Rapamycin (DrugBank: Rapamycin)
18 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 |
| 15 | 封入体筋炎 | 1 |
| 34 | 神経線維腫症 | 3 |
| 35 | 天疱瘡 | 1 |
| 46 | 悪性関節リウマチ | 1 |
| 49 | 全身性エリテマトーデス | 2 |
| 51 | 全身性強皮症 | 1 |
| 60 | 再生不良性貧血 | 1 |
| 61 | 自己免疫性溶血性貧血 | 1 |
| 67 | 多発性嚢胞腎 | 4 |
| 86 | 肺動脈性肺高血圧症 | 1 |
| 89 | リンパ脈管筋腫症 | 2 |
| 96 | クローン病 | 1 |
| 157 | スタージ・ウェーバー症候群 | 1 |
| 158 | 結節性硬化症 | 17 |
| 256 | 筋型糖原病 | 1 |
| 278 | 巨大リンパ管奇形（頚部顔面病変） | 1 |
| 279 | 巨大静脈奇形（頚部口腔咽頭びまん性病変） | 1 |
| 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 | NCT02481453 (ClinicalTrials.gov) | July 15, 2015 | 13/5/2015 | Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis | Étude de l'Effet de la Rapamycine Sur la Force Musculaire et la réponse Immunitaire au Cours de la Myosite à Inclusions: étude RAPAMI | Inclusion Body Myositis (IBM) | Drug: Rapamycin;Drug: Placebo | Institut National de la Santé Et de la Recherche Médicale, France | NULL | Completed | 45 Years | 85 Years | All | 44 | Phase 2;Phase 3 | 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-UMIN000015081 | 2014/09/08 | 07/09/2014 | Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 | neurofibromatosis type1 | 1) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks. 2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on two-thirds of the tumor once a day for another 2 weeks. 3) Check the skin findings, blood test results, blood level of rapamycin and adverse events after a total of 4 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on the whole surface of the tumor once a day for another 8 weeks. | Department of Dermatology Graduate School of Medicine, Osaka University | NULL | Recruiting | 20years-old | Not applicable | Male and Female | 3 | Phase 2;Phase 3 | Japan | |
| 2 | 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 |
| 3 | NCT00901849 (ClinicalTrials.gov) | May 2007 | 13/5/2009 | Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1) | Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1) | Low-grade Gliomas | Drug: Tarceva and Rapamycin | Roger Packer | Children's Research Institute | Completed | N/A | 21 Years | Both | 21 | 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 | 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 | ChiCTR-IPR-17010307 | 2017-01-01 | 2017-01-01 | The efficacy and safety of rapamycin in refractory rheumatoid arthritis: A multi-center randomized controlled trial in China | The efficacy and safety of rapamycin in refractory rheumatoid arthritis: A multi-center randomized controlled trial in China | Rheumatoid Arthritis;M05.901 | Rapamycin group:Rapamycin;Non-rapamycin group :conventional glucocorticoids and DMARDs; | the Second Hospital of Shanxi Medical University | NULL | Recruiting | 18 | 65 | Both | Rapamycin group:200;Non-rapamycin group :100; | 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-IPR-16009451 | 2016-11-01 | 2016-10-16 | The Efficacy and Safety of Low Dose IL-2 Conbined Rapamycin Therapy in Chinese Over-treated Patients with Systemic Lupus Erythematosus | The Efficacy and Safety of Low Dose IL-2 Conbined Rapamycin Therapy in Chinese Over-treated Patients with Systemic Lupus Erythematosus | Systemic Lupus Erythematosus | IL-2 group:IL-2;IL-2 + Rapamycin group :IL-2 and Rapamycin;non IL-2 or IL-6 antagonist group :Glucocorticoids and DMARDs; | the Second Hospital of Shanxi Medical University | NULL | Pending | 18 | 80 | Both | IL-2 group:20;IL-2 + Rapamycin group :20;non IL-2 or IL-6 antagonist group :20; | China | |
| 2 | NCT00779194 (ClinicalTrials.gov) | October 2008 | 23/10/2008 | Prospective Study of Rapamycin for the Treatment of SLE | Prospective Study of Rapamycin for the Treatment of SLE | Systemic Lupus Erythematosus (SLE) | Drug: Rapamycin | State University of New York - Upstate Medical University | Pfizer;Pfizer | Completed | 18 Years | N/A | Both | 40 | 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 | NCT00241189 (ClinicalTrials.gov) | August 2002 | 14/10/2005 | Rapamycin vs Methotrexate in Diffuse SSc | A 48-week, Double-blind, Randomized, Parallel Phase I/II Study of Oral Rapamycin Versus Methotrexate in Systemic Sclerosis (Scleroderma) | Systemic Sclerosis | Drug: rapamycin;Drug: methotrexate | University of California, Los Angeles | NULL | Completed | 18 Years | 70 Years | Both | 17 | 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 | 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 |
| 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 | ChiCTR1900023476 | 2019-07-01 | 2019-05-30 | Multicenter, prospective and open clinical study for rapamycin in the treatment of autoimmune hemolytic anemia | Multicenter, prospective and open clinical study for rapamycin in the treatment of autoimmune hemolytic anemia | Autoimmune | 1:glucocorticoid;2:glucocorticoid & Ciclosporin;3:glucocorticoid & rapamycin;4:rapamycin; | Tianjin Medical University General Hospital | NULL | Pending | Both | 1:20;2:20;3:20;4:20; | 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 | 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 |
