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Tests treatment safety and results for Hereditary Pulmonary Alveolar Proteinosis
Official title Safety and Efficacy of PMT Therapy of hPAP
ClinicalTrials.gov ID: NCT05761899
What this study is testing
What is Gene-Corrected Macrophages administered by bronchoscopic instillation?
Gene-Corrected Macrophages administered by bronchoscopic instillation is an investigational medicine, being studied as a potential treatment for hereditary pulmonary alveolar proteinosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The major goal of this study is to evaluate a new type of cell transplantation therapy for individuals with hereditary PAP, study a new treatment that may be useful for treatment of other diseases, and research mechanisms that drive the development and function of lung macrophages.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Patients must meet all of the following conditions to be eligible for participation in this study:
- Male or female with a confirmed diagnosis of hPAP defined as:
- Homozygous or compound heterozygous CSF2RA mutations - AND -
- A normal GM-CSF autoantibody test result - AND -
- An abnormal STAT5-PI test result - OR -
You likely can't join if
- Patients who meet any of the following conditions will not be eligible for participation in this study:
- History of a confirmed diagnosis of any other PAP-causing disease defined as:
- PAP caused by function-altering mutations in CSF2RB, adenosine triphosphate (ATP)-binding cassette subfamily A member 3 (ABCA3), SFTPB, SFTPC...
- PAP associated with an abnormal GM-CSF autoantibody test
- PAP associated with hematologic disorders including but not limited to myelodysplasia, aplastic anemia, leukemia, multiple myeloma, lymphoma
- PAP associated with non-hematologic malignancies
See the full eligibility criteria
- Patients must meet all of the following conditions to be eligible for participation in this study:
- Male or female with a confirmed diagnosis of hPAP defined as:
- Homozygous or compound heterozygous CSF2RA mutations - AND -
- A normal GM-CSF autoantibody test result - AND -
- An abnormal STAT5-PI test result - OR -
- An abnormal GM-CSF 50% effective concentration (EC50) test result
- Diffuse ground glass opacification of the lungs visualized on a chest computed tomogram (CT)
- History of prior receipt of WLL therapy or moderate hPAP lung disease severity requiring therapy in the opinion of the Clinical Site Investigator and/or Sponsor
- Able to undergo bone marrow collection by routine clinical aspiration
- 18 years of age or older on the date the Informed consent form (ICF) is signed
- Females who have been post-menopausal for \>2 years or females of child-bearing potential after a confirmed menstrual period using a highly efficient method of contraception (as described in Section 11.4.2) for the...
- Males of reproductive potential must agree to use condoms for the period from the 1st administration of gene-corrected macrophages until 12 months after the last dose of gene-corrected macrophages, have a partner who is...
- Signed written informed consent form (ICF)
- Patients who meet any of the following conditions will not be eligible for participation in this study:
- History of a confirmed diagnosis of any other PAP-causing disease defined as:
- PAP caused by function-altering mutations in CSF2RB, adenosine triphosphate (ATP)-binding cassette subfamily A member 3 (ABCA3), SFTPB, SFTPC, Thyroid Transcription Factor 1 (TTF-1), GATA-binding factor 2 (GATA2)...
- PAP associated with an abnormal GM-CSF autoantibody test
- PAP associated with hematologic disorders including but not limited to myelodysplasia, aplastic anemia, leukemia, multiple myeloma, lymphoma
- PAP associated with non-hematologic malignancies
- PAP associated with immune deficiency syndromes
- PAP associated with chronic inflammatory syndromes
- PAP associated with chronic infections including but not limited to human immunodeficiency virus, Mycobacteria tuberculosis or other Mycobacterial species, or other organisms
- PAP associated with inhaled materials including but not limited to inorganic dusts (e.g., silica, titanium, indium, aluminum), organic dusts (e.g., sawdust, fertilizer); or gases/vapors (e.g., cleaning products, paints...
- Pulmonary fibrosis that is clinically significant in the opinion of Clinical Site Investigator and/or Sponsor
- A confirmed (i.e., repeated) positive serum anti-GM-CSF receptor antibody test and/or a confirmed positive anti-lentiviral antibody test at the time of screening and prior to each administration of gene-corrected...
- History of receipt of any investigational agent within 3 months of Study Visit 3
- History of active chronic infection (e.g., HIV, Hepatitis, others) at the time of Screening
- History of significant alcohol consumption for a period of more than 3 consecutive months within 1 year prior to Study Visit 3, defined as more than 14 drinks/week for females or 21 drinks/week for males (1 drink - 5...
- History of medication or illicit drug abuse within 1 year prior to Study Visit 3, including but not limited to cocaine, heroin, or other opioids
- Currently or planning to become pregnant between the Screening visit and Visit 14 and/or currently breast-feeding
- Any other medical, behavioral, or psychiatric condition that would interfere with the completion of Study Visits or assessments in the opinion of the Clinical Site Investigator and/or Sponsor
The study team makes the final eligibility decision.
Where it's taking place
- Cincinnati, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Cincinnati, Ohio, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.