Unique ID issued by UMIN | UMIN000055081 |
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Receipt number | R000062921 |
Scientific Title | Long-term observational study after GM-CSF inhalation therapy in patients with autoimmune pulmonary alveolar proteinosis. |
Date of disclosure of the study information | 2024/07/26 |
Last modified on | 2024/07/26 09:45:55 |
Long-term observational study after GM-CSF inhalation therapy in patients with autoimmune pulmonary alveolar proteinosis
Real PAP History
Long-term observational study after GM-CSF inhalation therapy in patients with autoimmune pulmonary alveolar proteinosis.
Real PAP History Study
Japan |
Autoimmune Pulmonary Alveolar Proteinosis
Pneumology |
Others
NO
1.To observe the natural history of remission of autoimmune alveolar proteinosis after GM-CSF inhalation therapy
2.To explore background factors for responder/non-responder to GM-CSF inhalation therapy
Safety
Remission rate after GM-CSF inhalation therapy
Definition of remission=AaDO2 more than 10 torr from baseline and no additional treatment (whole lung lavage or GM-CSF inhalation)
Search for predictors of treatment response (responder/non-responder)
Trends in %DLco
Changes in CT imaging score
Changes in mMRC
transition of oxygen requirement, and number of patients who were able to wean off LTOT
Trends in QOL score
Changes in anti-GM-CSF antibodies
Changes in serum biomarkers
Observational
12 | years-old | <= |
85 | years-old | > |
Male and Female
1)Patients with autoimmune pulmonary alveolar proteinosis enrolled in the PAGE II specified clinical research* (cohort A)
2)Patients with disease severity III or higher who newly start GM-CSF inhalation therapy after GM-CSF is launched (Cohort B)
*PAGE II Specified Clinical Study of Sargramostim for Autoimmune Pulmonary Alveolar Proteinosis in 2022-2024.
(1) Patients diagnosed with secondary pulmonary alveolar proteinosis or hereditary pulmonary alveolar proteinosis
(ii) Patients with severe symptoms due to heart or blood vessel disease such as congestive heart failure or angina pectoris
(iii) Patients diagnosed with cancer within the past 5 years (excluding those whose condition has stabilized and who are in the follow-up period)
(iv) Patients with bronchial asthma, pulmonary infection (including pulmonary tuberculosis), or other pulmonary diseases (including pulmonary tuberculosis). However, patients with non-tuberculous mycobacterial infection, pulmonary aspergillosis, or pulmonary nocardiosis who are under treatment or untreated and whose symptoms are stable may participate at the discretion of their physician), pulmonary fibrosis, interstitial pneumonia, bronchiectasis or other respiratory diseases that are expected to make it difficult to evaluate this study
(v) Patients treated with whole lung lavage, repeated area lavage therapy, or rituximab within 1 month of enrollment (Cohort B only)
(vi) Patients who received GM-CSF inhalation therapy within 1 month of enrollment (Cohort B only)
(vii) Other patients deemed inappropriate by the physician in charge.
60
1st name | Takuro |
Middle name | |
Last name | Sakagami |
Faculty of Life Sciences, Kumamoto University
Department of Respiratory Medicine
860-8556
1-1-1 Honjyo, Chuo-ku, Kumamoto, JAPAN
0963735012
stakuro@kumamoto-u.ac.jp
1st name | Chieko |
Middle name | |
Last name | Yoshida |
Faculty of Life Sciences, Kumamoto University
Department of Respiratory Medicine
860-8556
1-1-1 Honjyo, Chuo-ku, Kumamoto, JAPAN
0963735012
c-yoshida@kumamoto-u.ac.jp
Kumamoto University
self funding
Self funding
Kumamoto University Research Ethics Committee
1-1-1 Honjyo, Chuo-ku, Kumamoto, JAPAN
0963735657
ski-shien@jimu.kumamoto-u.ac.jp
NO
北海道大学病院(北海道)、市立函館病院(北海道)、東北大学病院(宮城県)、福島県立医科大学病院(福島県)、新潟大学医歯学総合病院(新潟県)、千葉大学病院(千葉県)、杏林大学医学部付属病院(東京都)、さいたま赤十字病院(埼玉県)、愛知医科大学病院(愛知県)、京都大学医学部附属病院(京都府)、国立病院機機構近畿中央呼吸器センター(大阪府)、神戸市民病院機構神戸市立医療センター中央市民病院(兵庫県)、倉敷市立市民病院(岡山県)、広島大学病院(広島県)、福岡大学医学部付属病院(福岡県)、大分大学医学部付属病院(大分県)、長崎大学病院(長崎県)、琉球大学大学病院(沖縄県)、独立行政法人国立病院機構沖縄病院(沖縄県)
2024 | Year | 07 | Month | 26 | Day |
Unpublished
Open public recruiting
2024 | Year | 07 | Month | 11 | Day |
2024 | Year | 07 | Month | 11 | Day |
2024 | Year | 07 | Month | 26 | Day |
2030 | Year | 03 | Month | 31 | Day |
Prospective observational study with one visit every 6 months at a collaborating institution for a total of 11 visits over 5 years
2024 | Year | 07 | Month | 26 | Day |
2024 | Year | 07 | Month | 26 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000062921
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