DDrare: Database of Drug Development for Rare Diseases

310. 先天異常症候群
［臨床試験数：10，薬物数：18（DrugBank：10），標的遺伝子数：3，標的パスウェイ数：7］

Searched query = "Congenital anomalies syndrome", "Trisomy 1q", "9q34 deletion syndrome", "Cornelia de Lange syndrome", "CdLS", "Smith-Lemli-Opitz syndrome", "SLO syndrome"
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 = Antioxidants; Cholesterol; Cholesterol supplementation; Cholic Acid; Crystalline cholesterol oil-based suspension; Cysteine; DHC; Egg; Egg substitute, without cholesterol; Egg yolk; Egg yolk preparation with cholesterol; Gold; Lactose; Lovastatin; N-acetyl cysteine; OraPlus; Simvastatin; Simvastatin Susp.

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	NCT00114634 (ClinicalTrials.gov)	June 2005	15/6/2005	Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome	Short-Term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome	Smith-Lemli-Opitz Syndrome	Dietary Supplement: Egg yolk preparation with cholesterol;Dietary Supplement: Egg substitute, without cholesterol	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	NULL	Completed	4 Years	17 Years	All	13	Phase 2	United States

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

NCT00114634
(ClinicalTrials.gov)

June 2005

15/6/2005

Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome

Short-Term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome

Smith-Lemli-Opitz Syndrome

Dietary Supplement: Egg yolk preparation with cholesterol;Dietary Supplement: Egg substitute, without cholesterol

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NULL

Completed

4 Years

17 Years

All

Phase 2

United States