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

NCT01327703 | Completed Phase 4 Results

• 2 other identifiers: MA-PA25CF10-012010-019267-11
• Study Type: interventional
• Target: 87
• Locations: 2 countries
• Sites: 13

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).

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Keywords

Exocrine Pancreatic Insufficiency; Cystic Fibrosis; Steatorrhea; Malabsorption; Malnutrition

Outcome Measures

Primary Outcomes (1)

• 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.

  Day 12 up to Day 15 in first and second treatment periods

Secondary Outcomes (13)

• Mean Daily Number of Stools

  Day 12 up to Day 15 in first and second treatment periods

• Percentage of Stools With Normal Consistency

  Day 12 up to Day 15 in first and second treatment periods

• Total Weight of Stools

  Day 12 up to Day 15 in first and second treatment periods

• Mean Weight Per Stool Sample

  Day 12 up to Day 15 in first and second treatment periods

• Relative Frequency of Days With Abdominal Symptoms

  Day 1 up to Day 15 in first and second treatment periods

Study Arms (2)

Panzytrat® 25,000

EXPERIMENTAL

Drug: Panzytrat® 25,000

Kreon® 25,000

ACTIVE COMPARATOR

Drug: Kreon® 25,000

Interventions

Panzytrat® 25,000 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.

Panzytrat® 25,000

Kreon® 25,000 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.

Kreon® 25,000

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:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Forest Laboratories: lead

Study Sites (13)

Klinikum-Bochum

Bochum, Germany

Location

Universitätsklinikum Carl Gustav Carus

Dresden, Germany

Location

Universitaetsklinikum Erlangen

Erlangen, Germany

Location

Jena University Hospital, Universitaetsklinikum Jena

Jena, Germany

Location

Klinikum der Universitat Munchen Medizinische Klinik-Innenstadt

München, Germany

Location

University Children's Clinic Tubingen

Tübingen, Germany

Location

Specjalistyczny Zespół Opieki Zdrowotnej nad Matką i Dzieckiem Poradnia Leczenia Mukowiscydozy

Gdansk, Poland

Location

Wojewodzki Specjalistityczny Szpital Dziect Im Sw Ludwika

Krakow, Poland

Location

Dziecięcy Szpital Kliniczny im. Prof. Antoniego Gębali

Lublin, Poland

Location

Szpital Kliniczny im Karola Jonschera

Poznan, Poland

Location

NZOZ Sanatorium Cassia Villa Medica

Rabka-Zdrój, Poland

Location

NZOZ Podkarpacki Osrodek Pulmonologii i Alergologii

Rzeszów, Poland

Location

Children's Health Memorial Institute

Warsaw, Poland

Location

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis; Steatorrhea; Malabsorption Syndromes; Malnutrition

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; Intestinal Diseases; Gastrointestinal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Nutrition Disorders

Results Point of Contact

• Title: Robert Winkler, MD, VP, Clinical Development and Operations
• Organization: Aptalis Pharma US, Inc.

1-800-472-2634

Study Officials

• Aptalis Medical Information

  Forest Laboratories

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2011
• First Posted: April 4, 2011
• Study Start: April 1, 2011
• Primary Completion: May 1, 2012
• Study Completion: May 1, 2012
• Last Updated: April 10, 2014
• Results First Posted: April 10, 2014

Record last verified: 2014-03

Locations

DE (6); PL (7)