Clinical Trials Register

Summary
EudraCT Number:	2014-005260-15
Sponsor's Protocol Code Number:	FPCLI002
National Competent Authority:	Czechia - SUKL
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2017-06-29
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Czechia - SUKL

A.2

EudraCT number

2014-005260-15

A.3

Full title of the trial

A Phase III Double-blind, Randomised, Parallel Group Comparison of the Efficacy and Safety of FP-1201-lyo (Recombinant Human Interferon Beta-1a) and Placebo in the Treatment of Patients with Moderate or Severe Acute Respiratory Distress Syndrome

Dvojitě zaslepené randomizované klinické hodnocení fáze III s paralelními skupinami porovnávající účinnost a bezpečnost přípravku FP-1201-lyo (rekombinantní lidský interferon beta 1-a) oproti placebu v léčbě pacientů se středně těžkým až těžkým syndromem akutní dechové tísně

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A study to assess effectiveness and safety of a new drug FP-1201-lyo (recombinant human interferon beta-1a) in patients having acute respiratory distress syndrome (ARDS)

A.3.2

Name or abbreviated title of the trial where available

INTEREST study

A.4.1

Sponsor's protocol code number

FPCLI002

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02622724

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Faron Pharmaceuticals Ltd
B.1.3.4	Country	Finland
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Faron Pharmaceuticals Ltd
B.4.2	Country	Finland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Faron Pharmaceuticals Ltd
B.5.2	Functional name of contact point	CMO, VP Drug Development
B.5.3	Address:
B.5.3.1	Street Address	Joukahaisenkatu 6
B.5.3.2	Town/ city	Turku
B.5.3.3	Post code	FI-20520
B.5.3.4	Country	Finland
B.5.4	Telephone number	+35840149 5277
B.5.6	E-mail	matti.karvonen@faron.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/07/505
D.3 Description of the IMP
D.3.1	Product name	Traumakine
D.3.2	Product code	FP-1201-lyo
D.3.4	Pharmaceutical form	Lyophilisate for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Interferon beta-1a
D.3.9.1	CAS number	220581-49-7
D.3.9.2	Current sponsor code	FP-1201
D.3.9.4	EV Substance Code	SUB12440MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	11.2
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	Yes
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	Yes
D.3.11.13.1	Other medicinal product type	Pre-filled syringe containing 1.0 ml water for injection for reconstitue lyophilised IMP.

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Lyophilisate for solution for injection
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Adult patients diagnosed with moderate or severe Acute Respiratory Distress Syndrome (ARDS)

E.1.1.1

Medical condition in easily understood language

Adult acute respiratory failure.

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10003083
E.1.2	Term	ARDS
E.1.2	System Organ Class	100000004855

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To demonstrate the efficacy of FP-1201-lyo in improving the clinical course and outcome based on survival and need for mechanical ventilation in patients with moderate or severe acute respiratory distress syndrome (ARDS)

E.2.2

Secondary objectives of the trial

Safety
• the safety of FP-1201-lyo compared with placebo

Efficacy
• 28-day all-cause mortality of FP-1201-lyo compared with placebo
• all-cause mortality at other selected time points
• the efficacy of FP-1201-lyo compared with placebo by assessing:
- days free of organ failure, of renal support, of vasoactive support, of mechanical ventilation and number of intensive care unit (ICU) -free days
- number of days in hospital
• the immunogenicity
• the pharmacodynamics (PD) of FP-1201-lyo with myxovirus resistance protein A (MxA)
• patient outcomes for respiratory and neurological functioning and quality of life (QoL)

Pharmacoeconomics
• selected parameters

Exploratory
• gas exchange during mechanical ventilation
• the PD of FP-1201-lyo using the (CD)73 biomarker
• selected potential inflammatory markers (PIM)
• Blood sample for future pharmacogenetic analysis
• all-cause mortality, QoL, and respiratory and neurological functioning follow-up (Day 360)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

All patients must be intubated and mechanically ventilated to diagnose ARDS and be eligible for the study
1. Patient has a diagnosis of moderate or severe ARDS according to the Berlin definition of ARDS:
1.1 Acute onset of respiratory failure within 1 week of a known clinical insult or new or worsening respiratory symptoms
1.2 Respiratory failure associated with known ARDS risk factors and not fully explained by either cardiac failure or fluid overload (an objective assessment of cardiac failure or fluid overload is needed if no risk factors for ARDS [moderate or severe ARDS] are present)
1.3 Radiological abnormalities on chest X-ray or on computerised tomography scan, i.e., bilateral opacities, that are not fully explained by effusions, nodules, masses or lobar/lung collapse
1.4 Hypoxaemia:
• Moderate ARDS: PaO2/FiO2 >100 mmHg (>13.3 kPa) to ≤200 mmHg (≤26.6 kPa) with positive end expiratory pressure (PEEP) ≥5 cmH2O
• Severe ARDS: PaO2/FiO2 ≤100 mmHg (≤13.3 kPa) with PEEP ≥5 cmH2O
2. The radiological and hypoxaemia criteria (1.3 and 1.4) must be met within the same 24-hour period. The time of onset of ARDS is when the last of the two specified ARDS criteria is met
3. Administration of the first dose of study drug must be planned to take place within 48 hours of moderate or severe ARDS diagnosis
4. Patient is intubated and mechanically ventilated
5. A signed informed consent form from the patient or the patient’s personal legal representative or a professional legal representative must be available
6. Patient is aged ≥18 years

