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	NCT02061397 (ClinicalTrials.gov)	March 2014	23/1/2014	Safety of Simvastatin in LAM and TSC	The Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)	Lymphangioleiomyomatosis;Tuberous Sclerosis Complex	Drug: Simvastatin;Drug: Sirolimus Oral Product;Drug: Everolimus Oral Product	University of Pennsylvania	The LAM Foundation	Completed	18 Years	N/A	Female	10	Phase 1;Phase 2	United States

No.

TrialID

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registration

Public_title

Scientific_title

Condition

Intervention

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agemin

Inclusion_
agemax

Inclusion_
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NCT02061397
(ClinicalTrials.gov)

March 2014

23/1/2014

Safety of Simvastatin in LAM and TSC

The Safety of Simvastatin (SOS) in Patients With Pulmonary Lymphangioleiomyomatosis (LAM) and With Tuberous Sclerosis Complex (TSC)

Lymphangioleiomyomatosis;Tuberous Sclerosis Complex

Drug: Simvastatin;Drug: Sirolimus Oral Product;Drug: Everolimus Oral Product

University of Pennsylvania

The LAM Foundation

Completed

18 Years

N/A

Female

Phase 1;Phase 2

United States