DDrare: Database of Drug Development for Rare Diseases

89. リンパ脈管筋腫症
［臨床試験数：38，薬物数：42（DrugBank：19），標的遺伝子数：26，標的パスウェイ数：134］

Searched query = "Lymphangioleiomyomatosis", "LAM"
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 = 68Ga-NEB; Afinitor; Afinitor 5mg Tablets; Albuterol; Albuterol inhaler; Albuterol nebulizer; Bevacizumab; Celecoxib; Cetuximab; Doxycycline; Doxycycline 100 mg Capsules; EVEROLIMUS; Everolimus; Everolimus (RAD001); Everolimus Oral Product; Glutamine; Hydroxychloroquine; Imatinib; Imatinib Mesylate 400Mg Capsule; Letrozole; Nintedanib; Octreotide; Ofev; Other: Laboratory Biomarker Analysis; Other: Pharmacological Study; PFT; Procedure: PFT; RAD001; RAD001 5mg; Rapamycin; Rapamycin, sirolimus; Resveratrol; Saracatinib; Simvastatin; Sirolimus; Sirolimus Oral Product; Sirolimus and Hydroxychloroquine 200 mg; Sirolimus and Hydroxychloroquine 400 mg; Srolimus; Temsirolimus; Valproic Acid; Valproic acid

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	EUCTR2010-019825-32-FR (EUCTR)	29/06/2010	28/05/2010	An exploratory, open label, non-randomized, within-patient multiple dose-escalation safety, tolerability, PK and efficacy trial of RAD001 (everolimus) in patients withLymphangioleiomyomatosis	An exploratory, open label, non-randomized, within-patient multiple dose-escalation safety, tolerability, PK and efficacy trial of RAD001 (everolimus) in patients withLymphangioleiomyomatosis	Lymphangioleiomyomatosis MedDRA version: 12.1;Level: LLT;Classification code 10049459;Term: Lymphangioleiomyomatosis	Product Code: RAD001 INN or Proposed INN: everolimus Trade Name: Afinitor Product Code: RAD001 INN or Proposed INN: everolimus Trade Name: Afinitor Product Code: RAD001 INN or Proposed INN: everolimus	Novartis Pharma Service AG	NULL	Not Recruiting			Female: yes Male: no	24		France;Italy
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

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registration

Public_title

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Condition

Intervention

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sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
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Phase

Countries

EUCTR2010-019825-32-FR
(EUCTR)

29/06/2010

28/05/2010

An exploratory, open label, non-randomized, within-patient multiple dose-escalation safety, tolerability, PK and efficacy trial of RAD001 (everolimus) in patients withLymphangioleiomyomatosis

Lymphangioleiomyomatosis
MedDRA version: 12.1;Level: LLT;Classification code 10049459;Term: Lymphangioleiomyomatosis

Product Code: RAD001
INN or Proposed INN: everolimus
Trade Name: Afinitor
Product Code: RAD001
INN or Proposed INN: everolimus
Trade Name: Afinitor
Product Code: RAD001
INN or Proposed INN: everolimus

Novartis Pharma Service AG

NULL

Not Recruiting

Female: yes
Male: no

France;Italy

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