| 2 | NCT00920309 (ClinicalTrials.gov) | June 2009 | 12/6/2009 | Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy | Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy | Autosomal Dominant Polycystic Kidney Disease | Drug: Rapamycin;Other: Standard of Care-Placebo | Yale University | NULL | Terminated | 18 Years | 70 Years | All | 21 | Phase 2;Phase 3 | United States |
| 3 | 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 | ||||
| 4 | NCT00286156 (ClinicalTrials.gov) | October 2006 | 1/2/2006 | Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease | Polycystic Kidney Diseases | Drug: Rapamune | The Cleveland Clinic | Wyeth is now a wholly owned subsidiary of Pfizer | Completed | 18 Years | 75 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 | NCT02587325 (ClinicalTrials.gov) | April 1, 2017 | 23/10/2015 | ABI-009, an mTOR Inhibitor, for Patients With Severe Pulmonary Arterial Hypertension | A Phase 1 Clinical Trial of ABI-009, an mTOR Inhibitor, for Patients With Severe Pulmonary Arterial Hypertension (PAH) | Pulmonary Hypertension | Drug: ABI-009, nab-rapamycin, albumin-bound rapamycin | Aadi, LLC | NULL | Recruiting | 18 Years | N/A | All | 25 | 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 | 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 |
| 2 | 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 | NCT02675153 (ClinicalTrials.gov) | April 1, 2015 | 21/1/2016 | To Evaluate the Efficacy and Safety of Rapamycin for Crohn's Disease-related Stricture | Efficacy and Safety of Rapamycin in the Treatment of Crohn's Disease-related Stricture | Crohn's Diseases | Drug: Rapamycin | The Second Hospital of Nanjing Medical University | NULL | Recruiting | 18 Years | N/A | All | 15 | N/A | 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 | NCT02080624 (ClinicalTrials.gov) | January 2011 | 18/4/2013 | Efficacy and Safety Study of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber Syndrome | Phase II, Randomized, Triple Blind, Intra-individually Placebo-controlled Clinical Trial to Assess the Efficacy and Safety of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber Syndrome. | Sturge- Weber Syndrome | Drug: Drug: Topical Rapamycin | Clinica Universidad de Navarra, Universidad de Navarra | NULL | Completed | 16 Years | 65 Years | Both | 23 | Phase 2 | 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 | EUCTR2019-000752-34-SK (EUCTR) | 15/11/2019 | 01/10/2019 | 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] | DSLP | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Hungary;Slovakia;Spain;Australia;New Zealand;United Kingdom | |||
| 2 | EUCTR2019-000752-34-HU (EUCTR) | 14/11/2019 | 18/11/2019 | 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] | DSLP | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Hungary;Slovakia;Spain;Australia;New Zealand;United Kingdom | |||
| 3 | NCT03826628 (ClinicalTrials.gov) | July 28, 2019 | 29/1/2019 | Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex | 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 | Facial Angiofibroma;Tuberous Sclerosis | Drug: rapamycin;Drug: placebo | Dermatology Specialties Limited Partnership | NULL | Recruiting | 6 Years | 65 Years | All | 120 | Phase 2;Phase 3 | United States;Australia;Czechia;Hungary;New Zealand;Slovakia;Spain;United Kingdom |
| 4 | ChiCTR-OOB-15006535 | 2015-05-26 | 2015-05-29 | A Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis Complex | A Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis Complex | tuberous sclerosis complex | Case series:rapamycin; | Chinese PLA General Hospital | NULL | Recruiting | 1 | 18 | Both | Case series:500; | China | |
| 5 | JPRN-UMIN000015114 | 2014/09/11 | 10/09/2014 | Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex | Tuberous sclerosis complex | Application of 0.2% rapamycin gel on skin lesions of the face and trunk twice a day for 12 weeks. | Department of Dermatology Graduate School of Medicine, Osaka University | NULL | Recruiting | Not applicable | 70years-old | Male and Female | 24 | Phase 2;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 | NCT03140449 (ClinicalTrials.gov) | September 5, 2013 | 26/4/2017 | Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis | Study of Combination Therapy With Topical Rapamycin and Calcitriol for Cutaneous Lesions of Tuberous Sclerosis: A Double-blind Randomized Controlled Trial | Facial Angiofibroma | Drug: Rapamycin;Drug: Calcitriol;Drug: Rapamycin-calcitriol combination | National Taiwan University Hospital | NULL | Completed | 7 Years | 65 Years | All | 52 | Phase 3 | NULL |
