Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03231033
• Date of registration: 19/06/2017
• Prospective Registration: Yes
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis PioPAP
• Scientific title: First in Human Study of Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis
• Date of first enrolment: August 17, 2017
• Target sample size: 3
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03231033
• 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, MD
• 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 complete administration of study drug at home

- Able and willing to adhere to study visit schedule and study procedures

- Diagnosis of aPAP 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 aPAP

- 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 = 25 mm Hg

Exclusion Criteria:

- Diagnosis of any other PAP-causing disease

- aPAP complicated by:

- Severe disease at screening/enrollment (A-aD02<55)

- Clinically significant pulmonary fibrosis

- History of any clinically significant:

- Other lung disease

- Cardiovascular disease

- Disease requiring use of systemic steroids in past year

- History of Diabetes Mellitus

- History of untreated osteoporosis

- History of bladder cancer

- Active / serious lung or systemic infection

- Persistent or unexplained fever >101oF within 2 months of study

- Treatment with an investigational therapeutic agent for aPAP within 3 months
prior to enrollment, which includes inhaled GM-CSF

- Abnormal clinical and/or laboratory parameters at screening

- Women who are pregnant or plan to become pregnant

- 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: Pioglitazone

Primary Outcome(s)

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

Secondary Outcome(s)

Shortness of breath measured by San Diego Dyspnea Score [Time Frame: 1 year]

Alveolar-arterial oxygen concentration gradient (A-aDO2) [Time Frame: 1 year]

Parenchymal lung density determined by quantitative computed tomography (CT) densitometry [Time Frame: 1 year]

Quality of Life measured by SF-36 [Time Frame: 1 year]

Minimum SpO2 during a standardized treadmill exercise test [Time Frame: 1 year]

DLCO (Diffusion capacity of the lung for carbon monoxide) [Time Frame: 1 year]

PaO2 [Time Frame: 1 year]

Time during a standardized treadmill exercise test required for SpO2 to fall below 88% (or discontinuance of testing due to dyspnea) [Time Frame: 1 year]

Quality of Life measured by St. George's Respiratory Questionnaire [Time Frame: 1 year]

Secondary ID(s)

TPSC-114

Source(s) of Monetary Support

Please refer to primary and secondary sponsors