Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Chiesi USA, Inc. | INDUSTRY |
Not provided
Not provided
Not provided
This is a clinical trial with a cross over design investigating the effect of the proton pump inhibitor omeprazole on fat malabsorption in subjects with cystic fibrosis and pancreatic insufficiency. Participants will be randomized to receive either omeprazole or placebo for 28 days, then cross over and receive omeprazole or placebo for another 28 days. Markers of fat absorption will be measured after each treatment course.
Fat malabsorption contributes to poor nutritional status in people with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). Prescribing gastric acid-reducing agents such as proton pump inhibitors (PPIs) and histamine receptor antagonists (H2RAs) as an adjunct to pancreatic enzyme replacement therapy (PERT) to improve PERT efficacy and dietary fat absorption has become accepted clinical practice in CF, despite limited evidence to support the practice. Establishing the efficacy and true health benefit of acid suppression for nutritional status and outcomes in CF is particularly important in light of potential health risks and cost associated with long-term or even lifetime use of these medications.
This study aims to characterize changes in fat malabsorption using the coefficient of fat absorption (CFA) as the primary endpoint in subjects who are on and off acid suppression with a PPI in addition to PERT. Additionally, the SmartPill® will be used to evaluate duodenal power of hydrogen (pH) while on and off acid suppression, and the malabsorption blood test (MBT) will be used to characterize changes in fat absorption. Associations will be explored between changes in nutritional status (weight, height, BMI), clinical GI symptoms, and quality of life in subjects treated with PPI vs. placebo.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Omeprazole | Active Comparator | Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. |
|
| Placebo | Placebo Comparator | Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Omeprazole 20mg Capsule | Drug | Omeprazole 20mg daily for 28 days |
|
| Measure | Description | Time Frame |
|---|---|---|
| Coefficient of Fat Absorption | Gold standard measurement of fat malabsorption | After 28 days of treatment or placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Duodenal pH | Change in duodenal pH as measured by the motility testing system (SmartPill) | After 28 days of treatment or placebo |
| Fat Absorption Via Malabsorption Blood Test | Measurement of serum pentadecanoic acid and heptadecanoic acid |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Virginia A Stallings, MD | Children's Hospital of Philadelphia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19146 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 7472816 | Background | Borowitz DS, Grand RJ, Durie PR. Use of pancreatic enzyme supplements for patients with cystic fibrosis in the context of fibrosing colonopathy. Consensus Committee. J Pediatr. 1995 Nov;127(5):681-4. doi: 10.1016/s0022-3476(95)70153-2. No abstract available. | |
| 27546383 | Background | Ng SM, Moore HS. Drug therapies for reducing gastric acidity in people with cystic fibrosis. Cochrane Database Syst Rev. 2016 Aug 22;2016(8):CD003424. doi: 10.1002/14651858.CD003424.pub4. |
Not provided
Not provided
Individual participant data that underlie the results reported will be shared upon request, after deidentification.
The data will be available immediately upon publication.
Contact brownellj@email.chop.edu. Requestors will need to sign a data access agreement.
Not provided
19 subjects were randomized. There were 13 evaluable subjects who completed data collection at the end of the treatment period and 6 subjects did not complete data collection.
