Clinical trial in patients with the lung disease, autoimmune pulmonary alveolar proteinosis, to assess continued treatment with molgramostim by inhalation

AN OPEN-LABEL, NON-CONTROLLED, MULTICENTRE CLINICAL TRIAL OF INHALED MOLGRAMOSTIM IN AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS PATIENTS - IMPALA-EX

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-004078-32-NL

Enrollment

Registered

2017-12-18

Start date

2018-05-08

Completion date

Unknown

Last updated

2021-04-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Autoimmune Pulmonary Alveolar Proteinosis (aPAP) MedDRA version: 20.0 Level: LLT Classification code 10037316 Term: Pulmonary alveolar proteinosis System Organ Class: 100000004855

Interventions

Product Name: Molgramostim Nebuliser solution 300 mcg Pharmaceutical Form: Nebuliser solution INN or Proposed INN: MOLGRAMOSTIM CAS Number: 99283-10-0 Concentration unit: µg/ml microgram(s)/millilitre

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Completer of the IMPALA trial 2. Females who have been post-menopausal for >1 year, or females of childbearing potential after a confirmed menstrual period using a highly efficient method of contraception (i.e. a method with =65 years) yes F.1.3.1 Number of subjects for this age range 10

Exclusion criteria

Exclusion criteria: 1. Treatment with GM-CSF products other than molgramostim nebuliser solution within three months of Baseline. 2. Treatment with any IMP other than inhaled molgramostim within four weeks of Baseline. 3. History of allergic reactions to GM-CSF. 4. Connective tissue disease, inflammatory bowel disease or other autoimmune disorder requiring treatment associated with significant immunosuppression, e.g. more than 10 mg/day systemic prednisolone. 5. Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product. 6. History of, or present, myeloproliferative disease or leukaemia. 7. Apparent pre-existing concurrent pulmonary fibrosis. 8. Any other serious medical condition which in the opinion of the investigator would make the subject unsuitable for the trial.

Design outcomes

Primary

Measure	Time frame
Main Objective: To investigate safety of long term use of inhaled molgramostim.;Secondary Objective: • To investigate effects of long term use of inhaled molgramostim on oxygenation. • To investigate effects of long term use of inhaled molgramostim on exercise capacity. • To investigate effects of long term use of inhaled molgramostim on respiratory quality of life. • To investigate frequency of need for WLL during long term use of inhaled molgramostim. • To investigate effects of long term use of inhaled molgramostim on lung function. • To investigate maintenance of effect after discontinuation of inhaled molgramostim.;Primary end point(s): Number of AEs, SAEs, adverse drug reactions (ADRs), and AEs leading to treatment discontinuation.;Timepoint(s) of evaluation of this end point: End of trial	—

Measure

Time frame

Main Objective: To investigate safety of long term use of inhaled molgramostim.;Secondary Objective: • To investigate effects of long term use of inhaled molgramostim on oxygenation. • To investigate effects of long term use of inhaled molgramostim on exercise capacity. • To investigate effects of long term use of inhaled molgramostim on respiratory quality of life. • To investigate frequency of need for WLL during long term use of inhaled molgramostim. • To investigate effects of long term use of inhaled molgramostim on lung function. • To investigate maintenance of effect after discontinuation of inhaled molgramostim.;Primary end point(s): Number of AEs, SAEs, adverse drug reactions (ADRs), and AEs leading to treatment discontinuation.;Timepoint(s) of evaluation of this end point: End of trial

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Secondary

Measure	Time frame
Secondary end point(s): • (A-a)DO2 during the trial • 6MWD during the trial • SGRQ total score during the trial • Frequency of WLL during the trial • DLCO (% predicted), FEV1 (% predicted), and FVC (% predicted) during the trial • PaO2 and disease severity score (DSS) during the trial • Need for oxygen supplement therapy during the trial • Number of subjects not requiring treatment for aPAP and time off treatment after discontinuation of inhaled molgramostim;Timepoint(s) of evaluation of this end point: End of trial	—

Measure

Time frame

Secondary end point(s): • (A-a)DO2 during the trial • 6MWD during the trial • SGRQ total score during the trial • Frequency of WLL during the trial • DLCO (% predicted), FEV1 (% predicted), and FVC (% predicted) during the trial • PaO2 and disease severity score (DSS) during the trial • Need for oxygen supplement therapy during the trial • Number of subjects not requiring treatment for aPAP and time off treatment after discontinuation of inhaled molgramostim;Timepoint(s) of evaluation of this end point: End of trial

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Countries

Denmark, France, Germany, Greece, Israel, Italy, Netherlands, Russian Federation, United Kingdom

Contacts

Public ContactTrial Coordinator

Savara ApS

mette.vinge@savarapharma.com+4524885979

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026