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
| 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 | NCT04977986 (ClinicalTrials.gov) | September 13, 2021 | 20/7/2021 | Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome | A 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 - RECONNECT | Fragile X Syndrome | Drug: ZYN002 - transdermal gel;Drug: Placebo | Zynerba Pharmaceuticals, Inc. | NULL | Recruiting | 3 Years | 23 Years | All | 204 | Phase 3 | United States;Australia;Ireland;United Kingdom |
| 2 | NCT03614663 (ClinicalTrials.gov) | June 12, 2018 | 10/7/2018 | Clinical 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 Syndrome | Fragile X Syndrome | Drug: ZYN002 - CBD Transdermal Gel;Other: Placebo Transdermal Gel | Zynerba Pharmaceuticals, Inc. | NULL | Completed | 3 Years | 17 Years | All | 212 | Phase 2/Phase 3 | United States;Australia;New Zealand |
| 3 | EUCTR2021-002542-33-IE (EUCTR) | 10/08/2021 | Effects 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 Syndrome | 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 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. | NULL | NA | Female: yes Male: yes | 204 | Phase 3 | United States;Ireland;Australia;United Kingdom |