Correction of steathorrea associated with chronic exocrine pancreatic insufficiency in cystic fibrosis MedDRA version: 13.1 Level: PT Classification code 10033628 Term: Pancreatic insufficiency System Organ Class: 10017947 - Gastrointestinal disorders MedDRA version: 13.1 Level: LLT Classification code 10041968 Term: Steatorrhea System Organ Class: 10017947 - Gastrointestinal disorders MedDRA version: 13.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 100103
Conditions
Interventions
Trade Name: PANZYTRAT 25000
Product Name: PANZYTRAT 25000
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Pancreas Powder
CAS Number: 8049-47-6
Other descriptive name: PANCREATIN
Concentration
Sponsors
Axcan Pharma Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - After understanding the study procedures and agreeing to comply with the protocol, the subject or his/her legally authorized representative will sign an informed consent form (ICF) prior to starting any study procedures. - Subject has a diagnosis of CF that was based on one or more typical clinical features of CF phenotype, in addition to one of the following: a genotype that documents the presence of two (2) CF-causing mutation or a sweat chloride test = 60 mmol/L by quantitative pilocarpine iontophoresis on two separate occasions. - Male or female subjects aged 7 years or older. - Subject has a diagnosis of severe EPI confirmed by ELISA measurement of fecal elastase (FE-1) (test could have been performed in the past and result available). The severity of EPI will be defined by the laboratory according to their range. If EPI was never confirmed by FE-1 for a given subject, a stool sample will be collected prior to randomization and the result confirmed during the treatment phase. - Subject currently receives and had received a stable dose of lipase with either Panzytrat® 25 000 or Kreon® 25 000, for at least 30 days prior to ICF signature. - Subject is generally in good health, except for the underlying symptoms associated with CF and EPI and is clinically stable (no change in the last 30 days of physical examination) as evidenced by: medical and medication history, physical examination including vital signs during screening, and laboratory tests. - According to the qualification phase diary, subject will be able to maintain a CF standardized diet with a lipid content customized to his/her needs during the study. - Women of childbearing potential must have a negative pregnancy test at study entry and must use a medically acceptable contraceptive method for the duration of the study (i.e. from the qualification visit up to 30 days after the last study visit). Women who are not of childbearing potential will be defined as pre-menarcheal, post-menopausal (no presence of menses for at least 12 months if > 50 years of age, or no presence of menses for 24 months if = 50 years of age), surgically sterilized (tubal ligation for at least 6 months, ovariectomy or hysterectomy) or otherwise infertile. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Known contraindication, sensitivity or hypersensitivity to Panzytrat® 25 000 or Kreon® 25 000, or to any porcine protein. - Recent treatment of an emergent acute infection with oral or intravenous (IV) antibiotics that was not stopped at least 14 days prior to randomization. - Chronic use of narcotics that were not stopped at least 7 days prior to the qualification visit. - Use of any prohibited medications or products listed in the prohibited medication section of the protocol (see section 4.3.2) - History of significant bowel resection that could impair fat absorption. (N.B. Small bowel resection for meconium ileus at birth and appendectomy are acceptable.) - Presence of acute pancreatitis or exacerbation of chronic pancreatic disease - Presence of any significant gastrointestinal dysmotility disorders - Presence of chronic or severe abdominal pain - Use of enteral tube feeding over day and night - History or presence of clinically significant portal hypertension. - Presence of any condition known to increase fecal fat loss including but not limited to: celiac disease, Crohn's disease, tropical sprue, bacterial bowel infection, liver disease, lactose intolerance, pseudomembranous colitis, biliary and pancreatic cancer, radiation enteritis, Whipple’s disease, Whipple's procedure, etc. - Presence or history of complete Distal Intestinal Obstruction Syndrome (DIOS) in the past six (6) months, or two (2) or more incomplete DIOS in the past year, DIOS defined per the ESPGHAN criteria - Subject with poorly controlled diabetes as per the investigator’s opinion - Female subjects who are pregnant or breastfeeding - Presence of any condition or history of any illness, or pre-study laboratory abnormality which, in the opinion of the Investigator or sponsor, might put the subject at risk, prevent the subject from completing the study, or otherwise affect the outcome of the study. - Use of any investigational drug within 30 days prior to the date of signature of the ICF
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Assess the efficacy of Panzytrat® 25 000 compared to Kreon® 25 000 in the control of steatorrhea in cystic fibrosis (CF) subjects with exocrine pancreatic insufficiency (EPI).;Primary end point(s): The primary endpoint will be the coefficient of fat absorption (CFA%) estimated over a 3-day stool collection, after 11 days of stabilization under a specific formulation of lipase.;Secondary Objective: Assess and compare the stool frequency between Panzytrat 25000 (Panzytrat) and Kreon 25000 (Kreon) during each stool collection period in CF subjects with EPI. Assess and compare the stool consistency between Panzytrat and Kreon during each stool collection period in CF subjects with EPI. Assess and compare the stool weight between Panzytrat and Kreon during each stool collection period in CF subjects with EPI. Assess the frequency and severity of abdominal symptoms (abdominal pain and flatulence) between Panzytrat and Kreon treatment periods in CF subjects with EPI. Assess the presence of abdominal distention between Panzytrat and Kreon treatment periods in CF subject with EPI. Explore the effect of concomitant acid suppression or not on CFA% between Panzytrat and Kreon treatment periods in CF subjects with EPI. Assess the tolerability and the safety of Panzytrat in CF subjects with EPI as compared to Kreon. Describe and compare the baseline nutritional status of subjects. | — |
Countries
Germany
Contacts
Public ContactGilles Chauviere
AXCAN PHARMA SAS
Outcome results
None listed