Sirolimus    (DrugBank: Sirolimus)

35 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
13多発性硬化症/視神経脊髄炎1
17多系統萎縮症1
19ライソゾーム病2
34神経線維腫症5
35天疱瘡1
36表皮水疱症2
46悪性関節リウマチ3
49全身性エリテマトーデス1
51全身性強皮症2
60再生不良性貧血6
61自己免疫性溶血性貧血1
62発作性夜間ヘモグロビン尿症1
63特発性血小板減少性紫斑病2
65原発性免疫不全症候群7
66IgA腎症2
67多発性嚢胞腎10
84サルコイドーシス1
85特発性間質性肺炎1
89リンパ脈管筋腫症13
98好酸球性消化管疾患1
127前頭側頭葉変性症1
137限局性皮質異形成5
157スタージ・ウェーバー症候群1
158結節性硬化症14
192コケイン症候群2
222一次性ネフローゼ症候群3
277リンパ管腫症/ゴーハム病6
278巨大リンパ管奇形(頚部顔面病変)8
279巨大静脈奇形(頚部口腔咽頭びまん性病変)2
280巨大動静脈奇形(頚部顔面又は四肢病変)4
281クリッペル・トレノネー・ウェーバー症候群1
283後天性赤芽球癆3
285ファンコニ貧血1
331特発性多中心性キャッスルマン病3

6. パーキンソン病 [臨床試験数:2,123,薬物数:2,046(DrugBank:324),標的遺伝子数:183,標的パスウェイ数:198
Searched query = "Parkinson disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 2,123 trial found
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1NCT04127578January 3, 202014/10/2019Phase 1/2a Clinical Trial of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of PR001A in Patients With Parkinson's Disease With at Least One GBA1 MutationParkinson DiseaseBiological: PR001A;Drug: Methylprednisolone;Drug: Sirolimus;Drug: PrednisonePrevail TherapeuticsNULLRecruiting35 Years80 YearsAll12Phase 1/Phase 2United States

13. 多発性硬化症/視神経脊髄炎 [臨床試験数:3,050,薬物数:2,147(DrugBank:348),標的遺伝子数:244,標的パスウェイ数:228
Searched query = "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 3,050 trial found
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1NCT00095329May 20032/11/2004Treating Multiple Sclerosis With Sirolimus, an Immune System SuppressorA Phase I/II, Open-Label Pilot Trial to Evaluate the Safety of Rapamune (Sirolimus) in Patients With Multiple SclerosisMultiple Sclerosis (MS) - Relapsing-remittingBiological: sirolimusNational Institute of Allergy and Infectious Diseases (NIAID)Autoimmunity Centers of ExcellenceTerminated18 Years58 YearsBoth14Phase 1/Phase 2United States

17. 多系統萎縮症 [臨床試験数:107,薬物数:153(DrugBank:48),標的遺伝子数:59,標的パスウェイ数:104
Searched query = "Multiple system atrophy", "MSA-C", "MSA-P", "Olivopontocerebellar atrophy", "OPCA", "Striatonigral degeneration", "Shy-Drager syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 107 trial found
No.TrialIDDate_
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1NCT03589976September 1, 20185/7/2018A Futility Trial of Sirolimus in Multiple System AtrophyA Single Center Randomized,Double Blind, Placebo-controlled Futility Trial to Determine if Sirolimus is of Sufficient Promise to Slow the Progression of Multiple System AtrophyMultiple System AtrophyDrug: Sirolimus 2 MG;Other: PlaceboNYU Langone HealthNational Institute of Neurological Disorders and Stroke (NINDS)Recruiting30 Years80 YearsAll56Phase 2United States

19. ライソゾーム病 [臨床試験数:784,薬物数:673(DrugBank:101),標的遺伝子数:68,標的パスウェイ数:184
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 784 trials found
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1NCT04411654September 202011/5/2020Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants With Type 2 Gaucher DiseaseGaucher Disease, Type 2Biological: PR001;Drug: Methylprednisolone;Drug: Sirolimus;Drug: PrednisonePrevail TherapeuticsNULLRecruitingN/A24 MonthsAll15Phase 1/Phase 2United States
2NCT03952637August 19, 201915/5/2019A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type I and Type II GM1 GangliosidosisA Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 GangliosidosisLysosomal Diseases;Gangliosidosis;GM1Biological: AAV9-GLB1;Drug: Rituximab;Drug: Sirolimus;Drug: Methylprednisolone;Drug: Prednisone;Diagnostic Test: Audiology assessmentwith ABR;Diagnostic Test: Bone density scan (DEXA;Diagnostic Test: Electrocardiogram (EKG);Diagnostic Test: Echocardiogram;Other: Electroencephalogram (EEG) awake andextended overnight;Diagnostic Test: Laboratory tests;Procedure: Lumbar puncture;Procedure: Brain MRI/MRS/fMRI;Behavioral: Neurocognitive testing;Other: Neurology exam;Behavioral: PICC line placement;Procedure: Skeletal survey;Procedure: Skin biopsy;Procedure: Speech and modified barium swallow study;Procedure: Ophthalmology examNational Human Genome Research Institute (NHGRI)Axovant Sciences, Inc.Recruiting6 Months12 YearsAll45Phase 1/Phase 2United States

34. 神経線維腫症 [臨床試験数:120,薬物数:182(DrugBank:72),標的遺伝子数:84,標的パスウェイ数:194
Searched query = "Neurofibromatosis", "NF1", "von Recklinghausen disease", "NF2"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
5 / 120 trials found
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1NCT03433183October 2, 201929/1/2018SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath TumorsSARC031: A Phase 2 Trial of the MEK Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Combination With the mTOR Inhibitor Sirolimus for Patients With Unresectable or Metastatic Malignant Peripheral Nerve Sheath TumorsMalignant Peripheral Nerve Sheath Tumors;Neurofibromatosis 1Drug: Selumetinib;Drug: SirolimusSarcoma Alliance for Research through CollaborationUnited States Department of Defense;AstraZenecaRecruiting12 YearsN/AAll21Phase 2United States
2JPRN-UMIN0000210302016/03/1622/02/2016A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 Neurofibromatosis type 10.2% Sirolimus gel twice daily 24 weeks topical application
0.4% Sirolimus gel twice daily 24 weeks topical application
Placebo gel twice daily 24 weeks topical application
Osaka University HospitalNULLComplete: follow-up complete16years-old70years-oldMale and Female18Phase 2Japan
3NCT01031901December 200910/12/2009Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;NeurofibromaDrug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycinThe University of Texas Health Science Center, HoustonSociety for Pediatric DermatologyCompleted13 YearsN/ABoth52Phase 1United States
4NCT00634270April 200820/2/2008A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform NeurofibromasA Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform NeurofibromasNeurofibromatosis Type 1Drug: SirolimusUniversity of Alabama at BirminghamBoston Children’s Hospital;Children's Hospital of Philadelphia;Children's Research Institute;Children's Hospital Medical Center, Cincinnati;National Cancer Institute (NCI);University of Chicago;University of Utah;Washington University School of MedicineCompleted3 Years75 YearsAll58Phase 2United States
5NCT00652990March 20083/4/2008Sirolimus to Treat Plexiform Neurofibromas in Patients With Neurofibromatosis Type IA Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform NeurofibromasNeurofibromatosis Type 1;Plexiform Neurofibromas;Paraspinal Plexiform NeurofibromasDrug: SirolimusUniversity of Alabama at BirminghamNational Cancer Institute (NCI)Active, not recruiting3 YearsN/ABoth18Phase 2United States

35. 天疱瘡 [臨床試験数:87,薬物数:114(DrugBank:37),標的遺伝子数:17,標的パスウェイ数:158
Searched query = "Pemphigus"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 87 trial found
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1NCT01313923February 201110/3/2011Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis PemphigusEvaluation of Sirolimus for the Treatment of the Autoimmune Blistering Dermatosis PemphigusPemphigusDrug: Sirolimus (formerly known as Rapamycin)University of California, IrvineNULLTerminated18 YearsN/AAll3Early Phase 1United States

36. 表皮水疱症 [臨床試験数:147,薬物数:170(DrugBank:40),標的遺伝子数:32,標的パスウェイ数:113
Searched query = "Epidermolysis bullosa", "EBS", "JEB", "DDEB", "RDEB", "Kindler syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 147 trials found
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1NCT02960997May 201615/6/2016Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) StudyA Prospective, Double-Blind, Cross-Over, Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2% in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex (EBS)Epidermolysis Bullosa Simplex;Epidermolysis Bullosa Simplex Kobner;Weber-Cockayne SyndromeDrug: Sirolimus, 2%;Drug: VehicleStanford UniversityNULLActive, not recruiting4 YearsN/AAll8Phase 2United States
2NCT03016715May 20169/1/2017Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) StudyA Prospective, Double-Blind, Cross-Over, Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2% in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex (EBS)Epidermolysis Bullosa Simplex;Epidermolysis Bullosa Simplex Kobner;Weber-Cockayne SyndromeDrug: Sirolimus 2%;Drug: VehiclePremier Specialists, AustraliaNULLRecruiting5 YearsN/AAll8Phase 2Australia