E.4

Principal exclusion criteria

1. Woman known to be pregnant, lactating or with a positive (urine or serum test) or indeterminate (serum test) pregnancy test
2. Patient is simultaneously taking part in another pharmacotherapy protocol
3. Patient is not expected to survive for 24 hours
4. Patient has an underlying clinical condition where, in the opinion of the Investigator, it would be extremely unlikely that the patient would come off ventilation, e.g., motor neurone disease, Duchenne muscular dystrophy, or rapidly progressive interstitial pulmonary fibrosis
5. Patient has severe chronic obstructive pulmonary disease requiring long-term home oxygen therapy or mechanical ventilation (non-invasive ventilation or via tracheotomy) except for continuous positive airway pressure (CPAP) or bi-level positive airway pressure used solely for sleep-disordered breathing
6. Patient has congestive heart failure, defined as New York Heart Association class IV
7. Patient has acute left ventricular failure
8. Patient has liver failure (Child-Pugh grade C)
9. Patient has received any prior interferon
10. Patient has known hypersensitivity to natural or recombinant IFN beta or to any of the excipients
11. Patient is receiving renal dialysis therapy for chronic renal failure
12. Patient is receiving extra-corporeal membrane oxygenation, high-frequency oscillatory ventilation or any form of extra-corporeal lung support
13. Patient has had any form of mechanical ventilation (invasive or non-invasive, excluding CPAP alone) for longer than 48 hours prior to the diagnosis of ARDS
Non-invasive ventilation has to be continuously applied for at least 12 hrs per day in these 48 hours
14. Patient has burns to ≥15% of their total body surface area

E.5 End points

E.5.1

Primary end point(s)

Primary Efficacy Endpoint:
• Composite endpoint including any cause of death at D28 and days free of mechanical ventilation (VFDsurv) within 28 days among survivors

E.5.1.1

Timepoint(s) of evaluation of this end point

Day 28

E.5.2

Secondary end point(s)

•Secondary efficacy endpoints relating to the efficacy of FP-1201-lyo treatment:
- All-cause mortality at D28, D90 and D180
- Mortality in ICU up to D28
- Mortality in hospital up to D28
• Other secondary efficacy endpoints at D28:
- Days free of organ failure (Sequential Organ Failure Assessment methodology) (D28 or last day in ICU if patient has left the ICU earlier than D28, or at withdrawal)
- Days free of renal support
- Days free of vasoactive support
- Days free of mechanical ventilation
- Number of ICU free days
- Number of days in hospital
• Presence of neutralising antibodies to IFN beta-1a at baseline and D28 (or last day in ICU if patient has left the ICU earlier than D28, or at withdrawal)
• Evaluation of PD using MxA biomarker from baseline to D14
• Long-term secondary endpoints, relating to QoL, respiratory and neurological functioning at D180:
- EQ-5D-3L
- 6MWT
- FEV1

Evaluation of safety:
• Adverse events up to D28, AEs occurring after D28 if the investigator considers there is a causal relationship with the study drug and all deaths up to D360
• Physical examination, vital signs and laboratory results (biochemistry, haematology and urinalysis) up to D28 (or last day in ICU if patient has left the ICU earlier than D28, or at withdrawal)

Evaluation of pharmacoeconomics at D28:
− Days free of organ failure (D28 or last day in ICU if patient has left the ICU earlier than D28, or at withdrawal)
− Days free of renal support
− Days free of vasoactive support
− Days free of mechanical ventilation
− Number of ICU-free days
− Number of days in hospital

Exploratory endpoints relating to the efficacy of FP-1201-lyo treatment:
− Composite endpoint including mortality and days free of mechanical ventilation (VFDsurv) within 90 days among survivors
− Ordered categorical endpoint defined as improvement (severe to moderate/mild; from moderate to mild ARDS), no change or worsening (from moderate to severe/death; from severe to death) in terms of gas exchange (PaO2/FiO2 ratio) from baseline to D28 (or last day in ICU if patient has left the ICU earlier than D28, or at withdrawal)

• Change in the treatment-specific exploratory biomarker CD73 concentration from baseline to D14
• Changes in levels of PIMs, including but not limited to, interleukin-6 and -8 from baseline to D14
• A blood sample will be taken for pharmacogenetic analysis and correlation with other markers of the activity of FP-1201-lyo
• Extended long-term follow-up: 12-month mortality rate, EQ-5D-3L, 6MWT and FEV1

E.5.2.1

Timepoint(s) of evaluation of this end point

D14, D28, D90, D180 and D360

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

Yes

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Quality of life

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

180

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

120

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

Yes

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Patient treatment in ICU unit - availability of the legal representative

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

300

F.4.2.2

In the whole clinical trial

300

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None. According to normal practice.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2017-09-06

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-09-07

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2018-05-17

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