Clinical trial NCT01327703: Control of Steatorrhea in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
Official title: An Open-label, Multicenter, Randomized, Cross-over Study to Compare the Safety and Efficacy of PANZYTRAT® 25,000 to KREON® 25,000 in the Control of Steatorrhea in Subjects Aged 7 Years and Older With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (EPI)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Keywords: Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Steatorrhea
Malabsorption
Malnutrition
Lead sponsor: Forest Laboratories
Sponsor class: INDUSTRY
First posted: 2011-04-04
Enrollment: 87
Sex: ALL
Minimum age (years): 7
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Percent Coefficient of Fat Absorption (CFA)
timeFrame: Day 12 up to Day 15 in first and second treatment periods
Secondary outcomes:
measure: Mean Daily Number of Stools
timeFrame: Day 12 up to Day 15 in first and second treatment periods
measure: Percentage of Stools With Normal Consistency
timeFrame: Day 12 up to Day 15 in first and second treatment periods
measure: Total Weight of Stools
timeFrame: Day 12 up to Day 15 in first and second treatment periods
measure: Mean Weight Per Stool Sample
timeFrame: Day 12 up to Day 15 in first and second treatment periods
measure: Relative Frequency of Days With Abdominal Symptoms
timeFrame: Day 1 up to Day 15 in first and second treatment periods
measure: Percentage of Participants With Abdominal Distension
timeFrame: Day 1 up to Day 15 in first and second treatment periods
measure: Percent Coefficient of Fat Absorption (CFA) Based on Concomitant Use of Proton Pump Inhibitors (PPIs)
timeFrame: Day 12 up to Day 15 in first and second treatment periods
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs)
timeFrame: Baseline up to 30 days after last dose
measure: Nutritional Status as Assessed by Body Weight
timeFrame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation
measure: Nutritional Status as Assessed by Body Mass Index (BMI)
timeFrame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation
measure: Nutritional Status as Assessed by Electrolytes Level
timeFrame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation
measure: Nutritional Status as Assessed by Albumin, Serum Transferrin and Hemoglobin Level
timeFrame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation
measure: Nutritional Status as Assessed by Hematocrit Level
timeFrame: Baseline, end of treatment (within 3 days after Day 15 of first and second treatment periods) or early discontinuation
Other outcomes:
Locations:
city: Bochum
country: Germany
facility: Klinikum-Bochum
city: Dresden
country: Germany
facility: Universitätsklinikum Carl Gustav Carus
city: Erlangen
country: Germany
facility: Universitaetsklinikum Erlangen
city: Jena
country: Germany
facility: Jena University Hospital, Universitaetsklinikum Jena
city: München
country: Germany
facility: Klinikum der Universitat Munchen Medizinische Klinik-Innenstadt
city: Tubingen
country: Germany
facility: University Children's Clinic Tubingen
city: Gdansk
country: Poland
facility: Specjalistyczny Zespół Opieki Zdrowotnej nad Matką i Dzieckiem Poradnia Leczenia Mukowiscydozy
city: Krakow
country: Poland
facility: Wojewodzki Specjalistityczny Szpital Dziect Im Sw Ludwika
city: Lublin
country: Poland
facility: Dziecięcy Szpital Kliniczny im. Prof. Antoniego Gębali
city: Poznan
country: Poland
facility: Szpital Kliniczny im Karola Jonschera
city: Rabka Zdrój
country: Poland
facility: NZOZ Sanatorium Cassia Villa Medica
city: Rzeszow
country: Poland
facility: NZOZ Podkarpacki Osrodek Pulmonologii i Alergologii
city: Warszawa
country: Poland
facility: Children's Health Memorial Institute
Study officials:
affiliation: Forest Laboratories
name: Aptalis Medical Information
role: STUDY_DIRECTOR
References: