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NCT03231033 · ClinicalTrials.gov registry record · Phase 1
Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis
A Phase 1 study of Autoimmune Pulmonary Alveolar Proteinosis, sponsored by Children's Hospital Medical Center, Cincinnati.
- Completed
- Registry status
- Phase 1
- Development phase
- 3
- Enrollment target
- 1
- Study location
NCT03231033 is a Phase 1 study of Autoimmune Pulmonary Alveolar Proteinosis that has completed, run by Children's Hospital Medical Center, Cincinnati. The registered enrollment target is 3 participants. The trial reports 1 study location across 1 state.
The verdict
NCT03231033, a Phase 1 study of Autoimmune Pulmonary Alveolar Proteinosis, has completed, sponsored by Children's Hospital Medical Center, Cincinnati.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 3 participants
- Enrollment target
- 1
- Study location
Study Summary
Pulmonary alveolar proteinosis (PAP) is a syndrome of surfactant accumulation, respiratory failure, and innate immune deficiency for which therapy remains limited to whole lung lavage (WLL), an invasive physical procedure to remove surfactant unavailable at most medical centers. While PAP occurs in multiple diseases affecting men, women, and children of all ages and ethnic origins, in 85% of patients, it occurs as an idiopathic disease associated with neutralizing GM-CSF autoantibodies. Basic science and translational research has shown that idiopathic PAP is an autoimmune disease in which disruption of GM-CSF signaling impairs the ability of alveolar macrophages to clear surfactant and perform host defense functions. Recently, it has been shown that cholesterol toxicity drives pathogenesis in alveolar macrophages from GM-CSF deficient (Csf2-/-) mice and patients with autoimmune PAP. Loss of GM-CSF signaling reduces PU.1/CEBP-mediated expression of PPARγ and its downstream target ABCG1 (a cholesterol exporter important in macrophages). The cell responds by esterifying and storing cholesterol in vesicles to reduce toxicity. Eventually, vesicles fill the cell, impair intracellular transport and reduce uptake and clearance of surfactant from the lung surface resulting in disease manifestations. Recent data indicates that pioglitazone, a PPARγ agonist currently approved by the FDA for human use, increases cholesterol/surfactant clearance by alveolar macrophages from autoimmune PAP patients and Csf2-/- mice. Importantly, pioglitazone significantly reduced the severity of PAP lung disease in Csf2-/- mice after several months of therapy. Together, these observations suggest pioglitazone could be 'repurposed' as pharmacologic therapy for PAP.
Conditions Studied
Interventions
- DRUG Pioglitazone
Study Locations (1)
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2017-08-17 |
| Est. Completion | 2019-04-02 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03231033
The ClinicalTrials.gov registry entry for NCT03231033 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 3 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospital Medical Center, Cincinnati, which has 571 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Autoimmune Pulmonary Alveolar Proteinosis appearing as the primary indexed condition, and to 1 intervention - of which Pioglitazone is the first listed.
NCT03231033 reports 1 study location spanning 1 distinct geographic area - top geographies include Ohio.
Frequently Asked Questions
What is clinical trial NCT03231033 about?
NCT03231033 is a clinical study titled "Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis". Pulmonary alveolar proteinosis (PAP) is a syndrome of surfactant accumulation, respiratory failure, and innate immune deficiency for which therapy remains limited to whole lung lavage (WLL), an invasive physical procedure to remove surfactant unavailable at most medical centers. While PAP occurs in ...
What is the current status of trial NCT03231033?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2017-08-17. Estimated completion is 2019-04-02.
What conditions does trial NCT03231033 study?
This clinical trial studies the following conditions: Autoimmune Pulmonary Alveolar Proteinosis.
What interventions are being tested in trial NCT03231033?
The interventions under investigation include: Pioglitazone (DRUG).
Who is sponsoring clinical trial NCT03231033?
This trial is sponsored by Children's Hospital Medical Center, Cincinnati, which has 571 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03231033 being conducted?
This trial has 1 study location across Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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