Cystic Fibrosis Clinical Trial
Official title:
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center, Crossover Study to Evaluate the Effectiveness and Safety of PANCRECARB® MS-16 (Pancrelipase) in Reducing Steatorrhea in Children and Adults With Cystic Fibrosis
Verified date | February 2012 |
Source | Digestive Care, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
The primary objective of this study is to determine if PANCRECARB® MS-16 (pancrelipase) is safe and effective in reducing steatorrhea (as measured by 72-hour stool fat determinations) in children and adults with cystic fibrosis and pancreatic insufficiency.
Status | Completed |
Enrollment | 29 |
Est. completion date | September 2007 |
Est. primary completion date | September 2007 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 7 Years and older |
Eligibility |
Inclusion Criteria: - Male or female age = 7 years - Confirmed diagnosis of CF based on the following criteria: One or more clinical features consistent with the CF phenotype, AND Positive sweat chloride = 60 mEq/liter (by pilocarpine iontophoresis), OR Genotype with two identifiable mutations consistent with CF - Adequate nutritional status based on BMI: Age 7 years to 20 years old, Body Mass Index Percentile = 5th percentile; Age > 20 years old, Body Mass Index for females = 16.0, Body Mass Index for males = 16.5 - Pancreatic insufficiency documented by spot fecal elastase-1 (FE 1) <= 100 micrograms/g stool at the time of randomization - Currently receiving pancreatic enzyme replacement therapy with a commercially available pancreatic enzyme - Able to swallow size 0 capsules - Clinically stable with no evidence of an acute medical condition - Able to understand and sign a written informed consent or assent and comply with the requirements of the study Exclusion Criteria: - History of fibrosing colonopathy - History of significant bowel resection - History of being refractory to pancreatic enzyme replacement therapy - Solid organ transplant - Abdominal surgery within past five (5) years - A current diagnosis or a history of distal intestinal obstruction syndrome (DIOS) in the past six (6) months, or 2 or more episodes of DIOS in the past twelve (12) months - Conditions known to increase fecal fat loss including: inflammatory bowel disease , celiac disease, Crohn's disease, tropical Sprue, Whipple's disease - A known contraindication, sensitivity or hypersensitivity to porcine pancreatic enzymes or food dyes (i.e., FD&C Blue No. 2) - Active liver disease with liver enzymes (alanine aminotransferase (ALT/SGPT), aspartate aminotransferase (AST/SGOT) or bilirubin = 3 times the upper limit of normal - Acute pancreatitis or acute exacerbation of chronic pancreatitis - Acute treatment with any systemic (oral or IV) antibiotics two (2) weeks prior to screening - Treatment with erythromycin and unwilling to discontinue the treatment two (2) weeks prior to the screening. (azithromycin is allowed) - Change in chronic treatment with systemic (oral and IV) antibiotics during the trial NOTE: Study subject may remain on a chronic regimen of systemic (oral or IV) antibiotics (with exception of erythromycin), if he/she started the antibiotics at least 2 weeks prior to study screening, was at his/her usual bowel pattern at the time of screening, and does not stop or change these antibiotics during the study period. - Receiving enteral tube feeding during the study - Expected inability to cooperate with or be non-adherent to required study procedures - Pregnant, breast-feeding, or unwilling to practice birth control (for females of child-bearing potential) during participation in the study - Use of narcotics - Poorly controlled diabetes - Participation in an investigational study of a drug, biologic, or device not currently approved for marketing, within 30 days of screening visit - A medical condition which the investigator deems significant enough to interfere with the ability of the study patient to participate in the trial or interfering with assessment of effects of enzyme therapy on fat absorption |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Rainbow Babies and Children's Hospital | Cleveland | Ohio |
Lead Sponsor | Collaborator |
---|---|
Digestive Care, Inc. |
United States,
Borowitz D, Baker RD, Stallings V. Consensus report on nutrition for pediatric patients with cystic fibrosis. J Pediatr Gastroenterol Nutr. 2002 Sep;35(3):246-59. Review. — View Citation
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. — View Citation
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. Review. — View Citation
Brady MS, Garson JL, Krug SK, Kaul A, Rickard KA, Caffrey HH, Fineberg N, Balistreri WF, Stevens JC. An enteric-coated high-buffered pancrelipase reduces steatorrhea in patients with cystic fibrosis: a prospective, randomized study. J Am Diet Assoc. 2006 Aug;106(8):1181-6. — View Citation
Davis PB, Drumm M, Konstan MW. Cystic fibrosis. Am J Respir Crit Care Med. 1996 Nov;154(5):1229-56. Review. — View Citation
Konstan MW, Stern RC, Trout JR, Sherman JM, Eigen H, Wagener JS, Duggan C, Wohl ME, Colin P. Ultrase MT12 and Ultrase MT20 in the treatment of exocrine pancreatic insufficiency in cystic fibrosis: safety and efficacy. Aliment Pharmacol Ther. 2004 Dec;20(11-12):1365-71. — View Citation
Miller MR, Hankinson J, Brusasco V, Burgos F, Casaburi R, Coates A, Crapo R, Enright P, van der Grinten CP, Gustafsson P, Jensen R, Johnson DC, MacIntyre N, McKay R, Navajas D, Pedersen OF, Pellegrino R, Viegi G, Wanger J; ATS/ERS Task Force. Standardisation of spirometry. Eur Respir J. 2005 Aug;26(2):319-38. — View Citation
Pellegrino R, Viegi G, Brusasco V, Crapo RO, Burgos F, Casaburi R, Coates A, van der Grinten CP, Gustafsson P, Hankinson J, Jensen R, Johnson DC, MacIntyre N, McKay R, Miller MR, Navajas D, Pedersen OF, Wanger J. Interpretative strategies for lung function tests. Eur Respir J. 2005 Nov;26(5):948-68. — View Citation
Rosenstein BJ, Cutting GR. The diagnosis of cystic fibrosis: a consensus statement. Cystic Fibrosis Foundation Consensus Panel. J Pediatr. 1998 Apr;132(4):589-95. Review. — View Citation
Stern RC, Eisenberg JD, Wagener JS, Ahrens R, Rock M, doPico G, Orenstein DM. A comparison of the efficacy and tolerance of pancrelipase and placebo in the treatment of steatorrhea in cystic fibrosis patients with clinical exocrine pancreatic insufficiency. Am J Gastroenterol. 2000 Aug;95(8):1932-8. — View Citation
VAN DE KAMER JH, TEN BOKKEL HUININK H, WEYERS HA. Rapid method for the determination of fat in feces. J Biol Chem. 1949 Jan;177(1):347-55. — View Citation
* Note: There are 11 references in all — Click here to view all references
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | percent coefficient of fat absorption (% CFA) | calculated from the 72-hour stool collection and dietary records | No | |
Secondary | percent coefficient of nitrogen absorption (% CNA) | calculated from the 72-hour stool collections and dietary records | No | |
Secondary | change in stool frequency and stool weight | recorded over the 72-hour stool collection period | No |
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