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Cytokine Therapy

Molgramostim for Pulmonary Alveolar Proteinosis

Phase 3
Waitlist Available
Research Sponsored by Savara Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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
Be ≥6 and <18 years of age, at the time of signing the informed consent and informed assent (if applicable)
Must not have
Have undergone treatment with Lung Lavage (WLL) within 1 month of Baseline
Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 0, 4, 12,24,48 and 52 weeks
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial aims to study how molgramostim can help pediatric patients aged 6 to 18 with autoimmune pulmonary alveolar proteinosis (aPAP). The study will look at how mol

Who is the study for?
This trial is for children aged 6-18 with autoimmune pulmonary alveolar proteinosis (aPAP), a rare lung disease. Participants must be able to use a nebulizer daily and visit the clinic every 12 weeks. Specific eligibility criteria are not provided, but typically include having the condition being studied and meeting certain health requirements.
What is being tested?
The study tests molgramostim, an inhaled medication delivered via nebulizer, as a treatment for pediatric aPAP. Over 12 months, all participants will receive this drug daily to assess its impact on breathing function and activity levels without comparing it to another drug or placebo.
What are the potential side effects?
While specific side effects of molgramostim in children are not detailed here, common ones may include respiratory symptoms like coughing or wheezing, headaches, fatigue, and possible reactions at the site of inhalation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with pulmonary alveolar proteinosis.
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I am between 6 and 17 years old.
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I tested positive for aPAP.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I had a lung lavage treatment within the last month.
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I have a genetic or acquired lung condition affecting surfactant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~0, 4, 12,24,48 and 52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 0, 4, 12,24,48 and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
DLCO
Secondary study objectives
6-minute walk distance
Oxygen Saturation (SpO2)
PedsQL
Other study objectives
Adverse Events
Anti-GM-CSF Ab titer
FEV1
+1 more

Side effects data

From 2019 Phase 2 trial • 139 Patients • NCT02702180
33%
Cough
22%
Chest pain
15%
Nasopharyngitis
13%
Headache
11%
Dyspnoea
9%
Productive cough
7%
Alveolar proteinosis
7%
Weight increased
4%
Pyrexia
4%
Respiratory tract infection
4%
Back pain
4%
Arthralgia
4%
Dizziness
2%
Pneumonia bacterial
2%
Respiratory failure
2%
Pneumonia
2%
Aphasia
2%
Epilepsy
2%
Laryngeal oedema
100%
80%
60%
40%
20%
0%
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.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: molgramostimExperimental Treatment1 Intervention
Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Molgramostim
2015
Completed Phase 2
~190

Find a Location

Who is running the clinical trial?

Savara Inc.Lead Sponsor
10 Previous Clinical Trials
747 Total Patients Enrolled
Yasmine Wasfi, MD, Ph.D.Study DirectorSavara Inc.
~3 spots leftby Jan 2027
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