Sirolimus (DrugBank: Sirolimus)
40 diseases| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 2 | 筋萎縮性側索硬化症 | 2 |
| 6 | パーキンソン病 | 1 |
| 13 | 多発性硬化症／視神経脊髄炎 | 1 |
| 15 | 封入体筋炎 | 0 |
| 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 |
| 66 | IgA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 |
| 86 | 肺動脈性肺高血圧症 | 0 |
| 89 | リンパ脈管筋腫症 | 13 |
| 96 | クローン病 | 0 |
| 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 |
| 256 | 筋型糖原病 | 0 |
| 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 |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03359538 (ClinicalTrials.gov) | September 19, 2017 | 8/11/2017 | Rapamycin Treatment for ALS | Rapamycin (Sirolimus) Treatment for Amyotrophic Lateral Sclerosis | Amyotrophic Lateral Sclerosis | Drug: Rapamycin;Drug: Placebo Oral Tablet | Azienda Ospedaliero-Universitaria di Modena | University of Modena and Reggio Emilia;Azienda Ospedaliero Universitaria Maggiore della Carita;IRCCS Azienda Ospedaliera Universitaria San Martino - IST Istituto Nazionale per la Ricerca sul Cancro, Genoa, Italy;University of Turin, Italy;Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta;Azienda Ospedaliera Niguarda Cà Granda;Fondazione Salvatore Maugeri;University of Padova | Active, not recruiting | 18 Years | 75 Years | All | 63 | Phase 2 | Italy |
| 2 | EUCTR2016-002399-28-IT (EUCTR) | 14/07/2017 | 23/01/2018 | Rapamycin (Sirolimus) treatment for amyotrophic lateral sclerosis | Rapamycin (Sirolimus) treatment for amyotrophic lateral sclerosis - RAP.ALS | definite or probable ALS MedDRA version: 20.0;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: RAPAMUNE - 1 MG 100 COMPRESSE RIVESTITE IN BLISTER USO ORALE Product Name: Rapamune | AZIENDA OSPEDALIERO-UNIVERSITARIA POLICLINICO DI MODENA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 63 | Phase 2 | Italy |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04127578 (ClinicalTrials.gov) | January 3, 2020 | 14/10/2019 | Phase 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 Mutation | Parkinson Disease | Biological: PR001A;Drug: Methylprednisolone;Drug: Sirolimus;Drug: Prednisone | Prevail Therapeutics | NULL | Recruiting | 35 Years | 80 Years | All | 12 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT00095329 (ClinicalTrials.gov) | May 2003 | 2/11/2004 | Treating Multiple Sclerosis With Sirolimus, an Immune System Suppressor | A Phase I/II, Open-Label Pilot Trial to Evaluate the Safety of Rapamune (Sirolimus) in Patients With Multiple Sclerosis | Multiple Sclerosis (MS) - Relapsing-remitting | Biological: sirolimus | National Institute of Allergy and Infectious Diseases (NIAID) | Autoimmunity Centers of Excellence | Terminated | 18 Years | 58 Years | Both | 14 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03589976 (ClinicalTrials.gov) | September 1, 2018 | 5/7/2018 | A Futility Trial of Sirolimus in Multiple System Atrophy | A Single Center Randomized,Double Blind, Placebo-controlled Futility Trial to Determine if Sirolimus is of Sufficient Promise to Slow the Progression of Multiple System Atrophy | Multiple System Atrophy | Drug: Sirolimus 2 MG;Other: Placebo | NYU Langone Health | National Institute of Neurological Disorders and Stroke (NINDS) | Recruiting | 30 Years | 80 Years | All | 56 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04411654 (ClinicalTrials.gov) | September 2020 | 11/5/2020 | Phase 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 Disease | Gaucher Disease, Type 2 | Biological: PR001;Drug: Methylprednisolone;Drug: Sirolimus;Drug: Prednisone | Prevail Therapeutics | NULL | Recruiting | N/A | 24 Months | All | 15 | Phase 1;Phase 2 | United States |
| 2 | NCT03952637 (ClinicalTrials.gov) | August 19, 2019 | 15/5/2019 | A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type I and Type II GM1 Gangliosidosis | A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis | Lysosomal Diseases;Gangliosidosis;GM1 | Biological: 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 exam | National Human Genome Research Institute (NHGRI) | Axovant Sciences, Inc. | Recruiting | 6 Months | 12 Years | All | 45 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03433183 (ClinicalTrials.gov) | October 2, 2019 | 29/1/2018 | SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors | SARC031: 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 Tumors | Malignant Peripheral Nerve Sheath Tumors;Neurofibromatosis 1 | Drug: Selumetinib;Drug: Sirolimus | Sarcoma Alliance for Research through Collaboration | United States Department of Defense;AstraZeneca | Recruiting | 12 Years | N/A | All | 21 | Phase 2 | United States |
| 2 | JPRN-UMIN000021030 | 2016/03/16 | 22/02/2016 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 | Neurofibromatosis type 1 | 0.2% 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 Hospital | NULL | Complete: follow-up complete | 16years-old | 70years-old | Male and Female | 18 | Phase 2 | Japan | |
| 3 | NCT01031901 (ClinicalTrials.gov) | December 2009 | 10/12/2009 | Topical 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 1 | Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;Neurofibroma | Drug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycin | The University of Texas Health Science Center, Houston | Society for Pediatric Dermatology | Completed | 13 Years | N/A | Both | 52 | Phase 1 | United States |
| 4 | NCT00634270 (ClinicalTrials.gov) | April 2008 | 20/2/2008 | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | Neurofibromatosis Type 1 | Drug: Sirolimus | University of Alabama at Birmingham | Boston 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 Medicine | Completed | 3 Years | 75 Years | All | 58 | Phase 2 | United States |
