Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveola… (NCT03006146) | Clinical Trial Compass
CompletedPhase 1
Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
United States10 participantsStarted 2017-07-13
Plain-language 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.
Who can participate
Age range
18 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
Male or female
Age ≥ 18 years and ≤ 80 years
Able to understand and willing to sign a written informed consent document
Able and willing to use hand held nebulizer
Able and willing to adhere to study visit schedule and study procedures
Diagnosis of autoimmune PAP determined by:
* History of a diagnosis of PAP with or without supporting lung histology or BAL/cytology
and
* Abnormal serum GM-CSF autoantibody test (GMAb ELISA Test)
and
* Chest CT findings compatible with a diagnosis of autoimmune PAP
Evidence of impaired GM-CSF signaling demonstrated by an abnormal STAT5 phosphorylation index (STAT5-PI) test measured in heparinized whole blood at the time screening
A-aDO2 ≥ 15 mm Hg
Exclusion Criteria:
Diagnosis of any other PAP-causing disease
Autoimmune PAP complicated by:
* Severe disease at screening/enrollment (A-aD02\<50)
* Clinically significant pulmonary fibrosis
History of any clinically significant:
* Other lung disease
* Cardiovascular disease
* Disease requiring use of systemic steroids in past year
* Coagulopathy or other hematologic disease
* Active / serious lung or systemic infection
* Persistent or unexplained fever \>101oF within 2 months of study
* Use of any immunosuppressive medication within 3-6 months of screening
* Women who are pregnant or plan to become pregnant
* History of active tobacco/e-cig/marijuana use
* Concomitant or recent use of specific medicines
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Occurrence of any treatment-emergent adverse events and serious adverse events
Timeframe: 1 year
Trial details
NCT IDNCT03006146
SponsorChildren's Hospital Medical Center, Cincinnati