
20 days
Safety Population: Non-gastrointestinal Adverse Event by System Organ Class and Preferred Term (n=33)
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Clinical Treatment Practice: Period A: Days -7 to -1 | Period A was a 7-day baseline period. Patients received Peptamen 1.5 at their normal volume of enteral formula administration up to a maximum of 1000 mL per feeding. Current treatment practice was followed with normal use of oral pancreatic enzyme replacement therapy (PERT) during daily meals and nightly enteral feedings. A gastrointestinal symptom diary was completed for 7 consecutive days. Baseline Day -7 required a clinic visit followed by Days -6 to -1 at home. | 1 | 33 | 2 | 33 | ||
| EG001 | Double-Blind Crossover: Period B: Days 1 to 11 | Period B was the randomized, double-blind, placebo-controlled crossover period. Eligible patients were randomized in a 1:1 ratio to either Placebo-RELiZORB or RELiZORB-Placebo treatment sequences. On two separate administration Days 1 and 9, patients received 500 mL of Impact Peptide1.5 in clinic over a 4h period. Motility and acid suppression medications were discontinued 24h before arrival in clinic. No nocturnal feeding occurred between Days 1-2 and Days 9-10. During the home washout period Days 2 to 8, patients received Peptamen 1.5 for enteral nutrition up to a maximum volume of 1000 mL per feeding. Safety follow up calls were conducted on Days 2 and 10. | 0 | 33 | 0 | 33 | ||
| EG002 | Clinical Treatment Practice and Relizorb: Period C: Days 12-20 | Period C was the open label clinical treatment period with RELiZORB. All patients received RELiZORB with Impact Peptide 1.5 at their normal volume as in Period A up to a maximum of 1000 mL per feeding. Patients were instructed to use the same daily dose and schedule of oral PERT taken in Period C as in Period A. There were no dietary restrictions. A gastrointestinal symptom diary was completed for 7 consecutive days. Patients received enteral feeding at home from Days 12 to 18 and returned to clinic for their end of study Day 19. | 0 | 33 | 0 | 33 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cystic fibrosis pulmonary exacerbation | Infections and infestations | MedDRA 18.0 | Systematic Assessment | Cystic fibrosis (increased cough, vomiting, decreased PFTs, hospital care required supplemental oxygen, IV antibiotics, and supportive care). |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA 18.0 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Russell G. Clayton, D.O., Chief Medical Officer | Alcresta Therapeutics, Inc. | 215.813.5100 | rclayton@alcresta.com |
| ID | Term |
|---|---|
| D010188 | Exocrine Pancreatic Insufficiency |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Adverse Event by Severity (Mild) |
|
| Adverse Event by Severity (Moderate) |
|
| Adverse Event by Severity (Severe) |
|
| Patients With At Least One UADE |
|
|
|
|