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

  
24 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03773770
(ClinicalTrials.gov)
August 1, 201910/12/2018Expanded Access to TriheptanoinAn 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: TriheptanoinUltragenyx Pharmaceutical IncNULLAvailableN/AN/AAllNULL
2NCT03301532
(ClinicalTrials.gov)
June 5, 201829/8/2017Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1DCompatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 DeficiencyGLUT1DS1Drug: TriheptanoinUniversity of Texas Southwestern Medical CenterNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting30 Months35 YearsAll10Phase 2United States
3EUCTR2015-000389-69-DK
(EUCTR)
23/08/201713/06/2017A 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: 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 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): noUnited States;Spain;Australia;Denmark;Israel;United Kingdom
4EUCTR2015-005536-17-GB
(EUCTR)
04/08/201711/07/2017A 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 IncNULLNot 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): noFrance;United States;Spain;Australia;Israel;Germany;Italy;United Kingdom
5EUCTR2015-005536-17-FR
(EUCTR)
30/06/201716/05/2017A 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 IncNULLNot 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): noUnited States;France;Spain;Ireland;Australia;Israel;Netherlands;Germany;Italy;United Kingdom
6EUCTR2013-003771-35-DK
(EUCTR)
07/06/201711/04/2017A 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 IncNULLNot RecruitingFemale: yes
Male: yes
40Phase 2France;United States;Hungary;Spain;Australia;Denmark;Israel;Italy;United Kingdom
7EUCTR2015-000389-69-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
8EUCTR2015-005536-17-DE
(EUCTR)
17/05/201701/03/2017A 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 IncNULLNot 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): noFrance;United States;Spain;Australia;Israel;Germany;Italy;United Kingdom
9NCT03041363
(ClinicalTrials.gov)
March 29, 201731/1/2017Treatment Development of Triheptanoin (G1D)Treatment Development of Triheptanoin (C7) for Glucose Transporter Type I Deficiency (G1D): A Phase I Maximum Tolerable Dose TrialEpilepsy;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 SyndromeDrug: TriheptanoinJuan PascualNational Institute of Neurological Disorders and Stroke (NINDS)Completed2 Years35 YearsAll12Phase 1United States
10EUCTR2015-005536-17-ES
(EUCTR)
17/03/201710/03/2017A 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 IncNULLNot 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): noFrance;United States;Spain;Ireland;Australia;Israel;Netherlands;Germany;Italy;United Kingdom
11NCT02968953
(ClinicalTrials.gov)
March 17, 201716/11/2016Treatment With UX007 for a Single Patient With GLUT1 Deficiency SyndromeTreatment With UX007 (Triheptanoin) for a Single Patient (ERS) With Glucose Transporter 1 (GLUT1) Deficiency SyndromeGlucose Transporter 1 Deficiency SyndromeDrug: TriheptanoinJerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer available3 YearsN/AFemaleUnited States
12EUCTR2015-000389-69-GB
(EUCTR)
27/01/201720/07/2016A 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 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;Hungary;Spain;Denmark;Australia;Israel;Italy;United Kingdom
13EUCTR2013-003771-35-HU
(EUCTR)
13/04/201610/08/2015A 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 IncNULLNot RecruitingFemale: yes
Male: yes
40Phase 2United States;France;Hungary;Spain;Denmark;Australia;Israel;United Kingdom;Italy
14NCT02021526
(ClinicalTrials.gov)
December 201512/12/2013Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)Treatment Development of Triheptanoin for Glucose Transporter Type I DeficiencyGlucose Transporter Type 1 Deficiency Syndrome;Glut1 Deficiency SyndromeDrug: TriheptanoinJuan PascualNULLWithdrawn30 Months55 YearsAll0Phase 1/Phase 2United States
15EUCTR2013-003771-35-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
16EUCTR2013-003771-35-GB
(EUCTR)
19/03/201510/11/2014A 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 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;Hungary;Spain;Denmark;Italy;United Kingdom
17NCT02000960
(ClinicalTrials.gov)
April 201427/11/2013Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency SyndromeA Controlled N-of-1 Before-and-after Study to Determine Safety and Efficacy Triheptanoin in Patients With Glucose Transporter 1 Deficiency SyndromeGlucose Transporter Type 1 Deficiency SyndromeDrug: TriheptanoinUniversity of British ColumbiaUltragenyx Pharmaceutical IncActive, not recruiting1 Year18 YearsBoth3Phase 2Canada
18NCT01993186
(ClinicalTrials.gov)
February 28, 201431/10/2013Phase 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 SyndromeGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)Drug: UX007;Drug: PlaceboUltragenyx Pharmaceutical IncNULLCompleted1 Year100 YearsAll36Phase 2United States;Australia;France;Israel;Italy;Spain;United Kingdom;Hungary
19NCT02036853
(ClinicalTrials.gov)
February 20, 201413/1/2014An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency SyndromeAn 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: TriheptanoinAdrian LacyUltragenyx Pharmaceutical IncCompleted1 Year50 YearsAll20Phase 2United States
20EUCTR2013-003771-35-IT
(EUCTR)
11/02/201411/12/2013A 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 IncNULLNot RecruitingFemale: yes
Male: yes
80Phase 2France;United States;Hungary;Spain;Denmark;Netherlands;United Kingdom;Italy
21NCT02014883
(ClinicalTrials.gov)
December 4, 20133/12/2013Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DSPhase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DSGlut1 Deficiency SyndromeDrug: GLUT1 DSInstitut National de la Santé Et de la Recherche Médicale, FranceUltragenyx Pharmaceutical IncCompleted3 YearsN/AAll20Phase 2France
22NCT02018302
(ClinicalTrials.gov)
October 201311/12/2013Post Study Continuation of C7 for G1DPost Study Continuation of C7 for G1DGlut1 Deficiency Syndrome;Glucose Transporter Type 1 Deficiency SyndromeDrug: TriheptanoinJuan PascualNULLNo longer available1 Month28 YearsAllUnited States
23NCT02018315
(ClinicalTrials.gov)
January 201211/12/2013Treatment 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 SyndromeDrug: TriheptanoinJuan PascualNULLCompleted1 Month20 YearsAll14Phase 1United States
24EUCTR2013-003771-35-FR
(EUCTR)
17/06/2015A 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, 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 IncNULLNAFemale: yes
Male: yes
80Phase 2United States;France;Hungary;Spain;Denmark;Italy;United Kingdom