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	NCT03525834 (ClinicalTrials.gov)	November 9, 2018	3/5/2018	Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.	Phase IV, Single Arm Study of Safety and Efficacy of Everolimus in Chinese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma Not Requiring Immediate Surgery	Renal Angiomyolipoma	Drug: everolimus	Novartis Pharmaceuticals	NULL	Completed	18 Years	N/A	All	40	Phase 4	China
2	EUCTR2008-002113-48-DE (EUCTR)		12/02/2009	Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)	A randomized double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - EXIST-2	Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) MedDRA version: 16.1;Level: PT;Classification code 10051810;Term: Angiomyolipoma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Afinitor Product Name: RAD001 5mg Product Code: RAD001 INN or Proposed INN: EVEROLIMUS	Novartis Pharma Services AG	NULL	Not Recruiting			Female: yes Male: yes	118	Phase 3	France;United States;Canada;Spain;Russian Federation;Netherlands;Germany;Italy;United Kingdom;Japan
3	EUCTR2010-022583-13-FR (EUCTR)		01/03/2011	An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA - EFFECTS STUDY	An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA - EFFECTS STUDY	The study will evaluate the safety of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC).	Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Afinitor Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS Trade Name: Afinitor Product Name: everolimus Product Code: RAD001 INN or Proposed INN: everolimus Other descriptive name: EVEROLIMUS	Novartis Pharma Services AG	NULL	NA			Female: yes Male: yes	250	Phase 3	France;Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy

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

NCT03525834
(ClinicalTrials.gov)

November 9, 2018

3/5/2018

Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.

Phase IV, Single Arm Study of Safety and Efficacy of Everolimus in Chinese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma Not Requiring Immediate Surgery

Renal Angiomyolipoma

Drug: everolimus

Novartis Pharmaceuticals

NULL

Completed

18 Years

N/A

All

Phase 4

China

EUCTR2008-002113-48-DE
(EUCTR)

12/02/2009

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

A randomized double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - EXIST-2

Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
MedDRA version: 16.1;Level: PT;Classification code 10051810;Term: Angiomyolipoma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: Afinitor
Product Name: RAD001 5mg
Product Code: RAD001
INN or Proposed INN: EVEROLIMUS

Novartis Pharma Services AG

NULL

Not Recruiting

Female: yes
Male: yes

118

Phase 3

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

EUCTR2010-022583-13-FR
(EUCTR)

01/03/2011

An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA - EFFECTS STUDY

The study will evaluate the safety of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC).

Product Name: everolimus
Product Code: RAD001
INN or Proposed INN: everolimus
Other descriptive name: EVEROLIMUS
Trade Name: Afinitor
Product Name: everolimus
Product Code: RAD001
INN or Proposed INN: everolimus
Other descriptive name: EVEROLIMUS
Trade Name: Afinitor
Product Name: everolimus
Product Code: RAD001
INN or Proposed INN: everolimus
Other descriptive name: EVEROLIMUS

Novartis Pharma Services AG

NULL

Female: yes
Male: yes

250

Phase 3

France;Czech Republic;Hungary;Greece;Belgium;Spain;Germany;Italy