158. 結節性硬化症 ［臨床試験数：54，薬物数：37（DrugBank：14），標的遺伝子数：19，標的パスウェイ数：106］
Searched query = "Tuberous sclerosis", "Tuberous sclerosis complex"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03826628 | July 28, 2019 | 4 November 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 | Recruiting | 6 Years | 65 Years | All | 120 | Phase 2/Phase 3 | United States;Australia;New Zealand;Spain | |
| 2 | NCT03525834 | November 9, 2018 | 28 October 2019 | Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex. | Phase IV, Single Arm Study of Safety and Efficacy of Everolimus in Chinese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma Not Requiring Immediate Surgery | Renal Angiomyolipoma | Drug: everolimus | Novartis Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 40 | Phase 4 | China | |
| 3 | EUCTR2018-002531-18-FR | 31/10/2018 | 28 February 2019 | Topical Everolimus versus placebo for the treatment of facial angiofibromas in patients with tuberous sclerosis complex. | Topical Everolimus versus placebo for the treatment of facial angiofibromas in patients with tuberous sclerosis complex. A phase II/III, multicentre, randomized, double-blind, placebo-controlled study of 3 doses of topical Everolimus. EVEROST | angiofibromas MedDRA version: 20.0 Level: PT Classification code 10002429 Term: 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: Everolimus Pharmaceutical Form: Cream INN or Proposed INN: EVEROLIMUS CAS Number: 159351-69-6 Other descriptive name: EVEROLIMUS Concentration unit: % (V/V) percent volume/volume Concentration type: equal Concentration number: 0.3- Pharmaceutical form of the placebo: Cream Route of administration of the placebo: Topical use (Noncurrent) Product Name: Everolimus Pharmaceutical Form: Cream INN or Proposed INN: Everolimus CAS Number: 159351-69-6 Other descriptive name: EVEROLIMUS Concentration unit: % (V/V) percent volume/volume Concentration type: equal Concentration number: 0.7- Pharmaceutical form of the placebo: Cream Route of administration of the placebo: Topical use (Noncurrent) Product Name: Everolimus Pharmaceutical Form: Cream INN or Proposed INN: Everolimus CAS Number: 159351-69-6 Other descriptive name: EVEROLIMUS Concentration unit: % (V/V) percent volume/volume Concentration type: equal Concentration number: 1- Pharmaceutical form of the placebo: Cream Route of administration of the placebo: Topical use (Noncurrent) | Hospices Civils de Lyon | Authorised | Female: yes Male: yes | 146 | Phase 2;Phase 3 | France | |||
| 4 | NCT02860494 | September 2018 | 5 March 2018 | Topical Everolimus in Patients With Tuberous Sclerosis Complex | Topical Everolimus Versus Placebo for the Treatment of Facial Angiofibromas in Patients With Tuberous Sclerosis Complex. A Phase II/III, Multicentre, Randomized, Double-blind, Placebo-controlled Study of 3 Doses of Topical Everolimus. | Facial Angiofibromas | Drug: Placebo;Drug: Everolimus | Hospices Civils de Lyon | Not recruiting | N/A | 2 Years | All | 96 | Phase 2/Phase 3 | France | |
| 5 | NCT03254680 | March 2018 | 11 June 2018 | Turmeric as Treatment in Epilepsy | Turmeric as Treatment in Epilepsy | Epilepsy;Dravet Syndrome;Lennox-Gastaut Syndrome;Tuberous Sclerosis;Focal Seizures | Dietary Supplement: Turmeric | New York University School of Medicine | Not recruiting | 1 Year | 70 Years | All | 0 | N/A | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03356769 | November 20, 2017 | 16 December 2017 | Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex | A Placebo-controlled Study of Efficacy & Safety of Aspirin as an add-on Treatment in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Seizures | Tuberous Sclerosis Complex;Aspirin;Epilepsy;Cognitive Decline;Skin Lesions | Drug: Aspirin;Drug: AED;Drug: Placebo | Peking Union Medical College Hospital | Shijiazhuang Pharmaceutical Group Co. Ltd. | Recruiting | 6 Years | 30 Years | All | 98 | Phase 2 | China |
| 7 | NCT03363763 | April 12, 2017 | 30 September 2019 | 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 | Recruiting | 2 Years | 18 Years | All | 45 | Phase 2 | United States | |
