229. 肺胞蛋白症（自己免疫性又は先天性） Autoimmune pulmonary alveolar proteinosis Clinical trials / Disease details
臨床試験数 : 44 / 薬物数 : 22 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 14
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04811274 (ClinicalTrials.gov) | June 7, 2021 | 19/3/2021 | Macrophages, GM-CSF and MARS Proteinosis | Study of Macrophage Function and of the GM-CSF Signaling Pathway in Alveolar Proteinosis by Mutations of the MARS Gene | Pulmonary Alveolar Proteinosis (PAP);MARS;Genetic Mutation | Biological: Blood collection;Biological: Bronchoalveolar lavage fluid collection | Assistance Publique - Hôpitaux de Paris | NULL | Recruiting | N/A | 17 Years | All | 20 | France | |
| 2 | NCT02835742 (ClinicalTrials.gov) | September 1, 2016 | 13/7/2016 | Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan | Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan | Pulmonary Alveolar Proteinosis, Autoimmune | Drug: Sargramostim;Drug: Placebo | Niigata University Medical & Dental Hospital | NULL | Completed | 16 Years | 80 Years | All | 78 | Phase 2 | Japan |
| 3 | NCT03316651 (ClinicalTrials.gov) | August 2016 | 27/8/2017 | Sequential Therapy With WLL/Inhaling GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis | A Multicenter Clinical Study of the Sequential Therapy With Whole Lung Lavage/Inhaling Granulocyte-macrophage Colony Stimulating Factor in Adult Patients With Severe Autoimmune Pulmonary Alveolar Proteinosis in China | Pulmonary Alveolar Proteinosis;Treatment | Drug: GM-CSF | Dai Huaping | NULL | Recruiting | 18 Years | N/A | All | 60 | Phase 2 | China |
| 4 | JPRN-JMA-IIA00205 | 20/07/2016 | 07/01/2015 | Pulmonary alveolar proteinosis GM-CSF inhalation efficacy trial in Japan | Pulmonary alveolar proteinosis GM-CSF inhalation efficacy trial in Japan | autoimmune pulmonary alveolar proteinosis | Intervention type:DRUG. Intervention1:GM-CSF inhalation therapy, Dose form:INJECTION, Route of administration:INHALATIONAL, intended dose regimen:125mcg BID inhalation for 7days and 7days without inhalation, 12 cycles. Control intervention1:placebo controlled, Dose form:INJECTION, Route of administration:INHALATIONAL, Intended dose regimen:placebo BID inhalation for 7days and 7days without inhalation, 12 cycles. | Koh Nakata | Partner Therapeutics | Completed | >=16 YEARS | <=80 YEARS | BOTH | 80 | Phase 2-3 | Japan |
| 5 | NCT02840708 (ClinicalTrials.gov) | May 2016 | 13/7/2016 | SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study | SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study | Pulmonary Alveolar Proteinosis, Autoimmune | Drug: Sargramostim | Niigata University Medical & Dental Hospital | NULL | Completed | 20 Years | 80 Years | All | 14 | Phase 1 | Japan |
| 6 | NCT02243228 (ClinicalTrials.gov) | August 2014 | 14/9/2014 | Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP) | A Prospective Study of Inhaling Granulocyte-macrophage Colony Stimulating Factor in Adult Patients With Mild-to-moderate Autoimmune Pulmonary Alveolar Proteinosis in China: a Randomized Open-label Study | Autoimmune Pulmonary Alveolar Proteinosis | Drug: GM-CSF | Peking Union Medical College Hospital | The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School | Recruiting | 18 Years | N/A | Both | 42 | Phase 2 | China |
| 7 | NCT01511068 (ClinicalTrials.gov) | August 2012 | 12/12/2011 | Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP) | Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP) | Hereditary Pulmonary Alveolar Proteinosis | Drug: Leukine | Children's Hospital Medical Center, Cincinnati | Virginia Commonwealth University;Genzyme, a Sanofi Company | Completed | 8 Years | N/A | All | 2 | Phase 2 | United States |
| 8 | NCT00901511 (ClinicalTrials.gov) | July 2009 | 11/5/2009 | Whole Lung Lavage (WLL)/Inhaled Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis (PAP) | Whole Lung Lavage Followed by Inhaled Sargramostim in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis | Pulmonary Alveolar Proteinosis | Drug: GM-CSF [Leukine (Sargramostim)];Procedure: WLL | IRCCS Policlinico S. Matteo | Agenzia Italiana del Farmaco | Enrolling by invitation | 18 Years | N/A | Both | 18 | Phase 2/Phase 3 | Italy |
| 9 | EUCTR2008-007086-23-IT (EUCTR) | 12/12/2008 | 27/01/2009 | Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP | Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP | Autoimmune PAP MedDRA version: 9.1;Level: LLT;Classification code 10037316;Term: Pulmonary alveolar proteinosis | Trade Name: Leukine INN or Proposed INN: Sargramostim | OSPEDALE POLICLINICO S. MATTEO | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Italy | ||||
| 10 | NCT00030056 (ClinicalTrials.gov) | September 2001 | 30/1/2002 | GM-CSF in Patients With Pulmonary Alveolar Proteinosis | Trial of GM-CSF for Alveolar Proteinosis | Pulmonary Alveolar Proteinosis | Drug: GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim) | The Cleveland Clinic | NULL | Terminated | 18 Years | 70 Years | Both | 48 | Phase 2 | United States |
| 11 | JPRN-JMA-IIA00013 | 16/08/2007 | A Phase II Study of Inhaled GM-CSF in Patients With Idiopathic Pulmonary Alveolar Proteinosis (iPAP) | A Phase II Study of Inhaled GM-CSF in Patients With Idiopathic Pulmonary Alveolar Proteinosis (iPAP) | Idiopathic pulmonary alveolar proteinosis | Intervention type:DRUG. Intervention1:Granulocyte-macrophage colony stimulating factor (GM-CSF), Dose form:INJECTION, Route of administration:INHALATIONAL, intended dose regimen:Weeks 1-12: 6 cycles of inhaled GM-CSF 125 mcg twice daily on days 1 through 8 of a 14-day Weeks 13-24: 6 cycles of inhaled GM-CSF 125 mcg once daily on days 1 through 4 of a 14-day cycle. | Koh Nakata, M.D., Ph.D.Bioscience Medical Research Center (BMRC), Niigata University Medical & Dental Hospital | Toshihiro NUKIWA, M.D., Ph.D., Department of Respiratory Medicine, Tohoku University Medical SchoolYoshikazu INOUE, M.D., Ph.D., National Hospital Organization Kinki-Chuo Chest Medical CenterToshinori TAKADA M.D., Ph.D., Department of Internal Medicine, Niigata University Medical and Dental Hospital | Completed | >=16 YEARS | <=80 YEARS | BOTH | 40 | Phase 2 | Japan |