158. 結節性硬化症
[臨床試験数:100,薬物数:62(DrugBank:16),標的遺伝子数:35,標的パスウェイ数:115

Searched query = "Tuberous sclerosis", "Tuberous sclerosis complex"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT02860494
(ClinicalTrials.gov)
December 20204/8/2016Topical Everolimus in Patients With Tuberous Sclerosis ComplexTopical 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 AngiofibromasDrug: Everolimus;Drug: PlaceboHospices Civils de LyonNULLNot yet recruitingN/A2 YearsAll96Phase 2;Phase 3France
2NCT04595513
(ClinicalTrials.gov)
September 8, 20206/10/2020Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC InfantsStopping TSC Onset and Progression 2: Epilepsy Prevention in TSC InfantsTuberous Sclerosis Complex;EpilepsyDrug: TAVT-18 (sirolimus);Drug: PlaceboChildren's Hospital Medical Center, CincinnatiNULLRecruitingN/A6 MonthsAll65Phase 1;Phase 2United States
3NCT04485104
(ClinicalTrials.gov)
September 202021/7/2020Safety, Pharmacokinetics, and Exploratory Efficacy Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) Compared With Standard of Care Antiepileptic Therapy, in Patients Age 1 Month to <12 Months of Age With Tuberous Sclerosis Complex Who Experience Inadequately-controlled SeizuresAn Open-label, Randomized Trial to Assess the Safety, Pharmacokinetics, and Exploratory Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) Compared With Standard of Care Antiepileptic Therapy, in Patients Age 1 Month to Less Than 12 Months of Age With Tuberous Sclerosis Complex Who Experience Inadequately-controlled SeizuresSeizure in Participants With Tuberous Sclerosis ComplexDrug: GWP42003-P;Drug: Standard of careGW Research LtdNULLNot yet recruiting1 Month11 MonthsAll15Phase 3NULL
4ChiCTR2000031984
2020-05-012020-04-17Sirolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex and EpilepsySirolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex and Epilepsy Tuberous sclerosis complex; Epilepsyexperimental group:Antiepileptic drug + sirolimus;control group:antiepileptic drugs;West China Hospital, Sichuan UniversityNULLPendingBothexperimental group:31;control group:31;N/AChina
5NCT04285346
(ClinicalTrials.gov)
April 8, 202018/2/2020Adjunctive 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 SclerosisDrug: GanaxoloneMarinus PharmaceuticalsNULLRecruiting2 Years65 YearsAll30Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-000752-34-SK
(EUCTR)
15/11/201901/10/2019 Study to evaluate if the study medication, Rapamycin, topical cream, is effective and safe at treating facial angiofibroma in patients 6 years of age and over. A Phase 2/3, multi-center, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison of the efficacy and safety of a topical rapamycin cream for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and over - Dose-ranging efficacy and safety study of topical rapamycin cream Facial Angiofibromas Associated with Tuberous Sclerosis Complex
MedDRA version: 20.0;Level: PT;Classification code 10002429;Term: Angiofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0;Classification code 10073678;Term: Juvenile angiofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
DSLPNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120Phase 2;Phase 3United States;Hungary;Slovakia;Spain;Australia;New Zealand;United Kingdom
7EUCTR2019-000752-34-HU
(EUCTR)
14/11/201918/11/2019 Study to evaluate if the study medication, Rapamycin, topical cream, is effective and safe at treating facial angiofibroma in patients 6 years of age and over.A Phase 2/3, multi-center, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison of the efficacy and safety of a topical rapamycin cream for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and over - Dose-ranging efficacy and safety study of topical rapamycin cream Facial Angiofibromas Associated with Tuberous Sclerosis Complex
MedDRA version: 20.0;Level: PT;Classification code 10002429;Term: Angiofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0;Classification code 10073678;Term: Juvenile angiofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
DSLPNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120Phase 2;Phase 3United States;Hungary;Slovakia;Spain;Australia;New Zealand;United Kingdom
8NCT03826628
(ClinicalTrials.gov)
July 28, 201929/1/2019Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis ComplexA Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and OverFacial Angiofibroma;Tuberous SclerosisDrug: rapamycin;Drug: placeboDermatology Specialties Limited PartnershipNULLRecruiting6 Years65 YearsAll120Phase 2;Phase 3United States;Australia;Czechia;Hungary;New Zealand;Slovakia;Spain;United Kingdom
