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Safety and Efficacy of PMT Therapy of hPAP

NCT05761899 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-12-19

No results posted yet for this study

Summary

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

  • Hereditary Pulmonary Alveolar Proteinosis

Interventions

COMBINATION_PRODUCT

Gene-Corrected Macrophages administered by bronchoscopic instillation

This study will evaluate administration of autologous bone marrow CD34+ cell-derived, CSF2RA lentiviral vector-transduced macrophages (CSF2RA gene-corrected macrophages) by bronchoscopic instillation into individual lung segments on three occasions at 2-month intervals in patients with hPAP. The target (maximum) number of cells to be administered is 778 million gene-corrected macrophages per 70 kg patient, which is equal to 11.1 million cells/kg of ideal body weight.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • University of South Florida

    collaborator OTHER

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Christopher Towe, MD · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-26
Primary Completion
2026-09-01
Completion
2038-10-01
FDA Drug
Yes
FDA Device
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05761899 on ClinicalTrials.gov