206. 脆弱X症候群 Fragile X syndrome Clinical trials / Disease details


臨床試験数 : 108 薬物数 : 91 - (DrugBank : 36) / 標的遺伝子数 : 52 - 標的パスウェイ数 : 77

  
3 trials found
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1NCT04977986
(ClinicalTrials.gov)
September 13, 202120/7/2021Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X SyndromeA Randomized, Double-Blind, Placebo-Controlled, Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults With Fragile X Syndrome - RECONNECTFragile X SyndromeDrug: ZYN002 - transdermal gel;Drug: PlaceboZynerba Pharmaceuticals, Inc.NULLRecruiting3 Years23 YearsAll204Phase 3United States;Australia;Ireland;United Kingdom
2NCT03614663
(ClinicalTrials.gov)
June 12, 201810/7/2018Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With Fragile X SyndromeFragile X SyndromeDrug: ZYN002 - CBD Transdermal Gel;Other: Placebo Transdermal GelZynerba Pharmaceuticals, Inc.NULLCompleted3 Years17 YearsAll212Phase 2/Phase 3United States;Australia;New Zealand
3EUCTR2021-002542-33-IE
(EUCTR)
10/08/2021Effects and Safety of Test drug ZYN002, applied as a gel onto the skin of children and adolescents with a certain hereditary disease called Fragile X SyndromeA Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome - RECONNECT - RECONNECT Fragile X syndrome (FXS) is a rare genetic disorder caused by the deficiency or absence of Fragile X mental retardation protein (FMRP), an RNA-binding protein and the gene product of the FMR1 gene. FXS is most commonly caused by silencing of the FMR1 gene due to a trinucleotide repeat expansion. In FXS patients, the relative absence of functional FMRP is associated with critical impairments in neurodevelopment and learning, as well as disruption to other neuronal and non-neuronal functions.
MedDRA version: 20.1;Level: PT;Classification code 10017324;Term: Fragile X syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: ZYN002
INN or Proposed INN: n/a
Other descriptive name: CANNABIDIOL
Zynerba Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
204Phase 3United States;Ireland;Australia;United Kingdom