Assessment of the effect of Wharton's jelly mesenchymal stem cell preparation (WJMSCs) in the treatment of fibrotic interstitial lung diseases

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Medical University Of Gdansk

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

28 Sep 2023 → ongoing

Decision date (initial)

2024-11-07

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Medical Research Agency (Agencja Badań Medycznych)

External identifiers

EU CT number

2024-514433-38-00

EudraCT number

2021-001555-14

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Efficacy, Safety, Pharmacodynamic, Pharmacokinetic, Therapy

Evaluation of usage and safety of allogenic Mesenchymas cells of Wharton Jelly as a possible method of treatment of fibrotic interstitial lung disease especially diopathic pulmonry fibrosis and pulmonary fibrosis after COVID-19

Secondary objectives 1

1. Evaluation of: - changes in FVC - quality of life of patients - number of exacerbations during the annual total - survival during annual follow-up - pharmacokinetics and pharmacodynamics of the MSC preparation after administration

Conditions and MedDRA coding

fibrotic interstitial lung disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Group I, patients diagnosed with IPF: 1. Patients over 18 years of age 2. Diagnosis of some usual interstitial pneumonia (UIP) based on thoracic WRTK / lung parenchyma biopsy in IPF idiopathic pulmonary fibrosis 3. FVC> 40% nw 4. DLCO> 25% nw 5. FVC progression greater than 10% during the year excluding from the drug program 6. Informed consent form (ICF) to participate in the study Group II, patients diagnosed with restrictive fibrosis after COVID-19: 1. Patients over 18 years of age 2. History of severe COVID-19 pneumonia 3. Negative SARS-COV2 antigenic test , at least 4 weeks prior to study inclusion 4. Diagnosis of interstitial lung fibrosis 5. Informed consent form (ICF) to participate in the study

Exclusion criteria 1

1. 1. Inability to provide informed consent form (ICF) 2. Current or history of cancer. 3. Lower respiratory tract infection within 4 weeks prior to study enrollment. 4. Clinically proven active infection that in the investigator's opinion, may interfere with the course of the study, perform lung function measurements, or may affect the course of lung disease 5. Previous or active form of infection with HBV, HCV, HIV, mycobacterium tuberculosis, syphilis spirochete. Laboratory indicators of infection are sufficient for diagnosis without the need to identify clinical symptoms. 6. Participation in the study of an experimental medicinal product within 4 weeks prior to enrollment in the study (not less than 5 half-lives of the investigated product). 7. Significant coexisting diseases of other organs, including liver or kidney failure 8. History of liver failure, elevated levels of transaminase enzymes or exceeding the breakpoints of any of the following criteria in liver function tests: Total bilirubin above the upper limit of normal, Aspartic aminotransferase (AST) or alanine aminotransferase (ALT)> 2 × upper limit of normal, Alkaline phosphatase> 2.0 × ULN. 9. Creatinine clearance <30 ml / min by the Cockcroft-Gault formula. 10. Use of tobacco products within the 12 weeks prior to the start of the screening phase, or refusal to agree to stop using tobacco products until the last follow-up visit. 11. Pregnant women and breastfeeding or planning to become pregnant during the examination 12. Women - positive pregnancy test or no use of a medically recognized anti-conception during the study and up to 4 months after its completion, if applicable. 13. Men - expressed intention to have children during the study and up to 4 months after its completion, if applicable. 14. Excessive anxiety of the patient with regard to the procedures used in the study. 15. Any medical problem that in the opinion of the investigator, may adversely affect the patient's health, if included in the study 16. Diagnosed addiction to alcohol or psychoactive substances 17. Surgery scheduled during the study period. 18. Participation in the drug program 19. History of allergy to penicillin, streptomycin or amphotericin B

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 1. Number and severity of adverse events in the treated group and the control / placebo group 2. Inhibition of the decline in FVC (reduction by less than 10% over the course of the study, reduction significantly less than in the placebo group) 3. Improving the quality of life 4. Annual survival

Secondary endpoints 1

1. Evaluation of: - changes in FVC - quality of life of patients - number of exacerbations during the annual total - survival during annual follow-up - pharmacokinetics and pharmacodynamics of the MSC preparation after administration

Investigational products

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

Test 1

Comparator 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Medical University Of Gdansk

Sponsor organisation

Medical University Of Gdansk

Address

Ul. Marii Sklodowskiej-Curie 3a

City

Gdansk

Postcode

80-210

Country

Poland

Scientific contact point

Organisation

Medical University Of Gdansk

Contact name

Piotr Trzonkowski

Public contact point

Organisation

Medical University Of Gdansk

Contact name

Piotr Trzonkowski

Locations

1 EU/EEA country · 1 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 13 files

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

Application history

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