46. 悪性関節リウマチ [臨床試験数:4,183,薬物数:2,538(DrugBank:401),標的遺伝子数:183,標的パスウェイ数:219
Searched query = "Malignant rheumatoid arthritis", "Rheumatoid arthritis", "Rheumatoid arthritis with vasculitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 4,183 trials found
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1ChiCTR19000242612019-07-152019-07-03Efficacy and safety of sirolimus in new-onset rheumatoid arthritis: a prospective, double-blinded, randomized, placebo-controlled, monocentric study in ChinaEfficacy and safety of sirolimus in new-onset rheumatoid arthritis: a prospective, double-blinded, randomized, placebo-controlled, monocentric study in China rheumatoid arthritisA:methotrexate (7.5~15 mg/week)+ placbo (1 mg/d);B:sirolimus (1 mg/d) + methotrexate (7.5~15 mg/week);The Second Hospital of Shanxi Medical UniversityNULLPending1865BothA:30;B:30;China
2ChiCTR-IPR-170115662017-07-012017-06-05The efficacy and safety of sirolimus in refractory rheumatoid arthritis: A multi-center randomized controlled trial in ChinaThe efficacy and safety of sirolimus in refractory rheumatoid arthritis: A multi-center randomized controlled trial in China Rheumatoid Arthritis;M05.901Sirolimus group: Sirolimus; Non-Sirolimus group:Glucocorticoids and Immunosuppressant;the Second Hospital of Shanxi Medical UniversityNULLRecruiting1865BothSirolimus group:200; Non-Sirolimus group:100;China
3NCT00392951December 200624/10/2006Sirolimus for Autoimmune Disease of Blood CellsSirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot SeriesAutoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid ArthritisDrug: sirolimusChildren's Hospital of PhiladelphiaNULLCompleted1 Year30 YearsAll30Phase 1/Phase 2United States

49. 全身性エリテマトーデス [臨床試験数:827,薬物数:638(DrugBank:168),標的遺伝子数:108,標的パスウェイ数:191
Searched query = "Systemic lupus erythematosus", "SLE"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 827 trial found
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1NCT04582136November 20203/10/2020Efficacy and Safety of Sirolimus in Active Systemic Lupus ErythematosusEfficacy and Safety of Sirolimus in Patients With Active Systemic Lupus Erythematosus Despite Standard of Care: a Multi-center, Double Blinded, Randomized, Placebo-controlled, Phase 2 TrialSystemic Lupus ErythematosusDrug: Sirolimus;Drug: PlaceboChinese SLE Treatment And Research GroupBeijing Municipal Science & Technology Commission;North China Pharmaceutical Group CorporationNot yet recruiting18 Years65 YearsAll146Phase 2NULL

51. 全身性強皮症 [臨床試験数:466,薬物数:536(DrugBank:142),標的遺伝子数:110,標的パスウェイ数:210
Searched query = "Scleroderma", "Systemic sclerosis", "SSc", "dcSSc", "lcSSc"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 466 trials found
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1ChiCTR20000303702020-03-012020-02-29The efficacy and safety of sirolimus for the treatment of Systemic Sclerosis: a single-arm, single-center pilot studyThe efficacy and safety of sirolimus for the treatment of Systemic Sclerosis: a single-arm, single-center pilot study Systemic Sclerosistreatment group:sirolimus;The First Affiliated Hospital of China Medical UniversityNULLRecruiting18Bothtreatment group:20;China
2NCT03365869June 1, 20184/12/2017A Pilot-Study of Sirolimus for the Treatment of Systemic SclerosisA Phase ? Pilot-Study With Sirolimus for the Treatment of Systemic SclerosisSystemic SclerosisDrug: SirolimusPeking University People's HospitalNULLNot yet recruiting18 Years80 YearsAll72Phase 2NULL

60. 再生不良性貧血 [臨床試験数:218,薬物数:362(DrugBank:81),標的遺伝子数:39,標的パスウェイ数:155
Searched query = "Aplastic anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
6 / 218 trials found
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1NCT03192397August 3, 201716/6/2017Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell TransplantA Phase II Trial of Fludarabine/Melphalan/Total Body Irradiation With Post Transplant Cyclophosphamide as Graft Versus Host Disease Prophylaxis in Matched-Related and Matched-Unrelated Allogeneic Hematopoietic Cell TransplantationAcute Myeloid Leukemia in Remission;Adult Acute Lymphoblastic Leukemia in Complete Remission;Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission;Chronic Myelomonocytic Leukemia in Remission;Graft Versus Host Disease;Hodgkin Lymphoma;Minimal Residual Disease;Myelodysplastic Syndrome;Myeloproliferative Neoplasm;Non-Hodgkin Lymphoma;Plasma Cell Myeloma;Severe Aplastic Anemia;Waldenstrom MacroglobulinemiaProcedure: Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Melphalan Hydrochloride;Drug: Mycophenolate Mofetil;Drug: Sirolimus;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNational Cancer Institute (NCI)Recruiting18 Years79 YearsAll30Phase 2United States
2NCT02979873December 19, 20161/12/2016Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive TherapyA Randomized Trial of Sirolimus (Rapamune(R)) for Relapse Prevention in Patients With Severe Aplastic Anemia Responsive to Immunosuppressive TherapySevere Aplastic AnemiaDrug: SirolimusNational Heart, Lung, and Blood Institute (NHLBI)NULLRecruiting2 Years99 YearsAll118Phase 2United States
3EUCTR2006-006577-25-SE25/07/200711/06/2007A prospective randomized study comparing rapamune and tacrolimus vs. cyclosporine and methotrexate as immune prophylaxis in allogeneic hematopoietic stem cell transplantation, using HLA-A, -B, -DRB1 identical related or unrelated donors. A Nordic multicenter study. - Rapa + FK in stem cell transplantationA prospective randomized study comparing rapamune and tacrolimus vs. cyclosporine and methotrexate as immune prophylaxis in allogeneic hematopoietic stem cell transplantation, using HLA-A, -B, -DRB1 identical related or unrelated donors. A Nordic multicenter study. - Rapa + FK in stem cell transplantation Graft versus host disease prophylaxis in patients receiving stem cell transplantation due to: chronic myeloid leukemia (CML) in 1st or 2nd chronic phase, acute myeloid leukemia (AML) in complete remission, acute lymphoblastic leukemia (ALL) in complete remission, myelodysplastic syndrome, chronic lymphocytic leukemia, lymphoma, non-malignant disorders, severe aplastic anemia, hemoglobinopathies and metabolic disorders
MedDRA version: 9.1Level: LLTClassification code 10018799Term: GVHD
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: SIROLIMUS
Other descriptive name: Rapamycin
Trade Name: Prograf
Product Name: Prograf
INN or Proposed INN: TACROLIMUS
Other descriptive name: FK-506
Trade Name: Sandimmun Neoral
Product Name: Sandimmun Neoral
INN or Proposed INN: CICLOSPORIN
Other descriptive name: CsA
Trade Name: Methotrexate
Product Name: Methotrexate
INN or Proposed INN: METHOTREXATE
Other descriptive name: MTX
Karolinska InstitutetNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Finland;Sweden
4NCT00358657May 24, 200628/7/2006Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited DisordersHLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Active, not recruitingN/A55 YearsAll14Phase 2United States
5NCT00319878May 200628/4/2006Sirolimus and Cyclosporine for Treatment-Resistant Aplastic AnemiaA Phase I/II Trial of Sirolimus (Rapamune) and Cyclosporine in Patients With Refractory Aplastic AnemiaAnemia, AplasticDrug: Sirolimus;Drug: CyclosporineOffice of Rare Diseases (ORD)Rare Diseases Clinical Research NetworkRecruiting21 YearsN/ABoth52Phase 1/Phase 2United States
No.TrialIDDate_
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PhaseCountries
6NCT00061360June 26, 200323/5/2003Improving Immunosuppressive Treatment for Patients With Severe Aplastic AnemiaA Randomized Trial of a Novel Immunosuppressive Combination of ATG, CsA and Sirolimus (Rapamune) vs a Slow Taper Cyclosporine Regimen in Subjects With Severe Aplastic AnemiaSevere Aplastic AnemiaDrug: ATG+Rapamune+cyclosporine;Drug: ATG+cyclosporineNational Heart, Lung, and Blood Institute (NHLBI)NULLCompleted2 Years110 YearsAll77Phase 2United States

