Control of Steatorrhea in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

Forest Laboratories87 enrolled

Overview

This study by Aptalis (formerly Axcan) assesses the efficacy and safety of Panzytrat® 25,000 compared to Kreon® 25,000 in the control of steatorrhea in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).

This is an open-label, Phase IV, multicenter, randomized, two-period cross-over study to compare the efficacy and safety of Panzytrat® 25,000 to Kreon® 25,000 in participants aged 7 years and older suffering from CF and EPI. The study consists of a qualification phase (5 to 15 days); two treatment periods of 14 days each (plus a 3-day window if needed) and a 3-day stool collection will be performed from Days 12 to 15. A safety follow-up phone call will be arranged 7-10 days after completion of the treatment phase or after an early discontinuation.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Exocrine Pancreatic Insufficiency Cystic Fibrosis

Interventions

Panzytrat® 25,000DRUG

Panzytrat® 25,000 capsule will be given orally daily at a stabilized dose, as per investigator's discretion, for 14 days. Stabilized dose for a participant will be the optimal dose determined during a qualification phase that precedes the first treatment period and will be based upon the participant's usual lipase and lipid intake. Total dose will not exceed 10,000 European Pharmacopoeia (Ph.Eur.) units lipase/kilogram (kg) body weight/day in either first treatment period or second treatment period.

Kreon® 25,000DRUG

Kreon® 25,000 capsule will be given orally daily at a stabilized dose, as per investigator's discretion, for 14 days. Stabilized dose for a participant will be the optimal dose determined during a qualification phase that precedes the first treatment period and will be based upon the participant's usual lipase and lipid intake. Total dose will not exceed 10,000 Ph.Eur. units lipase/kg body weight/day in either first treatment period or second treatment period.

Eligibility

Sex: ALLMin age: 7 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Participant or his/her legal representative signed informed consent form (ICF) prior to starting any study procedures * Participant with clinical diagnosis of CF 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 2 CF-causing mutation, or a sweat chloride test greater than or equal to 60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions * Participant with severe EPI confirmed by enzyme-linked immunosorbent assay (ELISA) measurement of fecal elastase-1 (FE-1) * Male or female participant aged 7 years or older * Participant currently receiving and has received a stable dose of lipase with either Panzytrat® 25,000 or Kreon® 25,000 for at least 30 days prior to ICF signature * Participant 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 histories, physical examination including vital signs during screening and laboratory tests * Participant able to maintain a CF standardized diet with a lipid content customized to his/her needs during the study according to the qualification phase diary * 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 Exclusion Criteria: * Participant with known contraindication, sensitivity or hypersensitivity to Panzytrat® 25,000 or Kreon® 25,000, or to any porcine protein * Participant who recently received treatment of an emergent acute infection with oral or intravenous (IV) antibiotics that was not stopped at least 14 days prior to randomization * Participant with chronic use of narcotics that were not stopped at least 7 days prior to the qualification visit * Participant using of any prohibited medications or products listed in the prohibited medication section of the protocol * Participant with acute pancreatitis or exacerbation of chronic pancreatic disease * Participant with history of significant bowel resection that could impair fat absorption * Participant with 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 * Participant with any significant gastrointestinal dysmotility disorders * Participant with chronic abdominal pain or severe abdominal pain at study entry * Participant using enteral tube feeding over day and night * Participant with history or presence of clinically significant portal hypertension * Participant with history or presence of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months, or 2 or more episodes of DIOS in the past year * Participant with poorly controlled diabetes as per the investigator's opinion * Female participants who are pregnant or breastfeeding * Participant with any condition or history of any illness, or pre-study laboratory abnormality which, in the opinion of the investigator or sponsor, might put the participant at risk, prevent the participant from completing the study, or otherwise affect the outcome of the study * Participant using any investigational drug within 30 days prior to the date of signature of the ICF

Locations (13)

Klinikum-Bochum

Bochum, Germany

Universitätsklinikum Carl Gustav Carus

Dresden, Germany

Universitaetsklinikum Erlangen

Erlangen, Germany

Outcomes

Primary Outcomes

Percent Coefficient of Fat Absorption (CFA)

Percent CFA was calculated as (\[fat intake - fat excretion\]/fat intake)\*100, determined in the stools which were collected over a 3-day period (Day 12 to morning of Day 15) during each treatment period. Least squares mean percent (%) CFA was calculated for Day 12 to Day 15 in first and second treatment periods. Percent CFA was based on log transformed data.

Time frame: Day 12 up to Day 15 in first and second treatment periods

Secondary Outcomes

Mean Daily Number of Stools

Mean daily number of stools of each participant was calculated from frequency of stools by the participant per day. Mean daily number of stools during the collection period (Day 12 to Day 15 in first and second treatment periods) for total participants was summarized.

Time frame: Day 12 up to Day 15 in first and second treatment periods

Percentage of Stools With Normal Consistency

Normal consistency of stool was defined as formed hard, normal or soft stool and abnormal consistency was defined as loose and unformed, liquid stool and diarrhea. Percentage of stools with normal consistency of each participant was calculated as the number of stools with normal consistency relative to the total number of stools during the collection period. Mean percentage of stool with normal consistency during the collection period (Day 12 to Day 15 in first and second treatment periods) for total participants was summarized.

Time frame: Day 12 up to Day 15 in first and second treatment periods

Total Weight of Stools

Mean total weight of stools was calculated for Day 12 to Day 15 in first and second treatment periods.

Time frame: Day 12 up to Day 15 in first and second treatment periods

Mean Weight Per Stool Sample

Mean weight per stool sample was calculated for Day 12 to Day 15 in first and second treatment periods.

Data from ClinicalTrials.gov

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