Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 6:26 PM
Ignite Modification Date:
2025-12-24 @ 6:26 PM
NCT ID:
NCT01511068
Eligibility Criteria:
Inclusion Criteria:
* A diagnosis of PAP caused by bi-allelic mutations in CSF2RA or CSF2RB associated with impaired GM-CSF-R-alpha or GM-CSF-R-beta function, respectively, resulting in reduced but non-zero GM-CSF signaling
* Able and willing to give written informed consent / assent as necessary
* Clinically stable
Exclusion Criteria:
* Confirmed diagnosis of a disorder of surfactant production caused by bi-allelic mutations in ABCA3, SFTPB, or SFTPC
* Confirmed diagnosis of autoimmune PAP caused by a high level of GM-CSF autoantibody
* Confirmed diagnosis of secondary PAP caused by an underlying clinical disorder known to be associated with the development of PAP, e.g., inhalation of silica or titanium; myelodysplasia and others
* Treatment with any investigational agent in the 3 months prior to enrollment
* History of severe allergic or anaphylactic reactions to GM-CSF or other yeast-derived products
* History of asthma or other reactive airways disease
* Known active, viral, fungal, mycobacterial, or other infection
* A serious medical condition which, in the opinion of the investigator or data and safety monitoring committee, would make the patient unsuitable for the study
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
8 Years
Study:
NCT01511068
Study Brief:
Protocol Section:
NCT01511068