Glucocorticoids versus placebo for the treatment of acute exacerbation of idiopathic pulmonary fibrosis: a randomized controlled trial

Overview

Sponsor-declared trial summary

Key facts

Sponsor

GIE Groupe hospitalier Paris Saint-Joseph/Vinci

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

5 Nov 2024 → ongoing

Decision date (initial)

2024-11-05

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-514799-42-00

EudraCT number

2022-002464-75

ClinicalTrials.gov

NCT05674994

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

assess the efficacy of glucocorticoids compared to placebo on mortality at Day 30 among patients with IPF-AE

Secondary objectives 3

1. To evaluate among patients with IPF-AE the efficacy of glucocorticoids compared to placebo on: 1. Time to death 2. Overall mortality rate at Day 90 3. Death or transplantation at Day 90 4. Respiratory disease-specific mortality rate at Day 30 and 90 5. Time to worsening 6. Percentage of patients admitted to ICU 7. Percentage of patients requiring invasive ventilation 8. Length of hospital stay 9. Radiological evolution 10. Pulmonary function tests evolution
2. To evaluate among patients with IPF-AE the safety of glucocorticoids compared to placebo in particular on the occurence of: 1. Infectious disease 2. Diabetes mellitus 3. Cardiovascular disorder 4. Neuropsychological disturbances 5. Clinical laboratory evaluation
3. To compare both arms in terms of: 1. Dyspnea 2. Anxiety 3. Depression 4. Clinical status at day 15 as assessed on a 7-category ordinal scale

Conditions and MedDRA coding

IDIOPATHIC PULMONARY FIBROSIS

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Patient is ≥ 18 years of age
2. IPF or IPF (likely) diagnosis defined on 2018 international recommendations
3. Definite or suspected Acute Exacerbation defined by the international working group criteria after exclusion of alternative diagnoses of acute worsening
4. For women of childbearing age: efficient contraception for the duration of the study
5. Affiliation to the social security
6. Patient able to understand and sign a written informed consent form or in case of incapacity of the patient to a relative whom understand and sign a written informed consent form

Exclusion criteria 12

1. Identified etiology for acute worsening (i.e.: infectious disease)
2. Known hypersensitivity to glucocorticoids or to any component of the study treatment
3. Patient requiring mechanical ventilation or already on mechanical ventilation
4. Active bacterial, viral, fungal or parasitic infection. On swab collected, only positive for SARS-CoV-2, Influenzae A, Influenzae B and Respiratory Syncytial Virus (RSV) result, are considered active viral infection. The others viruses (i.e. Rhinovirus, Adenovirus…) are not considered to be responsible of pneumonia.
5. Active cancer
6. Patient on a lung transplantation waiting list
7. Treatment with glucocorticoids > 0.5 mg/kg/j for 3 consecutive days in the last 15 days OR treatment with glucocorticoids > 2 mg/kg/j for 3 consecutive days in the last 30 days Patient treated with long-term glucocorticoids ≤ 10mg/j for symptom management are eligible.
8. Patient participating to another interventional clinical trial
9. Documented pregnancy or lactation
10. Patient under tutorship or curatorship
11. Patient deprived of liberty
12. Patient under court protection

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Parameter: all-cause mortality rate; Timetable: Day 30.

Secondary endpoints 18

1. vital status assessment at Day 30 and Day 90 with time (in days) between randomization and death
2. Overall mortality at Day 90
3. Death or transplantation at Day 90
4. Mortality linked to the respiratory disease at Day 30 and Day 90
5. Time (in days) from randomization to worsening
6. percentage of patients admitted to ICU
7. Percentage of patients requiring invasive ventilation
8. length of hospital-stay
9. Progression of pulmonary fibrosis
10. Absolute change in percent Forced Vital Capacity and DLCO
11. Infectious disease
12. Capillary blood glucose monitoring and/or fasting blood glucose daily from D1 to hospital withdraw, discharge hospital visit, Day 30, Day 90
13. Cardiovascular disorder ( heart rate, blood pressure, clinical history daily)
14. Neuropsychological disturbances
15. Clinical laboratory evaluation (blood count, serum chemistries and creatinin measurement)
16. Dyspnea evaluation
17. Hospital Anxiety and Depression Scale (HADs)
18. 7-category ordinal scale at day 15

Investigational products

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

Test 2

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

GIE Groupe hospitalier Paris Saint-Joseph/Vinci

Sponsor organisation

GIE Groupe hospitalier Paris Saint-Joseph/Vinci

Address

185 Rue Raymond Losserand

City

Paris Cedex 14

Postcode

75674

Country

France

Scientific contact point

Organisation

GIE Groupe hospitalier Paris Saint-Joseph/Vinci

Contact name

Dr J-M NACCACHE

Public contact point

Organisation

GIE Groupe hospitalier Paris Saint-Joseph/Vinci

Contact name

Hélène BEAUSSIER

Locations

1 EU/EEA country · 29 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 10 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