Clinical Trials Register

Summary
EudraCT Number:	2018-000106-32
Sponsor's Protocol Code Number:	03-CL-1702
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2019-03-26
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

Poland - Office for Medicinal Products

A.2

EudraCT number

2018-000106-32

A.3

Full title of the trial

A Multinational, Multicenter, Masked, Randomized, Parallel Group, Controlled Study to Assess the Safety and Efficacy of Lucinactant for Inhalation versus nCPAP alone in Preterm Neonates 26 to 32 Weeks Gestational Age with Respiratory Distress Syndrome.

A.3.1

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

An international study at different sites testing the safety and effectiveness of an investigational drug called lucinactant, which is to be inhaled to treat breathing problems in babies born prematurely at a gestational age of 26 to 32 weeks.

A.4.1

Sponsor's protocol code number

03-CL-1702

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	Windtree Therapeutics, Inc.
B.1.3.4	Country	United States
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	Windtree Therapeutics, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Clinical Consulting Sp. z o.o.
B.5.2	Functional name of contact point	Clinical Operations
B.5.3	Address:
B.5.3.1	Street Address	Dzwonkowa 104
B.5.3.2	Town/ city	Tychy
B.5.3.3	Post code	43-100
B.5.3.4	Country	Poland
B.5.4	Telephone number	+48322272005
B.5.5	Fax number	+48323298426
B.5.6	E-mail	tadeusz.baron@clinicalconsulting.pl

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/3/04/216 and EU/3/04/217
D.3 Description of the IMP
D.3.1	Product name	Lyophilized lucinactant
D.3.2	Product code	R07AA-30
D.3.4	Pharmaceutical form	Nebuliser suspension
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Respiratory use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	dipalmitoylphosphatidylcholine
D.3.9.1	CAS number	63-89-8
D.3.9.2	Current sponsor code	401-000
D.3.9.3	Other descriptive name	1,2-DIPALMITOYL-SN-GLYCERO-3-PHOSPHATIDYLCHOLINE (DPPC)
D.3.9.4	EV Substance Code	SUB22681
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	22,5
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Palmitic acid
D.3.9.1	CAS number	57-10-3
D.3.9.2	Current sponsor code	401-002
D.3.9.3	Other descriptive name	PALMITIC ACID (hexadecanoic acid)
D.3.9.4	EV Substance Code	SUB14744MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	4,05
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	palmitoyloleoyl-phosphatidylglycerol, sodium salt
D.3.9.1	CAS number	268550-95-4
D.3.9.2	Current sponsor code	401-001
D.3.9.3	Other descriptive name	1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol, sodium salt
D.3.9.4	EV Substance Code	SUB126307
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	7,5
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	SINAPULTIDE
D.3.9.1	CAS number	138531-07-4
D.3.9.2	Current sponsor code	401-003
D.3.9.3	Other descriptive name	KL4 acetate
D.3.9.4	EV Substance Code	SUB10530MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.862
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	No
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	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Respiratory Distress Syndrome (RDS)

E.1.1.1

Medical condition in easily understood language

Breathing difficulty in babies born prematurely

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	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10021735
E.1.2	Term	Infant respiratory distress syndrome
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10038690
E.1.2	Term	Respiratory distress syndrome (neonatal)
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10028974
E.1.2	Term	Neonatal respiratory distress syndrome
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

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 evaluate the safety and efficacy of lucinactant for inhalation delivered by the next generation ADS device in conjunction with nCPAP, in comparison to nCPAP alone, in preterm neonates with RDS, as assessed by the incidence of and time to respiratory failure due to RDS in the first 72 hours and through 28 days of life, oxygen saturation and use of supplemental oxygen, all-cause mortality through 28 days of life, the incidence of bronchopulmonary dysplasia (BPD) at 36 weeks PMA, and the duration of mechanical ventilation. In addition, this study will evaluate the device performance and the ability to administer up to 3 lucinactant for inhalation repeat treatments.

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Signed ICF from legally authorized representative. It is recommended that consent is obtained antenatally (where permitted).
2. Gestational age: 26 + 0/7 to 32 + 6/7 weeks PMA
3. Successful implementation of non-invasive ventilation within 30 minutes after birth.
4. Spontaneous breathing.
5. Investigator determination of RDS. A chest x-ray should be obtained before treatment to confirm the diagnosis.
6. Within the first 6 hours after birth, requires an nCPAP of 5 to 7 cm H2O that is clinically indicated for at least 15 minutes with an FiO2 > 0.25 to ≤ 0.35 to maintain SpO2 of 90% to 95%. Transient (<5 minutes) FiO2 excursions outside this range do not reset the time requirement.

E.4

Principal exclusion criteria

1. A heart rate that cannot be stabilized above 100 beats per minute (bpm) within 5 minutes of birth.
2. Recurrent episodes of apnea requiring positive pressure ventilation (PPV) administered manually or mechanically through any patient interface.
3. A 5-minute Apgar score < 5.
4. Major congenital malformation(s) or craniofacial abnormalities that preclude the use of nCPAP, diagnosed antenatally or immediately after birth.
5. Clinically significant diseases or conditions other than RDS which could potentially interfere with cardiopulmonary function (eg, congenital heart disease, hydrops fetalis or congenital infection).
6. A known or suspected chromosomal abnormality or syndrome.
7. Premature rupture of membranes > 3 weeks.
8. Hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis.
9. A need for intubation and/or invasive mechanical ventilation at any time before enrollment into the study.
10. The administration (or plan for administration) of any the following:
a) Another investigational agent or investigational medical device
b) Any other surfactant agent
c) Systemic corticosteroids (other than antenatal steroids)
11. Presence of air leak (pneumothorax, pneumomediastinum, pneumopericardium, subcutaneous emphysema, or definite evidence of pulmonary interstitial emphysema [PIE]) on the baseline chest radiograph, or diagnosed via ultrasound or illumination.

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint for this study is the number of subjects with respiratory failure due to RDS or death within the first 28 days of life. Respiratory failure due to RDS is defined as intubation (including intratracheal catheter placement) for MV and/or surfactant administration.

E.5.1.1

Timepoint(s) of evaluation of this end point

within the first 72 hours of life or after 72 hours of life and within 28 days of life.

E.5.2

Secondary end point(s)

The key secondary endpoints of this study include the evaluation of the following:
• Respiratory failure through 72 hours and 28 days of life
• Time to respiratory failure through 72 hours and 28 days of life
• BPD and survival without BPD at 36 weeks PMA
• Severity of BPD at 36 weeks PMA
• Oxygen saturation and use of supplemental oxygen
• All cause mortality through 28 days of life and 36 weeks PMA

Other secondary endpoints of this study include the evaluation of the following:
• Common complications of prematurity through 36 weeks PMA
• Duration of MV and oxygen requirement through 36 weeks PMA
• Changes in fraction of inspired oxygen (FiO2), partial pressure of carbon dioxide (PCO2) and oxygen (O2) saturation, as determined by pulse oximetry (SpO2) over the first 72 hours of life, and over the first 7 days of life for FiO2.
• Number and duration of re-hospitalizations and reason for re-hospitalization through 12 months corrected age.
• Respiratory medications through 12 months corrected age.

E.5.2.1

Timepoint(s) of evaluation of this end point

the first 72 hours of life, the first 7 days of life and through 36 weeks PMA

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

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

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)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

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

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

nCPAP - Continuous positive airway pressure/nasal

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

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Chile

China

Poland

United States

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

Longer-Term Follow-Up-visit at 1-Year Corrected Age

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

Yes

F.1.1

Number of subjects for this age range:

130

F.1.1.1

In Utero

F.1.1.2

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

Yes

F.1.1.2.1

Number of subjects for this age range:

130

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)

F.1.3

Elderly (>=65 years)

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

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Paediatric patients

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

F.4.2.2

In the whole clinical trial

130

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

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

2019-06-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-06-14

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2021-01-21

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