DDrare: Database of Drug Development for Rare Diseases

102. ルビンシュタイン・テイビ症候群
［臨床試験数：3，薬物数：8（DrugBank：1），標的遺伝子数：7，標的パスウェイ数：17］

Searched query = "Rubinstein-Taybi syndrome", "RSTS"
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 = Culture of lymphoblastoid line from blood sample; Depakine; Other: Functional involvement of identified epigenetic alterations; Other: Generation of Induced Pluripotent Stem Cells (iPSC) from fibroblasts obtained by skin biopsy; Other: Histone acetylation profiles of cells of SRT patients with CREBBP mutations; Procedure: skin biopsy for the primary fibroblast culture and a 15 ml blood sample (3 unnamed samples of 5ml) in each of the 4 SRT patients included.; Sodium valproate; Valproate

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	NCT01619644 (ClinicalTrials.gov)	April 2012	12/6/2012	Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial	Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial	Rubinstein-Taybi Syndrome	Drug: sodium valproate;Drug: Placebo	University Hospital, Bordeaux	Fondation Syndrome de Rubinstein-Taybi	Completed	6 Years	21 Years	Both	41	Phase 2	France

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

NCT01619644
(ClinicalTrials.gov)

April 2012

12/6/2012

Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial

Rubinstein-Taybi Syndrome

Drug: sodium valproate;Drug: Placebo

University Hospital, Bordeaux

Fondation Syndrome de Rubinstein-Taybi

Completed

6 Years

21 Years

Both

Phase 2

France