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 : 30 / 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 | NCT03773770 (ClinicalTrials.gov) | January 2, 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) | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Drug: Triheptanoin | Ultragenyx Pharmaceutical Inc | NULL | Available | N/A | N/A | All | NULL | ||
| 2 | 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. | 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: 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] | Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | 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 | United States;Spain;Australia;Denmark;Israel;United Kingdom | ||
| 3 | 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 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) | 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] | Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | 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 | France;United States;Spain;Australia;Israel;Germany;Italy;United Kingdom | ||
| 4 | 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 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) | 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] | Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | 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 | United States;France;Spain;Ireland;Australia;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 5 | 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 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: 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] | Product Name: Triheptanoin 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;Australia;Denmark;Israel;Italy;United Kingdom | ||
| 6 | 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 | ||
| 7 | 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 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) | 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] | Product Name: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | 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 | France;United States;Spain;Australia;Israel;Germany;Italy;United Kingdom | ||
| 8 | NCT02960217 (ClinicalTrials.gov) | April 19, 2017 | 7/11/2016 | 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 Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Drug: UX007;Drug: Placebo | Ultragenyx Pharmaceutical Inc | NULL | Terminated | 6 Years | N/A | All | 44 | Phase 3 | United States;France;Germany;Italy;Spain;United Kingdom;Israel |
| 9 | 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 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) | 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: UX007 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | 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 | France;United States;Spain;Ireland;Australia;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 10 | NCT02968953 (ClinicalTrials.gov) | March 17, 2017 | 16/11/2016 | Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome | Treatment With UX007 (Triheptanoin) for a Single Patient (ERS) With Glucose Transporter 1 (GLUT1) Deficiency Syndrome | Glucose Transporter 1 Deficiency Syndrome | Drug: Triheptanoin | Jerry Vockley, MD, PhD | Ultragenyx Pharmaceutical Inc | No longer available | 3 Years | N/A | Female | United States | ||
| 11 | EUCTR2015-000389-69-GB (EUCTR) | 27/01/2017 | 20/07/2016 | 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: 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] | Product Name: Triheptanoin Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | 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;Hungary;Spain;Denmark;Australia;Israel;Italy;United Kingdom | ||
| 12 | EUCTR2013-003771-35-HU (EUCTR) | 13/04/2016 | 10/08/2015 | 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 Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome | Glucose Transporter Type 1 deficiency syndrome MedDRA version: 18.1;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 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 | United States;France;Hungary;Spain;Denmark;Australia;Israel;United Kingdom;Italy | ||
| 13 | 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 | ||
| 14 | NCT02599961 (ClinicalTrials.gov) | September 10, 2015 | 3/11/2015 | Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS) | 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 | Drug: UX007 | Ultragenyx Pharmaceutical Inc | NULL | Terminated | 1 Year | N/A | All | 15 | Phase 2 | United States;Australia;Denmark;Spain;United Kingdom |
| 15 | EUCTR2013-003771-35-GB (EUCTR) | 19/03/2015 | 10/11/2014 | 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: 17.1;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 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | 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;Hungary;Spain;Denmark;Italy;United Kingdom | ||
| 16 | NCT01993186 (ClinicalTrials.gov) | February 28, 2014 | 31/10/2013 | Phase 2 Study of Triheptanoin (UX007) for the Treatment of 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 (Glut1 DS) | Drug: UX007;Drug: Placebo | Ultragenyx Pharmaceutical Inc | NULL | Completed | 1 Year | 100 Years | All | 36 | Phase 2 | United States;Australia;France;Israel;Italy;Spain;United Kingdom;Hungary |
| 17 | EUCTR2013-003771-35-IT (EUCTR) | 11/02/2014 | 11/12/2013 | 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, Adaptive 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: 14.1;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 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | Ultragenyx Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 2 | France;United States;Hungary;Spain;Denmark;Netherlands;United Kingdom;Italy | ||
| 18 | EUCTR2013-003771-35-FR (EUCTR) | 17/06/2015 | 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, Adaptive 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 Product Code: UX007 INN or Proposed INN: Not available Other descriptive name: TRIHEPTANOIN | Ultragenyx Pharmaceutical Inc | NULL | NA | Female: yes Male: yes | 80 | Phase 2 | United States;France;Hungary;Spain;Denmark;Italy;United Kingdom |