A Phase II Study of Inhaled GM-CSF in Patients With Idiopathic Pulmonary Alveolar Proteinosis (iPAP)

Phase 2

Completed

• Conditions: Idiopathic pulmonary alveolar proteinosis

• Registration Number: JPRN-jRCT1091220013
• Lead Sponsor: Koh Nakata, M.D., Ph.D.Bioscience Medical Research Center (BMRC), Niigata University Medical & Dental Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2007-08-16
• Study Completion: 2012-02-28
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Male or female patients aged 16 - 80 (on the day of enrollment). Patients aged 16 - 19 must provide a signed consent from parents/guardians.
2. A written consent from the patient or his/her parents/guardians.
3. Able to be hospitalized for a short time.
4. Idiopathic pulmonary alveolar proteinosis with serum anti-GM-CSF autoantibody titer grater than 3 mcg/ml and meets A or B below:
A. typical pathological findings (accumulation of PAS-positive proteinaceous materials within the alveoli) on transbronchial lung biopsy or surgical lung biopsy (incl. thoracoscopy lung biopsy)
B. bronchoalveolar lavage (BAL) fluid showing features characteristic of iPAP (i.e., opaque appearance, proteinaceous materials, low alveolar macrophage count).
5. Resting Pao2 < 75 mmHg on room air.
6. Diagnosis of PAP
7. Life expectancy longer than 3 months.
8. Confirmed presence of anti-GM-CSF autoantibodies.

Exclusion Criteria

1. WBC count >/= 12000/mm3
2. Concomitant bronchial asthma.
3. Fever of >/= 38 degrees Celsius.
4. Edema of Grade 2 or more (CTCAE)
5. Myeloid malignancy.
6. Concomitant congestive heart failure (NYHA III - IV), angina, bleeding tendency, primary lung cancer, or metastatic lung cancer that will make assessment of treatment difficult.
7. Concurrent treatment with other cytokines.
8. Pregnant or possibly pregnant women or nursing mothers, and women planning to become pregnant during the study.
9. Whole lung lavage or sequential segmental lung lavage within 6 months prior to the start of treatment.
10. Upper lung lobe fibrosis with a honeycomb pattern or emphysema on x-ray or high-resolution computed tomography (HRCT) scan of the chest performed within 6 months prior to study enrollment.
11. For any reason, is judged by the investigator/co-investigators to be inappropriate for study, including inability to complete the protocol or cooperate with the investigator/co-investigators.
12. History of serious drug allergy.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Improvement in A-aDo2 at Week 24 compared with baseline

Secondary Outcome Measures

Name	Time	Method
1. Duration of response (defined as maintenance of >/= 10 mmHg increase from baseline in A-aDo2)<br>2. Changes from baseline in HRCT findings.<br>3. Changes from baseline in %VC and %DLco<br>4. Changes from baseline in serum markers (CEA, KL-6, SP-A, SP-D, LDH)