A study to test Inhaled Molgramostim in Pediatric Participants with Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Savara ApS

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

11 Sep 2025 → ongoing

Decision date (initial)

2025-04-24

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Savara ApS

External identifiers

EU CT number

2024-512039-66-00

ClinicalTrials.gov

NCT06431776

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Pharmacokinetic, Pharmacodynamic

To investigate efficacy of inhaled molgramostim in participants with aPAP, from 6 years to less than 18 years of age.

Secondary objectives 1

1. To investigate safety of inhaled molgramostim in participants with aPAP, from 6 years to less than 18 years of age.

Conditions and MedDRA coding

Autoimmune pulmonary alveolar proteinosis

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002282-PIP01-17

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Be ≥6 and <18 years of age, at the time of signing the informed consent and informed assent (if applicable).
2. Have a history of pulmonary alveolar proteinosis, based on examination of a lung biopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram of the chest.
3. Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP prior to screening.
4. Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening.

Exclusion criteria 2

1. Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production.
2. Have undergone treatment with WLL within 1 month of Baseline.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.

Secondary endpoints 7

1. Change in Hb-adjusted % predicted DLCO from Baseline to Week 48.
2. Absolute change from Baseline in oxygen saturation (SpO2) at rest after 24 and 48 weeks of treatment.
3. Absolute change from Baseline in 6-minute walk distance (6MWD) after 24 and 48 weeks of treatment.
4. Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score after 24 and 48 weeks of treatment.
5. AEs, including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain).
6. Titers of anti-GM-CSF antibodies at Baseline and after 4, 12, 24, 48, and 52 weeks.
7. Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted) and forced vital capacity (FVC) (% predicted) after 24 and 48 weeks of treatment.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Savara ApS

Sponsor organisation

Savara ApS

Address

Amaliegade 10

City

Copenhagen K

Postcode

1256

Country

Denmark

Scientific contact point

Organisation

Savara ApS

Contact name

Savara ApS

Public contact point

Organisation

Savara ApS

Contact name

Savara ApS

Third parties 3

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 26 files

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

Application history

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