248. グルコーストランスポーター1欠損症 Glucose transporter type 1 deficiency Clinical trials / Disease details
臨床試験数 : 29 / 薬物数 : 9 - (DrugBank : 1) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
| 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 | EUCTR2015-000389-69-ES (EUCTR) | 22/05/2017 | 27/03/2017 | A 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 Inc | NULL | Not 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): no | France;United States;Spain;Denmark;Australia;Israel;Italy;United Kingdom | ||
| 2 | EUCTR2013-003771-35-ES (EUCTR) | 19/11/2015 | 10/08/2015 | A 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 Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | France;United States;Hungary;Spain;Denmark;Italy;United Kingdom |