| 5 | NCT00652990 (ClinicalTrials.gov) | March 2008 | 3/4/2008 | Sirolimus to Treat Plexiform Neurofibromas in Patients With Neurofibromatosis Type I | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas | Neurofibromatosis Type 1;Plexiform Neurofibromas;Paraspinal Plexiform Neurofibromas | Drug: Sirolimus | University of Alabama at Birmingham | National Cancer Institute (NCI) | Active, not recruiting | 3 Years | N/A | Both | 18 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT01313923 (ClinicalTrials.gov) | February 2011 | 10/3/2011 | Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus | Evaluation of Sirolimus for the Treatment of the Autoimmune Blistering Dermatosis Pemphigus | Pemphigus | Drug: Sirolimus (formerly known as Rapamycin) | University of California, Irvine | NULL | Terminated | 18 Years | N/A | All | 3 | Early Phase 1 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02960997 (ClinicalTrials.gov) | May 2016 | 15/6/2016 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | A 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 Syndrome | Drug: Sirolimus, 2%;Drug: Vehicle | Stanford University | NULL | Active, not recruiting | 4 Years | N/A | All | 8 | Phase 2 | United States |
| 2 | NCT03016715 (ClinicalTrials.gov) | May 2016 | 9/1/2017 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | A 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 Syndrome | Drug: Sirolimus 2%;Drug: Vehicle | Premier Specialists, Australia | NULL | Recruiting | 5 Years | N/A | All | 8 | Phase 2 | Australia |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | ChiCTR1900024261 | 2019-07-15 | 2019-07-03 | Efficacy and safety of sirolimus in new-onset rheumatoid arthritis: a prospective, double-blinded, randomized, placebo-controlled, monocentric study in China | Efficacy and safety of sirolimus in new-onset rheumatoid arthritis: a prospective, double-blinded, randomized, placebo-controlled, monocentric study in China | rheumatoid arthritis | A: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 University | NULL | Pending | 18 | 65 | Both | A:30;B:30; | Phase 4 | China |
| 2 | ChiCTR-IPR-17011566 | 2017-07-01 | 2017-06-05 | The efficacy and safety of sirolimus in refractory rheumatoid arthritis: A multi-center randomized controlled trial in China | The efficacy and safety of sirolimus in refractory rheumatoid arthritis: A multi-center randomized controlled trial in China | Rheumatoid Arthritis;M05.901 | Sirolimus group: Sirolimus; Non-Sirolimus group:Glucocorticoids and Immunosuppressant; | the Second Hospital of Shanxi Medical University | NULL | Recruiting | 18 | 65 | Both | Sirolimus group:200; Non-Sirolimus group:100; | China | |
| 3 | NCT00392951 (ClinicalTrials.gov) | December 2006 | 24/10/2006 | Sirolimus for Autoimmune Disease of Blood Cells | Sirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot Series | Autoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid Arthritis | Drug: sirolimus | Children's Hospital of Philadelphia | NULL | Completed | 1 Year | 30 Years | All | 30 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04582136 (ClinicalTrials.gov) | November 2020 | 3/10/2020 | Efficacy and Safety of Sirolimus in Active Systemic Lupus Erythematosus | Efficacy 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 Trial | Systemic Lupus Erythematosus | Drug: Sirolimus;Drug: Placebo | Chinese SLE Treatment And Research Group | Beijing Municipal Science & Technology Commission;North China Pharmaceutical Group Corporation | Not yet recruiting | 18 Years | 65 Years | All | 146 | Phase 2 | NULL |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | ChiCTR2000030370 | 2020-03-01 | 2020-02-29 | The efficacy and safety of sirolimus for the treatment of Systemic Sclerosis: a single-arm, single-center pilot study | The efficacy and safety of sirolimus for the treatment of Systemic Sclerosis: a single-arm, single-center pilot study | Systemic Sclerosis | treatment group:sirolimus; | The First Affiliated Hospital of China Medical University | NULL | Recruiting | 18 | Both | treatment group:20; | China | ||
| 2 | NCT03365869 (ClinicalTrials.gov) | June 1, 2018 | 4/12/2017 | A Pilot-Study of Sirolimus for the Treatment of Systemic Sclerosis | A Phase ? Pilot-Study With Sirolimus for the Treatment of Systemic Sclerosis | Systemic Sclerosis | Drug: Sirolimus | Peking University People's Hospital | NULL | Not yet recruiting | 18 Years | 80 Years | All | 72 | Phase 2 | NULL |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03192397 (ClinicalTrials.gov) | August 3, 2017 | 16/6/2017 | Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant | A 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 Transplantation | Acute 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 Macroglobulinemia | Procedure: 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 Irradiation | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Recruiting | 18 Years | 79 Years | All | 30 | Phase 2 | United States |
| 2 | NCT02979873 (ClinicalTrials.gov) | December 19, 2016 | 1/12/2016 | Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy | A Randomized Trial of Sirolimus (Rapamune(R)) for Relapse Prevention in Patients With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy | Severe Aplastic Anemia | Drug: Sirolimus | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Recruiting | 2 Years | 99 Years | All | 118 | Phase 2 | United States |
| 3 | EUCTR2006-006577-25-SE (EUCTR) | 25/07/2007 | 11/06/2007 | A 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 | A 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: 9.1;Level: LLT;Classification code 10018799;Term: 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 Institutet | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Finland;Sweden | |||