| 8 | NCT02962414 | April 2, 2017 | 4 November 2019 | Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment | An Open-label, Multi-center Long-term Safety Roll-over Study in Patients With Tuberous Sclerosis Complex (TSC) and Refractory Seizures Who Are Judged by the Investigator to Benefit From Continued Treatment With Everolimus After Completion of Study CRAD001M2304. | Tuberous Sclerosis Complex | Drug: everolimus | Novartis Pharmaceuticals | Not recruiting | 2 Years | 65 Years | All | 206 | Phase 3 | United States;Australia;Belgium;Canada;Colombia;France;Hungary;Italy;Japan;Korea, Republic of;Mexico;Poland;Russian Federation;Spain;Taiwan;Thailand;Turkey;United Kingdom | |
| 9 | EUCTR2016-002977-37-HU | 24/02/2017 | 13 March 2017 | Roll-over study in patients with TSC and refractory seizures who are judged by the Investigator to benefit from continued treatment with everolimus after completion of study CRAD001M2304. | An open-label, multi-center long-term safety roll-over study in patients with tuberous sclerosis complex (TSC) and refractory seizures who are judged by the Investigator to benefit from continued treatment with everolimus after completion of study CRAD001M2304. | Refractory seizures associated with tuberous sclerosis complex MedDRA version: 19.1 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Votubia Product Name: Everolimus Product Code: RAD001 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Current Sponsor code: RAD001 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2- | Novartis Pharma AG | Authorised | Female: yes Male: yes | 216 | Phase 3 | United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of | |||
| 10 | EUCTR2016-002408-13-NL | 07/02/2017 | 2 October 2017 | Bumetanide to treat behavioral problems in TSC | Bumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors - BATSCH | Tuberous Sclerosis Complex MedDRA version: 20.0 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03] | Trade Name: bumetanide Product Name: bumetanide Product Code: bumetanide Pharmaceutical Form: Tablet | UMC Utrecht | Authorised | Female: yes Male: yes | Phase 2 | Netherlands | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT02849457 | December 2016 | 26 August 2019 | Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex | Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (PREVeNT Trial) A Randomized, Double-blind, Placebo-controlled Seizure Prevention Clinical Trial for Infants With TSC | Tuberous Sclerosis Complex | Drug: Vigabatrin;Drug: Placebo | Martina Bebin | National Institute of Neurological Disorders and Stroke (NINDS) | Recruiting | N/A | 6 Months | All | 80 | Phase 2 | United States |
| 12 | NCT02544750 | August 2016 | 9 October 2018 | An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) | A Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P, CBD) as Add-on Therapy in Patients With Tuberous Sclerosis Complex Who Experience Inadequately-controlled Seizures | Tuberous Sclerosis Complex;Seizures | Drug: GWP42003-P | GW Research Ltd | Recruiting | 1 Year | 65 Years | All | 210 | Phase 3 | United States | |
| 13 | ChiCTR-OPN-16008236 | 2016-06-01 | 18 April 2017 | TSC1/2 gene detection and Everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complex | TSC1/2 gene detection and Everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complex | renal angiomyolipoma | 10mg Everolimus:Everolimus 10mg QD for three months;5mg Everolimus:Everolimus 5mg QD for six months; | General Hospital of PLA | Not Recruiting | 18 | 60 | Both | 10mg Everolimus:50;5mg Everolimus:50; | Post-market | China | |
| 14 | NCT02634931 | December 2015 | 4 March 2019 | 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 | Not recruiting | 3 Years | N/A | All | 94 | Phase 3 | Japan | |
| 15 | NCT02635789 | December 2015 | 16 December 2017 | 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 | Not recruiting | 3 Years | N/A | All | 62 | Phase 3 | Japan | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | ChiCTR-OOB-15006535 | 2015-05-26 | 18 April 2017 | 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 | Recruiting | 1 | 18 | Both | Case series:500; | Other | China | |
| 17 | NCT02104011 | May 22, 2015 | 29 January 2018 | Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers | Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers: Pilot Trial | Renal Angiomyolipomas;Tuberous Sclerosis | Drug: Propranolol | University Hospital, Bordeaux | Not recruiting | 18 Years | N/A | All | 2 | Phase 2 | France | |
