DDrare: Database of Drug Development for Rare Diseases

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.

Drug = AED; Afinitor; Afinitor 5mg Tablets; Antiepileptic drug (1 to 3 only); Aspirin; Bumetanide; CANNABIDIOL; CBD; CBD - Oral Solution, is known as Epidiolex, and is the approved name in the USA.; CBD-Oral Solution, is known as Epidiolex, and is the approved name in the USA.; Calcitriol; Cannabidiol; Cannabidiol (CBD); Certican; Certican 1 mg tablets; Doxycycline; EVEROLIMUS; Everolimus; Everolimus (RAD001); Everolimus 2mg dispersible tablet; Everolimus Oral Product; GWP42003-P; Ganaxolone; Gold; L04AA18; Metformin; Metformin (generic); Metformin hydrochloride; NPC-12G gel; Open label RAD001 (only used for post-extension phase); Petrolatum; Propranolol; RAD001; RAD001 1mg; RAD001 2,5 mg; RAD001 5mg; RAD001 round tablets; RAPAMUNE; Rapamune; Rapamune 1 mg/ml solución oral; Rapamycin; Rapamycin cream, topical, 0.5%, 1.0% w/w; Rapamycin, sirolimus; Rapamycin-calcitriol combination; SIROLIMUS; Simvastatin; Sirolimus; Sirolimus 0.2%; Sirolimus 0.4%; Sirolimus Oral Product; Skincerity; Skincerity plus sirolimus/rapamycin; Skinercity plus sirolimus/rapamycin; Standard of care; TAVT-18 (sirolimus); Tacrolimus; Turmeric; VIGABATRIN; Vigabatrin; Vigabatrin (Sabril); Vigabatrin (sabril); Votubia

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01954693 (ClinicalTrials.gov)	June 2012	5/9/2013	A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis	TRON: A Randomised, Double Blind, Placebo-controlled Study of RAD001 (Everolimus) in the Treatment of Neurocognitive Problems in Tuberous Sclerosis	Tuberous Sclerosis	Drug: Placebo;Drug: Everolimus (RAD001)	Cardiff University	Novartis	Unknown status	16 Years	60 Years	All	48	Phase 2	United Kingdom
2	NCT01070316 (ClinicalTrials.gov)	January 2010	15/2/2010	Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)	Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex	Epilepsy;Tuberous Sclerosis Complex	Drug: Everolimus	Children's Hospital Medical Center, Cincinnati	Novartis	Completed	2 Years	N/A	All	20	Phase 1;Phase 2	United States
3	NCT00789828 (ClinicalTrials.gov)	August 2009	12/11/2008	Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)	A Randomized, Double-blind, Placebo-controlled Study of Everolimus in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC)	Tuberous Sclerosis;Subependymal Giant Cell Astrocytoma	Drug: Everolimus;Drug: Placebo	Novartis Pharmaceuticals	NULL	Completed	N/A	N/A	All	117	Phase 3	United States;Australia;Belgium;Canada;Germany;Italy;Netherlands;Poland;Russian Federation;United Kingdom
4	NCT00790400 (ClinicalTrials.gov)	April 2009	10/11/2008	Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)	A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)	Tuberous Sclerosis Complex (TSC);Lymphangioleiomyomatosis (LAM)	Drug: Everolimus (RAD001);Drug: Everolimus Placebo	Novartis Pharmaceuticals	NULL	Completed	18 Years	N/A	All	118	Phase 3	United States;Canada;France;Germany;Italy;Japan;Netherlands;Poland;Russian Federation;Spain;United Kingdom
5	NCT00792766 (ClinicalTrials.gov)	December 2008	17/11/2008	Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)	Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis	Tuberous Sclerosis;Angiolipoma	Drug: everolimus (RAD001)	Children's Hospital Medical Center, Cincinnati	Novartis	Completed	18 Years	65 Years	Both	20	Phase 1;Phase 2	United States
No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
6	NCT00411619 (ClinicalTrials.gov)	January 2007	12/12/2006	Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex	Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex	Tuberous Sclerosis;Subependymal Giant Cell Astrocytoma	Drug: Everolimus	Children's Hospital Medical Center, Cincinnati	Novartis	Completed	3 Years	N/A	All	28	Phase 1;Phase 2	United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT01954693
(ClinicalTrials.gov)

June 2012

5/9/2013

A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis

TRON: A Randomised, Double Blind, Placebo-controlled Study of RAD001 (Everolimus) in the Treatment of Neurocognitive Problems in Tuberous Sclerosis

Tuberous Sclerosis

Drug: Placebo;Drug: Everolimus (RAD001)

Cardiff University

Novartis

Unknown status

16 Years

60 Years

All

Phase 2

United Kingdom

NCT01070316
(ClinicalTrials.gov)

January 2010

15/2/2010

Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)

Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex

Epilepsy;Tuberous Sclerosis Complex

Drug: Everolimus

Children's Hospital Medical Center, Cincinnati

Novartis

Completed

2 Years

N/A

All

Phase 1;Phase 2

United States

NCT00789828
(ClinicalTrials.gov)

August 2009

12/11/2008

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)

A Randomized, Double-blind, Placebo-controlled Study of Everolimus in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC)

Tuberous Sclerosis;Subependymal Giant Cell Astrocytoma

Drug: Everolimus;Drug: Placebo

Novartis Pharmaceuticals

NULL

Completed

N/A

All

117

Phase 3

United States;Australia;Belgium;Canada;Germany;Italy;Netherlands;Poland;Russian Federation;United Kingdom

NCT00790400
(ClinicalTrials.gov)

April 2009

10/11/2008

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Tuberous Sclerosis Complex (TSC);Lymphangioleiomyomatosis (LAM)

Drug: Everolimus (RAD001);Drug: Everolimus Placebo

Novartis Pharmaceuticals

NULL

Completed

18 Years

N/A

All

118

Phase 3

United States;Canada;France;Germany;Italy;Japan;Netherlands;Poland;Russian Federation;Spain;United Kingdom

NCT00792766
(ClinicalTrials.gov)

December 2008

17/11/2008

Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)

Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Tuberous Sclerosis;Angiolipoma

Drug: everolimus (RAD001)

Children's Hospital Medical Center, Cincinnati

Novartis

Completed

18 Years

65 Years

Both

Phase 1;Phase 2

United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT00411619
(ClinicalTrials.gov)

January 2007

12/12/2006

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex

Tuberous Sclerosis;Subependymal Giant Cell Astrocytoma

Drug: Everolimus

Children's Hospital Medical Center, Cincinnati

Novartis

Completed

3 Years

N/A

All

Phase 1;Phase 2

United States