Investigation of breathable surfactant agent in patients with idiopathic lung fibrosis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Philipps-Universitaet Marburg

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Decision date (initial)

2024-07-05

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Deutsches Zentrum für Lungenforschung · Lyomark Pharma GmbH · Aerogen Limited

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the effect of inhalative Alveofact® on the Forced Vital Capacity of IPF patients after treatment compared to placebo in the same patients

Secondary objectives 5

1. To evaluate the effect of Alveofact® on the lung mechanics (except on the FVC) after treatment compared to placebo
2. To evaluate the effect of Alveofact® on the exercise capacity and gas exchange after treatment compared to placebo
3. To evaluate the effect of Alveofact® on the alveolar recruitment (imaging) after treatment compared to placebo
4. To evaluate the toxicity and safety of Alveofact® therapy during the course of the study
5. To evaluate the patient reported outcomes after treatment with Alveofact® compared to placebo

Conditions and MedDRA coding

Idiopathic pulmonary fibrosis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. IPF according to International Consensus Guidelines
2. Forced Vital Capacity (FVC) > 75% predicted
3. Diffusion Capacity for CO (TLCO) > 40% predicted
4. Capillary pO2 > 60 mm Hg at rest
5. No need for supplemental oxygen at rest
6. Treatment-naive patients or patients treated with standard of care for more than 3 months. Treatment naive patients can only be included if they have previously failed on SOC medication. No standard of care medication will be withheld from participants of the study.
7. Patient must be ≥ 18 years old
8. Signed written informed consent
9. Ability and willingness to comply with study procedures
10. Willingness of men and women of childbearing potential to use highly effective contraceptive methods

Exclusion criteria 15

1. Current / previous exacerbation / respiratory infection within the last four weeks
2. Concomitant emphysema > 15% of lung volume (by High Resolution Computed Tomography (HRCT))
3. Concomitant COPD with FEV1/FVC ratios < 70%
4. Concomitant malignant disease requiring therapy
5. Estimated life expectancy of less than 1 year for any reason
6. Renal insufficiency (GFR < 50% predicted and/or Crea > 2mg/dl)
7. Chronic liver cirrhosis > Child A
8. Any other liver disease with elevated ART/ARS >3 times the upper normal limit (UNL)
9. Any acute / chronic heart failure with ejection fraction of < 40%
10. Patients with known bronchial asthma
11. Simultaneous participation in another clinical trial with an experimental treatment
12. History of alcohol or drug abuse in the past year
13. Assessment by the investigator that the patient should not participate in the study if the patient is unlikely to be able to comply with the study procedures, limitations and requirements
14. Pregnancy or breastfeeding
15. Hypersensitivity to the active substance or any of the other ingredients

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is the mean intra-individual difference in the percentage change in FVC from baseline to the end of each inhalation phase

Secondary endpoints 17

1. Change in Total Lung Capacity (TLC) from baseline to the end of each inhalation phase
2. Change in Forced Expiratory Volume in one second (FEV1) from baseline to the end of each inhalation phase
3. Change in FEV1/FVC ratio
4. Change in parameters R5, R20, X5, RF and AX from oscillometry (IOS) from baseline to the end of each inhalation phase
5. Change in diffusion capacity for CO (TLCO), KCO from baseline to the end of each inhalation phase
6. Change in arterial partial pressure of O2 (paO2) from baseline to the end of each inhalation phase
7. Change in O2 saturation (SpO2) between rest and exercise (at the end of the 6MWT) from baseline to the end of each inhalation phase
8. Change in 6-minute walking distance (6MWT) from baseline to the end of each inhalation phase
9. Change in intratidal gas volume (ITV) from baseline to the end of each inhalation phase
10. Change in tidal impedance distribution (TID) from baseline to the end of each inhalation phase
11. Change in difference of TID over time (dTID) from baseline to the end of each inhalation phase
12. Change in surface of ventilated areas (SURF) from baseline to the end of each inhalation phase
13. Change in global inhomogeneity index (GI) from baseline to the end of each inhalation phase
14. Change in end-expiratory lung impedance (EELI) from baseline to the end of each inhalation phase
15. Change in difference of EELI over time (dEELI) from baseline to the end of each inhalation phase
16. Assessment of toxicity and safety during the course of the study: AEs, SAEs
17. Patient related outcome measures (PROMS): questionnaires KBILD, Leicester Cough and EQ-5D-5L from baseline to the end of each inhalation phase

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Philipps-Universitaet Marburg

Sponsor organisation

Philipps-Universitaet Marburg

Address

Karl-Von-Frisch-Strasse 4

City

Marburg

Postcode

35043

Country

Germany

Scientific contact point

Organisation

Philipps-Universitaet Marburg

Contact name

Dr. Sandrine Oberwinkler

Public contact point

Organisation

Philipps-Universitaet Marburg

Contact name

Dr. Sandrine Oberwinkler

Third parties 2

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Results and documents

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

Documents 10 files

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

Application history

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