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 158. 結節性硬化症 [臨床試験数:54,薬物数:37(DrugBank:14),標的遺伝子数:19,標的パスウェイ数:106] 

Searched query = "Tuberous sclerosis", "Tuberous sclerosis complex"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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PhaseCountries
1NCT03826628July 28, 20194 November 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 PartnershipRecruiting6 Years65 YearsAll120Phase 2/Phase 3United States;Australia;New Zealand;Spain
2NCT03525834November 9, 201828 October 2019Safety 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 PharmaceuticalsNot recruiting18 YearsN/AAll40Phase 4China
3EUCTR2018-002531-18-FR31/10/201828 February 2019Topical 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. EVEROSTangiofibromas
MedDRA version: 20.0 Level: PT Classification code 10002429 Term: Angiofibroma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]
Product Name: Everolimus
Pharmaceutical Form: Cream
INN or Proposed INN: EVEROLIMUS
CAS Number: 159351-69-6
Other descriptive name: EVEROLIMUS
Concentration unit: % (V/V) percent volume/volume
Concentration type: equal
Concentration number: 0.3-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)
Product Name: Everolimus
Pharmaceutical Form: Cream
INN or Proposed INN: Everolimus
CAS Number: 159351-69-6
Other descriptive name: EVEROLIMUS
Concentration unit: % (V/V) percent volume/volume
Concentration type: equal
Concentration number: 0.7-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)
Product Name: Everolimus
Pharmaceutical Form: Cream
INN or Proposed INN: Everolimus
CAS Number: 159351-69-6
Other descriptive name: EVEROLIMUS
Concentration unit: % (V/V) percent volume/volume
Concentration type: equal
Concentration number: 1-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)
Hospices Civils de LyonAuthorised Female: yes
Male: yes
146Phase 2;Phase 3France
4NCT02860494September 20185 March 2018Topical 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: Placebo;Drug: EverolimusHospices Civils de LyonNot recruitingN/A2 YearsAll96Phase 2/Phase 3France
5NCT03254680March 201811 June 2018Turmeric as Treatment in EpilepsyTurmeric as Treatment in EpilepsyEpilepsy;Dravet Syndrome;Lennox-Gastaut Syndrome;Tuberous Sclerosis;Focal SeizuresDietary Supplement: TurmericNew York University School of MedicineNot recruiting1 Year70 YearsAll0N/AUnited States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03356769November 20, 201716 December 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 Pharmaceutical Group Co. Ltd.Recruiting6 Years30 YearsAll98Phase 2China
7NCT03363763April 12, 201730 September 2019Topical 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, IncRecruiting2 Years18 YearsAll45Phase 2United States
8NCT02962414April 2, 20174 November 2019Roll-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 PharmaceuticalsNot 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
9EUCTR2016-002977-37-HU24/02/201713 March 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
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2-
Novartis Pharma AGAuthorisedFemale: 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
10EUCTR2016-002408-13-NL07/02/20172 October 2017Bumetanide to treat behavioral problems in TSCBumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors - BATSCHTuberous Sclerosis Complex
MedDRA version: 20.0 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]
Trade Name: bumetanide
Product Name: bumetanide
Product Code: bumetanide
Pharmaceutical Form: Tablet
UMC UtrechtAuthorisedFemale: yes
Male: yes
Phase 2Netherlands
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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Target_
size
PhaseCountries
11NCT02849457December 201626 August 2019Preventing 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)RecruitingN/A6 MonthsAll80Phase 2United States
12NCT02544750August 20169 October 2018An 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 LtdRecruiting1 Year65 YearsAll210Phase 3United States
13ChiCTR-OPN-160082362016-06-0118 April 2017TSC1/2 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 complexrenal angiomyolipoma10mg Everolimus:Everolimus 10mg QD for three months;5mg Everolimus:Everolimus 5mg QD for six months;General Hospital of PLANot Recruiting1860Both10mg Everolimus:50;5mg Everolimus:50;Post-marketChina
14NCT02634931December 20154 March 2019Long-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 gelNobelpharmaNot recruiting3 YearsN/AAll94Phase 3Japan
15NCT02635789December 201516 December 2017Phase 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 gelNobelpharmaNot recruiting3 YearsN/AAll62Phase 3Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16ChiCTR-OOB-150065352015-05-2618 April 2017A 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 Complextuberous sclerosis complexCase series:rapamycin;Chinese PLA General HospitalRecruiting118BothCase series:500;OtherChina
17NCT02104011May 22, 201529 January 2018Treatment 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, BordeauxNot recruiting18 YearsN/AAll2Phase 2France
18EUCTR2015-002154-12-PL28 February 2019A 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 seizuresTuberous Sclerosis Complex (TSC)
MedDRA version: 20.0 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Cannabidiol (CBD)
Product Code: GWP42003-P
Pharmaceutical Form: Oral solution
INN or Proposed INN: N/A
CAS Number: 13956-29-1
Current Sponsor code: GWP42003-P
Other descriptive name: CANNABIDIOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
GW Research Ltd.Not Available Female: yes
Male: yes
210Phase 3United States;Spain;Poland;Australia;Netherlands;United Kingdom