61. 自己免疫性溶血性貧血 [臨床試験数:90,薬物数:72(DrugBank:23),標的遺伝子数:19,標的パスウェイ数:147
Searched query = "Autoimmune hemolytic anemia", "AIHA", "WAIHA", "Cold agglutinin disease", "CAD", "Paroxysmal cold hemoglobinuria", "MAIHA", "Evans syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 90 trial found
No.TrialIDDate_
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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1NCT00392951December 200624/10/2006Sirolimus for Autoimmune Disease of Blood CellsSirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot SeriesAutoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid ArthritisDrug: sirolimusChildren's Hospital of PhiladelphiaNULLCompleted1 Year30 YearsAll30Phase 1/Phase 2United States

62. 発作性夜間ヘモグロビン尿症 [臨床試験数:202,薬物数:120(DrugBank:25),標的遺伝子数:15,標的パスウェイ数:90
Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 202 trial found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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1NCT03866681April 1, 201928/2/2019Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNHSirolimus Combined With Low-dose Warfarin for the Treatment of Refractory Classic Paroxysmal Nocturnal Hemoglobinuria ,a Prospective StudyParoxysmal Nocturnal HemoglobinuriaDrug: sirolimusPeking Union Medical College HospitalNULLNot yet recruiting18 Years70 YearsAll40Phase 4China

63. 特発性血小板減少性紫斑病 [臨床試験数:311,薬物数:185(DrugBank:39),標的遺伝子数:43,標的パスウェイ数:132
Searched query = "Idiopathic thrombocytopenic purpura", "Primary immune thrombocytopenia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 311 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1ChiCTR-ONC-170121262017-09-012017-07-25A multicenter, prospective, one-arm clinical study of Sirolimus treats refractory and recurrence primary immune thrombocytopeniaA multicenter, prospective, one-arm clinical study of Sirolimus treats refractory and recurrence primary immune thrombocytopenia refractory or recurrence primary immune thrombocytopeniaCase series:Sirolimus treats refractory or recurrence primary immune thrombocytopenia;Department of Hematology, Xiniao HospitalNULLPending1880BothCase series:103;China
2NCT00392951December 200624/10/2006Sirolimus for Autoimmune Disease of Blood CellsSirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot SeriesAutoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid ArthritisDrug: sirolimusChildren's Hospital of PhiladelphiaNULLCompleted1 Year30 YearsAll30Phase 1/Phase 2United States

65. 原発性免疫不全症候群 [臨床試験数:413,薬物数:581(DrugBank:97),標的遺伝子数:68,標的パスウェイ数:202
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
7 / 413 trials found
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT04370795December 17, 202030/4/2020Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and SirolimusMatched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and SirolimusSevere Combined Immunodeficiency (SCID)Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradiation (TBI)National Institute of Allergy and Infectious Diseases (NIAID)NULLEnrolling by invitation3 Years40 YearsAll30Phase 1/Phase 2United States
2NCT03910452October 28, 20199/4/2019Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant CyclophosphamideHaploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant CyclophosphamideChronic Granulomatous DiseaseDrug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cellNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting4 Years65 YearsAll30Early Phase 1United States
3NCT02629120December 17, 201510/12/2015High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseHigh Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseChronic Granulomatous Disease TransplantDrug: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cellsNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting4 Years65 YearsAll50Phase 1/Phase 2United States
4NCT02282904October 23, 20144/11/2014Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant CyclophosphamideHaploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant CyclophosphamideChronic Granulomatous DiseaseDrug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated2 Years65 YearsAll7Phase 1/Phase 2United States
5NCT02177760July 201419/6/2014Sirolimus Prophylaxis for aGVHD in TME SCIDSirolimus in Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID) Infants Receiving Unconditioned Hematopoietic Stem Cell Transplant (HSCT)Severe Combined Immunodeficiency;Transplacental Maternal Engraftment;Stem Cell TransplantDrug: SirolimusUniversity of California, San FranciscoNULLWithdrawnN/A1 YearBoth0Phase 2United States
No.TrialIDDate_
enrollment
Date_
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Public_titleScientific_titleConditionInterventionPrimary_
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Recruitment_
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agemin
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6NCT00392951December 200624/10/2006Sirolimus for Autoimmune Disease of Blood CellsSirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot SeriesAutoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid ArthritisDrug: sirolimusChildren's Hospital of PhiladelphiaNULLCompleted1 Year30 YearsAll30Phase 1/Phase 2United States
7NCT00358657May 24, 200628/7/2006Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited DisordersHLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Active, not recruitingN/A55 YearsAll14Phase 2United States

66. IgA腎症 [臨床試験数:199,薬物数:214(DrugBank:57),標的遺伝子数:32,標的パスウェイ数:128
Searched query = "IgA nephropathy", "IgA nephritis", "Berger disease", "IgA-IgG nephropathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 199 trials found
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Public_titleScientific_titleConditionInterventionPrimary_
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1NCT00396721January 20063/11/2006Sirolimus Therapy for Poor Prognosis Immunoglobulin A NephropathyPilot Trial of Treatment of Poor-Prognosis IgA Nephropathy With Low Exposure to Sirolimus.Glomerulonephritis, IGA;Nephropathy, IGA;IGA NephropathyDrug: ACE inhibitor + statin;Drug: Sirolimus (study drug)+ACE inhibitor + statinJosep m CruzadoWyeth is now a wholly owned subsidiary of PfizerCompleted18 Years70 YearsBoth23Phase 2Spain
2EUCTR2005-002610-37-ES05/12/200530/09/2005PILOT TRIAL OF TREATMENT OF POOR-PROGNOSIS IgA NEPHROPATHY WITH LOW EXPOSURE TO SIROLIMUS.Ensayo clínico piloto de tratamiento de la nefropatía IgA con factores de mal pronóstico con dosis bajas de sirolimusPILOT TRIAL OF TREATMENT OF POOR-PROGNOSIS IgA NEPHROPATHY WITH LOW EXPOSURE TO SIROLIMUS.Ensayo clínico piloto de tratamiento de la nefropatía IgA con factores de mal pronóstico con dosis bajas de sirolimus To test in a pilot trial the efficacy and tolerance of sirolimus oral (at low doses) in patient to treat poor-prognosis IgA Nephropathy.Trade Name: RAPAMUNE
Product Name: SIROLIMUS
INN or Proposed INN: Sirolimus
Trade Name: RAPAMUNE
Product Name: SIROLIMUS
INN or Proposed INN: Sirolimus
Trade Name: RAPAMUNE
Product Name: SIROLIMUS
INN or Proposed INN: Sirolimus
NEPHROLOGY DEPARTMENT (HOSPITAL UNIVERSITARY OF BELLVITGE)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Spain

