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 | NCT05252585 (ClinicalTrials.gov) | April 23, 2023 | 16/2/2022 | A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML) | Phase IV, Prospective Single Arm Study of Safety and Efficacy of Votubia (Everolimus) in Taiwanese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma | Renal Angiomyolipoma | Drug: Everolimus | Novartis Pharmaceuticals | NULL | Not yet recruiting | 20 Years | 65 Years | All | 10 | Phase 4 | NULL |
| 2 | NCT05495425 (ClinicalTrials.gov) | June 1, 2022 | 4/8/2022 | Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC | Placebo-controlled Comparative Study of NPC-12Y Gel in Patients With Skin Lesions Associated With Tuberous Sclerosis Complex | Tuberous Sclerosis Complex | Drug: NPC-12Y gel;Drug: NPC-12Y placebo gel | Nobelpharma | NULL | Recruiting | 3 Years | N/A | All | 40 | Phase 3 | Japan |
| 3 | NCT05604170 (ClinicalTrials.gov) | May 16, 2022 | 5/10/2022 | Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy | A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC OLE) | Tuberous Sclerosis Complex | Drug: Ganaxolone | Marinus Pharmaceuticals | NULL | Enrolling by invitation | 1 Year | 65 Years | All | 169 | Phase 3 | United States |
| 4 | NCT05323734 (ClinicalTrials.gov) | April 1, 2022 | 21/3/2022 | Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy | A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC) | Tuberous Sclerosis Complex | Drug: Ganaxalone;Drug: Placebo | Marinus Pharmaceuticals | NULL | Recruiting | 1 Year | 65 Years | All | 162 | Phase 3 | United States;Canada;France;Germany;Israel;Italy;Spain;United Kingdom |
| 5 | ChiCTR2200055795 | 2022-02-01 | 2022-01-20 | Effect of ultrapulse carbon dioxide laser combined with photodynamic therapy on facial angiofibroma in tuberous sclerosis complex | Effect of ultrapulse carbon dioxide laser combined with photodynamic therapy on facial angiofibroma in tuberous sclerosis complex | tuberous sclerosis complex | Treatment Group:ultrapulse carbon dioxide laser combined with photodynamic therapy; | The First Hospital of Hebei Medical University | NULL | Pending | 6 | 60 | Both | Treatment Group:5; | China | |
| 6 | 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 |
| 7 | NCT05044819 (ClinicalTrials.gov) | July 7, 2021 | 30/8/2021 | Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution | A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution | Lennox Gastaut Syndrome;Dravet Syndrome;Tuberous Sclerosis Complex | Drug: Cannabidiol | Jazz Pharmaceuticals | NULL | Recruiting | 1 Year | N/A | All | 150 | Phase 4 | United States |
| 8 | NCT04485104 (ClinicalTrials.gov) | May 19, 2021 | 21/7/2020 | Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures | An Open-label, Single-arm Study to Assess the Safety, Pharmacokinetics, and Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Participants With Tuberous Sclerosis Complex (Age 1 Month to < 2 Years of Age), Dravet Syndrome (1 Year to < 2 Years of Age), or Lennox-Gastaut Syndrome (1 Year to < 2 Years of Age) Who Experience Inadequately-controlled Seizures | Seizure in Participants With Tuberous Sclerosis Complex;Seizure in Participants With Dravet Syndrome;Seizure in Participants With Lennox-Gastaut Syndrome | Drug: GWP42003-P | Jazz Pharmaceuticals | NULL | Recruiting | 1 Month | 23 Months | All | 27 | Phase 3 | United States |
| 9 | 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 |
| 10 | NCT05059327 (ClinicalTrials.gov) | March 3, 2021 | 20/8/2021 | Basimglurant in Children, Adolescents, and Young Adults With TSC | A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults With Uncontrolled Seizures Associated With Tuberous Sclerosis Complex | Tuberous Sclerosis Complex | Drug: Basimglurant with crossover to Placebo;Drug: Placebo with crossover to Basimglurant | Noema Pharma AG | NULL | Recruiting | 5 Years | 30 Years | All | 54 | Phase 2 | United States;Australia;India;Israel;Italy;Poland;Spain;Turkey;United Kingdom |
| 11 | ChiCTR2000039523 | 2020-12-01 | 2020-10-30 | Safety and efficacy of everolimus in patients with renal angiomyolipoma associated with tuberous sclerosis: a case series study | Safety and efficacy of Everolimus in patients with renal angiomyolipoma associated with tuberous sclerosis: a case series study | tuberous sclerosis complex;LD2D.2 | case series:everolimus; | Department of Pharmacy, Peking University First Hospita | NULL | Recruiting | 0 | 100 | Both | case series:20; | Phase 4 | China |
| 12 | NCT02860494 (ClinicalTrials.gov) | December 2020 | 4/8/2016 | 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: Everolimus;Drug: Placebo | Hospices Civils de Lyon | NULL | Withdrawn | N/A | 2 Years | All | 0 | Phase 2/Phase 3 | France |
| 13 | 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 |
| 14 | 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 | ||
