TRIHEPTANOIN ( DrugBank: Triheptanoin )
10 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 2 | 筋萎縮性側索硬化症 | 1 |
| 8 | ハンチントン病 | 4 |
| 65 | 原発性免疫不全症候群 | 1 |
| 149 | 片側痙攣・片麻痺・てんかん症候群 | 1 |
| 156 | レット症候群 | 2 |
| 248 | グルコーストランスポーター1欠損症 | 25 |
| 256 | 筋型糖原病 | 5 |
| 257 | 肝型糖原病 | 3 |
| 317 | 三頭酵素欠損症 | 1 |
| 324 | メチルグルタコン酸尿症 | 1 |
2. 筋萎縮性側索硬化症
臨床試験数 : 624 / 薬物数 : 611 - (DrugBank : 160) / 標的遺伝子数 : 172 - 標的パスウェイ数 : 225
| 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 | NCT03506425 (ClinicalTrials.gov) | June 21, 2018 | 13/4/2018 | A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) | A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) | ALS | Drug: Triheptanoin | Richard Bedlack, M.D., Ph.D. | Ultragenyx Pharmaceutical Inc | Completed | 18 Years | N/A | All | 15 | Phase 1/Phase 2 | United States |
8. ハンチントン病
臨床試験数 : 229 / 薬物数 : 193 - (DrugBank : 60) / 標的遺伝子数 : 84 - 標的パスウェイ数 : 158
| 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 | EUCTR2014-005112-42-NL (EUCTR) | 11/12/2015 | 07/08/2015 | A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3) | A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3) | Huntington's disease MedDRA version: 20.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01] | Product Name: Triheptanoin Product Code: UX007 | INSERM | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 2 | France;Netherlands | ||
| 2 | NCT02453061 (ClinicalTrials.gov) | June 29, 2015 | 12/5/2015 | A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease | A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease | Huntington Disease | Drug: Triheptanoin oil;Drug: Placebo | Institut National de la Santé Et de la Recherche Médicale, France | Ultragenyx Pharmaceutical Inc | Completed | 18 Years | N/A | All | 100 | Phase 2 | France;Netherlands |
| 3 | EUCTR2014-005112-42-FR (EUCTR) | 22/06/2015 | 22/06/2015 | A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3) | A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (TRIHEP 3) | Huntington's disease MedDRA version: 18.0;Level: PT;Classification code 10070668;Term: Huntington's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Name: Triheptanoin Product Code: UX007 | INSERM | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 2 | France;Netherlands | ||
| 4 | NCT01882062 (ClinicalTrials.gov) | May 2013 | 18/6/2013 | Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease | Huntington Disease | Drug: Triheptanoin 1g/kg/day | Institut National de la Santé Et de la Recherche Médicale, France | NULL | Completed | 18 Years | N/A | All | 10 | Phase 2 | France |
65. 原発性免疫不全症候群
臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212
| 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 | NCT04513002 (ClinicalTrials.gov) | February 2022 | 26/7/2020 | Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis | A Phase 2A/2B Placebo-controlled Randomised Clinical Trial to Test the Ability of Triheptanoin to Protect Primary Airway Epithelial Cells Obtained From Participants With Ataxia-telangiectasia Against Death Induced by Glucose Deprivation | Ataxia Telangiectasia | Dietary Supplement: Triheptanoin | The University of Queensland | National Health and Medical Research Council, Australia | Not yet recruiting | N/A | N/A | All | 30 | Phase 2 | Australia |
149. 片側痙攣・片麻痺・てんかん症候群
臨床試験数 : 25 / 薬物数 : 40 - (DrugBank : 14) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 20
| 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 | NCT02408354 (ClinicalTrials.gov) | April 15, 2015 | 24/3/2015 | Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood | Etude Pilote, Comparative, Monocentrique, randomisée, en Cross Over, en Double Aveugle, Contre Placebo, Testant l'efficacité de l'Huile triheptanoïne Dans Les Hémiplégies Alternantes de l'Enfant HEMIHEP | Alternating Hemiplegia of Childhood | Drug: Triheptanoin;Drug: Placebo | Institut National de la Santé Et de la Recherche Médicale, France | NULL | Completed | 15 Years | N/A | All | 10 | Phase 2 | France |
156. レット症候群
臨床試験数 : 40 / 薬物数 : 53 - (DrugBank : 19) / 標的遺伝子数 : 77 - 標的パスウェイ数 : 113
| 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 | NCT03059160 (ClinicalTrials.gov) | April 1, 2017 | 2/2/2017 | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. | Rett Syndrome | Drug: Tridecanoic Acid | Sheba Medical Center | Ultragenyx Pharmaceutical Inc | Not yet recruiting | 5 Years | 18 Years | Female | 10 | Phase 2 | NULL |
| 2 | NCT02696044 (ClinicalTrials.gov) | June 2016 | 25/2/2016 | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin: An Open-label, 10-subject Clinical Trial of UX007 (Triheptanoin) in the Treatment of Mitochondrial Dysfunction in Participants With Rett Syndrome, Dyskinesia, and Epilepsy | Rett Syndrome | Drug: triheptanoin | Center for Rare Neurological Diseases, Norcross, GA | Ultragenyx Pharmaceutical Inc;Rett Syndrome Research Trust | Recruiting | 2 Years | N/A | Female | 12 | Phase 2 | United States |
