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
Showing 1 to 10 of 24 diseases
| 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 | NCT03773770 (ClinicalTrials.gov) | August 1, 2019 | 10/12/2018 | Expanded Access to Triheptanoin | An Open-label Intermediate-size Treatment Protocol for the Urgent Treatment of Seriously Ill Patients With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) With Triheptanoin (UX007) An Open-label Intermediate-size Treatment Protocol for the Urgent Treatment of Seriously Ill Patient ... | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Drug: Triheptanoin | Ultragenyx Pharmaceutical Inc | NULL | Available | N/A | N/A | All | NULL | ||
| 2 | NCT03301532 (ClinicalTrials.gov) | June 5, 2018 | 29/8/2017 | Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D | Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency Compatibility of Triheptanoin(C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transpo ... | GLUT1DS1 | Drug: Triheptanoin | University of Texas Southwestern Medical Center | National Institute of Neurological Disorders and Stroke (NINDS) | Active, not recruiting | 30 Months | 35 Years | All | 10 | Phase 2 | United States |
| 3 | EUCTR2015-000389-69-DK (EUCTR) | 23/08/2017 | 13/06/2017 | A trial to assess the long term safety and efficacy of UX007 in subjects who have already participated in a UX007 study. A trial to assess the long term safety and efficacy of UX007 in subjects who have already participat ... | An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with ... | Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;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] Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;Level: LLT;Classification code 1 ... | Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: Not available Other descriptive nam ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | United States;Spain;Australia;Denmark;Israel;United Kingdom | ||
| 4 | EUCTR2015-005536-17-GB (EUCTR) | 04/08/2017 | 11/07/2017 | A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated wi ... | A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and ... | Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;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] Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;Level: LLT;Classification code 1 ... | Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIH ... | Ultragenyx Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;United States;Spain;Australia;Israel;Germany;Italy;United Kingdom | ||
| 5 | EUCTR2015-005536-17-FR (EUCTR) | 30/06/2017 | 16/05/2017 | A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated wi ... | A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and ... | Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;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] Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;Level: LLT;Classification code 1 ... | Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIH ... | Ultragenyx Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | United States;France;Spain;Ireland;Australia;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 6 | EUCTR2013-003771-35-DK (EUCTR) | 07/06/2017 | 11/04/2017 | A trial to assess the safety and efficacy of UX007 in patients with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A trial to assess the safety and efficacy of UX007 in patients with Glucose Transporter Type 1 Defic ... | 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 A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Effica ... | Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;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] Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;Level: LLT;Classification code 1 ... | Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: Not available Other descriptive nam ... | Ultragenyx Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | France;United States;Hungary;Spain;Australia;Denmark;Israel;Italy;United Kingdom | ||
| 7 | 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. A trial to assess the long term safety and efficacy of UX007 in subjects who have already participat ... | An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with ... | 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] Glucose Transporter Type 1 deficiency syndrome MedDRA version: 19.1;Level: LLT;Classification code 1 ... | Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: no disponible Other descriptive name: TRIHEPTANOINA Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: no disponible Other descriptive nam ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | France;United States;Spain;Denmark;Australia;Israel;Italy;United Kingdom | ||
| 8 | EUCTR2015-005536-17-DE (EUCTR) | 17/05/2017 | 01/03/2017 | A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated wi ... | A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and ... | Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;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] Glucose Transporter Type 1 deficiency syndrome MedDRA version: 20.0;Level: LLT;Classification code 1 ... | Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIH ... | Ultragenyx Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;United States;Spain;Australia;Israel;Germany;Italy;United Kingdom | ||
| 9 | NCT03041363 (ClinicalTrials.gov) | March 29, 2017 | 31/1/2017 | Treatment Development of Triheptanoin (G1D) | Treatment Development of Triheptanoin (C7) for Glucose Transporter Type I Deficiency (G1D): A Phase I Maximum Tolerable Dose Trial Treatment Development of Triheptanoin(C7) for Glucose Transporter Type I Deficiency (G1D): A Phase I ... | Epilepsy;GLUT1DS1;Glut1 Deficiency Syndrome 1, Autosomal Recessive;Glucose Metabolism Disorders;Glucose Transport Defect;Glucose Transporter Type 1 Deficiency Syndrome;Glucose Transporter Protein Type 1 Deficiency Syndrome Epilepsy;GLUT1DS1;Glut1 Deficiency Syndrome 1, Autosomal Recessive;Glucose Metabolism Disorders;Gluc ... | Drug: Triheptanoin | Juan Pascual | National Institute of Neurological Disorders and Stroke (NINDS) | Completed | 2 Years | 35 Years | All | 12 | Phase 1 | United States |
| 10 | EUCTR2015-005536-17-ES (EUCTR) | 17/03/2017 | 10/03/2017 | A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated wi ... | A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and ... | 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] Glucose Transporter Type 1 deficiency syndrome MedDRA version: 19.1;Level: LLT;Classification code 1 ... | Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIH ... | Ultragenyx Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;United States;Spain;Ireland;Australia;Israel;Netherlands;Germany;Italy;United Kingdom |