Open-label single-arm, multicenter clinical trial to evaluate patient acceptability of a new CREON formulation of Pancreas Powder gastro-resistant pellets in patients with cystic fibrosis suffering from pancreatic exocrine insufficiency.
Pancreatic exocrine insufficiency
This is an open-label single-arm, multicenter Phase I study testing a new CREON formulation of Pancreas Powder gastro-resistant pellets in the clinic of existing patients with cystic fibrosis (CF) who suffer from pancreatic exocrine insufficiency (PEI) and meet eligibility criteria. The study will begin with a one-week Run-in period when participants will continue using their standard PERT treatment with CREON capsules provided by the sponsor. After that week, study participants will be enrolled into the study (Visit 2) and administer CREON new formulation in a dose-comparable manner for one week until Visit 3. Treatment will be administered per oral (PO) per meal/snack for 7 days. A final follow-up telephone call will be scheduled one week after Visit 3. CREON capsules are available in different dosage strengths, depending on the country. However, patients often require treatment with high doses. As capsule shell volume and the availability of highly active starting material used in the active substance production, currently limit high-strength capsule doses, subjects have no alternative than to take multiple, rather large capsules several times a day which is perceived as inconvenient by some subjects. Participants will be instructed to record the date, time, dose of CREON administered, number of capsules / new formulation, circumstance of use, location of use as well as clinical symptoms, averse-event occurrence, use of medication for acute conditions ( e.g. headache), as well as local tolerability throughout the medication period in a paper-based diary. At the end of the treatment, patients will be answering a questionnaire which will help to know about the acceptability of the new formulation.
1. Male and female subjects of any ethnic origin 2. At least 18 years of age 3. Cystic fibrosis (documented by two sweat tests or by gene analysis) 4. On treatment with PERT with CREON capsules on the current dose for at least 4 weeks before entry in the study and with satisfactory symptom control (e.g. stool frequency and consistency, meteorism/flatulence, abdominal pain) with a dose of at least two CREON capsules per main meal 5. Willing to comply with the requirements of the study protocol 6. Written informed consent
1. Evidence of severe disease, or any other relevant condition (other than CF) as revealed by history or physical examination which might limit participation in or completion of the study 2. History of allergic reaction or hypersensitivity to pancreatin or excipients of CREON (e.g. lipase, amylase, protease, dimethicone, gelatin, xanthan gum, citric acid, red colorant), or any porc or pig product) 3. Known predisposition to allergies 4. Positive β-human chorionic gonadotropin (HCG) pregnancy test, established pregnancy, or breast-feeding at Visit V1. Women of childbearing potential not using at least one effective method of contraception (preferably one whose effectiveness is not dependent on the user, such as an intrauterine device or implant). 5. Women on oral hormonal contraceptives or vaginal ring must agree to use another non-hormonal acceptable highly effective method of contraception in addition to the oral hormonal contraception or vaginal ring 6. Clinically relevant acute infections, febrile disease, or any acute condition (illness) two weeks prior to Visit 1 (V1), as judged by the investigator 7. Lack of suitability for the study: 7.1. History of alcohol or drug abuse within the last 2 years 7.2. Exposure to another investigational product within the last three months 8.0. Administrative reasons: 8.1. Lack of willingness to have personal study-related data collected, archived or 8.2. Transmitted according to protocol 9.3. Lack of willingness or inability to co-operate adequately 8.4. Anticipated non-availability for study visits/procedures 8.5. Vulnerable subjects (such as persons kept in detention) 8.6. Lack of ability or willingness to give informed consent