Safety and Efficacy of PMT Therapy of hPAP

Conditions

• Hereditary Pulmonary Alveolar Proteinosis

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Gene-Corrected Macrophages administered by bronchoscopic instillation — COMBINATION_PRODUCT
  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.

Study Details

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.

Key Dates

Start date

Jun 26, 2023

Status verified

Dec 2025

Primary completion

Sep 1, 2026

Completion

Oct 1, 2038

Study Design

Enrollment

3 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Gene-Corrected Macrophages
  Autologous bone marrow CD34+ cell-derived, CSF2RA lentiviral vector-transduced macrophages (CSF2RA gene-corrected macrophages) by bronchoscopic instillation into individual lung segments.

Primary Outcome Measure

Number of patients with adverse events (AEs) [ Time Frame: Pre- and Post-PMT Therapy for 15 years ]

Central Contacts

• Bruce Trapnell
  513-636-6361
  [email protected]
• Brenna Carey
  513-636-8916
  [email protected]

Locations (1)

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