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

NCT ID: NCT01327703

Last Updated: 2014-04-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 87 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-04-30
• Study Completion Date: 2012-05-31

Brief Summary

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).

Detailed Description

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.

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Panzytrat® 25,000

Group Type: EXPERIMENTAL

Panzytrat® 25,000

Intervention Type: DRUG

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® 25,000

Group Type: ACTIVE_COMPARATOR

Kreon® 25,000

Intervention Type: DRUG

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.

Interventions

Panzytrat® 25,000

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.

Intervention Type: DRUG

Kreon® 25,000

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.

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 7 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Forest Laboratories

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Aptalis Medical Information

Role: STUDY_DIRECTOR

Forest Laboratories

Locations

Klinikum-Bochum

Bochum, , Germany

Universitätsklinikum Carl Gustav Carus

Dresden, , Germany

Universitaetsklinikum Erlangen

Erlangen, , Germany

Jena University Hospital, Universitaetsklinikum Jena

Jena, , Germany

Klinikum der Universitat Munchen Medizinische Klinik-Innenstadt

München, , Germany

University Children's Clinic Tubingen

Tübingen, , Germany

Specjalistyczny Zespół Opieki Zdrowotnej nad Matką i Dzieckiem Poradnia Leczenia Mukowiscydozy

Gdansk, , Poland

Wojewodzki Specjalistityczny Szpital Dziect Im Sw Ludwika

Krakow, , Poland

Dziecięcy Szpital Kliniczny im. Prof. Antoniego Gębali

Lublin, , Poland

Szpital Kliniczny im Karola Jonschera

Poznan, , Poland

NZOZ Sanatorium Cassia Villa Medica

Rabka-Zdrój, , Poland

NZOZ Podkarpacki Osrodek Pulmonologii i Alergologii

Rzeszów, , Poland

Children's Health Memorial Institute

Warsaw, , Poland

Countries

Germany; Poland

Other Identifiers

2010-019267-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MA-PA25CF10-01

Identifier Type: -

Identifier Source: org_study_id