| 15 | NCT04285346 (ClinicalTrials.gov) | April 8, 2020 | 18/2/2020 | Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B) | A Phase 2 Open-label 12-Week Trial of Adjunctive Ganaxolone Treatment (Part A) in Tuberous Sclerosis Complex-related Epilepsy Followed by Long-term Treatment (Part B) | Tuberous Sclerosis | Drug: Ganaxolone | Marinus Pharmaceuticals | NULL | Completed | 2 Years | 65 Years | All | 24 | Phase 2 | United States |
| 16 | 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 | ||
| 17 | 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 | ||
| 18 | EUCTR2016-002977-37-IT (EUCTR) | 17/07/2019 | 01/06/2021 | Roll-over study in patients with TSC and refractory seizures who arejudged by the Investigator to benefit from continued treatment witheverolimus after completion of study CRAD001M2304. | An open-label, multi-center long-term safety roll-over study in patientswith tuberous sclerosis complex (TSC) and refractory seizures who arejudged by the Investigator to benefit from continued treatment witheverolimus after completion of study CRAD001M2304. - - | Refractory seizures associated with tuberous sclerosis complex MedDRA version: 21.0;Level: LLT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Votubia Product Name: - Product Code: [-] INN or Proposed INN: EVEROLIMUS Other descriptive name: EVEROLIMUS | NOVARTIS PHARMA AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 216 | Phase 3 | United States;Taiwan;Greece;Spain;Thailand;Korea, Democratic People's Republic of;Turkey;Russian Federation;Colombia;United Kingdom;Italy;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan | ||
| 19 | NCT03525834 (ClinicalTrials.gov) | November 9, 2018 | 3/5/2018 | 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 | NULL | Completed | 18 Years | N/A | All | 40 | Phase 4 | China |
| 20 | EUCTR2018-002531-18-FR (EUCTR) | 31/10/2018 | 11/07/2018 | 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 INN or Proposed INN: EVEROLIMUS Other descriptive name: EVEROLIMUS Product Name: Everolimus INN or Proposed INN: Everolimus Other descriptive name: EVEROLIMUS Product Name: Everolimus INN or Proposed INN: Everolimus Other descriptive name: EVEROLIMUS | Hospices Civils de Lyon | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 146 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France | ||
| 21 | NCT03254680 (ClinicalTrials.gov) | March 2018 | 15/8/2017 | Turmeric as Treatment in Epilepsy | Turmeric as Treatment in Epilepsy | Epilepsy;Dravet Syndrome;Lennox-Gastaut Syndrome;Tuberous Sclerosis;Focal Seizures | Dietary Supplement: Turmeric | NYU Langone Health | NULL | Withdrawn | 1 Year | 70 Years | All | 0 | N/A | United States |
| 22 | NCT03356769 (ClinicalTrials.gov) | November 20, 2017 | 2/11/2017 | 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 Yiling Pharmaceutical Co. Ltd | Recruiting | 6 Years | 30 Years | All | 98 | Phase 2 | China |
| 23 | EUCTR2015-002154-12-NL (EUCTR) | 01/09/2017 | 17/01/2017 | 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] | Trade Name: CBD-Oral Solution, is known as Epidiolex, and is the approved name in the USA. Product Name: Cannabidiol (CBD) Product Code: GWP42003-P INN or Proposed INN: N/A Other descriptive name: CANNABIDIOL | GW Research Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 210 | Phase 3 | United States;Spain;Poland;Australia;Netherlands;United Kingdom | ||
| 24 | EUCTR2016-002977-37-GB (EUCTR) | 25/08/2017 | 12/01/2017 | 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: 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 216 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of | ||
| 25 | EUCTR2016-002977-37-BE (EUCTR) | 06/07/2017 | 04/04/2017 | 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: 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | 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 | ||
| 26 | 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 |
| 27 | EUCTR2016-002977-37-ES (EUCTR) | 12/04/2017 | 10/03/2017 | 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Farmacéutica S.A | NULL | Not Recruiting | 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 | ||
| 28 | NCT02962414 (ClinicalTrials.gov) | April 2, 2017 | 9/11/2016 | 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 | NULL | Active, 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 |
| 29 | EUCTR2016-002977-37-FR (EUCTR) | 24/03/2017 | 16/05/2017 | 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: 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 216 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of | ||
| 30 | EUCTR2016-002977-37-HU (EUCTR) | 24/02/2017 | 18/01/2017 | 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | 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 | ||
| 31 | EUCTR2016-002408-13-NL (EUCTR) | 07/02/2017 | 25/07/2016 | 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 | UMC Utrecht | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | Netherlands | |||