| 4 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | Fludarabine 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 Disorders | HLA-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 Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: 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 Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| 5 | NCT00319878 (ClinicalTrials.gov) | May 2006 | 28/4/2006 | Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia | A Phase I/II Trial of Sirolimus (Rapamune) and Cyclosporine in Patients With Refractory Aplastic Anemia | Anemia, Aplastic | Drug: Sirolimus;Drug: Cyclosporine | Office of Rare Diseases (ORD) | Rare Diseases Clinical Research Network | Recruiting | 21 Years | N/A | Both | 52 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT00061360 (ClinicalTrials.gov) | June 26, 2003 | 23/5/2003 | Improving Immunosuppressive Treatment for Patients With Severe Aplastic Anemia | A Randomized Trial of a Novel Immunosuppressive Combination of ATG, CsA and Sirolimus (Rapamune) vs a Slow Taper Cyclosporine Regimen in Subjects With Severe Aplastic Anemia | Severe Aplastic Anemia | Drug: ATG+Rapamune+cyclosporine;Drug: ATG+cyclosporine | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 2 Years | 110 Years | All | 77 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT00392951 (ClinicalTrials.gov) | December 2006 | 24/10/2006 | Sirolimus for Autoimmune Disease of Blood Cells | Sirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot Series | Autoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid Arthritis | Drug: sirolimus | Children's Hospital of Philadelphia | NULL | Completed | 1 Year | 30 Years | All | 30 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03866681 (ClinicalTrials.gov) | April 1, 2019 | 28/2/2019 | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory Classic Paroxysmal Nocturnal Hemoglobinuria ,a Prospective Study | Paroxysmal Nocturnal Hemoglobinuria | Drug: sirolimus | Peking Union Medical College Hospital | NULL | Not yet recruiting | 18 Years | 70 Years | All | 40 | Phase 4 | China |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | ChiCTR-ONC-17012126 | 2017-09-01 | 2017-07-25 | A multicenter, prospective, one-arm clinical study of Sirolimus treats refractory and recurrence primary immune thrombocytopenia | A multicenter, prospective, one-arm clinical study of Sirolimus treats refractory and recurrence primary immune thrombocytopenia | refractory or recurrence primary immune thrombocytopenia | Case series:Sirolimus treats refractory or recurrence primary immune thrombocytopenia; | Department of Hematology, Xiniao Hospital | NULL | Pending | 18 | 80 | Both | Case series:103; | China | |
| 2 | NCT00392951 (ClinicalTrials.gov) | December 2006 | 24/10/2006 | Sirolimus for Autoimmune Disease of Blood Cells | Sirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot Series | Autoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid Arthritis | Drug: sirolimus | Children's Hospital of Philadelphia | NULL | Completed | 1 Year | 30 Years | All | 30 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04370795 (ClinicalTrials.gov) | December 17, 2020 | 30/4/2020 | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Severe 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) | NULL | Enrolling by invitation | 3 Years | 40 Years | All | 30 | Phase 1;Phase 2 | United States |
| 2 | NCT03910452 (ClinicalTrials.gov) | October 28, 2019 | 9/4/2019 | Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cell | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 4 Years | 65 Years | All | 30 | Early Phase 1 | United States |
| 3 | NCT02629120 (ClinicalTrials.gov) | December 17, 2015 | 10/12/2015 | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease Transplant | Drug: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cells | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 4 Years | 65 Years | All | 50 | Phase 1;Phase 2 | United States |
| 4 | NCT02282904 (ClinicalTrials.gov) | October 23, 2014 | 4/11/2014 | Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 2 Years | 65 Years | All | 7 | Phase 1;Phase 2 | United States |
| 5 | NCT02177760 (ClinicalTrials.gov) | July 2014 | 19/6/2014 | Sirolimus Prophylaxis for aGVHD in TME SCID | Sirolimus 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 Transplant | Drug: Sirolimus | University of California, San Francisco | NULL | Withdrawn | N/A | 1 Year | Both | 0 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT00392951 (ClinicalTrials.gov) | December 2006 | 24/10/2006 | Sirolimus for Autoimmune Disease of Blood Cells | Sirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot Series | Autoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid Arthritis | Drug: sirolimus | Children's Hospital of Philadelphia | NULL | Completed | 1 Year | 30 Years | All | 30 | Phase 1;Phase 2 | United States |
| 7 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | Fludarabine 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 Disorders | HLA-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 Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: 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 Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT00396721 (ClinicalTrials.gov) | January 2006 | 3/11/2006 | Sirolimus Therapy for Poor Prognosis Immunoglobulin A Nephropathy | Pilot Trial of Treatment of Poor-Prognosis IgA Nephropathy With Low Exposure to Sirolimus. | Glomerulonephritis, IGA;Nephropathy, IGA;IGA Nephropathy | Drug: ACE inhibitor + statin;Drug: Sirolimus (study drug)+ACE inhibitor + statin | Josep m Cruzado | Wyeth is now a wholly owned subsidiary of Pfizer | Completed | 18 Years | 70 Years | Both | 23 | Phase 2 | Spain |
| 2 | EUCTR2005-002610-37-ES (EUCTR) | 05/12/2005 | 30/09/2005 | PILOT 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 | PILOT 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) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Spain |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02055079 (ClinicalTrials.gov) | April 2014 | 31/1/2014 | Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease | Pulsed Oral Sirolimus in Autosomal Dominant Polycystic Kidney Disease - The Vienna RAP Study | Polycystic Kidney, Type 1 Autosomal Dominant Disease;Polycystic Kidney, Type 2 Autosomal Dominant Disease | Drug: Sirolimus;Drug: Placebo | Medical University of Vienna | NULL | Unknown status | 18 Years | N/A | All | 68 | Phase 3 | Austria |
| 2 | EUCTR2012-000550-60-AT (EUCTR) | 17/01/2014 | 27/11/2013 | Pulsed oral sirolimus in autosomal dominant polycystic kidney disease | Pulsed 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: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System 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 3 | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 68 | Phase 3 | Austria | ||
