158. 結節性硬化症 Tuberous sclerosis Clinical trials / Disease details
臨床試験数 : 112 / 薬物数 : 71 - (DrugBank : 19) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 118
| 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 | NCT05104983 (ClinicalTrials.gov) | October 13, 2021 | 22/10/2021 | Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study | Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study | Tuberous Sclerosis Complex;Epilepsy | Drug: Sirolimus;Drug: Placebo | Darcy Krueger | NULL | Recruiting | 1 Day | 6 Months | All | 64 | Phase 2 | United States |
| 2 | NCT04987463 (ClinicalTrials.gov) | May 7, 2021 | 28/5/2021 | Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants | Randomized, 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 | Tuberous Sclerosis Complex | Drug: Vigabatrin;Drug: Rapamycin;Drug: Placebo | Katarzyna Kotulska | NULL | Recruiting | 4 Weeks | 16 Weeks | All | 60 | Phase 2/Phase 3 | Poland |
| 3 | 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) | Children's Hospital Medical Center, Cincinnati | NULL | Active, not recruiting | N/A | 6 Months | All | 5 | Phase 1/Phase 2 | United States |
| 4 | 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 | ||
| 5 | 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] | 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 | DSLP | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;Czech Republic;Hungary;Slovakia;Spain;Australia;United Kingdom;New Zealand | ||
| 6 | 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] | Product Name: Rapamycin cream, topical, 0.5%, 1.0% w/w Product Code: Not applicable INN or Proposed INN: SIROLIMUS | DSLP | NULL | Authorised-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): no | United States;Hungary;Slovakia;Spain;Australia;New Zealand;United Kingdom | ||
| 7 | 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 | 21 Years | All | 45 | Phase 2 | United States;China |
| 8 | 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 |
| 9 | 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 |
| 10 | 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 | Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex - 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 2,3 | Japan |
| 11 | 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 |
| 12 | 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. | Randomized, double-blind, placebo-controlled, clinical trial with OSD-001 for skin lesions due to tuberous sclerosis complex. - Clinical trial for development of 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 1,2 | Japan |
| 13 | 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 |
| 14 | 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 |
| 15 | 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 | |||
| 16 | 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 |
| 17 | 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 | Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases - 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 2,3 | Japan |
| 18 | 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 | ||||
| 19 | 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 |
| 20 | JPRN-UMIN000002844 | 2009/09/01 | 03/12/2009 | Development of topical treatment with rapamycin for skin lesions of tuberous sclerosis complex | Development of topical treatment with rapamycin for skin lesions of tuberous sclerosis complex - 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 2,3 | Japan |
| 21 | 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 |
| 22 | 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 | ||||
| 23 | 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 |
| 24 | 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 |
| 25 | EUCTR2021-004548-64-PL (EUCTR) | 08/12/2022 | Placebo controlled study to assess the efficacy and safety of rapamycin in drug resistant epilepsy associated with tuberous sclerosis complex | Multicenter, randomized, double-blind, placebo controlled study to assess the efficacy and safety of Rapamycin in drug Resistant Epilepsy associated with TSC (RaRE-TS) - RaRE-TS | tuberous sclerosis complex, epilepsy, organ tumors associated with tuberous sclerosis;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Rapamune 1 mg/ml oral solution | The Children's Memorial Health Institute | NULL | NA | Female: yes Male: yes | 200 | Phase 3 | Poland | |||
| 26 | EUCTR2020-003231-19-PL (EUCTR) | 09/10/2020 | Comparison of the efficacy and safety of rapamycin versus vigabatrin in the prevention of Tuberous Sclerosis Complex symptoms in infants in the randomized clinical trial | Randomized, 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 Institute | NULL | NA | Female: yes Male: yes | 60 | Phase 2;Phase 3 | Poland | |||
| 27 | 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 |