9NCT03525834
(ClinicalTrials.gov)
November 9, 20183/5/2018Safety 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 SurgeryRenal AngiomyolipomaDrug: everolimusNovartis PharmaceuticalsNULLCompleted18 YearsN/AAll40Phase 4China
10EUCTR2018-002531-18-FR
(EUCTR)
31/10/201811/07/2018Topical 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]
Hospices Civils de LyonNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
146Phase 2;Phase 3France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
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Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03254680
(ClinicalTrials.gov)
March 201815/8/2017Turmeric as Treatment in EpilepsyTurmeric as Treatment in EpilepsyEpilepsy;Dravet Syndrome;Lennox-Gastaut Syndrome;Tuberous Sclerosis;Focal SeizuresDietary Supplement: TurmericNYU Langone HealthNULLWithdrawn1 Year70 YearsAll0N/AUnited States
12NCT03356769
(ClinicalTrials.gov)
November 20, 20172/11/2017Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis ComplexA Placebo-controlled Study of Efficacy & Safety of Aspirin as an add-on Treatment in Patients With Tuberous Sclerosis Complex (TSC) & Refractory SeizuresTuberous Sclerosis Complex;Aspirin;Epilepsy;Cognitive Decline;Skin LesionsDrug: Aspirin;Drug: AED;Drug: PlaceboPeking Union Medical College HospitalShijiazhuang Yiling Pharmaceutical Co. LtdRecruiting6 Years30 YearsAll98Phase 2China
13EUCTR2015-002154-12-NL
(EUCTR)
01/09/201717/01/2017A 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]
GW Research Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
210Phase 3United States;Poland;Spain;Australia;Netherlands;United Kingdom
14EUCTR2016-002977-37-GB
(EUCTR)
25/08/201712/01/2017Roll-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]
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
216Phase 3United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of
15EUCTR2016-002977-37-BE
(EUCTR)
06/07/201704/04/2017Roll-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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
216Phase 3United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT03363763
(ClinicalTrials.gov)
April 12, 20171/12/2017Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis ComplexPhase 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 LabelAngiofibroma of Face;Tuberous SclerosisDrug: Sirolimus 0.2%;Drug: Sirolimus 0.4%;Drug: Placebo ointmentAucta Pharmaceuticals, IncNULLRecruiting2 Years18 YearsAll45Phase 2United States;China
17EUCTR2016-002977-37-ES
(EUCTR)
12/04/201710/03/2017Roll-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.ANULLNot RecruitingFemale: yes
Male: yes
216Phase 3United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of
18NCT02962414
(ClinicalTrials.gov)
April 2, 20179/11/2016Roll-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 TreatmentAn 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 ComplexDrug: everolimusNovartis PharmaceuticalsNULLActive, not recruiting2 Years65 YearsAll206Phase 3United States;Australia;Belgium;Canada;Colombia;France;Hungary;Italy;Japan;Korea, Republic of;Mexico;Poland;Russian Federation;Spain;Taiwan;Thailand;Turkey;United Kingdom
19EUCTR2016-002977-37-FR
(EUCTR)
24/03/201716/05/2017Roll-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]
Novartis Pharma AGNULLNot Recruiting Female: yes
Male: yes
216Phase 3United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of
20EUCTR2016-002977-37-HU
(EUCTR)
24/02/201718/01/2017Roll-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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
216Phase 3United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2016-002408-13-NL
(EUCTR)
07/02/201725/07/2016Bumetanide to treat behavioral problems in TSCBumetanide 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 UtrechtNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Netherlands
22NCT02849457
(ClinicalTrials.gov)
December 201613/7/2016Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis ComplexPreventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (PREVeNT Trial) A Randomized, Double-blind, Placebo-controlled Seizure Prevention Clinical Trial for Infants With TSCTuberous Sclerosis ComplexDrug: Vigabatrin;Drug: PlaceboMartina BebinNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruitingN/A6 MonthsAll84Phase 2United States
23EUCTR2015-002154-12-ES
(EUCTR)
30/09/201615/07/2016A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
192Phase 3France;United States;Spain;Australia;United Kingdom
24NCT02544750
(ClinicalTrials.gov)
August 20167/9/2015An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)A Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P, CBD) as Add-on Therapy in Patients With Tuberous Sclerosis Complex Who Experience Inadequately-controlled SeizuresTuberous Sclerosis Complex;SeizuresDrug: GWP42003-PGW Research LtdNULLEnrolling by invitation1 Year65 YearsAll210Phase 3United States
25ChiCTR-OPN-16008236
2016-06-012016-04-07TSC1/2 gene detection and Everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complexTSC1/2 gene detection and Everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complex renal angiomyolipoma10mg Everolimus:Everolimus 10mg QD for three months;5mg Everolimus:Everolimus 5mg QD for six months;General Hospital of PLANULLPending1860Both10mg Everolimus:50;5mg Everolimus:50;China
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT02544763
(ClinicalTrials.gov)
April 6, 20167/9/2015A 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 SeizuresTuberous Sclerosis Complex;SeizuresDrug: GWP42003-P;Drug: PlaceboGW Research LtdNULLCompleted1 Year65 YearsAll224Phase 3United States;Australia;Netherlands;Poland;Spain;United Kingdom
27ChiCTR-DDD-17010586
2016-01-012017-02-08Prenatal diagnosis of tuberous sclerosis by fetal echocardiography combined with high throughput gene sequencingPrenatal diagnosis of TSC using high-throughput gene sequencing combined with fetal echocardiography Tuberous sclerosis complexGold 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 MedicineNULLRecruitingFemaleTarget condition:30;Difficult condition:5China
28NCT02635789
(ClinicalTrials.gov)
December 201515/12/2015Phase 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 ComplexTuberous Sclerosis;Angiofibroma;Hypomelanotic Macule;PlaqueDrug: NPC-12G gel;Drug: Placebo gelNobelpharmaNULLCompleted3 YearsN/AAll62Phase 3Japan
29NCT02634931
(ClinicalTrials.gov)
December 201516/12/2015Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis ComplexA 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 ComplexTuberous Sclerosis;Angiofibroma;Hypomelanotic Macule;PlaqueDrug: NPC-12G gelNobelpharmaNULLCompleted3 YearsN/AAll94Phase 3Japan
30ChiCTR-OOB-15006535
2015-05-262015-05-29A Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis ComplexA Study of mTOR Inhibitor as Adjunctive Therapy in children With Tuberous Sclerosis Complex tuberous sclerosis complexCase series:rapamycin;Chinese PLA General HospitalNULLRecruiting118BothCase series:500;China
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT02104011
(ClinicalTrials.gov)
May 22, 20151/4/2014Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockersTreatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers: Pilot TrialRenal Angiomyolipomas;Tuberous SclerosisDrug: PropranololUniversity Hospital, BordeauxNULLCompleted18 YearsN/AAll2Phase 2France
32EUCTR2013-005528-40-BE
(EUCTR)
11/12/201417/11/2014Long-term, prospective study evaluating clinical and molecular biomarkers of epileptogenesis in a genetic model of epilepsy – tuberous sclerosis complexLong-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 BrusselNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Belgium;Netherlands
33EUCTR2013-003795-13-BE
(EUCTR)
10/12/201417/11/2014Long-term monitoring of growth and development of pediatric patients previously treated with everolimusLong-term follow-up study to monitor the growth and development of pediatric patients previously treated with everolimus in study CRAD001M2301 - EXIST-LT Subependymal Giant Cell Astrocytoma (SEGA) associated with Tuberous Sclerosis Complex (TSC)
MedDRA version: 20.0;Level: PT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
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 AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 3;Phase 4United States;Canada;Poland;Belgium;Russian Federation
34EUCTR2013-005528-40-NL
(EUCTR)
05/12/201414/07/2014Long-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 InstituteNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Belgium;Netherlands
35JPRN-UMIN000015114
2014/09/1110/09/2014Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complex Tuberous sclerosis complexApplication of 0.2% rapamycin gel on skin lesions of the face and trunk twice a day for 12 weeks.Department of Dermatology Graduate School of Medicine, Osaka UniversityNULLRecruitingNot applicable70years-oldMale and Female24Phase 2;Phase 3Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT02201212
(ClinicalTrials.gov)