67. 多発性嚢胞腎 [臨床試験数:186,薬物数:196(DrugBank:47),標的遺伝子数:35,標的パスウェイ数:146
Searched query = "Polycystic kidney disease", "PKD", "PCKD", "Polycystic kidney", "ADPKD", "ARPKD"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
10 / 186 trials found
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Public_titleScientific_titleConditionInterventionPrimary_
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1NCT02055079April 201431/1/2014Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney DiseasePulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease - The Vienna RAP StudyPolycystic Kidney, Type 1 Autosomal Dominant Disease;Polycystic Kidney, Type 2 Autosomal Dominant DiseaseDrug: Sirolimus;Drug: PlaceboMedical University of ViennaNULLUnknown status18 YearsN/AAll68Phase 3Austria
2EUCTR2012-000550-60-AT17/01/201427/11/2013Pulsed oral sirolimus in autosomal dominant polycystic kidney diseasePulsed oral sirolimus in autosomal dominant polycystic kidney disease - The Vienna RAP Study Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by the development and uncontrolled proliferation of innumerable epithelial-lined cysts that stem from renal tubular cells, which compress and/or destroy vital renal tissue with a gradual decline in renal function, and terminal kidney failure with the need for renal reaplacement therapy. As yet, other than supportive care there is no viable therapy.
MedDRA version: 20.0Level: LLTClassification code 10036046Term: Polycystic kidney, autosomal dominantSystem Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Rapamune 1mg tablets
Product Name: Rapamune 1mg tablets
INN or Proposed INN: SIROLIMUS
Medizinische Universität Wien, Klinische Abteilung für Nephrologie und Dialyse, Universitätsklinik für Innere Medizin 3NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
68Phase 3Austria
3NCT01680250September 201130/8/2012Sirolimus for Massive Polycystic LiverAn Open-label, Prospective Clinical Trial to Evaluate the Effectiveness and Safety of Sirolimus to Reduce Cyst Growth in ADPKD Patients With Massive Polycystic LiverPolycystic Kidney DiseasesDrug: SirolimusSeoul National University HospitalWyeth is now a wholly owned subsidiary of PfizerRecruiting18 Years65 YearsBoth44Phase 2/Phase 3Korea, Republic of
4NCT01223755September 201012/10/2010Sirolimus In Autosomal Dominant Polycystic Kidney Disease And Severe Renal InsufficiencyEFFECTS OF SIROLIMUS ON DISEASE PROGRESSION IN PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE AND SEVERE RENAL INSUFFICIENCYAutosomal Dominant Polycystic Kidney Disease (ADPKD)Drug: Sirolimus;Drug: conventional therapyMario Negri Institute for Pharmacological ResearchNULLTerminated18 Years80 YearsBoth41Phase 2/Phase 3Italy
5NCT01632605November 200913/5/2012The Vienna RAP Pilot StudyRapamycin in Advanced Polycystic Kidney Disease Pilot StudyADPKDDrug: SirolimusMedical University of ViennaNULLCompleted18 YearsN/ABoth8N/AAustria
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
6EUCTR2007-005047-21-IT24/12/200702/07/2008Effects of Sirolimus on disease progression in patients with Autosomal Dominant Polycystic Kidney Disease and severe renal insufficiency - SIRENA IIEffects of Sirolimus on disease progression in patients with Autosomal Dominant Polycystic Kidney Disease and severe renal insufficiency - SIRENA II Autosomal Dominant Polycystic Kidney Disease (ADPKD)
MedDRA version: 9.1 Level: LLT Classification code 10010428 Term: Congenital cystic kidney disease
IST. DI RICERCHE FARMACOLOG. M. NEGRINULLNot Recruiting Female: yes
Male: yes
40 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noItaly
7EUCTR2007-006557-25-IT20/12/200718/12/2007RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDYRAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDY ADPKD type I
MedDRA version: 9.1Level: SOCClassification code 10038359Term: Renal and urinary disorders
Trade Name: RAPAMUNE*100CPR RIV 1MG
INN or Proposed INN: Sirolimus
Product Name: Ramipril
INN or Proposed INN: Ramipril
AZIENDA OSPEDALIERA OSPEDALE POLICLINICO CONSORZIALENULLNot RecruitingFemale: yes
Male: yes
Italy
8NCT00491517March 200725/6/2007Sirolimus Treatment in Patients With Autosomal Dominant Polycystic Kidney Disease: Renal Efficacy and SafetySirolimus Treatment in Patients With ADPKDPolycystic KidneyDrug: Sirolimus;Drug: conventional therapyMario Negri Institute for Pharmacological ResearchNULLCompleted18 Years80 YearsBoth22Phase 2Italy
9EUCTR2006-003427-37-IT05/02/200727/12/2006Sirolimus treatment in patients with autosomal dominant polycystic kidney disease renal efficacy and safety - NDSirolimus treatment in patients with autosomal dominant polycystic kidney disease renal efficacy and safety - ND Autosomal-Dominant Polycystic Kidney Disease ADPKD
MedDRA version: 9.1 Level: LLT Classification code 10010428 Term: Congenital cystic kidney disease
IST. DI RICERCHE FARMACOLOG. M. NEGRINULLNot Recruiting Female: yes
Male: yes
16 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noItaly
10NCT00346918June 200622/6/2006Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)Sirolimus (Rapamune®) for Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD): a Randomized Controlled Study.Autosomal Dominant Polycystic Kidney Disease (ADPKD)Drug: Sirolimus;Other: StandardUniversity of ZurichNULLCompleted18 Years40 YearsBoth100Phase 3Switzerland

84. サルコイドーシス [臨床試験数:143,薬物数:221(DrugBank:79),標的遺伝子数:82,標的パスウェイ数:165
Searched query = "Sarcoidosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 143 trial found
No.TrialIDDate_
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PhaseCountries
1EUCTR2017-004930-27-AT05/02/201920/12/2018Sirolimus as treatment for patients with sarcoidosis.Systems medicine analysis of sarcoidosis by targeting mTOR in apilot study of sirolimus as treatment in patients with sarcoidosis Sarcoidosis with cutaneous affections;Therapeutic area: Diseases [C] - Immune System Diseases [C20]Trade Name: Rapamune
INN or Proposed INN: SIROLIMUS
Other descriptive name: Rapamune
Product Name: Sirolimus Ointment
INN or Proposed INN: SIROLIMUS
Medical University of ViennaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2Austria

85. 特発性間質性肺炎 [臨床試験数:514,薬物数:377(DrugBank:108),標的遺伝子数:97,標的パスウェイ数:204
Searched query = "Idiopathic interstitial pneumonia", "IIPs", "Idiopathic pulmonary fibrosis", "IPF", "Usual interstitial pneumonia", "UIP", "Non-specific interstitial pneumonia", "NSIP", "Acute interstitial pneumonia", "AIP", "Diffuse alveolar damage", "DAD", "COP", "Organizing pneumonia", "OP", "Desquamative interstitial pneumonia", "DIP", "Respiratory bronchiolitis - associated interstitial lung disease", "RB-ILD", "Lymphocytic interstitial pneumonia", "LIP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 514 trial found
No.TrialIDDate_
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PhaseCountries
1NCT01462006October 201526/10/2011Double-blind Placebo-controlled Pilot Study of Sirolimus in Idiopathic Pulmonary Fibrosis (IPF)Double-blind Placebo-controlled Pilot Study of Sirolimus in IPFIdiopathic Pulmonary Fibrosis;Diffuse Parenchymal Lung Disease;Interstitial Lung DiseaseDrug: sirolimus;Other: PlaceboUniversity of VirginiaNational Heart, Lung, and Blood Institute (NHLBI)Unknown status21 Years85 YearsAll32N/AUnited States