| 3 | NCT01680250 (ClinicalTrials.gov) | September 2011 | 30/8/2012 | Sirolimus for Massive Polycystic Liver | An Open-label, Prospective Clinical Trial to Evaluate the Effectiveness and Safety of Sirolimus to Reduce Cyst Growth in ADPKD Patients With Massive Polycystic Liver | Polycystic Kidney Diseases | Drug: Sirolimus | Seoul National University Hospital | Wyeth is now a wholly owned subsidiary of Pfizer | Recruiting | 18 Years | 65 Years | Both | 44 | Phase 2;Phase 3 | Korea, Republic of |
| 4 | NCT01223755 (ClinicalTrials.gov) | September 2010 | 12/10/2010 | Sirolimus In Autosomal Dominant Polycystic Kidney Disease And Severe Renal Insufficiency | EFFECTS OF SIROLIMUS ON DISEASE PROGRESSION IN PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE AND SEVERE RENAL INSUFFICIENCY | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Drug: Sirolimus;Drug: conventional therapy | Mario Negri Institute for Pharmacological Research | NULL | Terminated | 18 Years | 80 Years | Both | 41 | Phase 2;Phase 3 | Italy |
| 5 | NCT01632605 (ClinicalTrials.gov) | November 2009 | 13/5/2012 | The Vienna RAP Pilot Study | Rapamycin in Advanced Polycystic Kidney Disease Pilot Study | ADPKD | Drug: Sirolimus | Medical University of Vienna | NULL | Completed | 18 Years | N/A | Both | 8 | N/A | Austria |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2007-005047-21-IT (EUCTR) | 24/12/2007 | 02/07/2008 | Effects of Sirolimus on disease progression in patients with Autosomal Dominant Polycystic Kidney Disease and severe renal insufficiency - SIRENA II | Effects 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. NEGRI | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | Italy | |||
| 7 | EUCTR2007-006557-25-IT (EUCTR) | 20/12/2007 | 18/12/2007 | RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDY | RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDY | ADPKD type I MedDRA version: 9.1;Level: SOC;Classification code 10038359;Term: 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 CONSORZIALE | NULL | Not Recruiting | Female: yes Male: yes | Italy | ||||
| 8 | NCT00491517 (ClinicalTrials.gov) | March 2007 | 25/6/2007 | Sirolimus Treatment in Patients With Autosomal Dominant Polycystic Kidney Disease: Renal Efficacy and Safety | Sirolimus Treatment in Patients With ADPKD | Polycystic Kidney | Drug: Sirolimus;Drug: conventional therapy | Mario Negri Institute for Pharmacological Research | NULL | Completed | 18 Years | 80 Years | Both | 22 | Phase 2 | Italy |
| 9 | EUCTR2006-003427-37-IT (EUCTR) | 05/02/2007 | 27/12/2006 | Sirolimus treatment in patients with autosomal dominant polycystic kidney disease renal efficacy and safety - ND | Sirolimus 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. NEGRI | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | Italy | |||
| 10 | NCT00346918 (ClinicalTrials.gov) | June 2006 | 22/6/2006 | Sirolimus (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: Standard | University of Zurich | NULL | Completed | 18 Years | 40 Years | Both | 100 | Phase 3 | Switzerland |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2017-004930-27-AT (EUCTR) | 05/02/2019 | 20/12/2018 | Sirolimus 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 Vienna | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Austria |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT01462006 (ClinicalTrials.gov) | October 2015 | 26/10/2011 | Double-blind Placebo-controlled Pilot Study of Sirolimus in Idiopathic Pulmonary Fibrosis (IPF) | Double-blind Placebo-controlled Pilot Study of Sirolimus in IPF | Idiopathic Pulmonary Fibrosis;Diffuse Parenchymal Lung Disease;Interstitial Lung Disease | Drug: sirolimus;Other: Placebo | University of Virginia | National Heart, Lung, and Blood Institute (NHLBI) | Unknown status | 21 Years | 85 Years | All | 32 | N/A | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03253913 (ClinicalTrials.gov) | March 31, 2018 | 10/8/2017 | Resveratrol and Sirolimus in Lymphangioleiomyomatosis Trial | Resveratrol and Sirolimus in Lymphangioleiomyomatosis Trial (RESULT) | Lymphangioleiomyomatosis | Drug: Sirolimus;Drug: Resveratrol | University of Cincinnati | National Heart, Lung, and Blood Institute (NHLBI) | Unknown status | 18 Years | N/A | Female | 25 | Phase 2 | United States |
| 2 | NCT03150914 (ClinicalTrials.gov) | January 1, 2018 | 10/5/2017 | Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial | Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial | LAM;Lymphangioleiomyomatosis | Drug: Sirolimus | University of Cincinnati | National Heart, Lung, and Blood Institute (NHLBI);National Center for Advancing Translational Science (NCATS);The LAM Foundation | Recruiting | 18 Years | N/A | Female | 60 | Phase 3 | United States |
| 3 | NCT02432560 (ClinicalTrials.gov) | March 2015 | 4/3/2015 | Safety and Durability of Sirolimus for Treatment of LAM | Multicenter International Durability and Safety of Sirolimus in LAM Trial (MIDAS) | Lymphangioleiomyomatosis | Drug: Sirolimus;Drug: Everolimus | University of Cincinnati | Rare Diseases Clinical Research Network;National Institutes of Health (NIH);National Heart, Lung, and Blood Institute (NHLBI);The LAM Foundation | Active, not recruiting | 18 Years | N/A | Female | 600 | United States | |
| 4 | JPRN-UMIN000016677 | 2015/01/01 | 02/03/2015 | Efficacy and safety of low dose sirolimus in lymphangioleiomyomatosis | lymphangioleiomyomatosis | sirolimus 1mg/day / sirolimus 2mg/day | National Hospital Organization Kinki-chuo Chest Medical Center | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 20 | Not selected | Japan | |
| 5 | NCT02061397 (ClinicalTrials.gov) | March 2014 | 23/1/2014 | Safety of Simvastatin in LAM and TSC | The Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC) | Lymphangioleiomyomatosis;Tuberous Sclerosis Complex | Drug: Simvastatin;Drug: Sirolimus Oral Product;Drug: Everolimus Oral Product | University of Pennsylvania | The LAM Foundation | Completed | 18 Years | N/A | Female | 10 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | JPRN-JMA-IIA00096 | 05/09/2012 | 17/08/2012 | multicenter lymphangioleiomyomatosis sirolimus trial for safety | multicenter lymphangioleiomyomatosis sirolimus trial for safety | lymphangioleiomyomatosis | Intervention 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, PhD | Yoshikazu InoueKuniaki SeyamaRyushi TazawaToshinori Takada | Completed | >=18 YEARS | No Limit | Female | 65 | Phase 2 | Japan |