| 7 | EUCTR2011-006308-12-ES (EUCTR) | 16/07/2012 | 29/03/2012 | Clinical trial in patients with tuberous sclerosis for the study of the effects of topical rapamycin in reducing facial tumors associated with the disease .. | phase II clinical trial, to study the efficacy and safety of topical rapamycin in reducing facial angiofibromas. | Facial tumors (angiofibromas) associated with tuberous sclerosis complex.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Trade Name: Rapamune 1 mg/ml solución oral INN or Proposed INN: RAPAMUNE | Fundación Investigación Hospital Ramón y Cajal | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | Spain | |||
| 8 | NCT01526356 (ClinicalTrials.gov) | May 2012 | 1/2/2012 | Topical Rapamycin to Erase Angiofibromas in TSC | Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy | Angiofibromas;Tuberous Sclerosis | Drug: Placebo;Drug: Rapamycin | The University of Texas Health Science Center, Houston | NULL | Completed | N/A | N/A | All | 179 | Phase 2 | United States;Australia |
| 9 | JPRN-UMIN000006108 | 2011/08/01 | 03/08/2011 | Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases | tuberous sclerosis complex, vitiligo vulgaris, white macules due to congenital diseases | Application of 0.2% rapamycin gel (base is carboxyvinyl polymer) on white macules of the face twice a day for 12 weeks. When it is possible, application of 0.2% rapamycin gel twice a day for 12 weeks on white macules of the non-exposed part to the sun. | Department of Dermatology Graduate School of Medicine, Osaka University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 21 | Phase 2;Phase 3 | Japan | |
| 10 | EUCTR2010-022655-29-NL (EUCTR) | 30/05/2011 | 22/10/2010 | Randomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATE | Randomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATE | Epilepsy in children with Tuberous Sclerosis Complex | Trade Name: Rapamune | Erasmus MC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Netherlands | ||||
| 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 | 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 |
| 12 | JPRN-UMIN000002844 | 2009/09/01 | 03/12/2009 | Development of topical treatment with rapamycin for skin lesions of tuberous sclerosis complex | tuberous sclerosis complex | Application of 0.2% Rapamycin ointment (ointment base is 0.03% tacrolimus ointment) on skin lesion of one side of the face twice a day for 12 weeks. Application of ointment base (control) on similar skin lesion of the other side of the face twice a day for 12 weeks. Application of 0.2% Rapamycin ointment (ointment base is white petrolatum) on skin lesion of one side of the face twice a day for 12 weeks. Application of ointment base (control) on similar skin lesion of the other side of the face twice a day for 12 weeks. Application of 0.2% Rapamycin gel (base is carboxyvinyl polymer) on skin lesion of one side of the face twice a day for 12 weeks. Application of gel base (control) on similar skin lesion of the other side of the face twice a day for 12 weeks. | Department of DermatologyGraduate School of Medicine, Osaka University | NULL | Complete: follow-up complete | Not applicable | Not applicable | Male and Female | 21 | Phase 2;Phase 3 | Japan | |
| 13 | 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 |
| 14 | 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 | ||||
| 15 | 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 |
| 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 |
| 16 | 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 |
| 17 | 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 | NCT02240407 (ClinicalTrials.gov) | October 17, 2017 | 11/9/2014 | Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease | Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD) | Pompe Disease | Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;Drug: Rapamycin;Other: saline;Drug: Rituxan;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: Lidocaine;Drug: LMX 4 Topical Cream | University of Florida | Lacerta Therapeutics, Inc | Recruiting | 18 Years | 50 Years | All | 7 | 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 | NCT03243019 (ClinicalTrials.gov) | June 25, 2018 | 1/8/2017 | Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations | Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis | Lymphatic Malformation;Pediatric | Drug: rapamycin;Device: MRI;Biological: Rapamycin dosage | University Hospital, Lille | Ministry of Health, France | Recruiting | 1 Year | 18 Years | All | 28 | 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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 |