This is a randomized, cross-over study for which 19 participants were consented/enrolled; not all participants initiated study drug/placebo. Participants were randomized to receive placebo or drug first.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Study Drug First, Then Placebo | Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days |
| FG001 | Placebo First, Then Study Drug |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 11, 2019 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo oral capsule | Drug | Identically-appearing capsule to omeprazole for 28 days |
|
|
| After 28 days of treatment or placebo |
| 23357842 | Background | Stallings VA, Mondick JT, Schall JI, Barrett JS, Wilson M, Mascarenhas MR. Diagnosing malabsorption with systemic lipid profiling: pharmacokinetics of pentadecanoic acid and triheptadecanoic acid following oral administration in healthy subjects and subjects with cystic fibrosis. Int J Clin Pharmacol Ther. 2013 Apr;51(4):263-73. doi: 10.5414/CP201793. |
| 25689042 | Background | Mascarenhas MR, Mondick J, Barrett JS, Wilson M, Stallings VA, Schall JI. Malabsorption blood test: Assessing fat absorption in patients with cystic fibrosis and pancreatic insufficiency. J Clin Pharmacol. 2015 Aug;55(8):854-65. doi: 10.1002/jcph.484. Epub 2015 Mar 23. |
| 23629618 | Background | Lightdale JR, Gremse DA; Section on Gastroenterology, Hepatology, and Nutrition. Gastroesophageal reflux: management guidance for the pediatrician. Pediatrics. 2013 May;131(5):e1684-95. doi: 10.1542/peds.2013-0421. Epub 2013 Apr 29. |
| 8420740 | Background | Heijerman HG, Lamers CB, Bakker W, Dijkman JH. Improvement of fecal fat excretion after addition of omeprazole to pancrease in cystic fibrosis is related to residual exocrine function of the pancreas. Dig Dis Sci. 1993 Jan;38(1):1-6. doi: 10.1007/BF01296765. |
| 1984743 | Background | Heijerman HG, Lamers CB, Bakker W. Omeprazole enhances the efficacy of pancreatin (pancrease) in cystic fibrosis. Ann Intern Med. 1991 Feb 1;114(3):200-1. doi: 10.7326/0003-4819-114-3-200. |
| 13680386 | Background | Proesmans M, De Boeck K. Omeprazole, a proton pump inhibitor, improves residual steatorrhoea in cystic fibrosis patients treated with high dose pancreatic enzymes. Eur J Pediatr. 2003 Nov;162(11):760-3. doi: 10.1007/s00431-003-1309-5. Epub 2003 Sep 17. |
| 423042 | Background | Cox KL, Isenberg JN, Osher AB, Dooley RR. The effect of cimetidine on maldigestion in cystic fibrosis. J Pediatr. 1979 Mar;94(3):488-92. doi: 10.1016/s0022-3476(79)80609-5. |
| 1505293 | Background | Carroccio A, Pardo F, Montalto G, Iapichino L, Soresi M, Averna MR, Iacono G, Notarbartolo A. Use of famotidine in severe exocrine pancreatic insufficiency with persistent maldigestion on enzymatic replacement therapy. A long-term study in cystic fibrosis. Dig Dis Sci. 1992 Sep;37(9):1441-6. doi: 10.1007/BF01296016. |
| 7380201 | Background | Boyle BJ, Long WB, Balistreri WF, Widzer SJ, Huang N. Effect of cimetidine and pancreatic enzymes on serum and fecal bile acids and fat absorption in cystic fibrosis. Gastroenterology. 1980 May;78(5 Pt 1):950-3. |
| 3885675 | Background | Chalmers DM, Brown RC, Miller MG, Clarke PC, Kelleher J, Littlewood JM, Losowsky MS. The influence of long-term cimetidine as an adjuvant to pancreatic enzyme therapy in cystic fibrosis. Acta Paediatr Scand. 1985 Jan;74(1):114-7. doi: 10.1111/j.1651-2227.1985.tb10930.x. |
| 8333766 | Background | Bowler IM, Green JH, Wolfe SP, Littlewood JM. Resting energy expenditure and substrate oxidation rates in cystic fibrosis. Arch Dis Child. 1993 Jun;68(6):754-9. doi: 10.1136/adc.68.6.754. |
| 12142815 | Background | Francisco MP, Wagner MH, Sherman JM, Theriaque D, Bowser E, Novak DA. Ranitidine and omeprazole as adjuvant therapy to pancrelipase to improve fat absorption in patients with cystic fibrosis. J Pediatr Gastroenterol Nutr. 2002 Jul;35(1):79-83. doi: 10.1097/00005176-200207000-00017. |
| 25019293 | Background | Ng SM, Franchini AJ. Drug therapies for reducing gastric acidity in people with cystic fibrosis. Cochrane Database Syst Rev. 2014 Jul 13;(7):CD003424. doi: 10.1002/14651858.CD003424.pub3. |