September 201423/7/2014Everolimus for Cancer With TSC1 or TSC2 MutationA Phase II Trial of Everolimus for Cancer Patients With Inactivating Mutations in TSC1 or TSC2 or Activating MTOR MutationsTSC1;TSC2;Tuberous Sclerosis Complex;MTORDrug: EverolimusDana-Farber Cancer InstituteNovartis PharmaceuticalsCompleted18 YearsN/AAll30Phase 2United States
37EUCTR2011-000860-90-IE
(EUCTR)
05/06/201410/04/2014A 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 seizuresA 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 AGNULLNot RecruitingFemale: yes
Male: yes
355Phase 3United 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
38NCT02061397
(ClinicalTrials.gov)
March 201423/1/2014Safety of Simvastatin in LAM and TSCThe Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)Lymphangioleiomyomatosis;Tuberous Sclerosis ComplexDrug: Simvastatin;Drug: Sirolimus Oral Product;Drug: Everolimus Oral ProductUniversity of PennsylvaniaThe LAM FoundationCompleted18 YearsN/AFemale10Phase 1;Phase 2United States
39NCT02451696
(ClinicalTrials.gov)
January 20148/10/2014A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCDA Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCDEpilepsy;Tuberous Sclerosis Complex;Focal Cortical DysplasiaDrug: EverolimusNYU Langone HealthNULLCompleted2 Years40 YearsAll15Phase 2United States
40JPRN-UMIN000012420
2013/12/1027/11/2013Randomized, double-blind, placebo-controlled, clinical trial with OSD-001 for skin lesions due to tuberous sclerosis complex. Tuberous sclerosis complex0.05% 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 UniversityNULLComplete: follow-up complete3years-old65years-oldMale and Female36Phase 1;Phase 2Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41NCT01853423
(ClinicalTrials.gov)
October 201312/4/2013Tuberous Sclerosis Complex: Facial Angiofibroma Skin CreamTuberous Sclerosis Complex: Facial Angiofibroma Skin CreamFacial AngiofibromaDrug: RapamuneLe Bonheur Children's HospitalPfizer;University of Tennessee Health Science CenterCompleted3 Years45 YearsBoth11Phase 1United States
42EUCTR2011-000860-90-BE
(EUCTR)
17/09/201305/06/2013A 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 seizuresA 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 AGNULLNot RecruitingFemale: yes
Male: yes
355Phase 3United 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
43EUCTR2011-000860-90-GR
(EUCTR)
12/09/201308/07/2013A 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 seizuresA 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 AGNULLNot RecruitingFemale: yes
Male: yes
345Phase 3United 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
44EUCTR2011-000860-90-DK
(EUCTR)
09/09/201309/09/2013A 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 seizuresA 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 AGNULLNot RecruitingFemale: yes
Male: yes
355Phase 3Taiwan;Greece;Spain;Ireland;Turkey;Colombia;Italy;United Kingdom;France;Hungary;Mexico;Argentina;Poland;Belgium;Denmark;Norway;Netherlands;Germany;Korea, Republic of
45NCT03140449
(ClinicalTrials.gov)
September 5, 201326/4/2017Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous SclerosisStudy of Combination Therapy With Topical Rapamycin and Calcitriol for Cutaneous Lesions of Tuberous Sclerosis: A Double-blind Randomized Controlled TrialFacial AngiofibromaDrug: Rapamycin;Drug: Calcitriol;Drug: Rapamycin-calcitriol combinationNational Taiwan University HospitalNULLCompleted7 Years65 YearsAll52Phase 3NULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46JPRN-UMIN000011559
2013/09/0101/09/2013Therpeutic drug monitoring of Everolimus in patients with renal cell carcinoma and tuberous sclerosis 1.advanced renal cell carcinoma2.renal angiomyolipoma associated with tuberous sclerosis complexEverolimus 5mg/day.Department of Urology, Keio University School of MedicineNULLComplete: follow-up complete20years-old90years-oldMale and Female43Not selectedJapan
47EUCTR2011-000860-90-NL
(EUCTR)
30/07/201305/02/2013A 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 seizuresA 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 AGNULLNot RecruitingFemale: yes
Male: yes
355Phase 3United 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
48EUCTR2011-000860-90-DE
(EUCTR)
19/07/201325/03/2013A 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 seizuresA 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 AGNULLNot RecruitingFemale: yes
Male: yes
355Phase 3United 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
49NCT01929642
(ClinicalTrials.gov)
July 20137/8/2013Rapalogues for Autism Phenotype in TSC: A Feasibility StudyRapalogues for Autism Phenotype in TSC: A Feasibility StudyTuberous Sclerosis Complex;Self-injury;AutismDrug: Sirolimus;Drug: EverolimusHugo W. Moser Research Institute at Kennedy Krieger, Inc.NULLCompleted2 Years30 YearsAll3Phase 2United States