| 18 | EUCTR2015-002154-12-PL | 28 February 2019 | A study of cannabidiol (GWP42003-P, CBD) in patients with tuberous sclerosis complex who experience seizures. | A double-blind, randomized, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P, CBD) as add-on therapy in patients with tuberous sclerosis complex who experience inadequately-controlled seizures | Tuberous Sclerosis Complex (TSC) MedDRA version: 20.0 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Cannabidiol (CBD) Product Code: GWP42003-P Pharmaceutical Form: Oral solution INN or Proposed INN: N/A CAS Number: 13956-29-1 Current Sponsor code: GWP42003-P Other descriptive name: CANNABIDIOL Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use | GW Research Ltd. | Not Available | Female: yes Male: yes | 210 | Phase 3 | United States;Spain;Poland;Australia;Netherlands;United Kingdom | ||||
| 19 | EUCTR2013-003795-13-BE | 10/12/2014 | 11 June 2018 | Long-term monitoring of growth and development of pediatric patients previously treated with everolimus | Long-term follow-up study to monitor the growth and development of pediatric patients previously treated with everolimus in study CRAD001M2301 - EXIST-LT | Subependymal Giant Cell Astrocytoma (SEGA) associated with Tuberous Sclerosis Complex (TSC) MedDRA version: 20.0 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Votubia Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Current Sponsor code: RAD001 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10- Trade Name: Votubia Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Current Sponsor code: RAD001 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2- Trade Name: Votubia Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Current Sponsor code: RAD001 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2.5- Trade Name: Votubia Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Current Sponsor code: RAD001 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Trade Name: Votubia Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Current Sponsor code: RAD001 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 3- Trade Name: Votubia Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Current Sponsor code: RAD001 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- | Novartis Pharma Services AG | Authorised | Female: yes Male: yes | 48 | Phase 3;Phase 4 | United States;Canada;Poland;Belgium;Russian Federation | |||
| 20 | JPRN-UMIN000015114 | 2014/09/11 | 2 April 2019 | 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 | Recruiting | Not applicable | 70years-old | Male and Female | 24 | Phase 2,3 | Japan | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT02201212 | September 2014 | 11 November 2019 | Everolimus for Cancer With TSC1 or TSC2 Mutation | A Phase II Trial of Everolimus for Cancer Patients With Inactivating Mutations in TSC1 or TSC2 or Activating MTOR Mutations | TSC1;TSC2;Tuberous Sclerosis Complex;MTOR | Drug: Everolimus | Dana-Farber Cancer Institute | Novartis Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 30 | Phase 2 | United States |
| 22 | NCT02061397 | March 2014 | 25 February 2019 | 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 | University of Pennsylvania | The LAM Foundation | Not recruiting | 18 Years | N/A | Female | 10 | Phase 1/Phase 2 | United States |
| 23 | NCT02451696 | January 2014 | 30 September 2019 | A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD | A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD | Epilepsy;Tuberous Sclerosis Complex;Focal Cortical Dysplasia | Drug: Everolimus | NYU Langone Health | Recruiting | 2 Years | 40 Years | All | 30 | Phase 2 | United States | |
| 24 | JPRN-UMIN000012420 | 2013/12/10 | 2 April 2019 | 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 Dermatology Graduate School of Medicine, Osaka University | Not Recruiting | 3years-old | 65years-old | Male and Female | 36 | Phase 1,2 | Japan | ||
| 25 | NCT01853423 | October 2013 | 29 August 2016 | Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream | Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream | Facial Angiofibroma | Drug: Rapamune | Le Bonheur Children's Hospital | Pfizer;University of Tennessee Health Science Center | Not recruiting | 3 Years | 45 Years | Both | 11 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT03140449 | September 5, 2013 | 16 December 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 | Not recruiting | 7 Years | 65 Years | All | 52 | Phase 3 | ||