89. リンパ脈管筋腫症 [臨床試験数:38,薬物数:42(DrugBank:19),標的遺伝子数:26,標的パスウェイ数:134
Searched query = "Lymphangioleiomyomatosis", "LAM"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
13 / 38 trials found
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Date_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT03253913March 31, 201810/8/2017Resveratrol and Sirolimus in Lymphangioleiomyomatosis TrialResveratrol and Sirolimus in Lymphangioleiomyomatosis Trial (RESULT)LymphangioleiomyomatosisDrug: Sirolimus;Drug: ResveratrolUniversity of CincinnatiNational Heart, Lung, and Blood Institute (NHLBI)Unknown status18 YearsN/AFemale25Phase 2United States
2NCT03150914January 1, 201810/5/2017Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease TrialMulticenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease TrialLAM;LymphangioleiomyomatosisDrug: SirolimusUniversity of CincinnatiNational Heart, Lung, and Blood Institute (NHLBI);National Center for Advancing Translational Science (NCATS);The LAM FoundationRecruiting18 YearsN/AFemale60Phase 3United States
3NCT02432560March 20154/3/2015Safety and Durability of Sirolimus for Treatment of LAMMulticenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS)LymphangioleiomyomatosisDrug: Sirolimus;Drug: EverolimusUniversity of CincinnatiRare Diseases Clinical Research Network;National Institutes of Health (NIH);National Heart, Lung, and Blood Institute (NHLBI);The LAM FoundationActive, not recruiting18 YearsN/AFemale600United States
4JPRN-UMIN0000166772015/01/0102/03/2015Efficacy and safety of low dose sirolimus in lymphangioleiomyomatosis lymphangioleiomyomatosissirolimus 1mg/day / sirolimus 2mg/dayNational Hospital Organization Kinki-chuo Chest Medical CenterNULLRecruitingNot applicableNot applicableMale and Female20Not selectedJapan
5NCT02061397March 201423/1/2014Safety of Simvastatin in LAM and TSCThe Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)Lymphangioleiomyomatosis;Tuberous Sclerosis ComplexDrug: Simvastatin;Drug: Sirolimus Oral Product;Drug: Everolimus Oral ProductUniversity of PennsylvaniaThe LAM FoundationCompleted18 YearsN/AFemale10Phase 1/Phase 2United States
No.TrialIDDate_
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6JPRN-JMA-IIA0009605/09/201217/08/2012multicenter lymphangioleiomyomatosis sirolimus trial for safetymulticenter lymphangioleiomyomatosis sirolimus trial for safety lymphangioleiomyomatosisIntervention type:DRUG. Intervention1:Srolimus, Dose form:TABLET, Route of administration:ORAL, intended dose regimen:LAM patients will be medicated with 2mg of sirolimus every day for 2 years and modified dosage between 1mg and 3mg QD depend on the plasma concentration monitoring..Koh Nakata, MD, PhDYoshikazu InoueKuniaki SeyamaRyushi TazawaToshinori TakadaCompleted>=18 YEARSNo LimitFemale65Japan
7NCT01687179September 20122/8/2012Safety Study of Sirolimus and Hydroxychloroquine in Women With LymphangioleiomyomatosisTargeting Autophagy for the Treatment of TSC and LAM: a Phase I Trial of Hydroxychloroquine and SirolimusLymphangioleiomyomatosisDrug: Sirolimus and Hydroxychloroquine 200 mg;Drug: Sirolimus and Hydroxychloroquine 400 mgBrigham and Women's HospitalNational Heart, Lung, and Blood Institute (NHLBI)Completed18 Years85 YearsFemale14Phase 1United States
8JPRN-UMIN0000073872012/03/3101/04/2012Clinical trials for long-term administration of sirolimus for lymphangioleiomyomatosisClinical trials for long-term administration of sirolimus for lymphangioleiomyomatosis - Long-term administration of sirolimus for lymphangioleiomyomatosis lymphangioleiomyomatosissirolimusKobe University Graduate School of MedicineNULLComplete: follow-up completeNot applicableNot applicableFemale1Not appl1cableJapan
9JPRN-JMA-IIA0003701/10/201115/03/2010MLLTS trialMulticenter Lymphangioleiomyomatosis Long Term Sirolimus Trial lymphangioleiomyomatosisIntervention type:DRUG. Intervention1:sirolimus, Dose form:TABLET, Route of administration:ORAL, intended dose regimen:LAM patients will be medicated with 2mg of sirolimus every day for 2 years..Koh Nakata, MD, Ph.DRyushi TazawaOther>=18 YEARSNo LimitFemale50Japan
10JPRN-JMA-IIA0001108/05/200829/06/2007Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus Trial (The MILES Trial)Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus Trial (The MILES Trial) Lymphangioleiomyomatosis (LAM)Intervention type:DRUG. Intervention1:Sirolimus, Dose form:TABLET, Route of administration:ORAL, intended dose regimen:12 months on, 12 months off. Control intervention1:Placebo, Dose form:TABLET, Route of administration:ORAL, Intended dose regimen:12 months on, 12 months off.Frank McCormack, M.D., University of Cincinnati Medical Center Director, Division of Pulmonary and Critical Care Medicinea.Alan F. Barker, M.D. - Oregon Health & Sciences University b.Kevin Brown, M.D., - National Jewish Medical & Research Center c.Edwin K. Silverman, M.D., Ph.D. - Harvard/Brigham & Women's Hospital d.James M. Stocks, M.D. - University of Texas Health Centere.James K. Stoller, M.D. - Cleveland Clinic Foundation f.Charlie Strange, M.D. - Medical University of South Carolina g.Bruce Trapnell, M.D.-Cincinnati Children's Medical Centerh.Mark Brantly, M.D.-University of Florida, Gainesvillei.Yosdhikazu Inoue, M.D., National Hospital Organization (NHO) Kinki-Chuo Chest Medical Centerj.Koh Nakata, M.D., Ph.D., Bioscience Medical Research Center, Niigata University Medical and Dental Hospitalk.Joel Moss, M.D., Ph.D-National Institutes of HealthCompleted>=18 YEARSFemale120Japan, United States, Canada
No.TrialIDDate_
enrollment
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11NCT00414648December 200620/12/2006Efficacy and Safety of Sirolimus for Treating Lymphangioleiomyomatosis (LAM)Lymphangioleiomyomatosis Efficacy and Safety TrialLymphangioleiomyomatosisDrug: Sirolimus;Drug: Placebo sirolimusOffice of Rare Diseases (ORD)FDA Office of Orphan Products DevelopmentActive, not recruiting18 YearsN/AFemale120Phase 3United States;Canada;Japan
12NCT00490789October 200521/6/2007Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAMA Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: sirolimusCardiff UniversityUniversity of Nottingham;St Georges Hospital Medical School;Royal Sussex County Hospital;The Tuberous Sclerosis Association;Wyeth is now a wholly owned subsidiary of PfizerActive, not recruiting18 Years65 YearsBoth14Phase 2United Kingdom
13NCT00457808December 20026/4/2007Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAMRapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: Rapamycin, sirolimusChildren's Hospital Medical Center, CincinnatiThe LAM Foundation;Tuberous Sclerosis AllianceCompleted18 Years65 YearsBoth25Phase 2United States

98. 好酸球性消化管疾患 [臨床試験数:128,薬物数:147(DrugBank:40),標的遺伝子数:34,標的パスウェイ数:132
Searched query = "Eosinophilic gastrointestinal disease", "Eosinophilic gastroenteritis", "Eosinophilic esophagitis", "Eosinophilic colitis", "Eosinophilic gastro-intestinal disorder", "EGID", "Neonatal food-protein induced enterocolitis", "N-FPIES"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 128 trial found
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1NCT01814059March 7, 201315/3/2013Sirolimus for Eosinophil-Associated Gastrointestinal DisordersA Phase 1, Open-Label Study of Sirolimus in Eosinophil-Associated Gastrointestinal DisordersEosinophilic Gastroenteritis;Eosinophilic EsophagitisDrug: sirolimusNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated18 Years65 YearsAll4Phase 1United States

127. 前頭側頭葉変性症 [臨床試験数:74,薬物数:83(DrugBank:28),標的遺伝子数:29,標的パスウェイ数:99
Searched query = "Frontotemporal lobar degeneration", "Frontotemporal dementia", "Semantic dementia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 74 trial found
No.TrialIDDate_
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1NCT04408625July 30, 202021/5/2020Phase 1/2 Clinical Trial of PR006 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of PR006A in Patients With Fronto-Temporal Dementia With Progranulin Mutations (FTD-GRN)Frontotemporal DementiaBiological: PR006;Drug: Methylprednisolone;Drug: Sirolimus;Drug: PrednisonePrevail TherapeuticsNULLRecruiting30 Years80 YearsAll15Phase 1/Phase 2United States

137. 限局性皮質異形成 [臨床試験数:8,薬物数:4(DrugBank:2),標的遺伝子数:1,標的パスウェイ数:50
Searched query = "Focal cortical dysplasia", "FCD"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
5 / 8 trials found
No.TrialIDDate_
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1JPRN-jRCTs03119015718/12/201911/12/2019Research of sirolimus administration for FCD II typeClinical study on the safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type II - FCDS-02 Epileptic patients with focal cortical dysplasia type 2
Epilepsy, FCD type 2
Sirolimus 0.5-4mg (0.5-4 tablets of the study drug) is administered orally once a day in the morning.Kato MitsuhiroNULLRecruiting>= 2age oldNot applicableBoth15Japan
2JPRN-jRCTs04119005909/08/201905/08/2019Study about safety of sirolimus: Shizuoka 2019-1Clinical study of sirolimus about safety for Japanese patients: Shizuoka 2019-1 - Sirolimus Shizuoka 2019-1 Epileptic patients with focal cortical dysplasia type 2
Epilepsy, FCD type 2
sirolimusTakahashi YukitoshiKato MitsuhiroRecruiting>= 6age oldNot applicableBoth5Japan
3JPRN-UMIN0000335042018/10/0125/07/2018An uncontrolled open-label study on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type-IIAn uncontrolled open-label study on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type-II - An investigator-initiated clinical trial on the efficacy and safety of sirolimus for epileptic seizures associated with FCD type-II focal cortical dysplasia (type-II)The investigational drug (sirolimus) is orally administered once a day with the following doses:
<Dose adjustment period>
Initial amount: 1 mg/day for body weight of less than 40 kg, and 2 mg/day for 40 kg or more.
Dose adjustment: to increase as necessary to adjust the trough concentration of sirolimus to the range of 5-15 ng/ml, and then, to shift to the maintenance therapy period.
<Maintenance therapy period>
To adopt dosage regimen and dose of sirolimus at trough concentration of 5-15 ng/ml.
Hokkaido University HospitalNishi-Niigata Chuo National HospitalNational Center Hospital, NCNPShizuoka Institute of Epilepsy and Neurological DisordersOkayama University HospitalNULLComplete: follow-up continuing6years-old65years-oldMale and Female15Phase 2Japan
4JPRN-UMIN0000309622018/02/2824/01/2018Clinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type IIClinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type II - Study of sirolimus administration for FCDII type Patients with epilepsy with focal cortical dysplasia type 2Prescribe the study medicine at the start of the gradual increase period (Visit 20 weeks) after the end of the preview phase. Administration of the study drug starts from the morning the following day of Visit 2. Take it once a day in the morning.
For test drugs (1 mg tablets), weighing less than 20 kg of sirolimus 0.5 mg / day, body weight of less than 20 kg to less than 40 kg is 1 mg / day, body weight of 40 mg or more is orally administered 2 mg / day once daily in the morning. The upper limit of the daily dose is 4 mg.
The dose was not changed in the first 4 weeks, the blood concentration of sirolimus was measured at the 4th week (visit 4), the next visit (4 weeks / visit 4-2 to 4-10) based on the examination result, From 0.5 mg / day for less than 20 kg to 1 mg / day for 20 kg or more, increase the dose and repeat the measurement of sirolimus blood concentration every 4 weeks until the trough concentration reaches 5 to 15 ng / mL, and the trough concentration. From the time when the target concentration is reached, the maintenance therapy period is entered.
As a result of the blood concentration at Visit 4, when the trough blood concentration is within the range of 5 - 15 ng / mL, it will shift to the maintenance therapy period from Visit 5 (Week 8).
Visit 5 (after week 8) is in the dose control phase and is fixed at the dose of sirolimus that has reached blood concentration of 5 - 15 ng / mL.
The maintenance therapy period will be visited 4 weeks, 8 weeks, 12 weeks after the start of maintenance therapy and will continue for 12 weeks.
The researcher who completed the procedure up to the end of the 12th week of maintenance therapy will complete the study
Hokkaido University HospitalNULLComplete: follow-up continuing2years-old65years-oldMale and Female2Not selectedJapan
5JPRN-UMIN0000307972017/12/0113/01/2018Uncontrolled open label study of sirolimus about efficacy for epileptic seizures and safety in patients with focal cortical dysplasia type 2Uncontrolled open label study of sirolimus about efficacy for epileptic seizures and safety in patients with focal cortical dysplasia type 2 - Clinical research-sirolimus FCD Shizuoka2017-1 focal cortical dysplasia type 2Period: 8 weeks to 2 years
Initial dose:
1mg/day for boy weight from 20 to 40Kg
2mg/day for body weight above 40Kg
Increasing dose: 1mg/day until the level of 5-15ng/ml (sirolimus)
National Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders, NHONULLComplete: follow-up continuing6years-old65years-oldMale and Female1Phase 2Japan

