Sargramostim ( DrugBank: Sargramostim )

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7 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
6	パーキンソン病	1
28	全身性アミロイドーシス	1
60	再生不良性貧血	2
62	発作性夜間ヘモグロビン尿症	1
96	クローン病	7
229	肺胞蛋白症（自己免疫性又は先天性）	6
285	ファンコニ貧血	1

6. パーキンソン病

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臨床試験数 : 2,307 /  薬物数 : 2,007 - (DrugBank : 349) / 標的遺伝子数 : 188 - 標的パスウェイ数 : 199

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	NCT03790670 (ClinicalTrials.gov)	January 30, 2019	18/12/2018	Biomarker Assessments of Leukine During Treatment of Parkinson's Disease	Safety, Tolerability and Biomarker Assessments of Leukine (Sargramostim) During Extended Timed Treatment for Parkinson's Disease: A Phase I Pilot Study	Parkinson Disease	Drug: sargramostim	University of Nebraska	NULL	Active, not recruiting	35 Years	85 Years	All	7	Phase 1	United States

28. 全身性アミロイドーシス

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臨床試験数 : 267 /  薬物数 : 241 - (DrugBank : 77) / 標的遺伝子数 : 68 - 標的パスウェイ数 : 180

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	NCT00007995 (ClinicalTrials.gov)	July 1999	6/1/2001	Chemotherapy Plus Peripheral Stem Cell Transplant in Treating Patients Who Have Multiple Myeloma or Primary Systemic Amyloidosis	Phase 2 Study Of High Dose Chemotherapy Followed By Autologous Hematopoietic Stem Cell Support In Patients With Multiple Myeloma And Primary Light Chain Amyloidosis	Multiple Myeloma and Plasma Cell Neoplasm	Biological: filgrastim;Biological: recombinant interferon alfa;Biological: sargramostim;Drug: busulfan;Drug: cyclophosphamide;Drug: melphalan;Procedure: autologous bone marrow transplantation;Procedure: bone marrow ablation with stem cell support;Procedure: peripheral blood stem cell transplantation	Herbert Irving Comprehensive Cancer Center	National Cancer Institute (NCI)	Completed	18 Years	N/A	Both	75	Phase 2	United States

60. 再生不良性貧血

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臨床試験数 : 245 /  薬物数 : 318 - (DrugBank : 86) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 166

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	NCT00053157 (ClinicalTrials.gov)	June 2002	27/1/2003	Sargramostim in Reducing Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplantation for Hematologic Cancer or Aplastic Anemia	Use Of Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) To Mobilize Donor Peripheral Blood Stem Cells Along With GM-CSF Administration Post Allogeneic Transplant - A Pilot Study	Chronic Myeloproliferative Disorders;Graft Versus Host Disease;Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Neoplasms	Biological: sargramostim	Roswell Park Cancer Institute	NULL	Completed	5 Years	60 Years	All	10	N/A	United States
2	NCT00053989 (ClinicalTrials.gov)	January 29, 2002	5/2/2003	NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders	Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders	Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Fanconi Anemia;Aplastic Anemia	Biological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Biological: sargramostim;Biological: therapeutic allogeneic lymphocytes;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation	Roswell Park Cancer Institute	NULL	Completed	4 Years	75 Years	All	41	Phase 2	United States

62. 発作性夜間ヘモグロビン尿症

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臨床試験数 : 292 /  薬物数 : 151 - (DrugBank : 49) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 108

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	NCT00012376 (ClinicalTrials.gov)	March 2001	3/3/2001	Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer	Dose Finding Study of Bryostatin-1 and GM-CSF in Refractory Myeloid Malignancies	Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Blastic Phase Chronic Myelogenous Leukemia;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Recurrent Adult Acute Myeloid Leukemia;Refractory Anemia;Refractory Anemia With Ringed Sideroblasts;Relapsing Chronic Myelogenous Leukemia;Thrombocytopenia;Untreated Adult Acute Myeloid Leukemia	Drug: bryostatin 1;Biological: sargramostim;Other: laboratory biomarker analysis;Other: pharmacological study	National Cancer Institute (NCI)	NULL	Completed	18 Years	N/A	Both	35	Phase 1	United States

96. クローン病

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臨床試験数 : 2,442 /  薬物数 : 1,278 - (DrugBank : 248) / 標的遺伝子数 : 142 - 標的パスウェイ数 : 209

