Rapamycin ( DrugBank: Rapamycin )


19 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18
193プラダー・ウィリ症候群1
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

2. 筋萎縮性側索硬化症


臨床試験数 : 624 薬物数 : 611 - (DrugBank : 160) / 標的遺伝子数 : 172 - 標的パスウェイ数 : 225
No.TrialIDDate_
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PhaseCountries
1NCT03359538
(ClinicalTrials.gov)
September 19, 20178/11/2017Rapamycin Treatment for ALSRapamycin (Sirolimus) Treatment for Amyotrophic Lateral SclerosisAmyotrophic Lateral SclerosisDrug: Rapamycin;Drug: Placebo Oral TabletAzienda Ospedaliero-Universitaria di ModenaUniversity 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 PadovaActive, not recruiting18 Years75 YearsAll63Phase 2Italy
2EUCTR2016-002399-28-IT
(EUCTR)
14/07/201723/01/2018Rapamycin (Sirolimus) treatment for amyotrophic lateral sclerosisRapamycin (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 MODENANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
63Phase 2Italy

15. 封入体筋炎


臨床試験数 : 42 薬物数 : 60 - (DrugBank : 16) / 標的遺伝子数 : 12 - 標的パスウェイ数 : 123
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT02481453
(ClinicalTrials.gov)
July 15, 201513/5/2015Rapamycine vs Placebo for the Treatment of Inclusion Body MyositisÉtude de l'Effet de la Rapamycine Sur la Force Musculaire et la réponse Immunitaire au Cours de la Myosite à Inclusions: étude RAPAMIInclusion Body Myositis (IBM)Drug: Rapamycin;Drug: PlaceboInstitut National de la Santé Et de la Recherche Médicale, FranceNULLCompleted45 Years85 YearsAll44Phase 2/Phase 3France

34. 神経線維腫症


臨床試験数 : 137 薬物数 : 213 - (DrugBank : 76) / 標的遺伝子数 : 87 - 標的パスウェイ数 : 200
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1JPRN-UMIN000015081
2014/09/0807/09/2014Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1 neurofibromatosis type11) 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 UniversityNULLRecruiting20years-oldNot applicableMale and Female3Phase 2,3Japan
2NCT01031901
(ClinicalTrials.gov)
December 200910/12/2009Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;NeurofibromaDrug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycinThe University of Texas Health Science Center, HoustonSociety for Pediatric DermatologyCompleted13 YearsN/ABoth52Phase 1United States
3NCT00901849
(ClinicalTrials.gov)
May 200713/5/2009Tarceva/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 GliomasDrug: Tarceva and RapamycinRoger PackerChildren's Research InstituteCompletedN/A21 YearsBoth21Phase 1United States

35. 天疱瘡


臨床試験数 : 98 薬物数 : 126 - (DrugBank : 41) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 168
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT01313923
(ClinicalTrials.gov)
February 201110/3/2011Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis PemphigusEvaluation of Sirolimus for the Treatment of the Autoimmune Blistering Dermatosis PemphigusPemphigusDrug: Sirolimus (formerly known as Rapamycin)University of California, IrvineNULLTerminated18 YearsN/AAll3Early Phase 1United States

46. 悪性関節リウマチ


臨床試験数 : 4,325 薬物数 : 2,671 - (DrugBank : 417) / 標的遺伝子数 : 187 - 標的パスウェイ数 : 224
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1ChiCTR-IPR-17010307
2017-01-012017-01-01The efficacy and safety of rapamycin in refractory rheumatoid arthritis: A multi-center randomized controlled trial in ChinaThe efficacy and safety of rapamycin in refractory rheumatoid arthritis: A multi-center randomized controlled trial in China Rheumatoid Arthritis;M05.901Rapamycin group:Rapamycin;Non-rapamycin group :conventional glucocorticoids and DMARDs;the Second Hospital of Shanxi Medical UniversityNULLRecruiting1865BothRapamycin group:200;Non-rapamycin group :100;China

