229. 肺胞蛋白症（自己免疫性又は先天性） ［臨床試験数：20，薬物数：15（DrugBank：7），標的遺伝子数：3，標的パスウェイ数：12］
Searched query = "Autoimmune pulmonary alveolar proteinosis", "Congenital pulmonary alveolar proteinosis", "Hereditary pulmonary alveolar proteinosis", "Pulmonary alveolar proteinosis"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03887169 | April 2019 | 1 April 2019 | Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene. | Oral or Enteral Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene. | Pulmonary Alveolar Proteinosis;Mutation Ala393Thr of the MARS Gene;mutationSer567Leu of the MARS Gene | Drug: Methionine;Drug: Vitamin B12, B9, B6, C supplementation;Diagnostic Test: Methionine/homocysteine Dosage;Diagnostic Test: Thoracic CT scan;Diagnostic Test: Abdominal and liver ultrasound.;Diagnostic Test: Brain MRI | Assistance Publique - Hôpitaux de Paris | Not recruiting | N/A | 10 Years | All | 3 | Phase 1/Phase 2 | France | |
| 2 | EUCTR2017-004078-32-NL | 08/05/2018 | 28 February 2019 | Clinical trial in patients with the lung disease, autoimmune pulmonary alveolar proteinosis, to assess continued treatment with molgramostim by inhalation | AN OPEN-LABEL, NON-CONTROLLED, MULTICENTRE CLINICAL TRIAL OF INHALED MOLGRAMOSTIM IN AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS PATIENTS - IMPALA-EX | Autoimmune Pulmonary Alveolar Proteinosis (aPAP) MedDRA version: 20.0 Level: LLT Classification code 10037316 Term: Pulmonary alveolar proteinosis System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Molgramostim Nebuliser solution 300 mcg Pharmaceutical Form: Nebuliser solution INN or Proposed INN: MOLGRAMOSTIM CAS Number: 99283-10-0 Concentration unit: µg/ml microgram(s)/millilitre Concentration type: equal Concentration number: 250- | Savara ApS | Authorised | Female: yes Male: yes | 90 | Phase 3 | France;Greece;Denmark;Russian Federation;Israel;Germany;Netherlands;Italy;United Kingdom | |||
| 3 | NCT03482752 | April 16, 2018 | 30 September 2019 | Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis | An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients | Autoimmune Pulmonary Alveolar Proteinosis | Drug: Molgramostim | Savara Inc. | Recruiting | 18 Years | N/A | All | 60 | Phase 3 | Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Russian Federation;Turkey;United Kingdom | |
| 4 | NCT03231033 | August 17, 2017 | 11 February 2019 | Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis | First in Human Study of Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis | Autoimmune Pulmonary Alveolar Proteinosis | Drug: Pioglitazone | Children's Hospital Medical Center, Cincinnati | Not recruiting | 18 Years | 80 Years | All | 3 | Phase 1 | United States | |
| 5 | NCT03006146 | July 13, 2017 | 11 February 2019 | Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis | Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis | Autoimmune Pulmonary Alveolar Proteinosis | Drug: Sargramostim | Children's Hospital Medical Center, Cincinnati | Rare Diseases Clinical Research Network;National Center for Advancing Translational Science (NCATS) | Recruiting | 18 Years | 80 Years | All | 10 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT02835742 | September 1, 2016 | 25 February 2019 | 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 | Not recruiting | 16 Years | 80 Years | All | 78 | Phase 2 | Japan | |
| 7 | NCT03316651 | August 2016 | 16 December 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 | Recruiting | 18 Years | N/A | All | 60 | Phase 2 | China | |
| 8 | JPRN-JMA-IIA00205 | 20/07/2016 | 2 April 2019 | 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 | Not Recruiting | >=16 YEARS | <=80 YEARS | BOTH | 80 | Phase 2-3 | Japan |
| 9 | NCT02840708 | May 2016 | 8 January 2018 | 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 | Not recruiting | 20 Years | 80 Years | All | 14 | Phase 1 | Japan | |