| 7 | NCT01687179 (ClinicalTrials.gov) | September 2012 | 2/8/2012 | Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis | Targeting Autophagy for the Treatment of TSC and LAM: a Phase I Trial of Hydroxychloroquine and Sirolimus | Lymphangioleiomyomatosis | Drug: Sirolimus and Hydroxychloroquine 200 mg;Drug: Sirolimus and Hydroxychloroquine 400 mg | Brigham and Women's Hospital | National Heart, Lung, and Blood Institute (NHLBI) | Completed | 18 Years | 85 Years | Female | 14 | Phase 1 | United States |
| 8 | JPRN-UMIN000007387 | 2012/03/31 | 01/04/2012 | Clinical trials for long-term administration of sirolimus for lymphangioleiomyomatosis | Clinical trials for long-term administration of sirolimus for lymphangioleiomyomatosis - Long-term administration of sirolimus for lymphangioleiomyomatosis | lymphangioleiomyomatosis | sirolimus | Kobe University Graduate School of Medicine | NULL | Complete: follow-up complete | Not applicable | Not applicable | Female | 1 | Not applicable | Japan |
| 9 | JPRN-JMA-IIA00037 | 01/10/2011 | 15/03/2010 | MLLTS trial | Multicenter Lymphangioleiomyomatosis Long Term Sirolimus Trial | lymphangioleiomyomatosis | Intervention 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.D | Ryushi Tazawa | Other | >=18 YEARS | No Limit | Female | 50 | Phase 2 | Japan |
| 10 | JPRN-JMA-IIA00011 | 08/05/2008 | 29/06/2007 | Multicenter 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 Medicine | a.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 Health | Completed | >=18 YEARS | Female | 120 | Phase 3 | Japan, United States, Canada | |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT00414648 (ClinicalTrials.gov) | December 2006 | 20/12/2006 | Efficacy and Safety of Sirolimus for Treating Lymphangioleiomyomatosis (LAM) | Lymphangioleiomyomatosis Efficacy and Safety Trial | Lymphangioleiomyomatosis | Drug: Sirolimus;Drug: Placebo sirolimus | Office of Rare Diseases (ORD) | FDA Office of Orphan Products Development | Active, not recruiting | 18 Years | N/A | Female | 120 | Phase 3 | United States;Canada;Japan |
| 12 | NCT00490789 (ClinicalTrials.gov) | October 2005 | 21/6/2007 | Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM | A Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis | Tuberous Sclerosis;Lymphangioleiomyomatosis | Drug: sirolimus | Cardiff University | University of Nottingham;St Georges Hospital Medical School;Royal Sussex County Hospital;The Tuberous Sclerosis Association;Wyeth is now a wholly owned subsidiary of Pfizer | Active, not recruiting | 18 Years | 65 Years | Both | 14 | Phase 2 | United Kingdom |
| 13 | NCT00457808 (ClinicalTrials.gov) | December 2002 | 6/4/2007 | Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM | Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis | Tuberous Sclerosis;Lymphangioleiomyomatosis | Drug: Rapamycin, sirolimus | Children's Hospital Medical Center, Cincinnati | The LAM Foundation;Tuberous Sclerosis Alliance | Completed | 18 Years | 65 Years | Both | 25 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT01814059 (ClinicalTrials.gov) | March 7, 2013 | 15/3/2013 | Sirolimus for Eosinophil-Associated Gastrointestinal Disorders | A Phase 1, Open-Label Study of Sirolimus in Eosinophil-Associated Gastrointestinal Disorders | Eosinophilic Gastroenteritis;Eosinophilic Esophagitis | Drug: sirolimus | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 18 Years | 65 Years | All | 4 | Phase 1 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04408625 (ClinicalTrials.gov) | July 30, 2020 | 21/5/2020 | Phase 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 Dementia | Biological: PR006;Drug: Methylprednisolone;Drug: Sirolimus;Drug: Prednisone | Prevail Therapeutics | NULL | Recruiting | 30 Years | 80 Years | All | 15 | Phase 1;Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-jRCTs031190157 | 18/12/2019 | 11/12/2019 | Research of sirolimus administration for FCD II type | Clinical 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 Mitsuhiro | NULL | Recruiting | >= 2age old | Not applicable | Both | 15 | Phase 2 | Japan |
| 2 | JPRN-jRCTs041190059 | 09/08/2019 | 05/08/2019 | Study about safety of sirolimus: Shizuoka 2019-1 | Clinical 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 | sirolimus | Takahashi Yukitoshi | Kato Mitsuhiro | Recruiting | >= 6age old | Not applicable | Both | 5 | Phase 2 | Japan |
| 3 | JPRN-UMIN000033504 | 2018/10/01 | 25/07/2018 | An uncontrolled open-label study on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type-II | An 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 Hospital | NULL | Complete: follow-up continuing | 6years-old | 65years-old | Male and Female | 15 | Phase 2 | Japan |
| 4 | JPRN-UMIN000030962 | 2018/02/28 | 24/01/2018 | Clinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type II | Clinical 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 2 | Prescribe 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 Hospital | NULL | Complete: follow-up continuing | 2years-old | 65years-old | Male and Female | 2 | Not selected | Japan |
