DDrare: Database of Drug Development for Rare Diseases

Searched query = "Klippel-Trenaunay-Weber syndrome", "Klippel-Trenauney-Weber syndrome", "Klippel-Trenaunay syndrome", "Klippel-Trenauney syndrome", "KTS", "Parkes Weber syndrome", "PWS"
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.

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	JPRN-UMIN000030522	2017/11/14	22/12/2017	A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies		Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformations	Body surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day. BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.	Gifu University	NULL	Recruiting	Not applicable	Not applicable	Male and Female	50	Not selected	Japan

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

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sponsor

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sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

JPRN-UMIN000030522

2017/11/14

22/12/2017

A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies

Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformations

Body surface area (BSA) >= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.
BSA < 1.0m2: an initial dose of sirolimus (1mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL. Maximum dose of sirolimus is 4 mg per day.

Gifu University

NULL

Recruiting

Not applicable

Male and Female

Not selected

Japan