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	NCT00329537 (ClinicalTrials.gov)	June 2006	23/5/2006	Study of Sargramostim in Moderately to Severely Active Crohn's Disease	Open-Label Phase 1 Study to Assess the Tolerability, Pharmacokinetics and Safety of 2 and 6 µg/kg Sargramostim (SH L 04023) Administered Subcutaneously Once Daily for 4 Weeks, and Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Assess the Efficacy and Safety of 6 µg/kg Sargramostim (SH L 04023) Administered Subcutaneously Once Daily for 8 Weeks in Patients With Active Crohn's Disease (Sargramostim Phase 1/2 Study)	Crohn's Disease	Drug: Sargramostim (Leukine);Drug: Placebo	Genzyme, a Sanofi Company	NULL	Terminated	18 Years	N/A	Both	11	Phase 2	Japan
2	NCT00295165 (ClinicalTrials.gov)	January 2006	21/2/2006	Efficacy (Induction of Response/Remission) and Safety Study in Patients With Moderate to Severe Crohn's Disease	Randomized, Double-Blind, Placebo-Controlled, Phase 3 Induction Study to Assess the Efficacy and Safety of 6µg Sargramostim (Leukine) Administered Subcutaneously Once Daily for 8 Weeks in Patients With Active Crohn's Disease	Crohn Disease	Drug: Sargramostim (Leukine);Drug: Placebo	Genzyme, a Sanofi Company	NULL	Terminated	18 Years	N/A	Both	33	Phase 3	United States;Australia;Brazil;Bulgaria;Canada;Israel;New Zealand;Romania;Russian Federation;South Africa;Ukraine
3	NCT00206661 (ClinicalTrials.gov)	December 2004	12/9/2005	Safety and Efficacy Study in the Treatment of Pediatric Crohn's Disease	Phase 1/2 Safety, Pharmacokinetic, and Pharmacodynamic Study of Sargramostim (Leukine®) in Pediatric Patients With Active Crohn's Disease, With One-year Surveillance and Retreatment Extension	Crohn Disease	Drug: Sargramostim (Leukine)	Genzyme, a Sanofi Company	NULL	Terminated	6 Years	16 Years	Both	22	Phase 1/Phase 2	United States
4	NCT00206674 (ClinicalTrials.gov)	September 2004	13/9/2005	Efficacy (Induction of Response/Remission) and Safety Study in Patients With Moderate to Severe Crohn's Disease	Phase 3 Randomized, Double-Blind, Placebo Controlled Induction Study of Sargramostim (Leukine®) in Patients With Active Crohn's Disease	Crohn Disease	Drug: Sargramostim (Leukine);Drug: Placebo	Genzyme, a Sanofi Company	NULL	Completed	18 Years	N/A	Both	286	Phase 3	Argentina;Australia;Brazil;Canada;New Zealand;Russian Federation;Switzerland;Ukraine;United Kingdom
5	NCT00206713 (ClinicalTrials.gov)	July 2004	13/9/2005	Efficacy Study of Sargramostim in the Retreatment of Patients Who Have Crohn's Disease Who Have Previously Responded to Treatment With Sargramostim	Phase 3 Randomized, Double-Blind, Placebo-Controlled Multicenter Retreatment Study of Sargramostim (Leukine®) in Patients With Active Crohn's Disease and Prior Treatment Response to Sargramostim	Crohn Disease	Drug: Sargramostim (Leukine);Drug: Placebo	Genzyme, a Sanofi Company	NULL	Terminated	18 Years	N/A	Both	264	Phase 3	United States
6	NCT00206596 (ClinicalTrials.gov)	July 2003	12/9/2005	Study in Patients With Crohn's Disease Who Are Steroid Dependent, Despite Previous Unsuccessful Attempts to Reduce Steroids Due to Worsening of Crohn's Disease	Randomized, Stratified, Double-Blind, Placebo-Controlled Phase 2 Study of Steroid-Sparing Properties of Sargramostim (Leukine) Therapy in Patients With Cortico-Dependent Crohn's Disease	Crohn Disease	Drug: Sargramostim (Leukine);Drug: Placebo	Genzyme, a Sanofi Company	NULL	Completed	18 Years	80 Years	Both	127	Phase 2	United States;Canada
7	NCT00206700 (ClinicalTrials.gov)	February 2003	13/9/2005	Open-label Trial of Leukine in Active Crohn's Disease	Open-label Trial of Leukine® (Sargramostim), A Recombinant Human Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF), in Active Crohn's Disease	Crohn Disease	Drug: Sargramostim (Leukine)	Genzyme, a Sanofi Company	NULL	Terminated	18 Years	N/A	Both	378	Phase 2	United States;Argentina;Australia;Brazil;Canada;New Zealand;Russian Federation;Switzerland;Ukraine;United Kingdom

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229. 肺胞蛋白症（自己免疫性又は先天性）

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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	NCT03006146 (ClinicalTrials.gov)	July 13, 2017	28/11/2016	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)	Completed	18 Years	80 Years	All	10	Phase 1	United States
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	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
4	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
5	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
6	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

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285. ファンコニ貧血

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臨床試験数 : 62 /  薬物数 : 93 - (DrugBank : 30) / 標的遺伝子数 : 30 - 標的パスウェイ数 : 144

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	NCT00053989 (ClinicalTrials.gov)	January 29, 2002	5/2/2003	NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders	Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders	Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Fanconi Anemia;Aplastic Anemia	Biological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Biological: sargramostim;Biological: therapeutic allogeneic lymphocytes;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation	Roswell Park Cancer Institute	NULL	Completed	4 Years	75 Years	All	41	Phase 2	United States

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