Evaluation of the Safety and Efficacy of Pancrecarb® MS-16 in Cystic Fibrosis

NCT00432861 | Completed Phase 3 DMC

• 2 other identifiers: PancrecarbDCI 06-001
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cystic Fibrosis; Pancreatic Insufficiency

Keywords

cystic fibrosis; pancreatic insufficiency; PANCRECARB® MS-16 (pancrelipase); steatorrhea

Outcome Measures

Primary Outcomes (1)

• percent coefficient of fat absorption (% CFA)

  calculated from the 72-hour stool collection and dietary records

Secondary Outcomes (2)

• percent coefficient of nitrogen absorption (% CNA)

  calculated from the 72-hour stool collections and dietary records

• change in stool frequency and stool weight

  recorded over the 72-hour stool collection period

Study Arms (2)

1

ACTIVE COMPARATOR

Pancrecarb(R) MS-16 Capsules

Drug: PANCRECARB® (pancrelipase)

2

PLACEBO COMPARATOR

Drug: Placebo

Interventions

PANCRECARB® (pancrelipase) DRUG

Capsules

Also known as: MS-16

1

Placebo DRUG

Capsules

2

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Digestive Care, Inc.: lead

Study Sites (1)

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Related Publications (11)

• Davis PB, Drumm M, Konstan MW. Cystic fibrosis. Am J Respir Crit Care Med. 1996 Nov;154(5):1229-56. doi: 10.1164/ajrccm.154.5.8912731. No abstract available.

  PMID: 8912731 BACKGROUND

• 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. doi: 10.1097/00005176-200209000-00004. No abstract available.

  PMID: 12352509 BACKGROUND

• 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. doi: 10.1016/j.jada.2006.05.011.

  PMID: 16863712 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.

  PMID: 7472816 BACKGROUND

• 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. doi: 10.1111/j.1365-2036.2004.02261.x.

  PMID: 15606399 BACKGROUND

• 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. doi: 10.1016/s0022-3476(98)70344-0.

  PMID: 9580754 BACKGROUND

• 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. doi: 10.1183/09031936.05.00034805. No abstract available.

  PMID: 16055882 BACKGROUND

• 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. doi: 10.1183/09031936.05.00035205. No abstract available.

  PMID: 16264058 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.

  PMID: 15343184 BACKGROUND

• 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. doi: 10.1111/j.1572-0241.2000.02244.x.

  PMID: 10950038 BACKGROUND

• 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. No abstract available.

  PMID: 18107439 BACKGROUND

Related Links

• Digestive Care, Inc.

MeSH Terms

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Steatorrhea

Interventions

Pancrelipase

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Lipase; Carboxylic Ester Hydrolases; Esterases; Hydrolases; Enzymes; Enzymes and Coenzymes; Pancreatic Extracts; Tissue Extracts; Complex Mixtures

Study Officials

• Michael W Konstan, MD

  University Hospitals Cleveland Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 7, 2007
• First Posted: February 8, 2007
• Study Start: January 1, 2007
• Primary Completion: September 1, 2007
• Study Completion: September 1, 2007
• Last Updated: February 22, 2012

Record last verified: 2012-02

Locations

US (1)