19EUCTR2013-003795-13-BE10/12/201411 June 2018Long-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-LTSubependymal Giant Cell Astrocytoma (SEGA) associated with Tuberous Sclerosis Complex (TSC)
MedDRA version: 20.0 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
Pharmaceutical Form: Tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2-
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
Pharmaceutical Form: Tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2.5-
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
Pharmaceutical Form: Tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 3-
Trade Name: Votubia
Product Name: everolimus
Product Code: RAD001
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Novartis Pharma Services AGAuthorisedFemale: yes
Male: yes
48Phase 3;Phase 4United States;Canada;Poland;Belgium;Russian Federation
20JPRN-UMIN0000151142014/09/112 April 2019Clinical study of safety and efficacy of rapamycin topical medication for skin lesions due to tuberous sclerosis complexTuberous 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 UniversityRecruitingNot applicable70years-oldMale and Female24Phase 2,3Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
21NCT02201212September 201411 November 2019Everolimus 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 PharmaceuticalsNot recruiting18 YearsN/AAll30Phase 2United States
22NCT02061397March 201425 February 2019Safety 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: SimvastatinUniversity of PennsylvaniaThe LAM FoundationNot recruiting18 YearsN/AFemale10Phase 1/Phase 2United States
23NCT02451696January 201430 September 2019A 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 HealthRecruiting2 Years40 YearsAll30Phase 2United States
24JPRN-UMIN0000124202013/12/102 April 2019Randomized, 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 Dermatology Graduate School of Medicine, Osaka UniversityNot Recruiting3years-old65years-oldMale and Female36Phase 1,2Japan
25NCT01853423October 201329 August 2016Tuberous Sclerosis Complex: Facial Angiofibroma Skin CreamTuberous Sclerosis Complex: Facial Angiofibroma Skin CreamFacial AngiofibromaDrug: RapamuneLe Bonheur Children's HospitalPfizer;University of Tennessee Health Science CenterNot recruiting3 Years45 YearsBoth11Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
26NCT03140449September 5, 201316 December 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 HospitalNot recruiting7 Years65 YearsAll52Phase 3
27JPRN-UMIN0000115592013/09/012 April 2019Therpeutic drug monitoring of Everolimus in patients with renal cell carcinoma and tuberous sclerosis1.advanced renal cell carcinoma 2.renal angiomyolipoma associated with tuberous sclerosis complexEverolimus 5mg/day.Department of Urology, Keio University School of MedicineNot Recruiting20years-old90years-oldMale and Female43Not selectedJapan
28NCT01929642July 20135 February 2018Rapalogues 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.Not recruiting2 Years30 YearsAll3Phase 2United States
29NCT01713946April 29, 201319 November 2018A 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 PharmaceuticalsNot recruiting2 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
30EUCTR2012-005397-63-ES19/04/201327 October 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
Pharmaceutical Form: Tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Novartis Farmacéutica, S.A,Not RecruitingFemale: yes
Male: yes
Spain
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
31NCT01730209November 201225 May 2015Efficacy 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
32EUCTR2011-006308-12-ES16/07/201214 August 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
Pharmaceutical Form: Oral solution
INN or Proposed INN: RAPAMUNE
Current Sponsor code: SIROLIMUS
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 3-
Pharmaceutical form of the placebo: Cutaneous solution
Route of administration of the placebo: Cutaneous use
Fundación Investigación Hospital Ramón y CajalAuthorisedFemale: yes
Male: yes
Phase 2Spain
33NCT01954693June 201212 February 2018A 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 UniversityNovartisNot recruiting16 Years60 YearsAll48Phase 2United Kingdom
34NCT01526356May 201229 June 2015Topical 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, HoustonNot recruitingN/AN/ABoth177Phase 2United States;Australia
35EUCTR2011-001319-30-GB04/04/201228 May 2018Metformin 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) V1.0Tuberous Sclerosis Complex
MedDRA version: 14.1 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: metformin (generic)
Product Name: Metformin
Product Code: n/a
Pharmaceutical Form: Tablet
INN or Proposed INN: metformin hydrochloride
CAS Number: 657-24-9
Current Sponsor code: -
Other descriptive name: -
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
University Hospitals Bristol NHS Foundation TrustNot RecruitingFemale: yes
Male: yes
100Phase 4United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
36EUCTR2010-019519-39-NL14/03/201218 February 2013Efficacy 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) - RAPITTuberous Sclerosis Complex;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]Trade Name: Votubia
Product Name: Everolimus
Product Code: L04AA18
Pharmaceutical Form: Tablet
INN or Proposed INN: Votubia
CAS Number: 159351-69-6
Other descriptive name: EVEROLIMUS
Concentration unit: ng nanogram(s)
Concentration type: range
Concentration number: 5-15
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Erasmus MC - Department of NeurologyAuthorisedFemale: yes
Male: yes
Netherlands
37EUCTR2011-004854-25-GB10/01/201228 February 2019TRON: A trial to examine the effectiveness of the study drug (Everolimus) in the treatment of cognitive problems experienced by patients with tuberous sclerosis.TRON: A randomised, double blind, placebo-controlled study of RAD001 (Everolimus) in the treatment of neurocognitive problems in tuberous sclerosis. - TRON (version 1.0)Tuberous Sclerosis Complex (TSC)
MedDRA version: 14.0 Level: PT Classification code 10002649 Term: Anorexia nervosa System Organ Class: 10037175 - Psychiatric disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Afinitor
Product Name: Everolimus
Pharmaceutical Form: Tablet
INN or Proposed INN: Everolimus
CAS Number: 159351-69-6
Other descriptive name: RAD001
Concentration unit: mg milligram(s)
Concentration type: equal
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Cardiff UniversityNot Recruiting Female: yes
Male: yes
48Phase 2United Kingdom
38JPRN-UMIN0000061082011/08/012 April 2019Clinical trial for development of topical rapamycin treatment for white macules due to tuberous sclerosis complex, vitiligo vulgaris, and other congenital diseasestuberous 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 UniversityRecruitingNot applicableNot applicableMale and Female21Phase 2,3Japan
39EUCTR2010-022655-29-NL30/05/201119 March 2012Randomised 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) - RATEEpilepsy in children with Tuberous Sclerosis ComplexTrade Name: Rapamune
Pharmaceutical Form: Oral solution
Erasmus MCAuthorisedFemale: yes
Male: yes
Netherlands
40NCT01289912January 20115 February 2018Trial 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 LLCNot recruiting6 Years21 YearsAll52Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
41NCT01266291August 201016 December 2017Sabril 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/SNot recruiting18 YearsN/AAll1Phase 4United States
42NCT01070316January 201016 December 2017Everolimus (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, CincinnatiNovartisNot recruiting2 YearsN/AAll20Phase 1/Phase 2United States
43EUCTR2010-022583-13-DE5 August 2014An 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 STUDYThe study will evaluate the safety of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC).Product Name: RAD001 2,5 mg
Product Code: RAD001
Pharmaceutical Form: Tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2.5-
Novartis Pharma Services AGNot RecruitingFemale: yes
Male: yes
250Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy
44NCT01031901December 200919 February 2015Topical 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 DermatologyNot recruiting13 YearsN/ABoth52Phase 1United States
45JPRN-UMIN0000028442009/09/012 April 2019Development of topical treatment with rapamycin for skin lesions of tuberous sclerosis complextuberous 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 Dermatology Graduate School of Medicine, Osaka UniversityNot RecruitingNot applicableNot applicableMale and Female21Phase 2,3Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46NCT00789828August 200919 October 2017Efficacy 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 PharmaceuticalsNot recruitingN/AN/AAll117Phase 3United States;Australia;Belgium;Canada;Germany;Italy;Netherlands;Poland;Russian Federation;United Kingdom
47NCT00989742July 200914 December 2015Doxycycline In Lymphangioleiomyomatosis (LAM)A Randomised, Double Blind, Placebo Controlled Trial of Doxycycline in Lymphangioleiomyomatosis.Lymphangioleiomyomatosis;Tuberous SclerosisDrug: Doxycycline;Drug: PlaceboUniversity of NottinghamNot recruiting18 YearsN/AFemale24Phase 4United Kingdom
48NCT00790400April 200916 December 2017Efficacy 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 PharmaceuticalsNot recruiting18 YearsN/AAll118Phase 3United States;Canada;France;Germany;Italy;Japan;Netherlands;Poland;Russian Federation;Spain;United Kingdom
49NCT00792766December 200819 February 2015Long 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, CincinnatiNovartisNot recruiting18 Years65 YearsBoth20Phase 1/Phase 2United States
50EUCTR2007-005978-30-ES22/01/200819 March 2012Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosa Clinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous SclerosisEnsayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosa Clinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous SclerosisAngiomiolipomas en pacientes con esclerosis tuberosa (angiomyolipoma of tuberous sclerosis patients)
MedDRA version: 9.1 Level: LLT Classification code 10045138 Term: Tuberous sclerosis
Trade Name: Rapamune
Product Name: Rapamune
Pharmaceutical Form: Oral solution
INN or Proposed INN: sirolimus
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1-
Trade Name: Rapamune
Product Name: Rapamune
Pharmaceutical Form: Coated tablet
INN or Proposed INN: sirolimus
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1-
Trade Name: Rapamune
Product Name: Rapamune
Pharmaceutical Form: Coated tablet
INN or Proposed INN: sirolimus
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2-
FUNDACIÓ PUIGVERTAuthorisedFemale: yes
Male: yes
Spain
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51NCT00411619January 200719 October 2017Everolimus (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, CincinnatiNovartisNot recruiting3 YearsN/AAll28Phase 1/Phase 2United States
52NCT00490789October 200519 February 2015Trial 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 PfizerNot recruiting18 Years65 YearsBoth14Phase 2United Kingdom
53NCT00457964August 200519 February 2015RAD001 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 PharmaceuticalsNot recruiting18 Years65 YearsBoth36Phase 1/Phase 2United States
54NCT00457808December 200219 February 2015Rapamycin 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 AllianceNot recruiting18 Years65 YearsBoth25Phase 2United States

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