157. スタージ・ウェーバー症候群 [臨床試験数:8,薬物数:11(DrugBank:4),標的遺伝子数:5,標的パスウェイ数:62
Searched query = "Sturge-Weber syndrome", "Síndrome de Sturge-Weber"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 8 trial found
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1NCT03047980January 20172/2/2017Trial of Sirolimus for Cognitive Impairment in Sturge-Weber SyndromeTrial of Sirolimus for Cognitive Impairment in Sturge-Weber SyndromeSturge-Weber SyndromeDrug: SirolimusAnne Comi, MDChildren's Hospital Medical Center, Cincinnati;Pfizer;National Institutes of Health (NIH);Faneca 66 Foundation;National Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting3 Years31 YearsAll10Phase 2/Phase 3United States

158. 結節性硬化症 [臨床試験数:100,薬物数:62(DrugBank:16),標的遺伝子数:35,標的パスウェイ数:115
Searched query = "Tuberous sclerosis", "Tuberous sclerosis complex"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
14 / 100 trials found
No.TrialIDDate_
enrollment
Date_
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Public_titleScientific_titleConditionInterventionPrimary_
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1NCT04595513September 8, 20206/10/2020Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC InfantsStopping TSC Onset and Progression 2: Epilepsy Prevention in TSC InfantsTuberous Sclerosis Complex;EpilepsyDrug: TAVT-18 (sirolimus);Drug: PlaceboChildren's Hospital Medical Center, CincinnatiNULLRecruitingN/A6 MonthsAll65Phase 1/Phase 2United States
2ChiCTR20000319842020-05-012020-04-17Sirolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex and EpilepsySirolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex and Epilepsy Tuberous sclerosis complex; Epilepsyexperimental group:Antiepileptic drug + sirolimus;control group:antiepileptic drugs;West China Hospital, Sichuan UniversityNULLPendingBothexperimental group:31;control group:31;N/AChina
3NCT03363763April 12, 20171/12/2017Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis ComplexPhase 2 Multi Center Prospective Rand. Double Blind Placebo Cont. Parallel Design Study to Evaluate Safety & Efficacy of Topical Sirolimus for Cutaneous Angiofibromas in Subjects W/ Tuberous Sclerosis Complex Followed by Opt. Open LabelAngiofibroma of Face;Tuberous SclerosisDrug: Sirolimus 0.2%;Drug: Sirolimus 0.4%;Drug: Placebo ointmentAucta Pharmaceuticals, IncNULLRecruiting2 Years18 YearsAll45Phase 2United States;China
4NCT02635789December 201515/12/2015Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)A Double-blind, Randomized, Placebo-controlled Phase III Trial to Investigate the Efficacy and Safety of NPC-12G Gel (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis ComplexTuberous Sclerosis;Angiofibroma;Hypomelanotic Macule;PlaqueDrug: NPC-12G gel;Drug: Placebo gelNobelpharmaNULLCompleted3 YearsN/AAll62Phase 3Japan
5NCT02634931December 201516/12/2015Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis ComplexA Long-term, Single-arm, Open-label Trial of NPC-12G (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis ComplexTuberous Sclerosis;Angiofibroma;Hypomelanotic Macule;PlaqueDrug: NPC-12G gelNobelpharmaNULLCompleted3 YearsN/AAll94Phase 3Japan
No.TrialIDDate_
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6NCT02061397March 201423/1/2014Safety of Simvastatin in LAM and TSCThe Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)Lymphangioleiomyomatosis;Tuberous Sclerosis ComplexDrug: Simvastatin;Drug: Sirolimus Oral Product;Drug: Everolimus Oral ProductUniversity of PennsylvaniaThe LAM FoundationCompleted18 YearsN/AFemale10Phase 1/Phase 2United States
7JPRN-UMIN0000124202013/12/1027/11/2013Randomized, double-blind, placebo-controlled, clinical trial with OSD-001 for skin lesions due to tuberous sclerosis complex. Tuberous sclerosis complex0.05% Sirolimus gel(adult)
0.1% Sirolimus gel(adult)
0.2% Sirolimus gel(adult)
0.05% Sirolimus gel(children)
0.1% Sirolimus gel(children)
0.2% Sirolimus gel(children)
Department of DermatologyGraduate School of Medicine, Osaka UniversityNULLComplete: follow-up complete3years-old65years-oldMale and Female36Phase 1,2Japan
8NCT01929642July 20137/8/2013Rapalogues for Autism Phenotype in TSC: A Feasibility StudyRapalogues for Autism Phenotype in TSC: A Feasibility StudyTuberous Sclerosis Complex;Self-injury;AutismDrug: Sirolimus;Drug: EverolimusHugo W. Moser Research Institute at Kennedy Krieger, Inc.NULLCompleted2 Years30 YearsAll3Phase 2United States
9NCT01031901December 200910/12/2009Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;NeurofibromaDrug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycinThe University of Texas Health Science Center, HoustonSociety for Pediatric DermatologyCompleted13 YearsN/ABoth52Phase 1United States
10NCT01217125October 20086/10/2010Rapamycin In Angiomyolipomas In Patients With Tuberous SclerosisCLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSISAngiomyolipomaDrug: SirolimusFundacio PuigvertMinistry of Health, SpainCompleted10 YearsN/ABoth18Phase 4NULL
No.TrialIDDate_
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11EUCTR2007-005978-30-ES22/01/200803/12/2007Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous SclerosisEnsayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous Sclerosis Angiomiolipomas en pacientes con esclerosis tuberosa (angiomyolipoma of tuberous sclerosis patients)
MedDRA version: 9.1Level: LLTClassification code 10045138Term: Tuberous sclerosis
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus
FUNDACIÓ PUIGVERTNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Spain
12NCT00490789October 200521/6/2007Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAMA Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: sirolimusCardiff UniversityUniversity of Nottingham;St Georges Hospital Medical School;Royal Sussex County Hospital;The Tuberous Sclerosis Association;Wyeth is now a wholly owned subsidiary of PfizerActive, not recruiting18 Years65 YearsBoth14Phase 2United Kingdom
13NCT00457808December 20026/4/2007Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAMRapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: Rapamycin, sirolimusChildren's Hospital Medical Center, CincinnatiThe LAM Foundation;Tuberous Sclerosis AllianceCompleted18 Years65 YearsBoth25Phase 2United States
14EUCTR2019-000752-34-CZ02/06/2020Study to evaluate if the study medication, Rapamycin, topical cream, is effective and safe at treating facial angiofibroma in patients 6 years of age and over.A Phase 2/3, multi-center, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison of the efficacy and safety of a topical rapamycin cream for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and over - Dose-ranging efficacy and safety study of topical rapamycin cream Facial Angiofibromas Associated with Tuberous Sclerosis Complex
MedDRA version: 20.0Level: PTClassification code 10002429Term: AngiofibromaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0Level: PTClassification code 10073678Term: Juvenile angiofibromaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps);Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Product Name: Rapamycin cream, topical, 0.5%, 1.0% w/w
Product Code: Not applicable
INN or Proposed INN: SIROLIMUS
Product Name: Rapamycin cream, topical, 0.5%, 1.0% w/w
Product Code: Not applicable
INN or Proposed INN: SIROLIMUS
Dermatology Specialities Limited Partnership (DSLP)NULLNAFemale: yes
Male: yes
120Phase 2;Phase 3United States;Serbia;Hungary;Czech Republic;Slovakia;Spain;Australia;New Zealand