| 10 | NCT02702180 | February 2016 | 30 September 2019 | Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) | A Randomised, Double-Blind, Placebo-Controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary AlveoLAr Proteinosis Patients IMPALA | Autoimmune Pulmonary Alveolar Proteinosis | Drug: molgramostim;Drug: placebo | Savara Inc. | Not recruiting | 18 Years | 75 Years | All | 135 | Phase 2/Phase 3 | United States;Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Portugal;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;Poland;Romania | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2015-003878-33-GB | 28/01/2016 | 12 March 2018 | A clinical trial where patients with the lung disease autoimmune Pulmonary Alveolar Proteinosis will be given the drug molgramostim by inhalation. | A Randomised, Double-Blind, Placebo-Controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary AlveoLAr Proteinosis Patients - IMPALA | Autoimmune Pulmonary Alveolar Proteinosis (aPAP) MedDRA version: 20.0 Level: LLT Classification code 10037316 Term: Pulmonary alveolar proteinosis System Organ Class: 100000004855 ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: Molgramostim 300 mcg nebuliser solution Pharmaceutical Form: Nebuliser solution INN or Proposed INN: MOLGRAMOSTIM CAS Number: 99283-10-0 Concentration unit: µg/ml microgram(s)/millilitre Concentration type: equal Concentration number: 250- Pharmaceutical form of the placebo: Nebuliser solution Route of administration of the placebo: Inhalation use | Savara ApS | Authorised | Female: yes Male: yes | 135 | Phase 2;Phase 3 | Portugal;United States;Slovakia;Greece;Spain;Turkey;Russian Federation;Israel;United Kingdom;Switzerland;Italy;France;Poland;Romania;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of | |||
| 12 | NCT02468908 | May 2015 | 16 December 2017 | Inhaled Molgramostim (rhGM-CSF) in Healthy Adult Subjects | A Randomized, Double-Blind, Placebo-Controlled, Single-Centre, Single Ascending Dose and Multiple Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Molgramostim When Administered by Inhalation to Healthy Adult Subjects | Pulmonary Alveolar Proteinosis;Bronchiectasis;Cystic Fibrosis;Acute Respiratory Distress Syndrome | Drug: Molgramostim;Drug: Placebo | Savara Inc. | Celerion | Not recruiting | 18 Years | 55 Years | All | 42 | Phase 1 | United Kingdom |
| 13 | NCT02243228 | August 2014 | 19 February 2015 | 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 |
| 14 | NCT01842386 | April 25, 2013 | 7 October 2019 | Rituximab for Anti-cytokine Autoantibody-Associated Diseases | Rituximab (Anti-CD20) for the Treatment of Subjects With Anticytokine Autoantibody-Associated Diseases | Pulmonary Alveolar Proteinosis (PAP);Severe Mucocutaneous Candidoasis | Drug: Rituximab | National Institute of Allergy and Infectious Diseases (NIAID) | Not recruiting | 18 Years | N/A | All | 7 | Phase 1 | United States | |
| 15 | NCT01511068 | August 2012 | 19 February 2015 | 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 | Not recruiting | 8 Years | N/A | Both | 2 | Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT01983657 | January 2012 | 19 February 2015 | Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP. | Study of Subcutaneous Injection of Low-dose Recombinant Granulocyte Macrophage-Colony Stimulating Factor (rhGM-CSF) +/- Whole Lung Lavage(WLL) in Pulmonary Alveolar Proteinosis. | Pulmonary Alveolar Proteinosis | Drug: rhGM-CSF;Procedure: Whole Lung Lavage(WLL) | Shanghai Pulmonary Hospital, Shanghai, China | Recruiting | 17 Years | 80 Years | Both | 40 | Phase 2 | China | |
| 17 | EUCTR2008-007086-23-IT | 12/12/2008 | 19 March 2012 | 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 Pharmaceutical Form: Solution for injection INN or Proposed INN: Sargramostim Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: .5- | OSPEDALE POLICLINICO S. MATTEO | Authorised | Female: yes Male: yes | Italy | |||||
| 18 | NCT00552461 | January 2007 | 19 February 2015 | Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis | Prospective Open-Label Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis | Pulmonary Alveolar Proteinosis;Primary Disease | Drug: rituximab | East Carolina University | Genentech, Inc. | Recruiting | 18 Years | N/A | Both | 10 | Phase 2 | United States |
| 19 | NCT00030056 | September 2001 | 7 April 2015 | 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 | Not recruiting | 18 Years | 70 Years | Both | 48 | Phase 2 | United States | |
| 20 | JPRN-JMA-IIA00013 | 2 April 2019 | 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 School Yoshikazu INOUE, M.D., Ph.D., National Hospital Organization Kinki-Chuo Chest Medical Center Toshinori TAKADA M.D., Ph.D., Department of Internal Medicine, Niigata University Medical and Dental Hospital | Not Recruiting | >=16 YEARS | <=80 YEARS | BOTH | 40 | Phase 2 | Japan |