Bezafibrate (INN 3968) ( DrugBank: Bezafibrate )

1 disease
告示番号疾患名（ページ内リンク）臨床試験数
324メチルグルタコン酸尿症1

324. メチルグルタコン酸尿症

臨床試験数 : 4 /  薬物数 : 4 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 10
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2015-001382-10-GB
(EUCTR)		21/01/201924/08/2015A study comparing the drug 'bezafibrate' against a placebo in the treatment of Barth SyndromeTreatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service - CARDIOMAN Barth Syndrome is a rare, life threatening, genetic disease which affects young males. It is caused by abnormal fats (lipids) in the powerhouses of cells (mitochondria) and those who suffer with it often develop heart failure, heart rhythm abnormalities, bacterial infections, poor growth or feeding, weak muscles, developmental delay, severe exercise intolerance, lethargy and fatigue.
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Trade Name: Bezafibrate
Product Name: Bezafibrate
INN or Proposed INN: Bezafibrate (INN 3968)		University Hospitals Bristol NHS Foundation TrustNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes		15 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom