Clinical Trials Register

Summary
EudraCT Number:	2022-000476-18
Sponsor's Protocol Code Number:	SPX-CF-001
National Competent Authority:	Slovakia - SIDC (Slovak)
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-04-05
Trial 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

Slovakia - SIDC (Slovak)

A.2

EudraCT number

2022-000476-18

A.3

Full title of the trial

Tolerability and Safety of Inhaled Colistimethate Sodium Administered Once Daily Compared to Twice Daily Dosing in Adult and Adolescent Subjects with Cystic Fibrosis and Chronic Pseudomonas Aeruginosa Lung Infection (COPILOT)

Bezpečnosť a znášanlivosť inhalačného kolistimetátu sodného podávaného raz denne v porovnaní s podaním dvakrát denne u dospelých a dospievajúcich účastníkov s cystickou fibrózou a chronickou pľúcnou infekciou spôsobenou Pseudomonas Aeruginosa (KOPILOT)

A.3.1

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

A study to test how well inhalation of Colistimethate Sodium once a day is tolerated and how safe it is compared with inhalation of Colistimethate Sodium two times a day in adult and children with Cystic Fibrosis and lung infection (COPILOT)

Klinické skúšanie toho, ako dobre je tolerovaná inhalácia kolistimetátu sodného podávaného raz denne v porovnaní s inhaláciou kolistimetátu sodného dvakrát denne u dospelých a detí s cystickou fibrózou a pľúcnou infekciou (KOPILOT)

A.3.2

Name or abbreviated title of the trial where available

COPILOT

A.4.1

Sponsor's protocol code number

SPX-CF-001

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	EnBiotix, 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	EnBiotix, 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	Accelsiors CRO and Consultancy Services Ltd
B.5.2	Functional name of contact point	Regulatory Affairs
B.5.3	Address:
B.5.3.1	Street Address	103 Haros Str.
B.5.3.2	Town/ city	Budapest
B.5.3.3	Post code	1222
B.5.3.4	Country	Hungary
B.5.4	Telephone number	003612990091
B.5.5	Fax number	00381216503380
B.5.6	E-mail	regulatory@accelsiors.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	Yes
D.2.1.1.1	Trade name	Colfinair 2 MIU Powder for Nebuliser Solution
D.2.1.1.2	Name of the Marketing Authorisation holder	PARI Pharma GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Italy
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Colistimethate sodium (CMS)
D.3.4	Pharmaceutical form	Powder for nebuliser solution
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Inhalation use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	COLISTIMETHATE SODIUM
D.3.9.1	CAS number	8068-28-8
D.3.9.4	EV Substance Code	SUB06801MIG
D.3.10	Strength
D.3.10.1	Concentration unit	million IU million international units
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	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	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

Chronic pulmonary infections due to Pseudomonas aeruginosa in patients with cystic fibrosis

E.1.1.1

Medical condition in easily understood language

Chronic lung infections caused by specific bacteria called Pseudomonas aeruginosa in patients with cystic fibrosis.

E.1.1.2

Therapeutic area

Diseases [C] - Bacterial Infections and Mycoses [C01]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10011763
E.1.2	Term	Cystic fibrosis lung
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic 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

Tolerability and Safety of Open Label inhaled CMS, comparing once daily (QD) with twice daily (BID) Administration for 28 days, measured by safety and respiratory tolerability events.

E.2.2

Secondary objectives of the trial

Additional assessments of pulmonary function and clinical events as measured by
• Pulmonary function (ppFEV1)
• Pulmonary exacerbations (PEx), severity of PEx, hospitalizations
• Additional antibacterials taken

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male and female subjects, ages 12 years and older:
a. Step A: ages 18 years and older
b. Step B: ages 12 years and older
2. Previous diagnosis of cystic fibrosis as confirmed by:
a. documented sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine iontophoresis test prior to initiation of therapy with CFTR modulators (e.g., elexacaftor, tezacaftor, ivacaftor), if applicable; or
b. two well-characterized genetic mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene; and
c. symptoms characteristic of cystic fibrosis
3. Persistent airways infection with P. aeruginosa as evidenced by:
a. positive P. aeruginosa in a sputum/deep-throat cough swab culture (or bronchoalveolar lavage [BAL]) within 6 months prior to screening; or
b. history of inhaled antibiotic treatment for suppression of P. aeruginosa
4. Stable treatment regimen of CF therapeutics for at least 2 months prior to randomization
defined as:
a. regimen of inhaled antibacterials for either at least 2 months of continuous therapy or at least a total of 56 days of consecutive on treatment periods in case of cyclical therapy (e.g. 28 day on treatment, followed by 28 days off treatment, followed by another 28 days on treatment) prior to randomization AND
b. no changes in either any current treatment regimen or initiation of treatment with hypertonic saline, dornase alfa, CFTR modulators or other CF specific therapeutics.
5. FEV1 at screening of ≥30% of predicted values
6. Arterial oxygen saturation (SpO2) ≥ 90% on room air at screening

E.4

Principal exclusion criteria

1. Current need for daily continuous O2 or requirement for > 2 liters/min at night
2. History of any investigational drug/device use within 28 days of screening or within 6 half-lives of investigational drug (whichever is longer)
3. History of lung or liver transplantation
4. History or current diagnosis of myasthenia gravis or porphyria
5. Abnormal renal or hepatic function measured in serum chemistry at screening (AST or ALT > 3x upper limit of normal (ULN); Creatinine > 2x ULN)
6. Positive pregnancy test at screening

E.5 End points

E.5.1

Primary end point(s)

Number of observed safety events during the 28-day treatment period in each treatment arm

E.5.1.1

Timepoint(s) of evaluation of this end point

28 days

E.5.2

Secondary end point(s)

1. Number of observed safety events during the 42-day follow-up period in each treatment arm
2. Number of observed safety events during the 169 days total follow-up period in each treatment arm
3. Absolute change from baseline (Day 1) to Day 14 and Day 28 in ppFEV1 in each treatment arm
4. Change in mean absolute ppFEV1 from baseline (after SABA) to post dose values during
serial spirometry at Day 1 and Day 28 in each treatment arm
5. Number of newly diagnosed total and severe PEx from baseline (Day 1) to Day 28, to Day 42 and to 169 in each treatment arm
6. Additional antibacterials taken
7. Number of hospitalizations from baseline (Day 1) to Day 28, to Day 42 and to Day 169 in each
treatment arm

E.5.2.1

Timepoint(s) of evaluation of this end point

1. 42 days
2. 169 days
3. Day 14 and Day 28
4. Day 1 and Day 28
5. Day 28, Day 42 and Day 169
6. Day 28
7. Day 28, Day 42 and Day 169

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

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

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)

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

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

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

different dose of the same medicinal product

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

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

Ukraine

Serbia

Greece

Hungary

Poland

Slovakia

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

E.8.9.2

In all countries concerned by the trial days

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:

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

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

Yes

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

F.3.3.7

Others

Yes

F.3.3.7.1

Details of other specific vulnerable populations

children aged 12-17 years

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

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)

After the end of the study the patient will return to standard treatment.

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

2022-06-28

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-09-30

P. End of Trial
P.	End of Trial Status	Ongoing

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