| 27 | JPRN-UMIN000011559 | 2013/09/01 | 2 April 2019 | Therpeutic drug monitoring of Everolimus in patients with renal cell carcinoma and tuberous sclerosis | 1.advanced renal cell carcinoma 2.renal angiomyolipoma associated with tuberous sclerosis complex | Everolimus 5mg/day. | Department of Urology, Keio University School of Medicine | Not Recruiting | 20years-old | 90years-old | Male and Female | 43 | Not selected | Japan | ||
| 28 | NCT01929642 | July 2013 | 5 February 2018 | 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. | Not recruiting | 2 Years | 30 Years | All | 3 | Phase 2 | United States | |
| 29 | NCT01713946 | April 29, 2013 | 19 November 2018 | A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures | A Three-arm, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of Two Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) Who Have Refractory Partial-onset Seizures | Tuberous Sclerosis Complex-associated Refractory Seizures | Drug: RAD001;Drug: Placebo;Drug: Antiepileptic drug (1 to 3 only);Drug: open label RAD001 (only used for post-extension phase) | Novartis Pharmaceuticals | Not recruiting | 2 Years | 65 Years | All | 366 | Phase 3 | United States;Argentina;Australia;Belgium;Canada;Colombia;Denmark;France;Germany;Greece;Hungary;Ireland;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Thailand;Turkey;United Kingdom;Sweden | |
| 30 | EUCTR2012-005397-63-ES | 19/04/2013 | 27 October 2014 | Expanded access study of RAD001 in patient with angiomyolipoma associated with tuberous sclerosis complex (TSC) | An open-label, multi-center, expanded access study of RAD001 in patients with angiomyolipoma associated with tuberous sclerosis complex (TSC) | Angiomyolipoma associated with either Tuberous Sclerosis Complex (TSC) MedDRA version: 16.1 Level: PT Classification code 10051810 Term: Angiomyolipoma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Votubia Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- | Novartis Farmacéutica, S.A, | Not Recruiting | Female: yes Male: yes | Spain | |||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT01730209 | November 2012 | 25 May 2015 | Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex | Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex | Tuberous Sclerosis Complex;TSC Related Cognitive Disability;TSC Related Autism;TSC Related Learning Problems | Drug: Everolimus;Drug: Placebo | Erasmus Medical Center | Utrecht University | Recruiting | 4 Years | 15 Years | Both | 60 | Phase 2/Phase 3 | Netherlands |
| 32 | EUCTR2011-006308-12-ES | 16/07/2012 | 14 August 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 Pharmaceutical Form: Oral solution INN or Proposed INN: RAPAMUNE Current Sponsor code: SIROLIMUS Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 3- Pharmaceutical form of the placebo: Cutaneous solution Route of administration of the placebo: Cutaneous use | Fundación Investigación Hospital Ramón y Cajal | Authorised | Female: yes Male: yes | Phase 2 | Spain | ||||
| 33 | NCT01954693 | June 2012 | 12 February 2018 | A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis | TRON: A Randomised, Double Blind, Placebo-controlled Study of RAD001 (Everolimus) in the Treatment of Neurocognitive Problems in Tuberous Sclerosis | Tuberous Sclerosis | Drug: Placebo;Drug: Everolimus (RAD001) | Cardiff University | Novartis | Not recruiting | 16 Years | 60 Years | All | 48 | Phase 2 | United Kingdom |
| 34 | NCT01526356 | May 2012 | 29 June 2015 | 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 | Not recruiting | N/A | N/A | Both | 177 | Phase 2 | United States;Australia | |