50EUCTR2011-000860-90-GB
(EUCTR)
30/05/201303/06/2013A 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 seizuresA 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]
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
355Phase 3United 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51EUCTR2011-000860-90-HU
(EUCTR)
24/05/201330/05/2013A 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 seizuresA 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 AGNULLNot RecruitingFemale: yes
Male: yes
355Phase 3United 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
52NCT01713946
(ClinicalTrials.gov)
April 29, 20139/10/2012A 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 SeizuresA 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 SeizuresTuberous Sclerosis Complex-associated Refractory SeizuresDrug: RAD001;Drug: Placebo;Drug: Antiepileptic drug (1 to 3 only);Drug: open label RAD001 (only used for post-extension phase)Novartis PharmaceuticalsNULLCompleted2 Years65 YearsAll366Phase 3United 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
53EUCTR2012-005397-63-ES
(EUCTR)
19/04/201316/01/2014Expanded 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,NULLNot RecruitingFemale: yes
Male: yes
Spain
54EUCTR2011-000860-90-IT
(EUCTR)
28/03/201304/02/2013A 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 seizuresA 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 FARMANULLNot RecruitingFemale: yes
Male: yes
345Phase 3United 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
55EUCTR2011-000860-90-ES
(EUCTR)
14/02/201321/12/2012A 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 seizuresA 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.NULLNot RecruitingFemale: yes
Male: yes
345Phase 3United 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
56NCT01730209
(ClinicalTrials.gov)
November 201226/10/2012Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis ComplexEfficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis ComplexTuberous Sclerosis Complex;TSC Related Cognitive Disability;TSC Related Autism;TSC Related Learning ProblemsDrug: Everolimus;Drug: PlaceboErasmus Medical CenterUtrecht UniversityRecruiting4 Years15 YearsBoth60Phase 2;Phase 3Netherlands
57EUCTR2011-006308-12-ES
(EUCTR)
16/07/201229/03/2012Clinical trial in patients with tuberous sclerosis for the study of the effects of topical rapamycin in reducing facial tumors associated with the disease ..phase II clinical trial, to study the efficacy and safety of topical rapamycin in reducing facial angiofibromas. Facial tumors (angiofibromas) associated with tuberous sclerosis complex.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Trade Name: Rapamune 1 mg/ml solución oral
INN or Proposed INN: RAPAMUNE
Fundación Investigación Hospital Ramón y CajalNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Spain
58NCT01954693
(ClinicalTrials.gov)
June 20125/9/2013A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous SclerosisTRON: A Randomised, Double Blind, Placebo-controlled Study of RAD001 (Everolimus) in the Treatment of Neurocognitive Problems in Tuberous SclerosisTuberous SclerosisDrug: Placebo;Drug: Everolimus (RAD001)Cardiff UniversityNovartisUnknown status16 Years60 YearsAll48Phase 2United Kingdom
59NCT01526356
(ClinicalTrials.gov)
May 20121/2/2012Topical Rapamycin to Erase Angiofibromas in TSCPhase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel TherapyAngiofibromas;Tuberous SclerosisDrug: Placebo;Drug: RapamycinThe University of Texas Health Science Center, HoustonNULLCompletedN/AN/AAll179Phase 2United States;Australia
60EUCTR2011-001319-30-GB
(EUCTR)
04/04/201205/09/2011Metformin 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 TrustNULLNot RecruitingFemale: yes
Male: yes
100Phase 4United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
61EUCTR2010-019519-39-NL
(EUCTR)
14/03/201201/12/2011Efficacy 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 NeurologyNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Netherlands
62EUCTR2011-004854-25-GB
(EUCTR)
10/01/201222/11/2011TRON: 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]
Cardiff UniversityNULLNot Recruiting Female: yes
Male: yes
48Phase 2United Kingdom
63JPRN-UMIN000006108
2011/08/0103/08/2011Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseases tuberous sclerosis complex, vitiligo vulgaris, white macules due to congenital diseasesApplication of 0.2% rapamycin gel (base is carboxyvinyl polymer) on white macules of the face twice a day for 12 weeks.
When it is possible, application of 0.2% rapamycin gel twice a day for 12 weeks on white macules of the non-exposed part to the sun.
Department of Dermatology Graduate School of Medicine, Osaka UniversityNULLRecruitingNot applicableNot applicableMale and Female21Phase 2;Phase 3Japan
64EUCTR2010-022583-13-IT
(EUCTR)
06/07/201127/04/2011An 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 STUDYAn 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 FARMANULLNot RecruitingFemale: yes
Male: yes
250Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy
65EUCTR2010-022583-13-BE
(EUCTR)
01/06/201103/03/2011An 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 STUDYAn 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 AGNULLNot RecruitingFemale: yes
Male: yes
250Phase 3Czech Republic;Hungary;Greece;Spain;Belgium;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
66EUCTR2007-006997-27-BE
(EUCTR)
01/06/201129/05/2009A 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/AA 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 AGNULLNot RecruitingFemale: yes
Male: yes
99Belgium;Netherlands;Germany;United Kingdom;Italy
67EUCTR2010-022583-13-CZ
(EUCTR)
01/06/201123/02/2011An 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 STUDYAn 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 AGNULLNot RecruitingFemale: yes
Male: yes
250Phase 3France;Hungary;Czech Republic;Greece;Belgium;Spain;Germany;Italy
68EUCTR2010-022655-29-NL
(EUCTR)
30/05/201122/10/2010Randomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATERandomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATE Epilepsy in children with Tuberous Sclerosis ComplexTrade Name: RapamuneErasmus MCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Netherlands
69EUCTR2010-022583-13-GR
(EUCTR)
18/04/201124/03/2011The 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 AGNULLNot RecruitingFemale: yes
Male: yes
250Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy
70EUCTR2010-022583-13-HU
(EUCTR)
31/03/201122/02/2011An 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 STUDYAn 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 AGNULLNot RecruitingFemale: yes
Male: yes
250Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
71NCT01289912
(ClinicalTrials.gov)
January 20112/2/2011Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)Randomized Double-Blind Phase 2 Trial Of RAD001 For Neurocognition In Individuals With Tuberous Sclerosis ComplexTuberous Sclerosis ComplexDrug: RAD001;Drug: PlaceboMustafa SahinTuberous Sclerosis Alliance;Autism Speaks;Novartis Pharmaceuticals;Seizure Tracker LLCCompleted6 Years21 YearsAll52Phase 2United States
72NCT01266291
(ClinicalTrials.gov)
August 201022/12/2010Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) PatientsSabril for Treatment of Adult Refractory Partial Seizures Symptomatic of Tuberous Sclerosis: An Open Label, Phase IV Prospective Safety and Tolerability StudyComplex Partial SeizuresDrug: vigabatrinUniversity of PennsylvaniaH. Lundbeck A/STerminated18 YearsN/AAll1Phase 4United States
73EUCTR2007-006997-27-NL
(EUCTR)
25/03/201003/12/2014Effectiveness 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 AGNULLNot RecruitingFemale: yes
Male: yes
99Canada;Poland;Belgium;Australia;Russian Federation;Germany;Netherlands;Italy;United Kingdom
74EUCTR2008-002113-48-NL
(EUCTR)
16/03/201002/04/2009A 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 AGNULLNot RecruitingFemale: yes
Male: yes
99France;Spain;Germany;Netherlands;Italy;United Kingdom
75EUCTR2007-006997-27-DE
(EUCTR)
12/03/201020/01/2010A 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/AA 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 AGNULLNot RecruitingFemale: yes
Male: yes
99Italy;Belgium;Netherlands;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
76NCT01070316
(ClinicalTrials.gov)
January 201015/2/2010Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis ComplexEpilepsy;Tuberous Sclerosis ComplexDrug: EverolimusChildren's Hospital Medical Center, CincinnatiNovartisCompleted2 YearsN/AAll20Phase 1;Phase 2United States
77NCT01031901
(ClinicalTrials.gov)
December 200910/12/2009Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;NeurofibromaDrug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycinThe University of Texas Health Science Center, HoustonSociety for Pediatric DermatologyCompleted13 YearsN/ABoth52Phase 1United States
78EUCTR2007-006997-27-GB
(EUCTR)
16/09/200906/07/2009A 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/AA 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).Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
99Phase 3Belgium;Netherlands;Germany;Italy;United Kingdom
79EUCTR2008-002113-48-IT
(EUCTR)
03/09/200907/09/2009A 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) - NDA 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 FARMANULLNot RecruitingFemale: yes
Male: yes
99France;Spain;Netherlands;Germany;United Kingdom;Italy
80JPRN-UMIN000002844
2009/09/0103/12/2009Development of topical treatment with rapamycin for skin lesions of tuberous sclerosis complex tuberous sclerosis complexApplication of 0.2% Rapamycin ointment (ointment base is 0.03% tacrolimus ointment) on skin lesion of one side of the face twice a day for 12 weeks.
Application of ointment base (control) on similar skin lesion of the other side of the face twice a day for 12 weeks.
Application of 0.2% Rapamycin ointment (ointment base is white petrolatum) on skin lesion of one side of the face twice a day for 12 weeks.
Application of ointment base (control) on similar skin lesion of the other side of the face twice a day for 12 weeks.
Application of 0.2% Rapamycin gel (base is carboxyvinyl polymer) on skin lesion of one side of the face twice a day for 12 weeks.
Application of gel base (control) on similar skin lesion of the other side of the face twice a day for 12 weeks.
Department of DermatologyGraduate School of Medicine, Osaka UniversityNULLComplete: follow-up completeNot applicableNot applicableMale and Female21Phase 2;Phase 3Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
81EUCTR2008-002113-48-GB
(EUCTR)
06/08/200922/05/2009Efficacy 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)
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
118Phase 3France;United States;Canada;Spain;Russian Federation;Netherlands;Germany;Italy;Japan;United Kingdom
82NCT00789828
(ClinicalTrials.gov)
August 200912/11/2008Efficacy 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 AstrocytomaDrug: Everolimus;Drug: PlaceboNovartis PharmaceuticalsNULLCompletedN/AN/AAll117Phase 3United States;Australia;Belgium;Canada;Germany;Italy;Netherlands;Poland;Russian Federation;United Kingdom
83EUCTR2007-006997-27-IT
(EUCTR)
27/07/200918/06/2009A 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) - NDA 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 FARMANULLNot RecruitingFemale: yes
Male: yes
99Belgium;Netherlands;Germany;United Kingdom;Italy
84NCT00989742
(ClinicalTrials.gov)
July 20092/10/2009Doxycycline In Lymphangioleiomyomatosis (LAM)A Randomised, Double Blind, Placebo Controlled Trial of Doxycycline in Lymphangioleiomyomatosis.Lymphangioleiomyomatosis;Tuberous SclerosisDrug: Doxycycline;Drug: PlaceboUniversity of NottinghamNULLCompleted18 YearsN/AFemale24Phase 4United Kingdom
85NCT00790400
(ClinicalTrials.gov)
April 200910/11/2008Efficacy 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 PlaceboNovartis PharmaceuticalsNULLCompleted18 YearsN/AAll118Phase 3United States;Canada;France;Germany;Italy;Japan;Netherlands;Poland;Russian Federation;Spain;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
86EUCTR2008-002113-48-FR
(EUCTR)
18/03/200919/03/2009A 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 AGNULLNot RecruitingFemale: yes
Male: yes
99Phase 3France;Spain;Netherlands;Germany;Italy;United Kingdom
87NCT00792766
(ClinicalTrials.gov)
December 200817/11/2008Long 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 LymphangioleiomyomatosisTuberous Sclerosis;AngiolipomaDrug: everolimus (RAD001)Children's Hospital Medical Center, CincinnatiNovartisCompleted18 Years65 YearsBoth20Phase 1;Phase 2United States
88NCT01217125
(ClinicalTrials.gov)
October 20086/10/2010Rapamycin In Angiomyolipomas In Patients With Tuberous SclerosisCLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSISAngiomyolipomaDrug: SirolimusFundacio PuigvertMinistry of Health, SpainCompleted10 YearsN/ABoth18Phase 4NULL
89EUCTR2007-005978-30-ES
(EUCTR)
22/01/200803/12/2007Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous SclerosisEnsayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous Sclerosis Angiomiolipomas en pacientes con esclerosis tuberosa (angiomyolipoma of tuberous sclerosis patients)
MedDRA version: 9.1;Level: LLT;Classification code 10045138;Term: Tuberous sclerosis
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus
FUNDACIÓ PUIGVERTNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Spain
90NCT00411619
(ClinicalTrials.gov)
January 200712/12/2006Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis ComplexEverolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis ComplexTuberous Sclerosis;Subependymal Giant Cell AstrocytomaDrug: EverolimusChildren's Hospital Medical Center, CincinnatiNovartisCompleted3 YearsN/AAll28Phase 1;Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
91NCT00490789
(ClinicalTrials.gov)
October 200521/6/2007Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAMA Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: sirolimusCardiff UniversityUniversity of Nottingham;St Georges Hospital Medical School;Royal Sussex County Hospital;The Tuberous Sclerosis Association;Wyeth is now a wholly owned subsidiary of PfizerActive, not recruiting18 Years65 YearsBoth14Phase 2United Kingdom
92NCT00457964
(ClinicalTrials.gov)
August 20056/4/2007RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAMRAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: RAD001Children's Hospital Medical Center, CincinnatiNovartis PharmaceuticalsCompleted18 Years65 YearsBoth36Phase 1;Phase 2United States
93NCT00457808
(ClinicalTrials.gov)
December 20026/4/2007Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAMRapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic LymphangioleiomyomatosisTuberous Sclerosis;LymphangioleiomyomatosisDrug: Rapamycin, sirolimusChildren's Hospital Medical Center, CincinnatiThe LAM Foundation;Tuberous Sclerosis AllianceCompleted18 Years65 YearsBoth25Phase 2United States
94EUCTR2010-022583-13-FR
(EUCTR)
01/03/2011An 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 STUDYAn 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 AGNULLNAFemale: yes
Male: yes
250Phase 3France;Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy
95EUCTR2010-022583-13-DE
(EUCTR)
23/12/2010An 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 STUDYAn 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 AGNULLNot RecruitingFemale: yes
Male: yes
250Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
96EUCTR2013-003795-13-PL
(EUCTR)
20/01/2015Long-term monitoring of growth and development of pediatric patients previously treated with everolimusLong-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 AGNULLNot RecruitingFemale: yes
Male: yes
48Phase 3United States;Canada;Belgium;Poland;Russian Federation
97EUCTR2019-000752-34-CZ
(EUCTR)
02/06/2020Study to evaluate if the study medication, Rapamycin, topical cream, is effective and safe at treating facial angiofibroma in patients 6 years of age and over.A Phase 2/3, multi-center, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison of the efficacy and safety of a topical rapamycin cream for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and over - Dose-ranging efficacy and safety study of topical rapamycin cream Facial Angiofibromas Associated with Tuberous Sclerosis Complex
MedDRA version: 20.0;Level: PT;Classification code 10002429;Term: Angiofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0;Classification code 10073678;Term: Juvenile angiofibroma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps);Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Product Name: Rapamycin cream, topical, 0.5%, 1.0% w/w
Product Code: Not applicable
INN or Proposed INN: SIROLIMUS
Product Name: Rapamycin cream, topical, 0.5%, 1.0% w/w
Product Code: Not applicable
INN or Proposed INN: SIROLIMUS
Dermatology Specialities Limited Partnership (DSLP)NULLNAFemale: yes
Male: yes
120Phase 2;Phase 3United States;Serbia;Hungary;Czech Republic;Slovakia;Spain;Australia;New Zealand
98EUCTR2008-002113-48-DE
(EUCTR)
12/02/2009Efficacy 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 AGNULLNot RecruitingFemale: yes
Male: yes
118Phase 3France;United States;Canada;Spain;Russian Federation;Netherlands;Germany;Italy;United Kingdom;Japan
99EUCTR2016-002977-37-PL
(EUCTR)
09/06/2017Roll-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 AGNULLNAFemale: yes
Male: yes
216Phase 3United States;Taiwan;Greece;Spain;Thailand;Turkey;Russian Federation;Colombia;United Kingdom;France;Hungary;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Japan;Korea, Republic of
100EUCTR2015-002154-12-PL
(EUCTR)
18/11/2016A 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 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.NULLNAFemale: yes
Male: yes
210Phase 3United States;Spain;Poland;Australia;Netherlands;United Kingdom