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Cytokine

Inhaled Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (IMPALA-2 Trial)

Phase 3

Waitlist Available

Led By Bruce Trapnell, Prof

Research Sponsored by Savara Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

History of PAP, based on examination of a lung biopsy, bronchoalveolar lavage (BAL) cytology, or a high-resolution computed tomogram (HRCT) of the chest.

Subject must be ≥18 years of age, at the time of signing the informed consent (≥20 in Japan).

Must not have

Inflammatory or autoimmune disease of a severity that necessitates significant (e.g. more than 10 mg/day systemic prednisolone) immunosuppression.

Treatment with any investigational medicinal product within 5 half-lives or 3 months (whichever is longer) prior to baseline.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from screening (6-week) until follow-up visit (week 100)

Awards & highlights

Pivotal Trial

Summary

This trial will study whether a daily inhaled drug can improve aPAP, and will last for 96 weeks total.

Who is the study for?

Adults diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP) who can perform certain physical tests without supplemental oxygen, have a history of PAP confirmed by specific lung tests, and are not on recent treatments like WLL or GM-CSF. They must be able to consent and follow trial procedures. Pregnant women, those with severe medical conditions or allergies to the treatment components, and individuals with other serious lung diseases are excluded.

What is being tested?

The study is testing an inhaled solution called Molgramostim against a placebo in people with aPAP. Participants will use their assigned treatment daily for 48 weeks; afterwards, all subjects receive Molgramostim for another 96 weeks. The goal is to see if this drug helps improve lung function compared to no active treatment.

What are the potential side effects?

Potential side effects of Molgramostim may include reactions at the site of inhalation such as coughing or throat irritation, general body discomforts like fatigue or headaches, possible immune system responses including allergic reactions, and respiratory symptoms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a history of PAP confirmed by lung tests or scans.

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I am at least 18 years old, or 20 if I am in Japan.

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I can stop using extra oxygen for tests like treadmill exercise, DLCO, and blood gas sampling.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I need strong medication (more than 10 mg/day of prednisolone) for my autoimmune disease.

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I haven't taken any experimental drugs recently.

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I have not had plasmapheresis in the last 6 weeks.

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I have lung scarring not related to my current condition.

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I have or had a blood disorder or leukemia.

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I need a whole lung lavage.

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I have not received GM-CSF treatment in the last 6 months.

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I had a whole lung lavage within the last 3 months.

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I have had severe reactions to any medicine given through inhalation.

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I cannot safely perform exercise tests due to a physical condition.

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I do not have heart or lung conditions worsened by exercise.

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I have been diagnosed with a rare lung condition related to surfactant production.

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I do not have any active infections that could interfere with the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from screening (6-week) until follow-up visit (week 100)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from screening (6-week) until follow-up visit (week 100)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from screening (6-week) until follow-up visit (week 100) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in percentage (%) predicted diffusing capacity of the lung for carbon monoxide (DLCO) to Week 24

Secondary study objectives

Change from baseline in EC, expressed as peak METs to Week 48

Change from baseline in SGRQ Activity component score to Week 24

Change from baseline in SGRQ Activity from baseline to Week 48

+10 more

Side effects data

From 2019 Phase 2 trial • 139 Patients • NCT02702180

33%

Cough

22%

Chest pain

15%

Nasopharyngitis

13%

Headache

11%

Dyspnoea

Productive cough

Alveolar proteinosis

Weight increased

Pyrexia

Respiratory tract infection

Back pain

Arthralgia

Dizziness

Pneumonia bacterial

Respiratory failure

Pneumonia

Aphasia

Epilepsy

Laryngeal oedema

100%

80%

60%

40%

20%

Study treatment Arm

Double-blind Molgramostim Once Daily

Double-blind Molgramostim Intermittent

Double-blind Placebo

Open-label Molgramostim Intermittent

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MolgramostimExperimental Treatment1 Intervention

Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks, followed by open-label treatment with molgramostim nebulizer solution 300 µg once daily for 96 weeks

Group II: PlaceboPlacebo Group2 Interventions

Double-blind treatment with placebo nebulizer solution once daily for 48 weeks, followed by open-label treatment with molgramostim nebulizer solution 300 µg once daily for 96 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Molgramostim

2015

Completed Phase 2

~190

0 / 16Eligibility criteria met