Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis

• Register: ClinicalTrials.gov
• Last refreshed on: 17 August 2021
• Main ID: NCT03006146
• Date of registration: 28/11/2016
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
• Scientific title: Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
• Date of first enrolment: July 13, 2017
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03006146
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Basic Science. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Bruce Trapnell
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Autoimmune Pulmonary Alveolar Proteinosis

Intervention(s)

Drug: Sargramostim

Primary Outcome(s)

Occurrence of any treatment-emergent adverse events and serious adverse events [Time Frame: 1 year]

Secondary Outcome(s)

Time to maximum plasma GM-CSF concentration (TMax) [Time Frame: 1 year]

GM-CSF autoantibody levels in blood and BAL fluid [Time Frame: 1 year]

GM-CSF signaling levels in blood and BAL fluid [Time Frame: 1 year]

Maximum plasma GM-CSF Concentration (Cmax) [Time Frame: 1 year]

Half-life of inhaled GM-CSF (t1/2) [Time Frame: 1 year]

GM-CSF Area Under the Curve (AUC) [Time Frame: 1 year]

Complete cell counts and differentials in blood and BAL fluid [Time Frame: 1 year]

Secondary ID(s)

UL1TR001425

TPSC-110

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Center for Advancing Translational Science (NCATS)

Rare Diseases Clinical Research Network