Actively Recruiting
Safety and Efficacy of PMT Therapy of hPAP
Led by Children's Hospital Medical Center, Cincinnati · Updated on 2025-12-19
3
Participants Needed
1
Research Sites
796 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety and Efficacy of PMT Therapy of hPAP
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female with confirmed hereditary pulmonary alveolar proteinosis caused by homozygous or compound heterozygous CSF2RA mutations
- Normal GM-CSF autoantibody test result
- Abnormal STAT5-PI test result or abnormal GM-CSF 50% effective concentration (EC50) test result
- Diffuse ground glass opacification in lungs seen on chest CT scan
- History of prior whole lung lavage therapy or moderate hPAP lung disease requiring treatment
- Able to undergo bone marrow collection by routine clinical aspiration
- 18 years of age or older at informed consent
- Females post-menopausal for more than 2 years or using effective contraception if of child-bearing potential, with negative pregnancy tests at screening and before treatment
- Males of reproductive potential agree to use condoms or have partners not of child-bearing potential
- Signed informed consent form
You will not qualify if you...
- Diagnosis of any other PAP-causing disease due to mutations in CSF2RB, ABCA3, SFTPB, SFTPC, TTF-1, GATA2, SLC7A7, MARS, or other known genes except CSF2RA
- PAP associated with abnormal GM-CSF autoantibody test
- PAP linked to hematologic disorders such as myelodysplasia, aplastic anemia, leukemia, multiple myeloma, lymphoma
- PAP linked to non-hematologic cancers
- PAP linked to immune deficiency syndromes
- PAP linked to chronic inflammatory syndromes
- PAP linked to chronic infections including HIV, tuberculosis, or other mycobacterial infections
- PAP linked to inhaled materials like silica, titanium, indium, aluminum, sawdust, fertilizer, or fumes from cleaning products, paints, welding
- Clinically significant pulmonary fibrosis
- Confirmed positive serum anti-GM-CSF receptor antibody or anti-lentiviral antibody at screening or before treatment
- Use of any investigational drug within 3 months before treatment
- Active chronic infection such as HIV or Hepatitis at screening
- Significant alcohol use (more than 14 drinks/week for females or 21 drinks/week for males) for over 3 months within 1 year before treatment
- History of medication or illicit drug abuse within 1 year before treatment
- Currently pregnant or breastfeeding
- Any medical, behavioral, or psychiatric condition interfering with study participation as judged by investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Research Team
B
Bruce Trapnell
CONTACT
B
Brenna Carey
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here