DDrare: Database of Drug Development for Rare Diseases

206. 脆弱X症候群
［臨床試験数：98，薬物数：73（DrugBank：27），標的遺伝子数：46，標的パスウェイ数：57］

Searched query = "Fragile X 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 = 11C-flumazenil; 18F-FTC-146; ACIDUM ASCORBICUM D6; AF056; AFQ056; AZD7325; AZD7325 (High-Dose); AZD7325 (Low-Dose); Acamprosate; Acetate; Acidum ascorbicum; Alpha-tocopherol; Ampalex; Arbaclofen; Aripiprazole; Ascorbic Acid (Vitamin C) and Alpha-tocopherol (Vitamin E); Ascorbic acid; BPN14770; CBD; CCD 1042, MD 9150000, Mepalon 1042, SPT316; CX516; CX516 (Ampalex®); Donepezil; EGCG; EGCG FontUp; Flumazenil; Fluoxetin; GANAXOLONE; Gaboxadol; Ganaxolone; Lovastatin; Lovastatin, then Minocycline/Lovastatin; MG01CI extended-release tablet; MGlu5 antagonist; Mavoglurant; Metformin; Methylphenidate; Methylphenidate, fluoxetin, risperidone; Minocycline; Minocycline hydrochloride; Minocycline, then Minocycline/Lovastatin; NNZ-2566; NPL-2009; OV101; OV101 (gaboxadol); Other: Biological samples; Other: Cognitive training; Other: Language Intervention; Other: MRI; Oxytocin; Oxytocin 24IU; Oxytocin 48IU; RO4917523; RO4917523 0.5 mg; RO4917523 1.5 mg; Riluzole; Risperidone; Ro 491-7523/F18; Ro 491-7523/F19; STX107; STX209; Sertraline; Sugar pill; TOCOPHERYL ACETATE; Tocopherol; Tocopheryl acetate; Vitamin C; Vitamin C 10mg/Kg Vitamin E 10 mg/Kg; Vitamin E; ZYN002 - CBD Transdermal Gel; [18F]FPEB; [18F]FPEB PET imaging; [18F]flumazenil

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	EUCTR2014-000251-89-BE (EUCTR)	22/10/2014	18/08/2014	A clinical study with an investigational drug called ganaxolone in children with fragile X syndrome	A controlled double-blind crossover trial of ganaxolone in children with fragile X syndrome - GNX study	behavioral problems in children with fragile X syndrome MedDRA version: 17.0;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 Name: Ganaxolone Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT316 INN or Proposed INN: GANAXOLONE	Antwerp University Hospital	NULL	Not Recruiting			Female: yes Male: yes	60		United States;Belgium
2	NCT01725152 (ClinicalTrials.gov)	November 2012	7/11/2012	Ganaxolone Treatment in Children With Fragile X Syndrome	A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome	Fragile x Syndrome	Drug: Ganaxolone;Drug: Placebo	Marinus Pharmaceuticals	University of California, Davis;U.S. Army Medical Research and Development Command	Completed	6 Years	17 Years	All	59	Phase 2	United States;Belgium

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

EUCTR2014-000251-89-BE
(EUCTR)

22/10/2014

18/08/2014

A clinical study with an investigational drug called ganaxolone in children with fragile X syndrome

A controlled double-blind crossover trial of ganaxolone in children with fragile X syndrome - GNX study

behavioral problems in children with fragile X syndrome
MedDRA version: 17.0;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 Name: Ganaxolone
Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT316
INN or Proposed INN: GANAXOLONE

Antwerp University Hospital

NULL

Not Recruiting

Female: yes
Male: yes

United States;Belgium

NCT01725152
(ClinicalTrials.gov)

November 2012

7/11/2012

Ganaxolone Treatment in Children With Fragile X Syndrome

A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome

Fragile x Syndrome

Drug: Ganaxolone;Drug: Placebo

Marinus Pharmaceuticals

University of California, Davis;U.S. Army Medical Research and Development Command

Completed

6 Years

17 Years

All

Phase 2

United States;Belgium