Clinical study to demonstrate the same clinical efficacy and tolerability of budesonide orodispersible tablets (taken twice a day compared to once a day) for the treatment of esophagus inflammation in adults

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Dr. Falk Pharma GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

21 May 2021 → ongoing

Decision date (initial)

2024-09-15

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2024-515229-26-00

EudraCT number

2020-001314-37

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

To prove the non-inferiority of 1 x 2 mg/d vs. 2 x 1 mg/d budesonide orodispersible tablets for the induction of histological remission in adult patients with active eosinophilic esophagitis (EoE)

Secondary objectives 3

1. To further assess EoE-associated clinical, endoscopic, and histological findings after 6 weeks treatment with budesonide orodispersible tablets
2. To study safety and tolerability as assessed by adverse events and laboratory parameters
3. To assess patients quality of life

Conditions and MedDRA coding

eosinophilic esophagitis

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

Yes

IPD plan description

It is not yet decided which data will be made available. Neither a time frame can be indicated yet.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Signed informed consent
2. Man or woman 18 to 75 years of age
3. Confirmed diagnosis of eosinophilic esophagitis (EoE)
4. Active symptomatic and histological EoE
5. At least 4 points in Patient's Global Assessment (PatGA) concerning the severity of EoE symptoms at baseline visit

Exclusion criteria 9

1. Gastroesophageal reflux disease (GERD)
2. Achalasia, scleroderma esophagus, or systemic sclerosis
3. Clinically evident causes for esophageal eosinophilia other than EoE
4. Any concomitant esophageal disease and relevant active gastrointestinal disease
5. Abnormal laboratory values, presence of or suspected relevant concomitant disease(s), that could affect study-specific assessments and/or their evaluation, or might compromise patient's safety and/or compliance (e.g. severe organic or psychiatric diseases/disorders, infectious diseases associated with clinical signs, liver cirrhosis, portal hypertension, cardiovascular disease, diabetes mellitus, osteoporosis, active peptic ulcer disease
6. History of cancer, gastrointestinal bleeding, esophageal surgery, esophageal dilation procedures or need for an immediate endoscopic intervention due to a stricture
7. Diagnosis of chickenpox, herpes zoster, or measles within the last 3 months prior to baseline
8. Treatment with medication, that could compromise/influence the effects of the study treatment, assessment of the endpoints and/or patients' safety, during or within too narrow timeframe of the clinical trial (e.g. immunosuppressants, CYP3A4 inhibitors, live vaccination, non-stable treatment with proton pump inhibitors)
9. Exiting or intended pregnancy or breast-feeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of patients with histological remission

Secondary endpoints 5

1. Change in the peak eos/mm2 hpf from baseline to week 6 (LOCF)
2. Change in the severity of dysphagia
3. Change in the severity of odynophagia
4. Change of overall severity of EoE symptoms
5. Change in the total weekly Eosinophilic Esophagitis Activity Index

Investigational products

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

Test 1

Comparator 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dr. Falk Pharma GmbH

Sponsor organisation

Dr. Falk Pharma GmbH

Address

Leinenweberstrasse 5, Hochdorf Hochdorf

City

Freiburg Im Breisgau

Postcode

79108

Country

Germany

Scientific contact point

Organisation

Dr. Falk Pharma GmbH

Contact name

CTIS – Scientific Request

Public contact point

Organisation

Dr. Falk Pharma GmbH

Contact name

Clinical Research and Development

Third parties 8

Locations

4 EU/EEA countries · 23 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

Documents 66 files

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

Application history

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