192. コケイン症候群 [臨床試験数:4,薬物数:7(DrugBank:3),標的遺伝子数:1,標的パスウェイ数:50
Searched query = "Cockayne syndrome", "CS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 4 trials found
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1NCT02960997May 201615/6/2016Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) StudyA Prospective, Double-Blind, Cross-Over, Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2% in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex (EBS)Epidermolysis Bullosa Simplex;Epidermolysis Bullosa Simplex Kobner;Weber-Cockayne SyndromeDrug: Sirolimus, 2%;Drug: VehicleStanford UniversityNULLActive, not recruiting4 YearsN/AAll8Phase 2United States
2NCT03016715May 20169/1/2017Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) StudyA Prospective, Double-Blind, Cross-Over, Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2% in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex (EBS)Epidermolysis Bullosa Simplex;Epidermolysis Bullosa Simplex Kobner;Weber-Cockayne SyndromeDrug: Sirolimus 2%;Drug: VehiclePremier Specialists, AustraliaNULLRecruiting5 YearsN/AAll8Phase 2Australia

222. 一次性ネフローゼ症候群 [臨床試験数:234,薬物数:241(DrugBank:78),標的遺伝子数:59,標的パスウェイ数:185
Searched query = "Primary nephrotic syndrome", "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Membranoproliferative glomerulonephritis", "MPGN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 234 trials found
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size
PhaseCountries
1ChiCTR-INR-170122122017-07-282017-08-01Use of sirolimus in patients with primary idiopathic membranous nephropathy: a prospective randomized control trialUse of sirolimus in patients with primary idiopathic membranous nephropathy: a prospective randomized control trial idiopathic membranous nephropathy1:Cyclosporine;2:cyclosporine combined with sirolimus;Renal Division, Peking University First HospitalNULLRecruiting1870Both1:35;2:35;China
2NCT00050713December 17, 200217/12/2002Sirolimus Therapy for Idiopathic and Lupus Membranous NephropathySirolimus Therapy in Idiopathic and Lupus Membranous NephropathyMembranous Glomerulonephritis;Lupus Membranous NepropathyDrug: SirolimusNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NULLCompleted13 YearsN/AAll12Phase 2United States
3NCT00040508June 200226/6/2002Sirolimus for Focal Segmental GlomerulosclerosisSirolimus for Focal Segmental GlomerulosclerosisFocal GlomerulosclerosisDrug: SirolimusNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NULLCompletedN/AN/ABoth30Phase 2United States

277. リンパ管腫症/ゴーハム病 [臨床試験数:7,薬物数:2(DrugBank:2),標的遺伝子数:1,標的パスウェイ数:50
Searched query = "Lymphangiomatosis", "Generalized lymphatic anomaly", "Gorham disease", "Gorham-Stout disease", "Diffuse lymphangiomatosis", "Mass osteolysis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
6 / 7 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-UMIN0000389732020/01/0625/12/2019A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomaliesA multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies - Sirolimus for Intractable Vascular Anomalies(SIVA) Kaposiform hemangioendothelioma or Tufted angiomaLymphangioma (cystic lymphatic malformation), lymphangiomatosis (generalized lymphatic anomaly) or Gorham-Stout diseaseVenous malformation or blue rubber bleb nevus syndromeComplex-combined vascular malformations or Klippel-Trenanay-Weber syndromeAn initial dose of sirolimus is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL.Gifu University HospitalNULLPending1months-oldNot applicableMale and Female10Phase 3Japan
2JPRN-jRCTs03118029016/11/201715/03/2019A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomaliesA multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies - Sirolimus for intractable vascular anomalies Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lympha
Vascular disorders
Body surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Ozeki MichioNULLRecruitingNot applicableNot applicableBoth100Japan
3JPRN-UMIN0000305222017/11/1422/12/2017A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformationsBody surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Gifu UniversityNULLRecruitingNot applicableNot applicableMale and Female50Not selectedJapan
4JPRN-UMIN0000298432017/11/0606/11/2017Sirolimus for the treatment of refractory lymphangiomatosis/ Gorham Stout disease Refractory lymphangiomatosis, Gorham-Stout diseaseOral sirolimusKyushu University HospitalNULLRecruiting8years-old30years-oldMale and Female2Not selectedJapan
5JPRN-UMIN0000165802015/02/0119/02/2015Sirolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomaliesSirolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomalies - Sirolimus for vascular anomalies Lymphangiomatosis, Gorham-Stout disease and vascular anomaliesOral sirolimusGifu universityNULLComplete: follow-up completeNot applicable30years-oldMale and Female30Phase 1,2Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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size
PhaseCountries
6NCT00975819October 200910/9/2009Safety and Efficacy Study of Sirolimus in Complicated Vascular AnomaliesA Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular AnomaliesKaposiform Hemangioendotheliomas;Tufted Angioma;Capillary Venous Lymphatic Malformation;Venous Lymphatic Malformation;Microcystic Lymphatic Malformation;Mucocutaneous Lymphangiomatosis and Thrombocytopenia;Capillary Lymphatic Arterial Venous Malformations;PTEN Overgrowth Syndrome With Vascular Anomaly;Lymphangiectasia SyndromesDrug: sirolimusChildren's Hospital Medical Center, CincinnatiNULLActive, not recruitingN/A31 YearsBoth60Phase 2United States

278. 巨大リンパ管奇形(頚部顔面病変) [臨床試験数:19,薬物数:23(DrugBank:7),標的遺伝子数:5,標的パスウェイ数:62
Searched query = "Huge lymphatic malformation with cervicofacial lesion", "Huge lymphatic malformation", "Lymphatic malformation"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
8 / 19 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04128722February 14, 202024/9/2019TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUNTOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUNLingual Microcystic Lymphatic MalformationsDrug: Sirolimus Oral Liquid Product 1mg/mLUniversity Hospital, ToursNULLRecruiting5 YearsN/AAll12Phase 2France
2JPRN-UMIN0000389732020/01/0625/12/2019A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomaliesA multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies - Sirolimus for Intractable Vascular Anomalies(SIVA) Kaposiform hemangioendothelioma or Tufted angiomaLymphangioma (cystic lymphatic malformation), lymphangiomatosis (generalized lymphatic anomaly) or Gorham-Stout diseaseVenous malformation or blue rubber bleb nevus syndromeComplex-combined vascular malformations or Klippel-Trenanay-Weber syndromeAn initial dose of sirolimus is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL.Gifu University HospitalNULLPending1months-oldNot applicableMale and Female10Phase 3Japan
3EUCTR2019-001530-33-FR29/06/201901/04/2019TOPical sirolimus in linGUal microkystic lymphatic malformation-TOPGUNTOPical sirolimus in linGUal microkystic lymphatic malformation-TOPGUN - TOPGUN Lingual microcystic lymphatic malformations (LMLM) in children and adults
MedDRA version: 20.0 Level: LLT Classification code 10003229 Term: Arteriovenous malformations System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
CHRU TOURSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
12 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance
4NCT03972592June 5, 201924/5/2019Topical Sirolimus in Cutaneous Lymphatic Malformations0.1% Topical Sirolimus in the Treatment of Cutaneous Microcystic Lymphatic Malformations in Children and Adults: Phase II, Split-body Randomized, Double-blind, Vehicle-controlled Clinical TrialVascular Malformations;Lymphatic MalformationDrug: Topical 0.1% Sirolimus;Drug: Topical VehicleUniversity Hospital, ToursUniversity Hospital, AngersRecruiting6 YearsN/AAll55Phase 2France
5EUCTR2018-001359-11-FR22/02/201923/07/20180.1% topical sirolimus in the treatment of cutaneous microcystic lymphatic malformations in children and adults: phase II, split-body randomized, double-blind, vehicle-controlled clinical trial0.1% topical sirolimus in the treatment of cutaneous microcystic lymphatic malformations in children and adults: phase II, split-body randomized, double-blind, vehicle-controlled clinical trial - TOPICAL Cutaneous microcystic lymphatic malformations (CMLM) in children and adults
MedDRA version: 20.0 Level: LLT Classification code 10003229 Term: Arteriovenous malformations System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
CHRU TOURSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
55 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6JPRN-jRCTs03118029016/11/201715/03/2019A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomaliesA multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies - Sirolimus for intractable vascular anomalies Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lympha
Vascular disorders
Body surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Ozeki MichioNULLRecruitingNot applicableNot applicableBoth100Japan
7JPRN-UMIN0000305222017/11/1422/12/2017A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformationsBody surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Gifu UniversityNULLRecruitingNot applicableNot applicableMale and Female50Not selectedJapan
8NCT00975819October 200910/9/2009Safety and Efficacy Study of Sirolimus in Complicated Vascular AnomaliesA Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular AnomaliesKaposiform Hemangioendotheliomas;Tufted Angioma;Capillary Venous Lymphatic Malformation;Venous Lymphatic Malformation;Microcystic Lymphatic Malformation;Mucocutaneous Lymphangiomatosis and Thrombocytopenia;Capillary Lymphatic Arterial Venous Malformations;PTEN Overgrowth Syndrome With Vascular Anomaly;Lymphangiectasia SyndromesDrug: sirolimusChildren's Hospital Medical Center, CincinnatiNULLActive, not recruitingN/A31 YearsBoth60Phase 2United States

