Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis (NCT03482752) | Clinical Trial Compass
TerminatedPhase 3
Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
Stopped: SAV006-03 was initiated before the evaluation of the IMPALA study (NCT02702180) results. Considering these results and authority advice there would not be adequate efficacy and safety data from SAV006-03 and the study was terminated.
Denmark, France, Germany60 participantsStarted 2018-04-16
Plain-language summary
SAV006-03 is an open-label extension study for participants who had completed the IMPALA study.
At the baseline visit, eligible participants may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor; GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once daily, and seven days off treatment.
Participants will be treated with inhaled molgramostim for up to 36 months.
During the trial, whole lung lavage will be applied as rescue therapy.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Completer of the IMPALA trial.
* Females who have been post menopausal for \>1 year, or females of child-bearing potential who are not pregnant or lactating and are using acceptable contraceptive methods.
* Males agreeing to use using acceptable contraceptive methods.
* Willing and able to provide signed informed consent.
Exclusion Criteria:
* Treatment with GM-CSF products other than molgramostim nebuliser solution within three months of Baseline.
* Treatment with any investigational medicinal product other than inhaled molgramostim within four weeks of Baseline.
* History of allergic reactions to GM-CSF.
* 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.
* Previous experience of severe and unexplained side effects during aerosol delivery of any kind of medicinal product.
* History of, or present, myeloproliferative disease or leukaemia.
* Apparent pre-existing concurrent pulmonary fibrosis.
* Any other serious medical condition which in the opinion of the investigator would make the subject unsuitable for the trial.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Treatment-emergent Adverse Events (TEAEs)
Timeframe: 139 weeks
2
Number of Serious TEAEs
Timeframe: 139 weeks
3
Number of Treatment-emergent Adverse Drug Reactions (ADRs)
Timeframe: 139 weeks
4
Number of TEAEs Leading to Treatment Discontinuation