| 5 | JPRN-UMIN000030797 | 2017/12/01 | 13/01/2018 | Uncontrolled open label study of sirolimus about efficacy for epileptic seizures and safety in patients with focal cortical dysplasia type 2 | Uncontrolled 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 2 | Period: 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, NHO | NULL | Complete: follow-up continuing | 6years-old | 65years-old | Male and Female | 1 | Phase 2 | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03047980 (ClinicalTrials.gov) | January 2017 | 2/2/2017 | Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome | Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome | Sturge-Weber Syndrome | Drug: Sirolimus | Anne Comi, MD | Children's Hospital Medical Center, Cincinnati;Pfizer;National Institutes of Health (NIH);Faneca 66 Foundation;National Institute of Neurological Disorders and Stroke (NINDS) | Active, not recruiting | 3 Years | 31 Years | All | 10 | Phase 2;Phase 3 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04595513 (ClinicalTrials.gov) | September 8, 2020 | 6/10/2020 | Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants | Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants | Tuberous Sclerosis Complex;Epilepsy | Drug: TAVT-18 (sirolimus);Drug: Placebo | Children's Hospital Medical Center, Cincinnati | NULL | Recruiting | N/A | 6 Months | All | 65 | Phase 1;Phase 2 | United States |
| 2 | ChiCTR2000031984 | 2020-05-01 | 2020-04-17 | Sirolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex and Epilepsy | Sirolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex and Epilepsy | Tuberous sclerosis complex; Epilepsy | experimental group:Antiepileptic drug + sirolimus;control group:antiepileptic drugs; | West China Hospital, Sichuan University | NULL | Pending | Both | experimental group:31;control group:31; | N/A | China | ||
| 3 | NCT03363763 (ClinicalTrials.gov) | April 12, 2017 | 1/12/2017 | Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex | Phase 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 Label | Angiofibroma of Face;Tuberous Sclerosis | Drug: Sirolimus 0.2%;Drug: Sirolimus 0.4%;Drug: Placebo ointment | Aucta Pharmaceuticals, Inc | NULL | Recruiting | 2 Years | 18 Years | All | 45 | Phase 2 | United States;China |
| 4 | NCT02635789 (ClinicalTrials.gov) | December 2015 | 15/12/2015 | Phase 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 Complex | Tuberous Sclerosis;Angiofibroma;Hypomelanotic Macule;Plaque | Drug: NPC-12G gel;Drug: Placebo gel | Nobelpharma | NULL | Completed | 3 Years | N/A | All | 62 | Phase 3 | Japan |
| 5 | NCT02634931 (ClinicalTrials.gov) | December 2015 | 16/12/2015 | Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex | A 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 Complex | Tuberous Sclerosis;Angiofibroma;Hypomelanotic Macule;Plaque | Drug: NPC-12G gel | Nobelpharma | NULL | Completed | 3 Years | N/A | All | 94 | Phase 3 | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT02061397 (ClinicalTrials.gov) | March 2014 | 23/1/2014 | Safety of Simvastatin in LAM and TSC | The Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC) | Lymphangioleiomyomatosis;Tuberous Sclerosis Complex | Drug: Simvastatin;Drug: Sirolimus Oral Product;Drug: Everolimus Oral Product | University of Pennsylvania | The LAM Foundation | Completed | 18 Years | N/A | Female | 10 | Phase 1;Phase 2 | United States |
| 7 | JPRN-UMIN000012420 | 2013/12/10 | 27/11/2013 | Randomized, double-blind, placebo-controlled, clinical trial with OSD-001 for skin lesions due to tuberous sclerosis complex. | Tuberous sclerosis complex | 0.05% 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 University | NULL | Complete: follow-up complete | 3years-old | 65years-old | Male and Female | 36 | Phase 1;Phase 2 | Japan | |
| 8 | NCT01929642 (ClinicalTrials.gov) | July 2013 | 7/8/2013 | Rapalogues for Autism Phenotype in TSC: A Feasibility Study | Rapalogues for Autism Phenotype in TSC: A Feasibility Study | Tuberous Sclerosis Complex;Self-injury;Autism | Drug: Sirolimus;Drug: Everolimus | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | NULL | Completed | 2 Years | 30 Years | All | 3 | Phase 2 | United States |
| 9 | NCT01031901 (ClinicalTrials.gov) | December 2009 | 10/12/2009 | Topical 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 1 | Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;Neurofibroma | Drug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycin | The University of Texas Health Science Center, Houston | Society for Pediatric Dermatology | Completed | 13 Years | N/A | Both | 52 | Phase 1 | United States |
| 10 | NCT01217125 (ClinicalTrials.gov) | October 2008 | 6/10/2010 | Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis | CLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSIS | Angiomyolipoma | Drug: Sirolimus | Fundacio Puigvert | Ministry of Health, Spain | Completed | 10 Years | N/A | Both | 18 | Phase 4 | NULL |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2007-005978-30-ES (EUCTR) | 22/01/2008 | 03/12/2007 | Ensayo 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 | Ensayo 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: 9.1;Level: LLT;Classification code 10045138;Term: 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 PUIGVERT | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Spain | ||||
| 12 | NCT00490789 (ClinicalTrials.gov) | October 2005 | 21/6/2007 | Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM | A Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis | Tuberous Sclerosis;Lymphangioleiomyomatosis | Drug: sirolimus | Cardiff University | University of Nottingham;St Georges Hospital Medical School;Royal Sussex County Hospital;The Tuberous Sclerosis Association;Wyeth is now a wholly owned subsidiary of Pfizer | Active, not recruiting | 18 Years | 65 Years | Both | 14 | Phase 2 | United Kingdom |
| 13 | NCT00457808 (ClinicalTrials.gov) | December 2002 | 6/4/2007 | Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM | Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis | Tuberous Sclerosis;Lymphangioleiomyomatosis | Drug: Rapamycin, sirolimus | Children's Hospital Medical Center, Cincinnati | The LAM Foundation;Tuberous Sclerosis Alliance | Completed | 18 Years | 65 Years | Both | 25 | Phase 2 | United States |
