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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 below

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and pre- and post-pmt therapy for 15 years for reporting.

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 more

Other 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.

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor

815 Previous Clinical Trials

6,531,588 Total Patients Enrolled

7 Trials studying Pulmonary Alveolar Proteinosis

626 Patients Enrolled for Pulmonary Alveolar Proteinosis

University of South FloridaOTHER

412 Previous Clinical Trials

187,056 Total Patients Enrolled

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

3,840 Previous Clinical Trials

47,852,121 Total Patients Enrolled

2 Trials studying Pulmonary Alveolar Proteinosis

529 Patients Enrolled for Pulmonary Alveolar Proteinosis

Media Library

PMT Therapy of hPAP (Cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05761899 — Phase 1 & 2

Pulmonary Alveolar Proteinosis Research Study Groups: Gene-Corrected Macrophages

Pulmonary Alveolar Proteinosis Clinical Trial 2023: PMT Therapy of hPAP Highlights & Side Effects. Trial Name: NCT05761899 — Phase 1 & 2

PMT Therapy of hPAP (Cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05761899 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this scientific experiment currently enrolling participants?

"That is accurate. According to clinicaltrials.gov, this medical experiment commenced recruitment on June 26th 2023 and was updated recently on August 21st of the same year. The trial requires 3 individuals from one location for participation in its clinical stages."

Answered by AI

How many participants are actively engaged in this research endeavor?

"Indeed, the clinicaltrials.gov portal confirms that this medical trial launched on June 26th 2023 and has been actively seeking applicants since its last update on August 21st 2023. Currently, it needs 3 participants at one site to be included in the study."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Safety and Efficacy of PMT Therapy of hPAP

2023. "Safety and Efficacy of PMT Therapy of hPAP". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05761899.