UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000055081 |
|---|---|
| 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 |
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Basic information
Public title
Long-term observational study after GM-CSF inhalation therapy in patients with autoimmune pulmonary alveolar proteinosis
Acronym
Real PAP History
Scientific Title
Long-term observational study after GM-CSF inhalation therapy in patients with autoimmune pulmonary alveolar proteinosis.
Scientific Title:Acronym
Real PAP History Study
Region
| Japan |
Condition
Condition
Autoimmune Pulmonary Alveolar Proteinosis
Classification by specialty
| Pneumology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
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
Basic objectives2
Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
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)
Key secondary outcomes
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
Base
Study type
Observational
Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| 12 | years-old | <= |
Age-upper limit
| 85 | years-old | > |
Gender
Male and Female
Key inclusion criteria
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.
Key exclusion criteria
(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.
Target sample size
60
Research contact person
Name of lead principal investigator
| 1st name | Takuro |
| Middle name | |
| Last name | Sakagami |
Organization
Faculty of Life Sciences, Kumamoto University
Division name
Department of Respiratory Medicine
Zip code
860-8556
Address
1-1-1 Honjyo, Chuo-ku, Kumamoto, JAPAN
TEL
0963735012
stakuro@kumamoto-u.ac.jp
Public contact
Name of contact person
| 1st name | Chieko |
| Middle name | |
| Last name | Yoshida |
Organization
Faculty of Life Sciences, Kumamoto University
Division name
Department of Respiratory Medicine
Zip code
860-8556
Address
1-1-1 Honjyo, Chuo-ku, Kumamoto, JAPAN
TEL
0963735012
Homepage URL
c-yoshida@kumamoto-u.ac.jp
Sponsor or person
Institute
Kumamoto University
Institute
Department
Personal name
Funding Source
Organization
self funding
Organization
Division
Category of Funding Organization
Self funding
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Kumamoto University Research Ethics Committee
Address
1-1-1 Honjyo, Chuo-ku, Kumamoto, JAPAN
Tel
0963735657
ski-shien@jimu.kumamoto-u.ac.jp
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
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）
Other administrative information
Date of disclosure of the study information
| 2024 | Year | 07 | Month | 26 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Open public recruiting
Date of protocol fixation
| 2024 | Year | 07 | Month | 11 | Day |
Date of IRB
| 2024 | Year | 07 | Month | 11 | Day |
Anticipated trial start date
| 2024 | Year | 07 | Month | 26 | Day |
Last follow-up date
| 2030 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Prospective observational study with one visit every 6 months at a collaborating institution for a total of 11 visits over 5 years
Management information
Registered date
| 2024 | Year | 07 | Month | 26 | Day |
Last modified on
| 2024 | Year | 07 | Month | 26 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000062921