| 14 | EUCTR2019-000752-34-CZ (EUCTR) | 02/06/2020 | Study 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: 20.0;Level: PT;Classification code 10002429;Term: Angiofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 20.0;Classification code 10073678;Term: Juvenile angiofibroma;System 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) | NULL | NA | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Serbia;Hungary;Czech Republic;Slovakia;Spain;Australia;New Zealand |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02960997 (ClinicalTrials.gov) | May 2016 | 15/6/2016 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | A 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 Syndrome | Drug: Sirolimus, 2%;Drug: Vehicle | Stanford University | NULL | Active, not recruiting | 4 Years | N/A | All | 8 | Phase 2 | United States |
| 2 | NCT03016715 (ClinicalTrials.gov) | May 2016 | 9/1/2017 | Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study | A 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 Syndrome | Drug: Sirolimus 2%;Drug: Vehicle | Premier Specialists, Australia | NULL | Recruiting | 5 Years | N/A | All | 8 | Phase 2 | Australia |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | ChiCTR-INR-17012212 | 2017-07-28 | 2017-08-01 | Use of sirolimus in patients with primary idiopathic membranous nephropathy: a prospective randomized control trial | Use of sirolimus in patients with primary idiopathic membranous nephropathy: a prospective randomized control trial | idiopathic membranous nephropathy | 1:Cyclosporine;2:cyclosporine combined with sirolimus; | Renal Division, Peking University First Hospital | NULL | Recruiting | 18 | 70 | Both | 1:35;2:35; | China | |
| 2 | NCT00050713 (ClinicalTrials.gov) | December 17, 2002 | 17/12/2002 | Sirolimus Therapy for Idiopathic and Lupus Membranous Nephropathy | Sirolimus Therapy in Idiopathic and Lupus Membranous Nephropathy | Membranous Glomerulonephritis;Lupus Membranous Nepropathy | Drug: Sirolimus | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | NULL | Completed | 13 Years | N/A | All | 12 | Phase 2 | United States |
| 3 | NCT00040508 (ClinicalTrials.gov) | June 2002 | 26/6/2002 | Sirolimus for Focal Segmental Glomerulosclerosis | Sirolimus for Focal Segmental Glomerulosclerosis | Focal Glomerulosclerosis | Drug: Sirolimus | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | NULL | Completed | N/A | N/A | Both | 30 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-UMIN000038973 | 2020/01/06 | 25/12/2019 | A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies | A 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 syndrome | An 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 Hospital | NULL | Pending | 1months-old | Not applicable | Male and Female | 10 | Phase 3 | Japan |
| 2 | JPRN-jRCTs031180290 | 16/11/2017 | 15/03/2019 | A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies | A 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 Michio | NULL | Recruiting | Not applicable | Not applicable | Both | 100 | Phase 3 | Japan |
| 3 | JPRN-UMIN000030522 | 2017/11/14 | 22/12/2017 | A 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 malformations | 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. | Gifu University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 50 | Not selected | Japan | |
| 4 | JPRN-UMIN000029843 | 2017/11/06 | 06/11/2017 | Sirolimus for the treatment of refractory lymphangiomatosis/ Gorham Stout disease | Refractory lymphangiomatosis, Gorham-Stout disease | Oral sirolimus | Kyushu University Hospital | NULL | Recruiting | 8years-old | 30years-old | Male and Female | 2 | Not selected | Japan | |
| 5 | JPRN-UMIN000016580 | 2015/02/01 | 19/02/2015 | Sirolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomalies | Sirolimus for the treatment of lymphangiomatosis, Gorham-Stout disease and vascular anomalies - Sirolimus for vascular anomalies | Lymphangiomatosis, Gorham-Stout disease and vascular anomalies | Oral sirolimus | Gifu university | NULL | Complete: follow-up complete | Not applicable | 30years-old | Male and Female | 30 | Phase 1;Phase 2 | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT00975819 (ClinicalTrials.gov) | October 2009 | 10/9/2009 | Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies | A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies | Kaposiform 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 Syndromes | Drug: sirolimus | Children's Hospital Medical Center, Cincinnati | NULL | Active, not recruiting | N/A | 31 Years | Both | 60 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04128722 (ClinicalTrials.gov) | February 14, 2020 | 24/9/2019 | TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN | TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN | Lingual Microcystic Lymphatic Malformations | Drug: Sirolimus Oral Liquid Product 1mg/mL | University Hospital, Tours | NULL | Recruiting | 5 Years | N/A | All | 12 | Phase 2 | France |
| 2 | JPRN-UMIN000038973 | 2020/01/06 | 25/12/2019 | A multicenter, phase 3 study assessing efficacy and safety of the Sirolimus (Granules and Tablets) in the Treatment of intractable vascular anomalies | A 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 syndrome | An 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 Hospital | NULL | Pending | 1months-old | Not applicable | Male and Female | 10 | Phase 3 | Japan |
| 3 | EUCTR2019-001530-33-FR (EUCTR) | 29/06/2019 | 01/04/2019 | TOPical sirolimus in linGUal microkystic lymphatic malformation-TOPGUN | TOPical 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 TOURS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | France | |||
| 4 | NCT03972592 (ClinicalTrials.gov) | June 5, 2019 | 24/5/2019 | Topical Sirolimus in Cutaneous Lymphatic Malformations | 0.1% 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 | Vascular Malformations;Lymphatic Malformation | Drug: Topical 0.1% Sirolimus;Drug: Topical Vehicle | University Hospital, Tours | University Hospital, Angers | Recruiting | 6 Years | N/A | All | 55 | Phase 2 | France |
| 5 | EUCTR2018-001359-11-FR (EUCTR) | 22/02/2019 | 23/07/2018 | 0.1% 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 | 0.1% 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 TOURS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 2 | France | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | JPRN-jRCTs031180290 | 16/11/2017 | 15/03/2019 | A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies | A 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 Michio | NULL | Recruiting | Not applicable | Not applicable | Both | 100 | Phase 3 | Japan |