| 32 | NCT02849457 (ClinicalTrials.gov) | December 2016 | 13/7/2016 | 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) | Active, not recruiting | N/A | 6 Months | All | 84 | Phase 2 | United States |
| 33 | EUCTR2015-002154-12-ES (EUCTR) | 30/09/2016 | 15/07/2016 | 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: 19.0;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 INN or Proposed INN: N/A Other descriptive name: CANNABIDIOL | GW Research Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 192 | Phase 3 | United States;France;Poland;Spain;Australia;Netherlands;United Kingdom | ||
| 34 | ChiCTR-OPN-16008236 | 2016-06-01 | 2016-04-07 | 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 | NULL | Pending | 18 | 60 | Both | 10mg Everolimus:50;5mg Everolimus:50; | China | |
| 35 | NCT02544763 (ClinicalTrials.gov) | April 6, 2016 | 7/9/2015 | A Randomized Controlled 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;Drug: Placebo | Jazz Pharmaceuticals | NULL | Completed | 1 Year | 65 Years | All | 224 | Phase 3 | United States;Australia;Netherlands;Poland;Spain;United Kingdom |
| 36 | ChiCTR-DDD-17010586 | 2016-01-01 | 2017-02-08 | Prenatal diagnosis of tuberous sclerosis by fetal echocardiography combined with high throughput gene sequencing | Prenatal diagnosis of TSC using high-throughput gene sequencing combined with fetal echocardiography | Tuberous sclerosis complex | Gold Standard:A Clinical diagnostic criteria of tuberous sclerosis (TSC) for the postnatal population 1. Definite diagnosis: 2 major features or 1 major feature and 2 minor teatures 2. Possible diagnosis: 1 major feature or 2 minor features [major features: (1) Facial angiofibro or forehead plaque, (2) 'Nontraumatic' periungual&#;Index test:1. Prenatal diagnosis of TSC high-risk fetus by the traditional method (amniocentesis) using the gold standard; 2. Prenatal diagnosis of TSC high-risk fetus by maternal peripheral blood gene detection.; | An Zhen Hospital affiliated to Capital Medical University, Beijing;Beijing Key Laboratory of Fetal Heart Diseases and Maternal-Fetal Medicine | NULL | Recruiting | Female | Target condition:30;Difficult condition:5 | China | |||
| 37 | 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 |
| 38 | 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 |
| 39 | ChiCTR-OOB-15006535 | 2015-05-26 | 2015-05-29 | A Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis Complex | A Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis Complex | tuberous sclerosis complex | Case series:rapamycin; | Chinese PLA General Hospital | NULL | Recruiting | 1 | 18 | Both | Case series:500; | China | |
| 40 | NCT02104011 (ClinicalTrials.gov) | May 22, 2015 | 1/4/2014 | 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 | NULL | Completed | 18 Years | N/A | All | 2 | Phase 2 | France |
| 41 | EUCTR2013-005528-40-BE (EUCTR) | 11/12/2014 | 17/11/2014 | Long-term, prospective study evaluating clinical and molecular biomarkers of epileptogenesis in a genetic model of epilepsy – tuberous sclerosis complex | Long-term, prospective study evaluating clinical and molecular biomarkers of epileptogenesis in a genetic model of epilepsy – tuberous sclerosis complex - EPISTOP | epilepsy in tuberous sclerosis complex (TSC);Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: vigabatrin (sabril) Product Name: vigabatrin INN or Proposed INN: VIGABATRIN | Vrije Universiteit Brussel | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Belgium;Netherlands | |||
| 42 | EUCTR2013-003795-13-BE (EUCTR) | 10/12/2014 | 17/11/2014 | 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: 21.0;Level: LLT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 3;Phase 4 | United States;Canada;Poland;Belgium;Russian Federation | ||
| 43 | EUCTR2013-005528-40-NL (EUCTR) | 05/12/2014 | 14/07/2014 | Long-term, prospective study evaluating clinical and molecular biomarkers of epileptogenesis in a genetic model of epilepsy – Tuberous Sclerosis Complex. | Long-term, prospective study evaluating clinical and molecular biomarkers of epileptogenesis in a genetic model of epilepsy – Tuberous Sclerosis Complex. - EPISTOP | Epilepsy in tuberous sclerosis complex (TSC) MedDRA version: 17.0;Level: LLT;Classification code 10032061;Term: Other forms of epilepsy;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Vigabatrin (Sabril) Product Name: vigabatrin | The Children's Memorial Health Institute | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Belgium;Netherlands | |||
| 44 | 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 |
| 45 | NCT02201212 (ClinicalTrials.gov) | September 2014 | 23/7/2014 | 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 | Completed | 18 Years | N/A | All | 30 | Phase 2 | United States |
