TRIHEPTANOINA ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
248グルコーストランスポーター1欠損症2

248. グルコーストランスポーター1欠損症


臨床試験数 : 30 薬物数 : 9 - (DrugBank : 1) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2015-000389-69-ES
(EUCTR)
22/05/201727/03/2017A trial to assess the long term safety and efficacy of UX007 in subjects who have already participated in a UX007 study.An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome Glucose Transporter Type 1 deficiency syndrome
MedDRA version: 19.1;Level: LLT;Classification code 10061032;Term: Carbohydrate transport disorder;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Triheptanoin
Product Code: UX007
INN or Proposed INN: no disponible
Other descriptive name: TRIHEPTANOINA
Ultragenyx Pharmaceutical IncNULLNot Recruiting Female: yes
Male: yes
40 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Spain;Denmark;Australia;Israel;Italy;United Kingdom
2EUCTR2013-003771-35-ES
(EUCTR)
19/11/201510/08/2015A trial to assess the safety and efficacy of UX007 in patients with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome Glucose Transporter Type 1 deficiency syndrome
MedDRA version: 18.0;Level: HLGT;Classification code 10039911;Term: Seizures (incl subtypes);System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Triheptanoin / Triheptanoina
Product Code: UX007
INN or Proposed INN: Not available
Other descriptive name: TRIHEPTANOIN
Ultragenyx Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
40Phase 2France;United States;Hungary;Spain;Denmark;Italy;United Kingdom