| 22513912 | Background | Ng SM, Francini AJ. Drug therapies for reducing gastric acidity in people with cystic fibrosis. Cochrane Database Syst Rev. 2012 Apr 18;(4):CD003424. doi: 10.1002/14651858.CD003424.pub2. |
| 12804466 | Background | Ng SM, Jones AP. Drug therapies for reducing gastric acidity in people with cystic fibrosis. Cochrane Database Syst Rev. 2003;(2):CD003424. doi: 10.1002/14651858.CD003424. |
| 23748712 | Background | Kaunitz JD, Akiba Y. Wireless telemetry and cystic fibrosis: just the pHacts. Dig Dis Sci. 2013 Aug;58(8):2129-30. doi: 10.1007/s10620-013-2714-x. Epub 2013 Jun 9. No abstract available. |
| 22592630 | Background | Gelfond D, Ma C, Semler J, Borowitz D. Intestinal pH and gastrointestinal transit profiles in cystic fibrosis patients measured by wireless motility capsule. Dig Dis Sci. 2013 Aug;58(8):2275-81. doi: 10.1007/s10620-012-2209-1. Epub 2012 May 17. |
| 15343184 | Background | Borowitz D, Baker SS, Duffy L, Baker RD, Fitzpatrick L, Gyamfi J, Jarembek K. Use of fecal elastase-1 to classify pancreatic status in patients with cystic fibrosis. J Pediatr. 2004 Sep;145(3):322-6. doi: 10.1016/j.jpeds.2004.04.049. |
| 17255835 | Background | Borowitz D, Lin R, Baker SS. Comparison of monoclonal and polyclonal ELISAs for fecal elastase in patients with cystic fibrosis and pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2007 Feb;44(2):219-23. doi: 10.1097/MPG.0b013e31802c41de. |
Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption.
Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days
| COMPLETED |
|
| NOT COMPLETED |
|
|
This randomized, crossover study includes baseline characteristics for 13 participants who both or either active and/or placebo treatment.
Only data for 11 participants were available for analysis.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Study Drug First, Then Placebo | Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days |
| BG001 | Placebo First, Then Study Drug | Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| 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 | Coefficient of Fat Absorption | Gold standard measurement of fat malabsorption | The primary outcome measurement (CFA) was not available for 2 participants who got study drug first and 2 participants who got placebo first. | Posted | Mean | Standard Deviation | % fat absorption | After 28 days of treatment or placebo |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Duodenal pH | Change in duodenal pH as measured by the motility testing system (SmartPill) | Posted | Mean | Standard Deviation | pH | After 28 days of treatment or placebo |
|
| ||||||||||||||||||||||||||||||
| Secondary | Fat Absorption Via Malabsorption Blood Test | Measurement of serum pentadecanoic acid and heptadecanoic acid | Posted | Mean | Standard Deviation | mg*h/dl | After 28 days of treatment or placebo |
|
|
28 days (while participant was on active treatment or placebo); total of 56 days.
Not provided
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 | Omeprazole | Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days | 0 | 11 | 0 | 11 | 0 | 11 |
| EG001 | Placebo | Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days | 0 | 11 | 0 | 11 | 0 | 11 |
Not provided
Not provided
Analysis of the secondary measures remains ongoing and results are anticipated in the Spring of 2024. Results from secondary outcomes will be reported when data analysis are complete.
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Virginia Stallings | Children's Hospital of Philadelphia | 267-425-1633 | stallingsv@chop.edu |
| Dec 19, 2023 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D010188 | Exocrine Pancreatic Insufficiency |
| D003550 | Cystic Fibrosis |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| D009853 | Omeprazole |
| ID | Term |
|---|---|
| D053799 | 2-Pyridinylmethylsulfinylbenzimidazoles |
| D013454 | Sulfoxides |
| D013457 | Sulfur Compounds |
| D009930 | Organic Chemicals |
| D011725 | Pyridines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D001562 | Benzimidazoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
Not provided
Not provided
| Male |
|
| African American |
|
|
|