49. 全身性エリテマトーデス


臨床試験数 : 946 薬物数 : 722 - (DrugBank : 186) / 標的遺伝子数 : 117 - 標的パスウェイ数 : 199
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1ChiCTR-IPR-16009451
2016-11-012016-10-16The Efficacy and Safety of Low Dose IL-2 Conbined Rapamycin Therapy in Chinese Over-treated Patients with Systemic Lupus ErythematosusThe Efficacy and Safety of Low Dose IL-2 Conbined Rapamycin Therapy in Chinese Over-treated Patients with Systemic Lupus Erythematosus Systemic Lupus ErythematosusIL-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 UniversityNULLPending1880BothIL-2 group:20;IL-2 + Rapamycin group :20;non IL-2 or IL-6 antagonist group :20;China
2NCT00779194
(ClinicalTrials.gov)
October 200823/10/2008Prospective Study of Rapamycin for the Treatment of SLEProspective Study of Rapamycin for the Treatment of SLESystemic Lupus Erythematosus (SLE)Drug: RapamycinState University of New York - Upstate Medical UniversityPfizer;PfizerCompleted18 YearsN/ABoth40Phase 2United States

51. 全身性強皮症


臨床試験数 : 523 薬物数 : 608 - (DrugBank : 156) / 標的遺伝子数 : 114 - 標的パスウェイ数 : 215
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT00241189
(ClinicalTrials.gov)
August 200214/10/2005Rapamycin vs Methotrexate in Diffuse SScA 48-week, Double-blind, Randomized, Parallel Phase I/II Study of Oral Rapamycin Versus Methotrexate in Systemic Sclerosis (Scleroderma)Systemic SclerosisDrug: rapamycin;Drug: methotrexateUniversity of California, Los AngelesNULLCompleted18 Years70 YearsBoth17Phase 1/Phase 2United States

60. 再生不良性貧血


臨床試験数 : 235 薬物数 : 381 - (DrugBank : 83) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 160
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1EUCTR2006-006577-25-SE
(EUCTR)
25/07/200711/06/2007A prospective randomized study comparing rapamune and tacrolimus vs. cyclosporine and methotrexate as immune prophylaxis in allogeneic hematopoietic stem cell transplantation, using HLA-A, -B, -DRB1 identical related or unrelated donors. A Nordic multicenter study. - Rapa + FK in stem cell transplantationA prospective randomized study comparing rapamune and tacrolimus vs. cyclosporine and methotrexate as immune prophylaxis in allogeneic hematopoietic stem cell transplantation, using HLA-A, -B, -DRB1 identical related or unrelated donors. A Nordic multicenter study. - Rapa + FK in stem cell transplantation Graft versus host disease prophylaxis in patients receiving stem cell transplantation due to: chronic myeloid leukemia (CML) in 1st or 2nd chronic phase, acute myeloid leukemia (AML) in complete remission, acute lymphoblastic leukemia (ALL) in complete remission, myelodysplastic syndrome, chronic lymphocytic leukemia, lymphoma, non-malignant disorders, severe aplastic anemia, hemoglobinopathies and metabolic disorders
MedDRA version: 9.1;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 InstitutetNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Finland;Sweden

61. 自己免疫性溶血性貧血


臨床試験数 : 137 薬物数 : 127 - (DrugBank : 55) / 標的遺伝子数 : 26 - 標的パスウェイ数 : 153
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1ChiCTR1900023476
2019-07-012019-05-30Multicenter, prospective and open clinical study for rapamycin in the treatment of autoimmune hemolytic anemiaMulticenter, prospective and open clinical study for rapamycin in the treatment of autoimmune hemolytic anemia Autoimmune1:glucocorticoid;2:glucocorticoid & Ciclosporin;3:glucocorticoid & rapamycin;4:rapamycin;Tianjin Medical University General HospitalNULLPendingBoth1:20;2:20;3:20;4:20;China

