Clinical Trials Register

Summary
EudraCT Number:	2016-001651-49
Sponsor's Protocol Code Number:	ALX0171-C201
National Competent Authority:	Slovakia - SIDC (Slovak)
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2016-09-07
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

Slovakia - SIDC (Slovak)

A.2

EudraCT number

2016-001651-49

A.3

Full title of the trial

A randomized, double-blind, placebo-controlled, multicenter dose ranging study of ALX-0171 in infants and young children hospitalized for respiratory syncytial virus lower respiratory tract infection

Randomizová, dvojito zaslepená, placebom kontrolovaná multicentrická klinická študia skúmajúca dávkovanie pre ALX-0171 u dojčiat a malých detí s infekciou dolných dýchacích ciest spôsobenou respiračným syncyciálnym vírusom

A.3.1

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

Phase 2b dose-ranging study of ALX-0171 in infants and young children hospitalized for RSV lower respiratory tract infection

A.3.2

Name or abbreviated title of the trial where available

Respire

A.4.1

Sponsor's protocol code number

ALX0171-C201

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/246/2014

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Ablynx NV
B.1.3.4	Country	Belgium
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	Ablynx NV
B.4.2	Country	Belgium
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Ablynx NV
B.5.2	Functional name of contact point	clinicaltrials@ablynx.com
B.5.3	Address:
B.5.3.1	Street Address	Technologiepark 21
B.5.3.2	Town/ city	Zwijnaarde
B.5.3.3	Post code	9052
B.5.3.4	Country	Belgium
B.5.4	Telephone number	+32(0)9262 00 00
B.5.5	Fax number	+32(0)9262 00 35
B.5.6	E-mail	clinicaltrials@ablynx.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	ALX-0171 Nanobody
D.3.2	Product code	ALX-0171
D.3.4	Pharmaceutical form	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	not assigned yet
D.3.9.1	CAS number	not assigned
D.3.9.2	Current sponsor code	ALX-0171
D.3.9.3	Other descriptive name	ALX-0171 Nanobody
D.3.9.4	EV Substance Code	SUB117525
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	50
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	No
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	No

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	Nebuliser solution
D.8.4	Route of administration of the placebo	Inhalation use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

RSV lower respiratory tract infection

E.1.1.1

Medical condition in easily understood language

RSV infection

E.1.1.2

Therapeutic area

Diseases [C] - Virus Diseases [C02]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	PT
E.1.2	Classification code	10061603
E.1.2	Term	Respiratory syncytial virus infection
E.1.2	System Organ Class	10021881 - Infections and infestations

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the anti-viral effect and safety of different doses of inhaled ALX-0171 in subjects hospitalized for RSV lower respiratory tract infection.

E.2.2

Secondary objectives of the trial

To evaluate the clinical activity, pharmacokinetic (PK) properties, pharmacodynamic (PD) effect and immunogenicity of different doses of inhaled ALX-0171.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Subject is a male or female infant or young child aged 28 days to < 2 years with gestational age ≥ 33 weeks at screening.
- Subject is otherwise healthy, but is hospitalized for and clinically diagnosed with RSV lower respiratory tract infection.
- Subject has a positive RSV diagnostic test at screening.
- Subject is expected to have to stay in the hospital for at least 24 hours.
- Parent(s)/legal guardian(s) provide written informed consent in accordance with locally approved consent process at screening.

E.4

Principal exclusion criteria

- Subject is known to have significant comorbidities
- Subject is known to be HIV-positive (or if the child is <6 months of age, the mother is known to be HIV-positive).
- Subject is known to be immunocompromised.
- Subject has or is suspected to have an active, clinically relevant concurrent infection
- Subject is critically ill and/or is expected to require invasive mechanical ventilation
- Subject is currently participating in any other study with investigational drug or has received an investigational drug within 4 weeks or 5 half-lives of the concerned drug (whichever is longer) prior to screening
- Subject was previously enrolled in a clinical study of ALX-0171

E.5 End points

E.5.1

Primary end point(s)

Anti-viral effect, as measured by the time needed for the viral load to drop below the assay quantification limit.

E.5.1.1

Timepoint(s) of evaluation of this end point

Throughout the in-hospital period, up to Day 14 after randomization

E.5.2

Secondary end point(s)

- Safety assessment (assessed by physical examination, AEs, laboratory assessments and vital signs) of different doses of ALX-0171
- Change from baseline in Global Severity Score
- Time to clinical response
- PK properties of ALX-0171
- Viral load profile over time
- Evaluation of serum anti-drug antibodies (ADA)

E.5.2.1

Timepoint(s) of evaluation of this end point

Throughout the study, up to Day 28 after randomization

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

Yes

E.6.7

Pharmacodynamic

Yes

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

Yes

E.6.13.1

Other scope of the trial description

Phase 2b dose-ranging study

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

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

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

Argentina

Belgium

Brazil

Bulgaria

Chile

Colombia

Croatia

Czech Republic

Estonia

Germany

Hungary

Israel

Latvia

Lithuania

Malaysia

Philippines

Poland

Slovakia

South Africa

Spain

Thailand

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

last subject last visit

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 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:

180

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

180

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

Infants and toddlers (28 days-23 months)

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

180

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, each subject will be treated according to standard clinical 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

2016-11-29

Ethics Committee Opinion of the trial application

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status	Completed

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