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.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

MEDA Pharma GmbH & Co. KG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

18 Apr 2024 → 20 Dec 2024

Decision date (initial)

2024-02-19

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Meda Pharma GmbH & Co. KG (A Viatris Company) Benzstraße 1 61352 Bad Homburg, Germany

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others

Evaluation of acceptability of a new CREON formulation containing Pancreas Powder gastro-resistant pellets.

Secondary objectives 4

1. Evaluation of lipase dose use (per main meal, snack and over the day, number of capsules/new formulation)
2. Evaluation of switch potential from CREON capsules to new formulation or to a combination of capsule and new formulation including situations eligible for switch
3. Evaluation of clinical symptoms (stool frequency, stool consistency, abdominal pain, flatulence)
4. Evaluation of safety and tolerability (including local tolerability)

Conditions and MedDRA coding

patients with cystic fibrosis suffering from pancreatic exocrine insufficiency

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

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 prior to 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.

Exclusion criteria 13

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 pork or pig product).
3. Evidence of rare hereditary problems of fructose intolerance, glucose-galactose malabsorption or sucrase-isomaltase insufficiency.
4. Known predisposition to allergies (relevance as judged by the investigator).
5. 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). 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 V1, as judged by investigator.
7. History of alcohol or drug abuse within the last 2 years.
8. Exposure to another investigational product within the last three months.
9. Lack of willingness to have personal study-related data collected, archived, or transmitted according to protocol.
10. Lack of willingness or inability to co-operate adequately.
11. Anticipated non-availability for study visits/procedures.
12. Vulnerable subjects (such as persons kept in detention).
13. Lack of ability or willingness to give informed consent.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Acceptability of study treatment assessed at Visit 3 (Day 8): • Opening the container • Emptying the dose from container • Taste • Tactile sensation • Color • Time spent taking the dose • General convenience taking the dose • Ease of swallowing • Preparation of final dose to be ingested • Number of dose units to be ingested • Experience of aftertaste after ingestion of treatment • Feeling of fulness after ingestion of the treatment

Secondary endpoints 4

1. Lipase dose used per main meal, snack, and over the day, used number of capsules/new formulation.
2. Closing switch questions: o Would you switch your current treatment to the new formulation? o If you would switch to a combination of capsules and new formulation: Under which of the following circumstances, would you use the new formulation? To what extent would you use the new formulation:
3. Clinical symptoms o Stool frequency per day o Stool consistency (hard, formed, loose) o Abdominal pain on average (none, mild, moderate, severe) o Flatulence on average (none, mild, moderate, severe)
4. Safety and tolerability o Incidence of Adverse events o Incidence of stomatitis/ oral mucosal irritation (investigator) o Inspection of oral cavity /Local tolerability assessment:  Inspection of oral cavity (stomatitis/ oral mucosal irritation, bleeding, ulcera) (by investigator)  Question regarding pain in mouth (subject)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Comparator 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

MEDA Pharma GmbH & Co. KG

Sponsor organisation

MEDA Pharma GmbH & Co. KG

Address

Benzstrasse 1

City

Bad Homburg

Postcode

61352

Country

Germany

Scientific contact point

Organisation

MEDA Pharma GmbH & Co. KG

Contact name

EUClinicalTrials@viatris

Public contact point

Organisation

MEDA Pharma GmbH & Co. KG

Contact name

EUClinicalTrials@viatris

Third parties 2

Locations

1 EU/EEA country · 6 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 16 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications