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	EUCTR2011-001319-30-GB (EUCTR)	04/04/2012	05/09/2011	Metformin as a treatment for people with the genetic disease Tuberous Sclerosis Complex.	A randomized, double-blind, parallel group, placebo-controlled trial of metformin in tuberous sclerosis complex. - Metformin in Tuberous Sclerosis Complex (MiTS) V1.0	Tuberous Sclerosis Complex MedDRA version: 14.1;Level: PT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: metformin (generic) Product Name: Metformin Product Code: n/a INN or Proposed INN: metformin hydrochloride Other descriptive name: -	University Hospitals Bristol NHS Foundation Trust	NULL	Not Recruiting			Female: yes Male: yes	100	Phase 4	United Kingdom

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

EUCTR2011-001319-30-GB
(EUCTR)

04/04/2012

05/09/2011

Metformin as a treatment for people with the genetic disease Tuberous Sclerosis Complex.

A randomized, double-blind, parallel group, placebo-controlled trial of metformin in tuberous sclerosis complex. - Metformin in Tuberous Sclerosis Complex (MiTS) V1.0

Tuberous Sclerosis Complex
MedDRA version: 14.1;Level: PT;Classification code 10045138;Term: Tuberous sclerosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: metformin (generic)
Product Name: Metformin
Product Code: n/a
INN or Proposed INN: metformin hydrochloride
Other descriptive name: -

University Hospitals Bristol NHS Foundation Trust

NULL

Not Recruiting

Female: yes
Male: yes

100

Phase 4

United Kingdom