| 7 | JPRN-UMIN000030522 | 2017/11/14 | 22/12/2017 | A 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 malformations | 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. | Gifu University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 50 | Not selected | Japan | |
| 8 | NCT00975819 (ClinicalTrials.gov) | October 2009 | 10/9/2009 | Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies | A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies | Kaposiform 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 Syndromes | Drug: sirolimus | Children's Hospital Medical Center, Cincinnati | NULL | Active, not recruiting | N/A | 31 Years | Both | 60 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03767660 (ClinicalTrials.gov) | July 31, 2018 | 24/11/2018 | Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation | Efficacy of Rapamycin (Sirolimus) in the Treatment of Blue Rubber Bleb Nevus Syndrome, Hereditary or Sporadic Venous Malformation | Blue Rubber Bleb Nevus Syndrome;Venous Malformation | Drug: Rapamycin | Peking Union Medical College Hospital | Air Force General Hospital of the PLA;Chinese Academy of Medical Sciences | Recruiting | N/A | N/A | All | 20 | Phase 4 | China |
| 2 | NCT00975819 (ClinicalTrials.gov) | October 2009 | 10/9/2009 | Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies | A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies | Kaposiform 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 Syndromes | Drug: sirolimus | Children's Hospital Medical Center, Cincinnati | NULL | Active, not recruiting | N/A | 31 Years | Both | 60 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-001530-33-FR (EUCTR) | 29/06/2019 | 01/04/2019 | TOPical sirolimus in linGUal microkystic lymphatic malformation-TOPGUN | TOPical 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 TOURS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | France | |||
| 2 | EUCTR2018-001359-11-FR (EUCTR) | 22/02/2019 | 23/07/2018 | 0.1% 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 | 0.1% 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 TOURS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 2 | France | |||
| 3 | JPRN-UMIN000030522 | 2017/11/14 | 22/12/2017 | A 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 malformations | 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. | Gifu University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 50 | Not selected | Japan | |
| 4 | NCT02042326 (ClinicalTrials.gov) | September 12, 2014 | 20/1/2014 | Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations | Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations | Arteriovenous Malformations | Drug: Sirolimus | Centre Hospitalier Universitaire, Amiens | NULL | Recruiting | 2 Years | N/A | All | 50 | Phase 2 | Belgium;France |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-UMIN000030522 | 2017/11/14 | 22/12/2017 | A 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 malformations | 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. | Gifu University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 50 | Not selected | Japan |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04470804 (ClinicalTrials.gov) | August 1, 2020 | 9/7/2020 | Sirolimus Treatment for Newly Diagnosed Primary Acquired PRCA | Sirolimus Treatment for Newly Diagnosed Primary Acquired Pure Red Cell Aplasia: a Single Center Prospective Study | Pure Red Cell Aplasia, Acquired | Drug: Sirolimus;Drug: Cyclosporine A | Bing Han | NULL | Not yet recruiting | 18 Years | 70 Years | All | 40 | Phase 4 | China |
| 2 | NCT03364764 (ClinicalTrials.gov) | January 2018 | 21/11/2017 | Sirolimus Treatment for Refractory PRCA | Sirolimus Treatment for Refractory Pure Red Cell Aplasia, a Prospective Study | Pure Red Cell Aplasia | Drug: Sirolimus | Bing Han | NULL | Recruiting | 18 Years | 80 Years | All | 30 | Phase 4 | China |
| 3 | NCT03214354 (ClinicalTrials.gov) | July 5, 2017 | 5/7/2017 | Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor | A 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 Aplasia | Drug: Alemtuzumab;Radiation: Total Body Irradiation;Drug: Sirolimus | University of Calgary | NULL | Recruiting | 1 Year | 19 Years | All | 12 | Phase 2 | Canada |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT00352976 (ClinicalTrials.gov) | May 18, 2006 | 14/7/2006 | TBI Dose De-escalation for Fanconi Anemia | Total Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation | Fanconi Anemia | Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Bone Marrow Transplantation;Drug: Mycophenolate Mofetil;Drug: Sirolimus | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | N/A | All | 88 | Phase 2;Phase 3 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-jRCT2071190029 | 25/05/2020 | 09/10/2019 | Randomized, 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 Atsushi | NULL | Recruiting | >= 18age old | Not applicable | Both | 20 | Phase 2 | Japan |
| 2 | NCT03933904 (ClinicalTrials.gov) | September 25, 2019 | 29/4/2019 | Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease | A Phase II, Single-arm Open-label Multi-center Study of Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease | Castleman Disease;Castleman's Disease, Multicentric | Drug: Sirolimus | University of Pennsylvania | NULL | Recruiting | 18 Years | 80 Years | All | 24 | Phase 2 | United States |
| 3 | NCT00092222 (ClinicalTrials.gov) | October 28, 2004 | 21/9/2004 | Virotherapy and Natural History Study of KHSV-Associated Multricentric Castleman s Disease With Correlates of Disease Activity | Targeted Oncolytic Virotherapy and Natural History Study of KSHV-Associated Multicentric Castleman's Disease With Laboratory and Clinical Correlates of Disease Activity | Lymphoproliferative Disorder;HHV-8;Malignancy;HIV | Drug: 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 Only | National Cancer Institute (NCI) | NULL | Recruiting | 12 Years | N/A | All | 72 | Phase 2 | United States |