Clinical trial NCT01641393: Safety and Efficacy Study of 2 Pancreatic Enzymes for Treatment of Exocrine Pancreatic Insufficiency in Cystic Fibrosis.
Official title: A Randomised, Double-Blind, Active-Controlled, Two-Treatment, Crossover, Multinational, Multicentre Study to Compare Two Pancreatic Enzyme Products in the Treatment of Exocrine Pancreatic Insufficiency in Subjects With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Exocrine Pancreatic Insufficiency: Cystic Fibrosis
Keywords: Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Pancreatic Insufficiency
Lead sponsor: Forest Laboratories
Sponsor class: INDUSTRY
First posted: 2012-07-16
Enrollment: 96
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to further evaluate the safety and efficacy of EUR-1008 as compared to Kreon® in the treatment of exocrine pancreatic insufficiency associated with Cystic Fibrosis in subjects 12 years of age and older.


Eligibility criteria: Inclusion Criteria:

1. Definitive diagnosis of CF based on the following:

   * One clinical feature consistent with CF and
   * Either a genotype with 2 identifiable mutations known to cause CF or a sweat chloride concentration \>60 mEq/L by pilocarpine iontophoresis
2. Pancreatic insufficiency documented by a monoclonal faecal elastase (FE) 100 μg/g stool at screening (test results within the previous 12 months are acceptable)
3. Currently receiving pancreatic enzyme replacement therapy
4. Adequate nutritional status based on the following: body mass index (BMI) \>19 kg/m2 in adult subjects or a BMI percentile 10th percentile for age in adolescent (12 to 17 years age group) subjects
5. Are clinically stable with no evidence of concomitant illness or acute upper or lower respiratory tract infection that requires antibiotics during the 7-day interval prior to screening and preceding entry into this clinical study

Exclusion Criteria:

1. Age \<12 years
2. Known contraindication, hypersensitivity, or intolerance to pork or other porcine PEPs
3. Current uncontrolled diabetes mellitus
4. History of solid organ transplantation
5. History of surgery affecting the bowel function and weight gain

Primary outcomes:
measure: Coefficient of Fat Absorption over 72 hours (CFA-72h)
timeFrame: 72 hours
Secondary outcomes:
measure: Body weight
timeFrame: 58 days.
measure: Coefficient of nitrogen absorption
timeFrame: 72 hours
measure: Control of signs and symptoms of EPI
timeFrame: 2- 14 day periods
measure: Impact on overall health, daily life, perceived well-being, and symptoms
timeFrame: 58 days
measure: Total cholesterol, calculated LDL-C, HDL-C
timeFrame: 58 days
measure: Treatment Emergent Adverse Events
timeFrame: 78 days
measure: Standard safety laboratory tests
timeFrame: 58 days
measure: Vital signs
timeFrame: 78 days
measure: Fat-soluble vitamins A, D, and E
timeFrame: 58 days
Other outcomes:
Locations:
city: Antwerp
country: Belgium
facility: University Hospital Antwerp - Universitair Ziekenhuis Antwerpen (UZA)
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent, Centrum voor Mucoviscidose
city: Pleven
country: Bulgaria
facility: Clinic of Paediatric Diseases at UMHAT Dr Georgi Stranski
city: Plovdiv
country: Bulgaria
facility: Clinic of Genetic and Paediatric Diseases at UMHAT Sveti Georgi
city: Ruse
country: Bulgaria
facility: Specialized Hospital for Active Treatment of Pulmological and Phtisiatric Disease
city: Varna
country: Bulgaria
facility: Multiprofile Clinic for Specialized Pediatric Clinic at MHAT Sveta Marina
city: Montpellier Cedex 5
country: France
facility: Hôpital Arnaud de Villeneuve
city: Strasbourg Cedex
country: France
facility: Nouvel Hôpital Civil
city: Bochum
country: Germany
facility: Ruhr-Universitaet Bochum - St. Josef Hospital
city: Dresden
country: Germany
facility: Universitaetsklinikum Carl Gustav Carus an der TU Dresden
city: Essen
country: Germany
facility: Ruhrlandklinik, Westdeutsches Lungenzentrum am Universitaetsklinikum Essen gGmbH
city: Essen
country: Germany
facility: Universitätsklinikum Essen Zentrum f.Kinderheilkunde Klinik f.Kinderheilkunde III
city: Jena
country: Germany
facility: Universitatsklinikum Jena, Klinik fur Kinder und Jugendmedizin
city: Muenchen
country: Germany
facility: Medizinische Klinik Innenstadt
city: Tuebingen
country: Germany
facility: Universitaetsklinik für Kinder- und Jugendmedizin Tuebingen
city: Ancona
country: Italy
facility: Azienda Ospedaliero Universitaria, Ospedali Riuniti, Ospedale Pediatrico G. Salesi
city: Bari
country: Italy
facility: Azienda Ospedale Policlinico di Bari
city: Catania
country: Italy
facility: University of Catania
city: Contesse -Messina
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico G. Martino, Gastroenterologia Pediatrica E Fibrosi Cistica
city: Naples
country: Italy
facility: University Federico II of Naples, Pediatrics Department
city: Orbassano (Torino)
country: Italy
facility: Clinica di Malattie dell'Apparato Respiratorio
city: Parma
country: Italy
facility: Istituto Azienda Ospedaliera Universitaria
city: Roma
country: Italy
facility: Universita degli Studi di Roma La Sapienza, Azienda Policlinico Umberto I, Dipartimento di Pediatria, Centro Fibrosi Cistica Regione Lazio
city: Rome
country: Italy
facility: Bambino Gesu Hospital, Cystic Fibrosis Unit
city: Turin
country: Italy
facility: Ospedale Infantile Regina Margherita
city: Verona
country: Italy
facility: Azienda Ospedaliera Universitaria Integrata, Centro Fibrosi Cistica-Ospedale Civile Maggiore
city: Gdansk
country: Poland
facility: Specjalistyczny Zespot Opieki Zdrowotnej nad Matka i Dzieckiem Poradnia Leczenia Mukowiscydozy
city: Karpacz
country: Poland
facility: Centrum Pulmonologii i Alergologii w Karpaczu
city: Lodzi
country: Poland
facility: Wojewodzki Szpital Specjalistyczny im Kopernika
city: Lublin
country: Poland
facility: ALERGOTEST s.c Specjalistyczne Centrum Medyczne
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: Centrum Zdrowia Matki, Dziecka i Mlodziezy
city: Warszawa
country: Poland
facility: IRMED Irena Wojciechowska
city: Liverpool
country: United Kingdom
facility: Liverpool Heart and Chest Hospital
city: Sheffield
country: United Kingdom
facility: Sheffield Children's Hospital, The Academic Unit of Child Health
Study officials:
affiliation: Sheffield Children's Hospital, The Academic Unit of Child Health
name: Christopher Taylor, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Somaraju URR, Solis-Moya A. Pancreatic enzyme replacement therapy for people with cystic fibrosis. Cochrane Database Syst Rev. 2020 Aug 5;8(8):CD008227. doi: 10.1002/14651858.CD008227.pub4.
citation: Taylor CJ, Thieroff-Ekerdt R, Shiff S, Magnus L, Fleming R, Gommoll C. Comparison of two pancreatic enzyme products for exocrine insufficiency in patients with cystic fibrosis. J Cyst Fibros. 2016 Sep;15(5):675-80. doi: 10.1016/j.jcf.2016.02.010. Epub 2016 Mar 21.