Potential of FX06 to improve disease severity in Acute Respiratory Distress Syndrome patients (Ixion 2.0)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

F4-Pharma Ges.m.b.H.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

8 Mar 2022 → 31 Mar 2025

Decision date (initial)

2024-12-02

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-519104-27-00

EudraCT number

2021-005059-35

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

The primary objective is to demonstrate a difference in the time to initial unassisted breathing (UAB) in patients receiving FX06 compared to patients receiving placebo until day 28.
• Time to initial unassisted breathing (UAB) until day 28 in both treatment groups.

Secondary objectives 8

1. Assessment and treatment comparison of the following objectives at all available visits and time points between the FX06 and placebo group: Disease progression/improvement
2. Lung function (partial oxygen pressure in the blood, fraction of inspired oxygen, Horowitz index) at BL, D3, D6, D10 and D28
3. Systemic inflammation (changes and normalisation in troponin, procalcitonin, CRP, leukocyte count, ferritin, D-dimers, lactate, lactate dehydrogenase, albumin, IL-6)
4. Survival / Mortality
5. Capillary refill time
6. Duration of hospital and ICU stay
7. Subgroup analysis by concomitant medication, age, sex, and ARDS cause
8. Safety parameters (evaluated for safety set)

Conditions and MedDRA coding

Acute Respiratory Distress Syndrome (ARDS)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Hospitalised patients with mild to moderate ARDS according to the Berlin Definition of ARDS or patients with assisted breathing (without the use of PEEP) and high oxygen demand (e.g., HFNO ≥ 30 L/min) who fulfil the other criteria of the Berlin Definition of ARDS (modified Berlin criteria) (for clarification see Table 1) (Note: Patients requiring PEEP will be eligible if they fulfil the criteria of the Berlin Definition as long as they are not defined as severe ARDS according to exclusion criterion 2.)
2. Patients ≥ 18 years
3. Randomization within 48h of ARDS diagnosis
4. Written informed consent obtained prior to the initiation of any protocol-required procedures by the patient or his/her legal representative
5. Patients or their legal representatives able to understand the requirements of the study and give written informed consent

Exclusion criteria 11

1. 1. >48 hours of invasive mechanical ventilation before randomization
2. 2. Severe ARDS according to the Berlin Definition
3. Evidence of other significant uncontrolled concomitant diseases or serious and/or uncontrolled diseases with a bad prognosis that are likely to interfere with the evaluation of the patient’s safety and with the study outcome as judged by the treating physician, e.g.: o Other severe advanced or chronic lung diseases (e.g., COPD Gold ≥ 3, severe silicosis) o Acute respiratory failure due to cardiac failure (NYHA ≥ III) or fluid overload o Pre-existing haematological disease with severe leukopenia, severe thrombocytopenia or severe anaemia (*1) o Severe renal impairment (*1) o Advanced hepatic insufficiency or severe liver disease (e.g., liver cirrhosis CHILD C) o Use of chronic (> 3 months) long-term oxygen therapy before randomization o Exacerbation of asthma o Septic shock
4. Any contraindications to use the IMP (e.g., allergy or intolerance against the IMP or its ingredients)
5. Is currently being detained in an institution by a governmental or judicial order
6. Do not intubate order
7. Women pregnant or breastfeeding
8. Males or females of reproductive potential not willing to use effective contraception for the duration of the study period (defined as PEARL index –1 - e.g., contraceptive pill, IUD or true sexual abstinence, bilateral tubal occlusion or male partner with vasectomy; also see chapter 19.2 for guidance)
9. Current participation in another interventional clinical trial with IMP or participation within the last 30 days
10. History of or current acohol or drug abuse (*1) (*1: text are additions only available in the protocol in France version FR V2.3 dated 15.01.2024)
11. Protected patients (i.e. those patients under guardianship and/or curatorship) (*1) (*1: text are additions only available in the protocol in France version FR V2.3 dated 15.01.2024)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary objective is to demonstrate a difference in the time to initial unassisted breathing (UAB) in patients receiving FX06 compared to patients receiving placebo until day 28. • Time to initial unassisted breathing (UAB) until day 28 in both treatment groups

Secondary endpoints 8

1. at all available visits and time points between the FX06 and placebo group • Disease progression/improvement
2. Lung function (partial oxygen pressure in the blood, fraction of inspired oxygen, Horowitz index) at BL, D3, D6, D10 and D28
3. Systemic inflammation (changes and normalisation in troponin, procalcitonin, CRP, leukocyte count, ferritin, D-dimers, lactate, lactate dehydrogenase, albumin, IL-6)
4. Survival / Mortality
5. Capillary refill time
6. Duration of hospital and ICU stay
7. Subgroup analysis by concomitant medication, age, sex, and ARDS cause
8. Safety parameters (evaluated for safety set)

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

F4-Pharma Ges.m.b.H.

Sponsor organisation

F4-Pharma Ges.m.b.H.

Address

Gersthoferstrasse 103/23

City

Vienna

Postcode

1180

Country

Austria

Scientific contact point

Organisation

F4-Pharma Ges.m.b.H.

Contact name

Dr. Petra Wülfroth

Public contact point

Organisation

F4-Pharma Ges.m.b.H.

Contact name

Dr. Petra Wülfroth

Third parties 4

Locations

5 EU/EEA countries · 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 31 files

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

Application history

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