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	NCT01853423 (ClinicalTrials.gov)	October 2013	12/4/2013	Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream	Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream	Facial Angiofibroma	Drug: Rapamune	Le Bonheur Children's Hospital	Pfizer;University of Tennessee Health Science Center	Completed	3 Years	45 Years	Both	11	Phase 1	United States
2	EUCTR2011-006308-12-ES (EUCTR)	16/07/2012	29/03/2012	Clinical 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 INN or Proposed INN: RAPAMUNE	Fundación Investigación Hospital Ramón y Cajal	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 2	Spain
3	EUCTR2010-022655-29-NL (EUCTR)	30/05/2011	22/10/2010	Randomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATE	Randomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATE	Epilepsy in children with Tuberous Sclerosis Complex	Trade Name: Rapamune	Erasmus MC	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes			Netherlands
4	EUCTR2007-005978-30-ES (EUCTR)	22/01/2008	03/12/2007	Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous Sclerosis	Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous Sclerosis	Angiomiolipomas 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 INN or Proposed INN: sirolimus Trade Name: Rapamune Product Name: Rapamune INN or Proposed INN: sirolimus Trade Name: Rapamune Product Name: Rapamune INN or Proposed INN: sirolimus	FUNDACIÓ PUIGVERT	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes			Spain

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

NCT01853423
(ClinicalTrials.gov)

October 2013

12/4/2013

Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

Facial Angiofibroma

Drug: Rapamune

Le Bonheur Children's Hospital

Pfizer;University of Tennessee Health Science Center

Completed

3 Years

45 Years

Both

Phase 1

United States

EUCTR2011-006308-12-ES
(EUCTR)

16/07/2012

29/03/2012

Clinical 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
INN or Proposed INN: RAPAMUNE

Fundación Investigación Hospital Ramón y Cajal

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Spain

EUCTR2010-022655-29-NL
(EUCTR)

30/05/2011

22/10/2010

Randomised open label cross-over study into the efficacy of RApamycin in children with Tuberous sclerosis complex with intractable Epilepsy (RATE-trial) - RATE

Epilepsy in children with Tuberous Sclerosis Complex

Trade Name: Rapamune

Erasmus MC

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Netherlands

EUCTR2007-005978-30-ES
(EUCTR)

22/01/2008

03/12/2007

Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosaClinical Trial to Determine the Efficacy and Safety of Rapamycin in Angiomyolipomas in Pacients with Tuberous Sclerosis

Angiomiolipomas 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
INN or Proposed INN: sirolimus
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus
Trade Name: Rapamune
Product Name: Rapamune
INN or Proposed INN: sirolimus

FUNDACIÓ PUIGVERT

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Spain