| 46 | EUCTR2011-000860-90-IE (EUCTR) | 05/06/2014 | 10/04/2014 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) MedDRA version: 17.1;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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 355 | Phase 3 | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 47 | 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 |
| 48 | NCT02451696 (ClinicalTrials.gov) | January 2014 | 8/10/2014 | 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 | NULL | Completed | 2 Years | 40 Years | All | 15 | Phase 2 | United States |
| 49 | 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 |
| 50 | NCT01853423 (ClinicalTrials.gov) | October 2013 | 12/4/2013 | 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 | Completed | 3 Years | 45 Years | Both | 11 | Phase 1 | United States |
| 51 | EUCTR2011-000860-90-BE (EUCTR) | 17/09/2013 | 05/06/2013 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) MedDRA version: 17.0;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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 355 | Phase 3 | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 52 | EUCTR2011-000860-90-GR (EUCTR) | 12/09/2013 | 08/07/2013 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) MedDRA version: 16.0;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: Everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 345 | Phase 3 | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 53 | EUCTR2011-000860-90-DK (EUCTR) | 09/09/2013 | 09/09/2013 | A 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 | 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 | Refractory partial-onset seizures 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 355 | Phase 3 | Taiwan;Greece;Spain;Ireland;Turkey;Colombia;Italy;United Kingdom;France;Hungary;Mexico;Argentina;Poland;Belgium;Denmark;Norway;Netherlands;Germany;Korea, Republic of | ||
| 54 | 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 |
| 55 | JPRN-UMIN000011559 | 2013/09/01 | 01/09/2013 | Therpeutic drug monitoring of Everolimus in patients with renal cell carcinoma and tuberous sclerosis | Therpeutic drug monitoring of Everolimus in patients with renal cell carcinoma and tuberous sclerosis - TDM study of Everolimus | 1.advanced renal cell carcinoma2.renal angiomyolipoma associated with tuberous sclerosis complex | Everolimus 5mg/day. | Department of Urology, Keio University School of Medicine | NULL | Complete: follow-up complete | 20years-old | 90years-old | Male and Female | 43 | Not selected | Japan |
| 56 | EUCTR2011-000860-90-NL (EUCTR) | 30/07/2013 | 05/02/2013 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) MedDRA version: 19.0;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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 355 | Phase 3 | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 57 | EUCTR2011-000860-90-DE (EUCTR) | 19/07/2013 | 25/03/2013 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) 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] | Product Name: Everolimus 2mg dispersible tablet Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 355 | Phase 3 | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Netherlands;Germany;Japan;Korea, Republic of | ||
| 58 | 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 |
| 59 | EUCTR2011-000860-90-GB (EUCTR) | 30/05/2013 | 03/06/2013 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) MedDRA version: 19.0;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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 355 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;Italy;United Kingdom;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 60 | EUCTR2011-000860-90-HU (EUCTR) | 24/05/2013 | 30/05/2013 | A 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 | 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 | Refractory partial-onset seizures 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 355 | Phase 3 | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 61 | NCT01713946 (ClinicalTrials.gov) | April 29, 2013 | 9/10/2012 | 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 | NULL | Completed | 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 |
| 62 | EUCTR2012-005397-63-ES (EUCTR) | 19/04/2013 | 16/01/2014 | 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Farmacéutica, S.A | ,NULL | Not Recruiting | Female: yes Male: yes | Spain | ||||
| 63 | EUCTR2011-000860-90-IT (EUCTR) | 28/03/2013 | 04/02/2013 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) MedDRA version: 15.1;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] | Product Name: Everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 345 | Phase 3 | United States;Taiwan;Greece;Thailand;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 64 | EUCTR2011-000860-90-ES (EUCTR) | 14/02/2013 | 21/12/2012 | A 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 | 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 | Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC) 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] | Product Name: Everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Farmacéutica, S.A. | NULL | Not Recruiting | Female: yes Male: yes | 345 | Phase 3 | United Kingdom;Italy;Hungary;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of;United States;Taiwan;Greece;Spain;Thailand;Ireland;Turkey;Russian Federation;Colombia | ||
