Other: Quality-of-Life Assessment ( DrugBank: - )
9 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 34 | Neurofibromatosis | 3 |
| 36 | Epidermolysis bullosa | 1 |
| 51 | Scleroderma | 1 |
| 62 | Paroxysmal nocturnal hemoglobinuria | 1 |
| 65 | Primary immunodeficiency | 1 |
| 96 | Crohn disease | 1 |
| 283 | Acquired pure red cell aplasia | 1 |
| 286 | Hereditary sideroblastic anemia | 1 |
| 298 | Hereditary pancreatitis | 1 |
34. Neurofibromatosis
Clinical trials : 137 / Drugs : 213 - (DrugBank : 76) / Drug target genes : 87 - Drug target pathways : 200
| 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 | NCT04166409 (ClinicalTrials.gov) | January 3, 2020 | 15/11/2019 | A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma | A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1) | Low Grade Astrocytoma;Low Grade Glioma;Metastatic Low Grade Astrocytoma;Metastatic Low Grade Glioma | Drug: Carboplatin;Other: Quality-of-Life Assessment;Other: Questionnaire Administration;Drug: Selumetinib Sulfate;Drug: Vincristine Sulfate | National Cancer Institute (NCI) | NULL | Recruiting | 2 Years | 21 Years | All | 220 | Phase 3 | United States;Canada;Puerto Rico |
| 2 | NCT03871257 (ClinicalTrials.gov) | October 4, 2019 | 11/3/2019 | A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma | A Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) | Low Grade Glioma;Neurofibromatosis Type 1;Visual Pathway Glioma | Drug: Carboplatin;Other: Quality-of-Life Assessment;Other: Questionnaire Administration;Drug: Selumetinib Sulfate;Drug: Vincristine Sulfate | National Cancer Institute (NCI) | NULL | Recruiting | 2 Years | 21 Years | All | 290 | Phase 3 | United States;Canada;Puerto Rico |
| 3 | NCT02700230 (ClinicalTrials.gov) | December 8, 2017 | 25/2/2016 | Vaccine Therapy in Treating Patients With Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by Surgery | Phase I Trial of Intratumoral Administration of a NIS-Expressing Derivative Manufactured From a Genetically Engineered Strain of Measles Virus in Patients With Unresectable or Recurrent Malignant Peripheral Nerve Sheath Tumor | Metastatic Malignant Peripheral Nerve Sheath Tumor;Neurofibromatosis Type 1;Recurrent Malignant Peripheral Nerve Sheath Tumor | Procedure: Computed Tomography;Other: Laboratory Biomarker Analysis;Biological: Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter;Other: Quality-of-Life Assessment;Procedure: Single Photon Emission Computed Tomography | Mayo Clinic | National Cancer Institute (NCI) | Recruiting | 18 Years | N/A | All | 30 | Phase 1 | United States |
36. Epidermolysis bullosa
Clinical trials : 160 / Drugs : 195 - (DrugBank : 47) / Drug target genes : 50 - Drug target pathways : 124
| 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 | NCT04177498 (ClinicalTrials.gov) | August 24, 2021 | 7/11/2019 | Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC | A Pilot, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma | Recessive Dystrophic Epidermolysis Bullosa | Drug: Rigosertib Sodium;Other: Quality-of-Life Assessment | Thomas Jefferson University | Onconova Therapeutics, Inc. | Recruiting | 18 Years | 79 Years | All | 6 | Early Phase 1 | United States |
51. Scleroderma
Clinical trials : 523 / Drugs : 608 - (DrugBank : 156) / Drug target genes : 114 - Drug target pathways : 215
| 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 | NCT01413100 (ClinicalTrials.gov) | September 15, 2011 | 8/8/2011 | Scleroderma Treatment With Autologous Transplant (STAT) Study | A Phase II Multi-center Study of High-Dose Cyclophosphamide and Antithymocyte Globulin Followed by Autologous Hematopoietic Cell Transplantation With Post Transplant Maintenance for the Treatment of Systemic Sclerosis | Systemic Scleroderma | Biological: Anti-Thymocyte Globulin;Procedure: Autologous Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Biological: Filgrastim;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Peripheral Blood Stem Cell Transplantation;Drug: Plerixafor;Other: Quality-of-Life Assessment;Other: Questionnaire Administration | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI) | Active, not recruiting | N/A | 70 Years | All | 21 | Phase 2 | United States;Canada |
62. Paroxysmal nocturnal hemoglobinuria
Clinical trials : 271 / Drugs : 163 - (DrugBank : 49) / Drug target genes : 22 - Drug target pathways : 106
| 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 | NCT04965597 (ClinicalTrials.gov) | March 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Research Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
65. Primary immunodeficiency
Clinical trials : 482 / Drugs : 653 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 212
| 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 | NCT04965597 (ClinicalTrials.gov) | March 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Research Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
96. Crohn disease
Clinical trials : 2,400 / Drugs : 1,391 - (DrugBank : 267) / Drug target genes : 170 - Drug target pathways : 215
| 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 | NCT01570348 (ClinicalTrials.gov) | July 17, 2012 | 2/4/2012 | Crohn's Allogeneic Transplant Study | Allogeneic Hematopoietic Cell Transplantation for Patients With Treatment-Refractory Crohn's Disease: A Phase 2 Study | Crohn Disease | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Drug: Mycophenolic Acid;Other: Quality-of-Life Assessment;Drug: Tacrolimus;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Institutes of Health (NIH) | Terminated | 18 Years | 60 Years | All | 2 | Phase 2 | United States |
283. Acquired pure red cell aplasia
Clinical trials : 18 / Drugs : 42 - (DrugBank : 22) / Drug target genes : 19 - Drug target pathways : 98
| 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 | NCT04965597 (ClinicalTrials.gov) | March 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Research Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
286. Hereditary sideroblastic anemia
Clinical trials : 7 / Drugs : 23 - (DrugBank : 10) / Drug target genes : 8 - Drug target pathways : 42
| 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 | NCT04965597 (ClinicalTrials.gov) | March 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Research Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
298. Hereditary pancreatitis
Clinical trials : 98 / Drugs : 180 - (DrugBank : 57) / Drug target genes : 53 - Drug target pathways : 142
| 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 | NCT02743364 (ClinicalTrials.gov) | September 19, 2016 | 15/4/2016 | Simvastatin in Reducing Pancreatitis in Patients With Recurrent, Acute or Chronic Pancreatitis | Statin Therapy to Reduce the Risk of Recurrent Pancreatitis | Acute Pancreatitis | Other: Laboratory Biomarker Analysis;Other: Placebo Administration;Other: Quality-of-Life Assessment;Other: Questionnaire Administration;Drug: Simvastatin | National Cancer Institute (NCI) | NULL | Active, not recruiting | 18 Years | N/A | All | 8 | Phase 2 | United States |