Status:
COMPLETED
Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Conditions:
Autoimmune Pulmonary Alveolar Proteinosis
Eligibility:
All Genders
18-80 years
Phase:
PHASE1
Brief Summary
Pulmonary alveolar proteinosis (PAP) is a syndrome of surfactant accumulation, respiratory failure, and innate immune deficiency for which therapy remains limited to whole lung lavage (WLL), an invasi...
Detailed Description
PAP is a rare syndrome of surfactant accumulation and resulting hypoxemic respiratory failure that occurs in multiple diseases that can be classified on the basis of pathogenesis into three groups: pr...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
August 17 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 2 2019
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT03231033
Start Date
August 17 2017
End Date
April 2 2019
Last Update
January 18 2020
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229