| 35 | EUCTR2011-001319-30-GB | 04/04/2012 | 28 May 2018 | Metformin as a treatment for people with the genetic disease Tuberous Sclerosis Complex. | A randomized, double-blind, parallel group, placebo-controlled trial of metformin in tuberous sclerosis complex. - Metformin in Tuberous Sclerosis Complex (MiTS) V1.0 | Tuberous Sclerosis Complex MedDRA version: 14.1 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: metformin (generic) Product Name: Metformin Product Code: n/a Pharmaceutical Form: Tablet INN or Proposed INN: metformin hydrochloride CAS Number: 657-24-9 Current Sponsor code: - Other descriptive name: - Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | University Hospitals Bristol NHS Foundation Trust | Not Recruiting | Female: yes Male: yes | 100 | Phase 4 | United Kingdom | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2010-019519-39-NL | 14/03/2012 | 18 February 2013 | Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with tuberous sclerosis complex (RAPIT-trial) | Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (RAPIT-trial) - RAPIT | Tuberous Sclerosis Complex;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Votubia Product Name: Everolimus Product Code: L04AA18 Pharmaceutical Form: Tablet INN or Proposed INN: Votubia CAS Number: 159351-69-6 Other descriptive name: EVEROLIMUS Concentration unit: ng nanogram(s) Concentration type: range Concentration number: 5-15 Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Erasmus MC - Department of Neurology | Authorised | Female: yes Male: yes | Netherlands | |||||
| 37 | EUCTR2011-004854-25-GB | 10/01/2012 | 28 February 2019 | TRON: A trial to examine the effectiveness of the study drug (Everolimus) in the treatment of cognitive problems experienced by patients with tuberous sclerosis. | TRON: A randomised, double blind, placebo-controlled study of RAD001 (Everolimus) in the treatment of neurocognitive problems in tuberous sclerosis. - TRON (version 1.0) | Tuberous Sclerosis Complex (TSC) MedDRA version: 14.0 Level: PT Classification code 10002649 Term: Anorexia nervosa System Organ Class: 10037175 - Psychiatric disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Afinitor Product Name: Everolimus Pharmaceutical Form: Tablet INN or Proposed INN: Everolimus CAS Number: 159351-69-6 Other descriptive name: RAD001 Concentration unit: mg milligram(s) Concentration type: equal Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Cardiff University | Not Recruiting | Female: yes Male: yes | 48 | Phase 2 | United Kingdom | |||
| 38 | JPRN-UMIN000006108 | 2011/08/01 | 2 April 2019 | 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 | Recruiting | Not applicable | Not applicable | Male and Female | 21 | Phase 2,3 | Japan | ||
| 39 | EUCTR2010-022655-29-NL | 30/05/2011 | 19 March 2012 | 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 Pharmaceutical Form: Oral solution | Erasmus MC | Authorised | Female: yes Male: yes | Netherlands | |||||
| 40 | NCT01289912 | January 2011 | 5 February 2018 | Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) | Randomized Double-Blind Phase 2 Trial Of RAD001 For Neurocognition In Individuals With Tuberous Sclerosis Complex | Tuberous Sclerosis Complex | Drug: RAD001;Drug: Placebo | Mustafa Sahin | Tuberous Sclerosis Alliance;Autism Speaks;Novartis Pharmaceuticals;Seizure Tracker LLC | Not recruiting | 6 Years | 21 Years | All | 52 | Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | NCT01266291 | August 2010 | 16 December 2017 | Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients | Sabril for Treatment of Adult Refractory Partial Seizures Symptomatic of Tuberous Sclerosis: An Open Label, Phase IV Prospective Safety and Tolerability Study | Complex Partial Seizures | Drug: vigabatrin | University of Pennsylvania | H. Lundbeck A/S | Not recruiting | 18 Years | N/A | All | 1 | Phase 4 | United States |
| 42 | NCT01070316 | January 2010 | 16 December 2017 | Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) | Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex | Epilepsy;Tuberous Sclerosis Complex | Drug: Everolimus | Children's Hospital Medical Center, Cincinnati | Novartis | Not recruiting | 2 Years | N/A | All | 20 | Phase 1/Phase 2 | United States |
| 43 | EUCTR2010-022583-13-DE | 5 August 2014 | An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA - EFFECTS STUDY | An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA - EFFECTS STUDY | The study will evaluate the safety of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC). | Product Name: RAD001 2,5 mg Product Code: RAD001 Pharmaceutical Form: Tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Other descriptive name: EVEROLIMUS Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2.5- | Novartis Pharma Services AG | Not Recruiting | Female: yes Male: yes | 250 | Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy | |||||