| 65 | NCT01730209 (ClinicalTrials.gov) | November 2012 | 26/10/2012 | 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 |
| 66 | 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 | |||
| 67 | NCT01954693 (ClinicalTrials.gov) | June 2012 | 5/9/2013 | 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 | Unknown status | 16 Years | 60 Years | All | 48 | Phase 2 | United Kingdom |
| 68 | 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 |
| 69 | EUCTR2011-001319-30-GB (EUCTR) | 04/04/2012 | 05/09/2011 | 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 INN or Proposed INN: metformin hydrochloride Other descriptive name: - | University Hospitals Bristol NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 4 | United Kingdom | ||
| 70 | EUCTR2010-019519-39-NL (EUCTR) | 14/03/2012 | 01/12/2011 | 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 INN or Proposed INN: Votubia Other descriptive name: EVEROLIMUS | Erasmus MC - Department of Neurology | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Netherlands | ||||
| 71 | EUCTR2011-004854-25-GB (EUCTR) | 10/01/2012 | 22/11/2011 | 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 INN or Proposed INN: Everolimus Other descriptive name: RAD001 | Cardiff University | NULL | Not Recruiting | Female: yes Male: yes | 48 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 72 | 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 |
| 73 | EUCTR2010-022583-13-IT (EUCTR) | 06/07/2011 | 27/04/2011 | 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 | Subependymal giant cell astrocytomas (SEGA)associated with Tuberous Sclerosis Complex (TSC) MedDRA version: 13.1;Level: PT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 250 | Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy | |||
| 74 | EUCTR2010-022583-13-CZ (EUCTR) | 01/06/2011 | 23/02/2011 | 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: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 250 | Phase 3 | France;Hungary;Czech Republic;Greece;Belgium;Spain;Germany;Italy | ||
| 75 | EUCTR2007-006997-27-BE (EUCTR) | 01/06/2011 | 29/05/2009 | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - N/A | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - N/A | This study will evaluate the antitumor activity of RAD001 versus placebo in patientswith subependymal giant cell astrocytomas (SEGA) associated with TuberousSclerosis Complex (TSC). | Trade Name: Certican 1 mg tablets Product Name: RAD001 Product Code: RAD001 INN or Proposed INN: everolimus | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 99 | Belgium;Netherlands;Germany;United Kingdom;Italy | |||
| 76 | EUCTR2010-022583-13-BE (EUCTR) | 01/06/2011 | 03/03/2011 | 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). | Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 250 | Phase 3 | Czech Republic;Hungary;Greece;Spain;Belgium;Germany;Italy | ||
| 77 | 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 | ||||
| 78 | EUCTR2010-022583-13-GR (EUCTR) | 18/04/2011 | 24/03/2011 | The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA. | 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).;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 250 | Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy | |||
| 79 | EUCTR2010-022583-13-HU (EUCTR) | 31/03/2011 | 22/02/2011 | 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). 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] - Cancer [C04] | Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 250 | Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy | |||
| 80 | NCT01289912 (ClinicalTrials.gov) | January 2011 | 2/2/2011 | 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 | Completed | 6 Years | 21 Years | All | 52 | Phase 2 | United States |
| 81 | NCT01266291 (ClinicalTrials.gov) | August 2010 | 22/12/2010 | 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 | Terminated | 18 Years | N/A | All | 1 | Phase 4 | United States |
| 82 | EUCTR2007-006997-27-NL (EUCTR) | 25/03/2010 | 03/12/2014 | Effectiveness and safety of RAD001 in patients of all ages with Subependymal Giant Cell Astrocytoma associated with Tuberous Sclerosis Complex (TSC) | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - N/A | This study will evaluate the antitumor activity of RAD001 versus placebo in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC). MedDRA version: 17.1;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] | Product Name: RAD001 Product Code: RAD001 INN or Proposed INN: everolimus | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 99 | Canada;Poland;Belgium;Australia;Russian Federation;Germany;Netherlands;Italy;United Kingdom | |||