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. | 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 | 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 | 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 | NCT03181399 (ClinicalTrials.gov) | April 18, 2018 | 25/4/2017 | Diet Treatment Glucose Transporter Type 1 Deficiency (G1D) | Dietary Treatment of Glucose Transporter Type 1 Deficiency (G1D) | GLUT1DS1;Epilepsy;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 | Drug: Triheptanoin | University of Texas Southwestern Medical Center | National Institute of Neurological Disorders and Stroke (NINDS) | Active, not recruiting | 24 Months | 35 Years | All | 45 | Phase 2 | United States |
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | 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 | Drug: Triheptanoin | Juan Pascual | National Institute of Neurological Disorders and Stroke (NINDS) | Active, not recruiting | 2 Years | 35 Years | All | 12 | Phase 1 | United States |
| 11 | 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 | ||
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | NCT02021526 (ClinicalTrials.gov) | December 2015 | 12/12/2013 | Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D) | Treatment Development of Triheptanoin for Glucose Transporter Type I Deficiency | Glucose Transporter Type 1 Deficiency Syndrome;Glut1 Deficiency Syndrome | Drug: Triheptanoin | Juan Pascual | NULL | Withdrawn | 30 Months | 55 Years | All | 0 | Phase 1/Phase 2 | United States |
| 16 | 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 | ||
| 17 | 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 | ||
| 18 | NCT02000960 (ClinicalTrials.gov) | April 2014 | 27/11/2013 | Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome | A Controlled N-of-1 Before-and-after Study to Determine Safety and Efficacy Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome | Glucose Transporter Type 1 Deficiency Syndrome | Drug: Triheptanoin | University of British Columbia | Ultragenyx Pharmaceutical Inc | Active, not recruiting | 1 Year | 18 Years | Both | 3 | Phase 2 | Canada |
| 19 | 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 |
| 20 | NCT02036853 (ClinicalTrials.gov) | February 20, 2014 | 13/1/2014 | An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome | An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome (GLUT1 DS) | Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS) | Drug: Triheptanoin | Adrian Lacy | Ultragenyx Pharmaceutical Inc | Completed | 1 Year | 50 Years | All | 20 | Phase 2 | United States |
| 21 | 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 | ||
| 22 | NCT02014883 (ClinicalTrials.gov) | December 4, 2013 | 3/12/2013 | Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS | Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS | Glut1 Deficiency Syndrome | Drug: GLUT1 DS | Institut National de la Santé Et de la Recherche Médicale, France | Ultragenyx Pharmaceutical Inc | Completed | 3 Years | N/A | All | 20 | Phase 2 | France |
| 23 | NCT02018302 (ClinicalTrials.gov) | October 2013 | 11/12/2013 | Post Study Continuation of C7 for G1D | Post Study Continuation of C7 for G1D | Glut1 Deficiency Syndrome;Glucose Transporter Type 1 Deficiency Syndrome | Drug: Triheptanoin | Juan Pascual | NULL | No longer available | 1 Month | 28 Years | All | United States | ||
| 24 | NCT02018315 (ClinicalTrials.gov) | January 2012 | 11/12/2013 | Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D) | Clinical Trial of Citric Acid Cycle Stimulation in Energy-deficiency States: Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D) (NMTUT 2010B) | Glucose Transporter Type 1 Deficiency Syndrome;GLUT1 Deficiency Syndrome | Drug: Triheptanoin | Juan Pascual | NULL | Completed | 1 Month | 20 Years | All | 14 | Phase 1 | United States |
| 25 | 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 |
256. 筋型糖原病
臨床試験数 : 180 / 薬物数 : 153 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 134
| 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 | NCT03642860 (ClinicalTrials.gov) | August 15, 2018 | 20/8/2018 | The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses | Triheptanoin's Effect on Fatty Acid Oxidation and Exercise Tolerance in Patients With Debrancher Deficiency, Glycogenin-1 Deficiency and Phosphofructoinase Deficiency at Rest and During Exercise. A Randomized, Double-blind, Placebo-controlled, Cross-over Study | Tarui Disease;Debrancher Deficiency;GYG1 DEFICIENCY | Drug: Triheptanoin;Drug: Placebo Oil | Rigshospitalet, Denmark | Ultragenyx Pharmaceutical Inc | Terminated | 15 Years | 85 Years | All | 3 | Phase 2 | Denmark |
| 2 | EUCTR2017-004153-17-DK (EUCTR) | 03/05/2018 | 26/03/2018 | The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses | Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study. - Triheptanoin in Glycogenoses | Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency.Tarui's diseaseAlso called: glycogen storage disease Type VII or phosphofructokinase deficiency.Glycogenin-1 deficiency or glycogen storage disease Type XV. MedDRA version: 20.1;Level: PT;Classification code 10053241;Term: Glycogen storage disease type VII;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Classification code 10053250;Term: Glycogen storage disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10053255;Term: Tarui disease;Classification code 10016983;Term: Forbes' disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;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 | Copenhagen Neuromuscular Center | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Denmark | ||
| 3 | NCT02919631 (ClinicalTrials.gov) | October 2016 | 28/9/2016 | Triheptanoin in Mc Ardle | The Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V) | Glycogen Storage Disease Type V | Drug: Triheptanoin;Drug: Placebo oil | Institut National de la Santé Et de la Recherche Médicale, France | Rigshospitalet, Denmark;Rigshospitalet, Denmark | Not yet recruiting | 18 Years | 65 Years | Both | 12 | Phase 2 | NULL |
| 4 | NCT02432768 (ClinicalTrials.gov) | April 2015 | 4/11/2014 | The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) | The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V) | Glycogen Storage Disease Type V | Drug: Triheptanoin;Other: Placebo oil | Rigshospitalet, Denmark | Groupe Hospitalier Pitie-Salpetriere;University of Texas Southwestern Medical Center;Ultragenyx Pharmaceutical Inc | Completed | 18 Years | 65 Years | All | 22 | Phase 2 | Denmark |
| 5 | EUCTR2014-003644-12-DK (EUCTR) | 20/02/2015 | 04/11/2014 | The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V) | The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V) - Triheptanoin in McArdle | McArdle Disease Also called: Glycogen Storage Disease Type V or Myophosphorylase Deficiency MedDRA version: 19.0;Level: LLT;Classification code 10026970;Term: McArdles disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0;Classification code 10026969;Term: McArdle's disease;Level: PT;Classification code 10018462;Term: Glycogen storage disease type V;System Organ Class: 10010331 - Congenital, familial and genetic disorders;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 | Neuromuscular Research Unit, Rigshospitalet | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | Denmark |
257. 肝型糖原病
臨床試験数 : 14 / 薬物数 : 28 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 8
| 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 | NCT03665636 (ClinicalTrials.gov) | October 16, 2020 | 23/8/2018 | Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I | Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I | Glycogen Storage Disease Type I | Drug: Triheptanoin | Areeg El-Gharbawy | Ultragenyx Pharmaceutical Inc | Completed | 1 Month | 65 Years | All | 4 | Early Phase 1 | United States |
| 2 | EUCTR2017-004153-17-DK (EUCTR) | 03/05/2018 | 26/03/2018 | The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses | Evaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study. - Triheptanoin in Glycogenoses | Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency.Tarui's diseaseAlso called: glycogen storage disease Type VII or phosphofructokinase deficiency.Glycogenin-1 deficiency or glycogen storage disease Type XV. MedDRA version: 20.1;Level: PT;Classification code 10053241;Term: Glycogen storage disease type VII;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Classification code 10053250;Term: Glycogen storage disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10053255;Term: Tarui disease;Classification code 10016983;Term: Forbes' disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;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 | Copenhagen Neuromuscular Center | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Denmark | ||
| 3 | NCT00947960 (ClinicalTrials.gov) | June 2009 | 24/7/2009 | Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease | A Treatment Trial of Triheptanoin in Patients With Adult Polyglucosan Body Disease - A Randomized Controlled Study | Adult Polyglucosan Body Disease;Glycogen Brancher Enzyme Deficiency;Glycogen Storage Disease Type IV | Drug: Triheptanoin;Other: Vegetable Oil | Baylor Research Institute | Ultragenyx Pharmaceutical Inc | Completed | 18 Years | 75 Years | All | 23 | Phase 2 | France;United States |
317. 三頭酵素欠損症
臨床試験数 : 3 / 薬物数 : 8 - (DrugBank : 4) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 1
| 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 | NCT01461304 (ClinicalTrials.gov) | April 17, 2009 | 25/10/2011 | Dietary Therapy for Inherited Disorders of Energy Metabolism | Dietary Therapy for Inherited Disorders of Energy Metabolism | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);Mitochondrial Trifunctional Protein Deficiency;Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;Glycogen Storage Disorders;Pyruvate Carboxylase Deficiency Disease;ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of;Barth Syndrome | Drug: triheptanoin | Jerry Vockley, MD, PhD | Ultragenyx Pharmaceutical Inc | No longer available | 1 Month | N/A | All | United States |
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. | 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 | NCT01461304 (ClinicalTrials.gov) | April 17, 2009 | 25/10/2011 | Dietary Therapy for Inherited Disorders of Energy Metabolism | Dietary Therapy for Inherited Disorders of Energy Metabolism | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);Mitochondrial Trifunctional Protein Deficiency;Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;Glycogen Storage Disorders;Pyruvate Carboxylase Deficiency Disease;ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of;Barth Syndrome | Drug: triheptanoin | Jerry Vockley, MD, PhD | Ultragenyx Pharmaceutical Inc | No longer available | 1 Month | N/A | All | United States |