67. 多発性嚢胞腎


臨床試験数 : 216 薬物数 : 219 - (DrugBank : 50) / 標的遺伝子数 : 39 - 標的パスウェイ数 : 151
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT01632605
(ClinicalTrials.gov)
November 200913/5/2012The Vienna RAP Pilot StudyRapamycin in Advanced Polycystic Kidney Disease Pilot StudyADPKDDrug: SirolimusMedical University of ViennaNULLCompleted18 YearsN/ABoth8N/AAustria
2NCT00920309
(ClinicalTrials.gov)
June 200912/6/2009Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to TherapyRapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to TherapyAutosomal Dominant Polycystic Kidney DiseaseDrug: Rapamycin;Other: Standard of Care-PlaceboYale UniversityNULLTerminated18 Years70 YearsAll21Phase 2/Phase 3United States
3EUCTR2007-006557-25-IT
(EUCTR)
20/12/200718/12/2007RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDYRAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE - RAPYD-STUDY ADPKD type I
MedDRA version: 9.1;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 CONSORZIALENULLNot RecruitingFemale: yes
Male: yes
Italy
4NCT00286156
(ClinicalTrials.gov)
October 20061/2/2006Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD)Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney DiseasePolycystic Kidney DiseasesDrug: RapamuneThe Cleveland ClinicWyeth is now a wholly owned subsidiary of PfizerCompleted18 Years75 YearsAll30Phase 1/Phase 2United States

86. 肺動脈性肺高血圧症


臨床試験数 : 1,181 薬物数 : 701 - (DrugBank : 126) / 標的遺伝子数 : 105 - 標的パスウェイ数 : 192
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1NCT02587325
(ClinicalTrials.gov)
April 1, 201723/10/2015ABI-009, an mTOR Inhibitor, for Patients With Severe Pulmonary Arterial HypertensionA Phase 1 Clinical Trial of ABI-009, an mTOR Inhibitor, for Patients With Severe Pulmonary Arterial Hypertension (PAH)Pulmonary HypertensionDrug: ABI-009, nab-rapamycin, albumin-bound rapamycinAadi, LLCNULLRecruiting18 YearsN/AAll25Phase 1United States

89. リンパ脈管筋腫症


臨床試験数 : 39 薬物数 : 46 - (DrugBank : 20) / 標的遺伝子数 : 27 - 標的パスウェイ数 : 137
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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agemax
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PhaseCountries
1NCT00490789
(ClinicalTrials.gov)
October 200521/6/2007Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAMA Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: sirolimusCardiff UniversityUniversity of Nottingham;St Georges Hospital Medical School;Royal Sussex County Hospital;The Tuberous Sclerosis Association;Wyeth is now a wholly owned subsidiary of PfizerActive, not recruiting18 Years65 YearsBoth14Phase 2United Kingdom
2NCT00457808
(ClinicalTrials.gov)
December 20026/4/2007Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAMRapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: Rapamycin, sirolimusChildren's Hospital Medical Center, CincinnatiThe LAM Foundation;Tuberous Sclerosis AllianceCompleted18 Years65 YearsBoth25Phase 2United States

96. クローン病


臨床試験数 : 2,400 薬物数 : 1,391 - (DrugBank : 267) / 標的遺伝子数 : 170 - 標的パスウェイ数 : 215
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT02675153
(ClinicalTrials.gov)
April 1, 201521/1/2016To Evaluate the Efficacy and Safety of Rapamycin for Crohn's Disease-related StrictureEfficacy and Safety of Rapamycin in the Treatment of Crohn's Disease-related StrictureCrohn's DiseasesDrug: RapamycinThe Second Hospital of Nanjing Medical UniversityNULLRecruiting18 YearsN/AAll15N/AChina

157. スタージ・ウェーバー症候群


臨床試験数 : 10 薬物数 : 14 - (DrugBank : 4) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 63
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT02080624
(ClinicalTrials.gov)
January 201118/4/2013Efficacy and Safety Study of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber SyndromePhase 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 SyndromeDrug: Drug: Topical RapamycinClinica Universidad de Navarra, Universidad de NavarraNULLCompleted16 Years65 YearsBoth23Phase 2Spain

158. 結節性硬化症


臨床試験数 : 108 薬物数 : 67 - (DrugBank : 17) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 118
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Recruitment_
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agemin
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PhaseCountries
1EUCTR2019-000752-34-SK
(EUCTR)
15/11/201901/10/2019 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]
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
DSLPNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Hungary;Slovakia;Spain;Australia;New Zealand;United Kingdom
2EUCTR2019-000752-34-HU
(EUCTR)
14/11/201918/11/2019 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]
Product Name: Rapamycin cream, topical, 0.5%, 1.0% w/w
Product Code: Not applicable
INN or Proposed INN: SIROLIMUS
DSLPNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Hungary;Slovakia;Spain;Australia;New Zealand;United Kingdom
3NCT03826628
(ClinicalTrials.gov)
July 28, 201929/1/2019Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis ComplexA 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 OverFacial Angiofibroma;Tuberous SclerosisDrug: rapamycin;Drug: placeboDermatology Specialties Limited PartnershipNULLRecruiting6 Years65 YearsAll120Phase 2/Phase 3United States;Australia;Czechia;Hungary;New Zealand;Serbia;Slovakia;Spain;Taiwan;United Kingdom
4ChiCTR-OOB-15006535
2015-05-262015-05-29A Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis ComplexA Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis Complex tuberous sclerosis complexCase series:rapamycin;Chinese PLA General HospitalNULLRecruiting118BothCase series:500;China
5JPRN-UMIN000015114
2014/09/1110/09/2014Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex Tuberous sclerosis complexApplication 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 UniversityNULLRecruitingNot applicable70years-oldMale and Female24Phase 2,3Japan
6NCT03140449
(ClinicalTrials.gov)
September 5, 201326/4/2017Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous SclerosisStudy of Combination Therapy With Topical Rapamycin and Calcitriol for Cutaneous Lesions of Tuberous Sclerosis: A Double-blind Randomized Controlled TrialFacial AngiofibromaDrug: Rapamycin;Drug: Calcitriol;Drug: Rapamycin-calcitriol combinationNational Taiwan University HospitalNULLCompleted7 Years65 YearsAll52Phase 3NULL
7EUCTR2011-006308-12-ES
(EUCTR)
16/07/201229/03/2012Clinical 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 CajalNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Spain
8NCT01526356
(ClinicalTrials.gov)
May 20121/2/2012Topical Rapamycin to Erase Angiofibromas in TSCPhase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel TherapyAngiofibromas;Tuberous SclerosisDrug: Placebo;Drug: RapamycinThe University of Texas Health Science Center, HoustonNULLCompletedN/AN/AAll179Phase 2United States;Australia
9JPRN-UMIN000006108
2011/08/0103/08/2011Clinical 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 diseasesApplication 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 UniversityNULLRecruitingNot applicableNot applicableMale and Female21Phase 2,3Japan
10EUCTR2010-022655-29-NL
(EUCTR)
30/05/201122/10/2010Randomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATERandomised 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 ComplexTrade Name: RapamuneErasmus MCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Netherlands
11NCT01031901
(ClinicalTrials.gov)
December 200910/12/2009Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;NeurofibromaDrug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycinThe University of Texas Health Science Center, HoustonSociety for Pediatric DermatologyCompleted13 YearsN/ABoth52Phase 1United States
12JPRN-UMIN000002844
2009/09/0103/12/2009Development of topical treatment with rapamycin for skin lesions of tuberous sclerosis complex tuberous sclerosis complexApplication 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 UniversityNULLComplete: follow-up completeNot applicableNot applicableMale and Female21Phase 2,3Japan
13NCT01217125
(ClinicalTrials.gov)
October 20086/10/2010Rapamycin In Angiomyolipomas In Patients With Tuberous SclerosisCLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSISAngiomyolipomaDrug: SirolimusFundacio PuigvertMinistry of Health, SpainCompleted10 YearsN/ABoth18Phase 4NULL
14EUCTR2007-005978-30-ES
(EUCTR)
22/01/200803/12/2007Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous SclerosisEnsayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous Sclerosis Angiomiolipomas en pacientes con esclerosis tuberosa (angiomyolipoma of tuberous sclerosis patients)
MedDRA version: 9.1;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 PUIGVERTNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Spain
15NCT00490789
(ClinicalTrials.gov)
October 200521/6/2007Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAMA Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: sirolimusCardiff UniversityUniversity of Nottingham;St Georges Hospital Medical School;Royal Sussex County Hospital;The Tuberous Sclerosis Association;Wyeth is now a wholly owned subsidiary of PfizerActive, not recruiting18 Years65 YearsBoth14Phase 2United Kingdom
16NCT00457808
(ClinicalTrials.gov)
December 20026/4/2007Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAMRapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: Rapamycin, sirolimusChildren's Hospital Medical Center, CincinnatiThe LAM Foundation;Tuberous Sclerosis AllianceCompleted18 Years65 YearsBoth25Phase 2United States
17EUCTR2019-000752-34-CZ
(EUCTR)
02/06/2020Study to evaluate if the study medication, Rapamycin, topical cream, is effective and safe at treating facial angiofibroma in patients 6 years of age and over.A Phase 2/3, multi-center, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison of the efficacy and safety of a topical rapamycin cream for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and over - Dose-ranging efficacy and safety study of topical rapamycin cream Facial Angiofibromas Associated with Tuberous Sclerosis Complex
MedDRA version: 20.0;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)NULLNAFemale: yes
Male: yes
120Phase 2;Phase 3United States;Serbia;Hungary;Czech Republic;Slovakia;Spain;Australia;New Zealand
18EUCTR2020-003231-19-PL
(EUCTR)
09/10/2020Comparison of the efficacy and safety of rapamycin versus vigabatrin in the prevention of Tuberous Sclerosis Complex symptoms in infants in the randomized clinical trialRandomized, placebo-controlled, double-blind and double-dummy clinical trial comparing the safety, tolerability, and efficacy of vigabatrin and rapamycin in a preventive treatment of infants with Tuberous Sclerosis Complex (ViRap) - ViRap Tuberous Sclerosis ComplexEpilepsyTumors associated with Tuberous Sclerosis Complex;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Trade Name: SABRIL 500 mg, granules for oral solution
Trade Name: RAPAMUNE 1 mg/ml oral solution
The Children's Memorial Health InstituteNULLNAFemale: yes
Male: yes
60Phase 2;Phase 3Poland

193. プラダー・ウィリ症候群


臨床試験数 : 111 薬物数 : 120 - (DrugBank : 30) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 103
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1ChiCTR2000040947
2021-01-012020-12-16The safety and effectiveness of rapamycin (sirolimus) in the treatment of Prader-Willi syndromeThe safety and effectiveness of rapamycin (sirolimus) in the treatment of Prader-Willi syndrome Prader-Willi syndromerapamycin group:rapamycin 1mg qd po;Peking Union Medical College HospitalNULLRecruitingBothrapamycin group:30;China

256. 筋型糖原病


臨床試験数 : 180 薬物数 : 153 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 134
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02240407
(ClinicalTrials.gov)
October 17, 201711/9/2014Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe DiseaseEvaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD)Pompe DiseaseGenetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;Drug: Rapamycin;Other: saline;Drug: Rituxan;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: Lidocaine;Drug: LMX 4 Topical CreamUniversity of FloridaNULLActive, not recruiting18 Years50 YearsAll2Phase 1United States

278. 巨大リンパ管奇形(頚部顔面病変)


臨床試験数 : 22 薬物数 : 27 - (DrugBank : 7) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 63
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03243019
(ClinicalTrials.gov)
June 25, 20181/8/2017Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic MalformationsEvaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor PrognosisLymphatic Malformation;PediatricDrug: rapamycin;Device: MRI;Biological: Rapamycin dosageUniversity Hospital, LilleMinistry of Health, FranceRecruiting1 Year18 YearsAll28Phase 2France

279. 巨大静脈奇形(頚部口腔咽頭びまん性病変)


臨床試験数 : 14 薬物数 : 24 - (DrugBank : 9) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 105
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03767660
(ClinicalTrials.gov)
July 31, 201824/11/2018Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous MalformationEfficacy of Rapamycin (Sirolimus) in the Treatment of Blue Rubber Bleb Nevus Syndrome, Hereditary or Sporadic Venous MalformationBlue Rubber Bleb Nevus Syndrome;Venous MalformationDrug: RapamycinPeking Union Medical College HospitalAir Force General Hospital of the PLA;Chinese Academy of Medical SciencesRecruitingN/AN/AAll20Phase 4China