| 83 | EUCTR2008-002113-48-NL (EUCTR) | 16/03/2010 | 02/04/2009 | A randomized double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) | A randomized double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) | Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) MedDRA version: 9.1;Level: LLT;Classification code 10051810;Term: Angiomyolipoma | Product Name: RAD001 Product Code: RAD001 INN or Proposed INN: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 99 | France;Spain;Germany;Netherlands;Italy;United Kingdom | |||
| 84 | EUCTR2007-006997-27-DE (EUCTR) | 12/03/2010 | 20/01/2010 | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - N/A | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - N/A | This study will evaluate the antitumor activity of RAD001 versus placebo in patientswith subependymal giant cell astrocytomas (SEGA) associated with TuberousSclerosis Complex (TSC). | Product Name: RAD001 1mg Product Code: RAD001 INN or Proposed INN: everolimus | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 99 | Italy;Belgium;Netherlands;Germany;United Kingdom | |||
| 85 | NCT01070316 (ClinicalTrials.gov) | January 2010 | 15/2/2010 | 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 | Completed | 2 Years | N/A | All | 20 | Phase 1/Phase 2 | United States |
| 86 | 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 |
| 87 | EUCTR2007-006997-27-GB (EUCTR) | 16/09/2009 | 06/07/2009 | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - N/A | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - N/A | This study will evaluate the antitumor activity of RAD001 versus placebo in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC). | Product Name: RAD001 round tablets Product Code: RAD001 INN or Proposed INN: everolimus | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 99 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 88 | EUCTR2008-002113-48-IT (EUCTR) | 03/09/2009 | 07/09/2009 | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - ND | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - ND | Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) MedDRA version: 12.0;Level: LLT;Classification code 10051810;Term: Angiomyolipoma | Product Name: RAD001 Product Code: RAD001 INN or Proposed INN: Everolimus | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 99 | France;Spain;Netherlands;Germany;United Kingdom;Italy | |||
| 89 | 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 |
| 90 | EUCTR2008-002113-48-GB (EUCTR) | 06/08/2009 | 22/05/2009 | 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 RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - EXIST-2 | Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) MedDRA version: 17.0;Level: PT;Classification code 10051810;Term: Angiomyolipoma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Trade Name: Afinitor 5mg Tablets Product Name: RAD001 Product Code: RAD001 INN or Proposed INN: Everolimus | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 118 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Spain;Russian Federation;Netherlands;Germany;Italy;Japan;United Kingdom | ||
| 91 | NCT00789828 (ClinicalTrials.gov) | August 2009 | 12/11/2008 | 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 | NULL | Completed | N/A | N/A | All | 117 | Phase 3 | United States;Australia;Belgium;Canada;Germany;Italy;Netherlands;Poland;Russian Federation;United Kingdom |
| 92 | EUCTR2007-006997-27-IT (EUCTR) | 27/07/2009 | 18/06/2009 | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - ND | A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC) - ND | This study will evaluate the antitumor activity of RAD001 versus placebo in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC). MedDRA version: 12.0;Level: LLT;Classification code 10060971;Term: Astrocytoma malignant | Product Code: RAD001 INN or Proposed INN: Everolimus | NOVARTIS FARMA | NULL | Not Recruiting | Female: yes Male: yes | 99 | Belgium;Netherlands;Germany;United Kingdom;Italy | |||
| 93 | NCT00989742 (ClinicalTrials.gov) | July 2009 | 2/10/2009 | 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 | NULL | Completed | 18 Years | N/A | Female | 24 | Phase 4 | United Kingdom |
| 94 | NCT00790400 (ClinicalTrials.gov) | April 2009 | 10/11/2008 | 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 | NULL | Completed | 18 Years | N/A | All | 118 | Phase 3 | United States;Canada;France;Germany;Italy;Japan;Netherlands;Poland;Russian Federation;Spain;United Kingdom |
