Clinical trial
An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
Name
SAV006-03
Description
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.
Trial arms
Trial start
2018-04-16
Estimated PCD
2021-01-14
Trial end
2021-01-14
Status
Terminated
Phase
Early phase I
Treatment
Molgramostim
300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.
Arms:
Molgramostim nebulizer solution (300 μg)
Other names:
Recombinant human GM-CSF
Size
60
Primary endpoint
Number of Treatment-emergent Adverse Events (TEAEs)
139 weeks
Number of Serious TEAEs
139 weeks
Number of Treatment-emergent Adverse Drug Reactions (ADRs)
139 weeks
Number of TEAEs Leading to Treatment Discontinuation
139 weeks
Eligibility criteria
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open label, non-controlled.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 60, 'type': 'ACTUAL'}}
Updated at
2024-07-03
1 organization
1 product
1 indication
Organization
SavaraProduct
Molgramostim