279. 巨大静脈奇形(頚部口腔咽頭びまん性病変) [臨床試験数:12,薬物数:23(DrugBank:9),標的遺伝子数:3,標的パスウェイ数:103
Searched query = "Huge venous malformation with cervical, oral and pharyngeal diffuse lesion", "Huge venous malformation", "Venous malformation"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 12 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03767660July 31, 201824/11/2018Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous MalformationEfficacy of Rapamycin (Sirolimus) in the Treatment of Blue Rubber Bleb Nevus Syndrome, Hereditary or Sporadic Venous MalformationBlue Rubber Bleb Nevus Syndrome;Venous MalformationDrug: RapamycinPeking Union Medical College HospitalAir Force General Hospital of the PLA;Chinese Academy of Medical SciencesRecruitingN/AN/AAll20Phase 4China
2NCT00975819October 200910/9/2009Safety and Efficacy Study of Sirolimus in Complicated Vascular AnomaliesA Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular AnomaliesKaposiform Hemangioendotheliomas;Tufted Angioma;Capillary Venous Lymphatic Malformation;Venous Lymphatic Malformation;Microcystic Lymphatic Malformation;Mucocutaneous Lymphangiomatosis and Thrombocytopenia;Capillary Lymphatic Arterial Venous Malformations;PTEN Overgrowth Syndrome With Vascular Anomaly;Lymphangiectasia SyndromesDrug: sirolimusChildren's Hospital Medical Center, CincinnatiNULLActive, not recruitingN/A31 YearsBoth60Phase 2United States

280. 巨大動静脈奇形(頚部顔面又は四肢病変) [臨床試験数:22,薬物数:26(DrugBank:13),標的遺伝子数:11,標的パスウェイ数:147
Searched query = "Huge arteriovenous malformation with cervicofacial or limb lesion", "Huge arteriovenous malformation", "Arteriovenous malformation"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
4 / 22 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-001530-33-FR29/06/201901/04/2019TOPical sirolimus in linGUal microkystic lymphatic malformation-TOPGUNTOPical sirolimus in linGUal microkystic lymphatic malformation-TOPGUN - TOPGUN Lingual microcystic lymphatic malformations (LMLM) in children and adults
MedDRA version: 20.0 Level: LLT Classification code 10003229 Term: Arteriovenous malformations System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
CHRU TOURSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
12 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance
2EUCTR2018-001359-11-FR22/02/201923/07/20180.1% topical sirolimus in the treatment of cutaneous microcystic lymphatic malformations in children and adults: phase II, split-body randomized, double-blind, vehicle-controlled clinical trial0.1% topical sirolimus in the treatment of cutaneous microcystic lymphatic malformations in children and adults: phase II, split-body randomized, double-blind, vehicle-controlled clinical trial - TOPICAL Cutaneous microcystic lymphatic malformations (CMLM) in children and adults
MedDRA version: 20.0 Level: LLT Classification code 10003229 Term: Arteriovenous malformations System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
CHRU TOURSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
55 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance
3JPRN-UMIN0000305222017/11/1422/12/2017A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformationsBody surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Gifu UniversityNULLRecruitingNot applicableNot applicableMale and Female50Not selectedJapan
4NCT02042326September 12, 201420/1/2014Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous MalformationsProspective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous MalformationsArteriovenous MalformationsDrug: SirolimusCentre Hospitalier Universitaire, AmiensNULLRecruiting2 YearsN/AAll50Phase 2Belgium;France

281. クリッペル・トレノネー・ウェーバー症候群 [臨床試験数:1,薬物数:1(DrugBank:1),標的遺伝子数:1,標的パスウェイ数:50
Searched query = "Klippel-Trenaunay-Weber syndrome", "Klippel-Trenauney-Weber syndrome", "Klippel-Trenaunay syndrome", "Klippel-Trenauney syndrome", "KTS", "Parkes Weber syndrome", "PWS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-UMIN0000305222017/11/1422/12/2017A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformationsBody surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
Gifu UniversityNULLRecruitingNot applicableNot applicableMale and Female50Not selectedJapan

283. 後天性赤芽球癆 [臨床試験数:17,薬物数:36(DrugBank:19),標的遺伝子数:16,標的パスウェイ数:91
Searched query = "Acquired pure red cell aplasia", "Pure red cell aplasia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 17 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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Target_
size
PhaseCountries
1NCT04470804August 1, 20209/7/2020Sirolimus Treatment for Newly Diagnosed Primary Acquired PRCASirolimus Treatment for Newly Diagnosed Primary Acquired Pure Red Cell Aplasia: a Single Center Prospective StudyPure Red Cell Aplasia, AcquiredDrug: Sirolimus;Drug: Cyclosporine ABing HanNULLNot yet recruiting18 Years70 YearsAll40Phase 4China
2NCT03364764January 201821/11/2017Sirolimus Treatment for Refractory PRCASirolimus Treatment for Refractory Pure Red Cell Aplasia, a Prospective StudyPure Red Cell AplasiaDrug: SirolimusBing HanNULLRecruiting18 Years80 YearsAll30Phase 4China
3NCT03214354July 5, 20175/7/2017Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling DonorA Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease Who Have a Matched Related Major ABO-Incompatible Donor (Sickle-MAID)Sickle Cell Disease;Stem Cell Transplant Complications;Red Blood Cell Disorder;Pure Red Cell AplasiaDrug: Alemtuzumab;Radiation: Total Body Irradiation;Drug: SirolimusUniversity of CalgaryNULLRecruiting1 Year19 YearsAll12Phase 2Canada

285. ファンコニ貧血 [臨床試験数:56,薬物数:111(DrugBank:31),標的遺伝子数:30,標的パスウェイ数:151
Searched query = "Fanconi anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 56 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
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agemin
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agemax
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size
PhaseCountries
1NCT00352976May 18, 200614/7/2006TBI Dose De-escalation for Fanconi AnemiaTotal Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell TransplantationFanconi AnemiaDrug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Bone Marrow Transplantation;Drug: Mycophenolate Mofetil;Drug: SirolimusMasonic Cancer Center, University of MinnesotaNULLCompletedN/AN/AAll88Phase 2/Phase 3United States

331. 特発性多中心性キャッスルマン病 [臨床試験数:30,薬物数:44(DrugBank:23),標的遺伝子数:30,標的パスウェイ数:148
Searched query = "Idiopathic multicentric castleman disease", "Castleman disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 30 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-jRCT207119002925/05/202009/10/2019Randomized, double-blind, placebo-controlled, parallel-group trial of sirolimus for tocilizumab-resistant idiopathic multicentric Castleman disease: Study protocol for clinical trial (SPIRIT Compliant)Randomized, double-blind, placebo-controlled, parallel-group trial of sirolimus for tocilizumab-resistant idiopathic multicentric Castleman disease: Study protocol for clinical trial (SPIRIT Compliant) idiopathic multicentric Castleman's disease
Castleman's disease
Treatment: Sirolimus 2 mg po once/day.
Control: Placebo given orally once daily
Kawakami AtsushiNULLRecruiting>= 18age oldNot applicableBoth20Japan
2NCT03933904September 25, 201929/4/2019Sirolimus in Previously Treated Idiopathic Multicentric Castleman DiseaseA Phase II, Single-arm Open-label Multi-center Study of Sirolimus in Previously Treated Idiopathic Multicentric Castleman DiseaseCastleman Disease;Castleman's Disease, MulticentricDrug: SirolimusUniversity of PennsylvaniaNULLRecruiting18 Years80 YearsAll24Phase 2United States
3NCT00092222October 28, 200421/9/2004Virotherapy and Natural History Study of KHSV-Associated Multricentric Castleman s Disease With Correlates of Disease ActivityTargeted Oncolytic Virotherapy and Natural History Study of KSHV-Associated Multicentric Castleman's Disease With Laboratory and Clinical Correlates of Disease ActivityLymphoproliferative Disorder;HHV-8;Malignancy;HIVDrug: Etoposide;Drug: Interferon-alpha;Drug: Rituximab;Drug: Zidovudine;Drug: Liposomal Doxorubicin;Drug: Bortezomib;Drug: Valganciclovir;Drug: Doxorubicin;Drug: Vincristine;Drug: Cyclophosphamide;Drug: Filgrastim (G-CSF);Drug: Prednisone;Drug: Sirolimus;Other: Observation OnlyNational Cancer Institute (NCI)NULLRecruiting12 YearsN/AAll72Phase 2United States