| 95 | EUCTR2008-002113-48-FR (EUCTR) | 18/03/2009 | 19/03/2009 | A randomized double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) | A randomized double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) | Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) MedDRA version: 9.1;Level: LLT;Classification code 10051810;Term: Angiomyolipoma | Product Name: RAD001 Product Code: RAD001 INN or Proposed INN: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 99 | Phase 3 | France;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 96 | NCT00792766 (ClinicalTrials.gov) | December 2008 | 17/11/2008 | 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 | Completed | 18 Years | 65 Years | Both | 20 | Phase 1/Phase 2 | United States |
| 97 | 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 |
| 98 | 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 | ||||
| 99 | NCT00411619 (ClinicalTrials.gov) | January 2007 | 12/12/2006 | 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 | Completed | 3 Years | N/A | All | 28 | Phase 1/Phase 2 | United States |
| 100 | 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 |
| 101 | NCT00457964 (ClinicalTrials.gov) | August 2005 | 6/4/2007 | 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 | Completed | 18 Years | 65 Years | Both | 36 | Phase 1/Phase 2 | United States |
| 102 | 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 |
| 103 | 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 | |||
| 104 | 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 | |||
| 105 | EUCTR2016-002977-37-PL (EUCTR) | 09/06/2017 | 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: 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma AG | NULL | NA | 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 | |||
| 106 | 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 | |||
| 107 | EUCTR2020-002132-67-Outside-EU/EEA (EUCTR) | 22/10/2021 | Safety, Pharmacokinetics, and Exploratory Efficacy Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) Compared With Standard of Care Antiseizure Medication, in Patients Age 1 Month to <12 Months of Age With Tuberous Sclerosis Complex Who Experience Inadequately-controlled Seizures | An Open-Label, Randomized Trial to Assess the Safety, Pharmacokinetics, and Exploratory Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) Compared with Standard of Care Antiseizure Medication, in Patients Age 1 Month to Less than 12 Months of Age with Tuberous Sclerosis Complex who Experience Inadequately-Controlled SeizuresP/0047/2020P/0350/2020P/0033/2021 | Tuberous Sclerosis Complex (TSC) MedDRA version: 21.0;Level: LLT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: PT;Classification code 10080584;Term: Tuberous sclerosis complex;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Cannabidiol Oral Solution, is known as Epidyolex 100 mg/mL oral solution and is the approved name in the EU. Epidiolex (cannabidiol) oral solution is the approved name in the US. Product Name: Cannabidiol (CBD) Product Code: GWP42003-P | GW Research Ltd | NULL | NA | Female: yes Male: yes | Phase 3 | United States | ||||
| 108 | EUCTR2010-022583-13-FR (EUCTR) | 01/03/2011 | 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: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Afinitor Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Afinitor Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 250 | Phase 3 | France;Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy | |||
| 109 | EUCTR2015-002154-12-PL (EUCTR) | 18/11/2016 | 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: 21.0;Level: LLT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: CBD - Oral Solution, is known as Epidyolex 100 mg/ml oral solution, Product Name: Cannabidiol (CBD) Product Code: GWP42003-P INN or Proposed INN: N/A Other descriptive name: CANNABIDIOL | GW Research Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 210 | Phase 3 | United States;Spain;Poland;Australia;Netherlands;United Kingdom | |||
| 110 | EUCTR2013-003795-13-PL (EUCTR) | 20/01/2015 | 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: 17.1;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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Votubia Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 48 | Phase 3 | United States;Canada;Belgium;Poland;Russian Federation | |||
| 111 | EUCTR2008-002113-48-DE (EUCTR) | 12/02/2009 | 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 RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - EXIST-2 | Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) 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: Afinitor Product Name: RAD001 5mg Product Code: RAD001 INN or Proposed INN: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 118 | Phase 3 | France;United States;Canada;Spain;Russian Federation;Netherlands;Germany;Italy;United Kingdom;Japan | |||
| 112 | EUCTR2010-022583-13-DE (EUCTR) | 23/12/2010 | 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 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 250 | Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy |