DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2015-002154-12-ES (EUCTR)	30/09/2016	15/07/2016	A 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.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	192	Phase 3	France;United States;Spain;Australia;United Kingdom
2	EUCTR2015-002154-12-PL (EUCTR)		18/11/2016	A 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.	NULL	NA			Female: yes Male: yes	210	Phase 3	United States;Spain;Poland;Australia;Netherlands;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

EUCTR2015-002154-12-ES
(EUCTR)

30/09/2016

15/07/2016

A 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.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

192

Phase 3

France;United States;Spain;Australia;United Kingdom

EUCTR2015-002154-12-PL
(EUCTR)

18/11/2016

A study of cannabidiol (GWP42003-P, CBD) in patients with tuberous sclerosis complex who experience 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.

NULL

Female: yes
Male: yes

210

Phase 3

United States;Spain;Poland;Australia;Netherlands;United Kingdom