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Cell Therapy
PMT Therapy for Pulmonary Alveolar Proteinosis
Phase 1 & 2
Recruiting
Led By Christopher Towe, MD
Research Sponsored by Children's Hospital Medical Center, Cincinnati
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
18 years of age or older on the date the Informed consent form (ICF) is signed
Diffuse ground glass opacification of the lungs visualized on a chest computed tomogram (CT)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up pre- and post-pmt therapy for 15 years
Awards & highlights
Study Summary
This trial is researching a new cell transplantation therapy for genetic diseases & studying new treatments that may help other diseases.
Who is the study for?
Adults diagnosed with hereditary Pulmonary Alveolar Proteinosis (hPAP) who have either received Whole Lung Lavage therapy or have moderate disease severity needing treatment. Participants must be able to undergo bone marrow collection, use effective contraception if of childbearing potential, and provide written consent.Check my eligibility
What is being tested?
The trial is testing a new cell transplantation therapy where gene-corrected macrophages are delivered directly into the lungs via bronchoscopy. The aim is to assess safety and effectiveness for hPAP and understand lung macrophage functions.See study design
What are the potential side effects?
Potential side effects may include reactions related to bronchoscopic procedures such as coughing or wheezing, discomfort at the bone marrow collection site, and immune responses against the introduced cells or vector.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 18 years old or older.
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My CT scan shows widespread cloudy areas in my lungs.
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I have been diagnosed with hPAP.
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I am a woman who has been post-menopausal for more than 2 years or can use reliable birth control.
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I am a man who will use condoms or my partner cannot become pregnant.
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I can have a bone marrow biopsy.
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I have had WLL therapy or my lung disease is severe enough to need treatment.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ pre- and post-pmt therapy for 15 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~pre- and post-pmt therapy for 15 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of patients with adverse events (AEs)
Secondary outcome measures
Biodistribution of transplanted macrophages via detection of lentiviral DNA in BAL and blood
Concentration of PAP biomarkers in bronchoalveolar lavage fluid
Difference in Health Status/Quality of Life Score via the 36-Item Short Form Survey (SF-36) Questionnaire
+22 moreOther outcome measures
Cell population dynamics of transplanted macrophages via RNA sequencing t-distributed stochastic neighborhood embedding analysis (t-SNE)
Percentage of bronchoalveolar lavage cells that respond to GM-CSF stimulation
Transcriptional regulation of alveolar macrophage specification via an assay for transposable-accessible chromatin with sequencing
Trial Design
1Treatment groups
Experimental Treatment
Group I: Gene-Corrected MacrophagesExperimental Treatment1 Intervention
Autologous bone marrow CD34+ cell-derived, CSF2RA lentiviral vector-transduced macrophages (CSF2RA gene-corrected macrophages) by bronchoscopic instillation into individual lung segments.
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Who is running the clinical trial?
Children's Hospital Medical Center, CincinnatiLead Sponsor
815 Previous Clinical Trials
6,530,667 Total Patients Enrolled
7 Trials studying Pulmonary Alveolar Proteinosis
626 Patients Enrolled for Pulmonary Alveolar Proteinosis
University of South FloridaOTHER
414 Previous Clinical Trials
188,294 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,847 Previous Clinical Trials
47,818,233 Total Patients Enrolled
2 Trials studying Pulmonary Alveolar Proteinosis
529 Patients Enrolled for Pulmonary Alveolar Proteinosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am 18 years old or older.My CT scan shows widespread cloudy areas in my lungs.I have been diagnosed with hPAP.I am a woman who has been post-menopausal for more than 2 years or can use reliable birth control.I am a man who will use condoms or my partner cannot become pregnant.My lung condition is linked to an immune system problem.I have PAP linked to long-term inflammation.My cancer is not related to blood and is associated with high calcium levels.I have PAP linked to long-term infections.I tested positive for anti-GM-CSF receptor or anti-lentiviral antibodies.I have been diagnosed with a disease that causes PAP.I have serious lung scarring.You have consumed a lot of alcohol within the past year before the third study visit.I currently have a long-term infection.I can have a bone marrow biopsy.My PAP is linked to a blood disorder like leukemia or anemia.I have had WLL therapy or my lung disease is severe enough to need treatment.
Research Study Groups:
This trial has the following groups:- Group 1: Gene-Corrected Macrophages
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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