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NCT03006146 · ClinicalTrials.gov registry record · Phase 1
Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
A Phase 1 study, sponsored by Children's Hospital Medical Center, Cincinnati.
- Completed
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT03006146 is a Phase 1 study that has completed, run by Children's Hospital Medical Center, Cincinnati. The registered enrollment target is 10 participants.
The verdict
NCT03006146, a Phase 1 study, has completed, sponsored by Children's Hospital Medical Center, Cincinnati.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
Study Summary
Autoimmune PAP is a rare lung disease affecting less than 5,000 individuals in US with no FDA-approved pharmacologic therapy. Results from "off-label" use in case reports and clinical studies completed outside of the US indicate that inhaled rhGM-CSF may be a safe and effective thera-py for autoimmune PAP. Preliminary clinical trials of inhaled rhGM-CSF in autoimmune PAP patients show promising results, 62%-96% therapeutic response rate without any identifiable drug-related adverse effects in at least 73 autoimmune PAP patients. However, the pharmacokinetics (PK), pharmacodynamics (PD), optimal dose, and treatment duration to maximize efficacy are unknown. The goal is to begin to address these knowledge gaps for inhaled sargramostim for autoimmune PAP patients with a pilot safety and PK/PD study (TPSC-110). TPSC-110, PharmPAP, which is a self-controlled open-label, phase I study to evaluate the safety, PK, and PD of inhaled sargra-mostim in autoimmune PAP patients. These results will impact the field by 1) confirming existing published data, 2) monitoring the local effects of inhaled sargramostim in autoimmune PAP patients, 3) potentially demonstrating a safe starting dose for a later trial to evaluate the therapeutic efficacy of inhaled sargramostim for autoimmune PAP.
Interventions
- DRUG Sargramostim
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2017-07-13 |
| Est. Completion | 2021-06-07 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03006146
The ClinicalTrials.gov registry entry for NCT03006146 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 10 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 0 conditions, and to 1 intervention - of which Sargramostim is the first listed.
NCT03006146 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03006146 about?
NCT03006146 is a clinical study titled "Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis". Autoimmune PAP is a rare lung disease affecting less than 5,000 individuals in US with no FDA-approved pharmacologic therapy. Results from "off-label" use in case reports and clinical studies completed outside of the US indicate that inhaled rhGM-CSF may be a safe and effective thera-py for autoimmu...
What is the current status of trial NCT03006146?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2017-07-13. Estimated completion is 2021-06-07.
What interventions are being tested in trial NCT03006146?
The interventions under investigation include: Sargramostim (DRUG).
Who is sponsoring clinical trial NCT03006146?
This trial is sponsored by Children's Hospital Medical Center, Cincinnati, which has 571 total clinical trials registered on ClinicalTrials.gov.
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