| 44 | NCT01031901 | December 2009 | 19 February 2015 | 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 | Not recruiting | 13 Years | N/A | Both | 52 | Phase 1 | United States |
| 45 | JPRN-UMIN000002844 | 2009/09/01 | 2 April 2019 | 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 Dermatology Graduate School of Medicine, Osaka University | Not Recruiting | Not applicable | Not applicable | Male and Female | 21 | Phase 2,3 | Japan | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | NCT00789828 | August 2009 | 19 October 2017 | Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) | A Randomized, Double-blind, Placebo-controlled Study of Everolimus in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC) | Tuberous Sclerosis;Subependymal Giant Cell Astrocytoma | Drug: Everolimus;Drug: Placebo | Novartis Pharmaceuticals | Not recruiting | N/A | N/A | All | 117 | Phase 3 | United States;Australia;Belgium;Canada;Germany;Italy;Netherlands;Poland;Russian Federation;United Kingdom | |
| 47 | NCT00989742 | July 2009 | 14 December 2015 | Doxycycline In Lymphangioleiomyomatosis (LAM) | A Randomised, Double Blind, Placebo Controlled Trial of Doxycycline in Lymphangioleiomyomatosis. | Lymphangioleiomyomatosis;Tuberous Sclerosis | Drug: Doxycycline;Drug: Placebo | University of Nottingham | Not recruiting | 18 Years | N/A | Female | 24 | Phase 4 | United Kingdom | |
| 48 | NCT00790400 | April 2009 | 16 December 2017 | Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) | A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) | Tuberous Sclerosis Complex (TSC);Lymphangioleiomyomatosis (LAM) | Drug: Everolimus (RAD001);Drug: Everolimus Placebo | Novartis Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 118 | Phase 3 | United States;Canada;France;Germany;Italy;Japan;Netherlands;Poland;Russian Federation;Spain;United Kingdom | |
| 49 | NCT00792766 | December 2008 | 19 February 2015 | Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM) | Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis | Tuberous Sclerosis;Angiolipoma | Drug: everolimus (RAD001) | Children's Hospital Medical Center, Cincinnati | Novartis | Not recruiting | 18 Years | 65 Years | Both | 20 | Phase 1/Phase 2 | United States |
| 50 | EUCTR2007-005978-30-ES | 22/01/2008 | 19 March 2012 | Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosa Clinical 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 tuberosa Clinical 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 Pharmaceutical Form: Oral solution INN or Proposed INN: sirolimus Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 1- Trade Name: Rapamune Product Name: Rapamune Pharmaceutical Form: Coated tablet INN or Proposed INN: sirolimus Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1- Trade Name: Rapamune Product Name: Rapamune Pharmaceutical Form: Coated tablet INN or Proposed INN: sirolimus Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2- | FUNDACIÓ PUIGVERT | Authorised | Female: yes Male: yes | Spain | |||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | NCT00411619 | January 2007 | 19 October 2017 | Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex | Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex | Tuberous Sclerosis;Subependymal Giant Cell Astrocytoma | Drug: Everolimus | Children's Hospital Medical Center, Cincinnati | Novartis | Not recruiting | 3 Years | N/A | All | 28 | Phase 1/Phase 2 | United States |
| 52 | NCT00490789 | October 2005 | 19 February 2015 | 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 | Not recruiting | 18 Years | 65 Years | Both | 14 | Phase 2 | United Kingdom |
| 53 | NCT00457964 | August 2005 | 19 February 2015 | RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM | RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis | Tuberous Sclerosis;Lymphangioleiomyomatosis | Drug: RAD001 | Children's Hospital Medical Center, Cincinnati | Novartis Pharmaceuticals | Not recruiting | 18 Years | 65 Years | Both | 36 | Phase 1/Phase 2 | United States |
| 54 | NCT00457808 | December 2002 | 19 February 2015 | 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 | Not recruiting | 18 Years | 65 Years | Both | 25 | Phase 2 | United States |