Tralulala-Azi

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Rigshospitalet

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Bacterial Infections and Mycoses [C01], Diseases [C] - Respiratory Tract Diseases [C08]

Decision date (initial)

2025-11-14

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Boernelungefonden · Rigshospitalets Forskningsfond

External identifiers

EU CT number

2024-518730-88-00

ClinicalTrials.gov

NCT06409299

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To determine the efficacy of 6 months of maintenance treatment with AZI on incidence of respiratory exacerbations in subjects with structural lung damage or congenital lung and airway malformations, 0-72 months of age

Secondary objectives 1

1. To determine the efficacy of 6 months of maintenance treatment with AZI on the severity of respiratory exacerbations in subjects with structural lung damage or congenital lung and airway malformations, 0-72 months of age

Conditions and MedDRA coding

Chronic Atelectasis

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Radiologically or/and bronchoscopy verified structural lung damage or congenital lung and airway malformations
2. Outpatient affiliation with one of the three specialized pediatric pulmonology centers in Denmark.
3. At risk of or already documented respiratory infections requiring antibiotics.
4. Age between 0-72 months.
5. Parents provide written informed consent for their child to participate in the trial, according to local regulations.

Exclusion criteria 10

1. Asthmatic challenges: Patients without any of the conditions mentioned in table 1 repeatedly experiencing asthmatic problems are not eligible for this study
2. Cystic fibrosis (CF) or Primary Ciliary Dyskinesia (PCD): Patients with a CF or PCD diagnosis will be excluded
3. Children already receiving AZI 3 times a week to prevent infections
4. Impaired liver function: Children with an alanine transaminase (ALAT) > twice the upper limits of normal will be excluded
5. Impaired kidney function: Children with GFR < 50 ml/min will be excluded
6. Neurological or psychiatric disorders will be excluded
7. Prolonged QT interval: Patients with either congenital or acquired prolonged QT interval will be excluded
8. Heart disease: Patients with clinically relevant bradycardia, cardiac arrhythmia or severe heart failure are not eligible for this study
9. Allergy to macrolide antibiotics: documented allergy to macrolide antibiotics (extremely rare) will result in exclusion from the study
10. Current treatment with medication known to interact with AZI: ciclosporin, coumarin-like oral anticoagulants (e.g. warfarin), digoxin, ergotamine derivatives (e.g. methylergometrine), nelfinavir, rifabutin and active substances known to prolong QT interval such as amiodarone and other class ΙA and class ΙΙΙ antiarrhythmics, cisapride, terfenadin, moxifloxacin and levofloxacin, antipsychotic agents such as pimozide and antidepressants such as citalopram

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Difference in the number of respiratory exacerbations between the treatment groups in the intervention period in the intervention period

Secondary endpoints 3

1. Difference in duration of exacerbations between the treatment groups in the intervention period
2. Difference in number of hospital admissions between the treatment groups in the intervention period
3. Difference in duration of hospital admissions between the treatment groups in the intervention period

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Rigshospitalet

Sponsor organisation

Rigshospitalet

Address

Blegdamsvej 9

City

Copenhagen Oe

Postcode

2100

Country

Denmark

Scientific contact point

Organisation

Rigshospitalet

Contact name

Frederik Buchvald

Public contact point

Organisation

Rigshospitalet

Contact name

Marika Nathalie Rønne

Third parties 2

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 9 files

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

Application history

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