UX007 ( DrugBank: - )


4 diseases
告示番号疾患名(ページ内リンク)臨床試験数
8ハンチントン病2
248グルコーストランスポーター1欠損症18
256筋型糖原病2
257肝型糖原病1

8. ハンチントン病


臨床試験数 : 242 薬物数 : 205 - (DrugBank : 62) / 標的遺伝子数 : 85 - 標的パスウェイ数 : 159
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2014-005112-42-NL
(EUCTR)
11/12/201507/08/2015A 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
INSERMNULLNot RecruitingFemale: yes
Male: yes
100Phase 2France;Netherlands
2EUCTR2014-005112-42-FR
(EUCTR)
22/06/201522/06/2015A 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
INSERMNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 2France;Netherlands

248. グルコーストランスポーター1欠損症


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.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
2EUCTR2015-000389-69-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
3EUCTR2015-005536-17-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
4EUCTR2015-005536-17-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
5EUCTR2013-003771-35-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
6EUCTR2015-000389-69-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
7EUCTR2015-005536-17-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
8NCT02960217
(ClinicalTrials.gov)
April 19, 20177/11/2016Crossover 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: PlaceboUltragenyx Pharmaceutical IncNULLTerminated6 YearsN/AAll44Phase 3United States;France;Germany;Italy;Spain;United Kingdom;Israel
9NCT02968953
(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
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
11EUCTR2015-000389-69-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
12EUCTR2013-003771-35-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
13EUCTR2013-003771-35-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
14NCT02599961
(ClinicalTrials.gov)
September 10, 20153/11/2015Study 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 SyndromeGlucose Transporter Type 1 Deficiency SyndromeDrug: UX007Ultragenyx Pharmaceutical IncNULLTerminated1 YearN/AAll15Phase 2United States;Australia;Denmark;Spain;United Kingdom
15EUCTR2013-003771-35-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
16NCT01993186
(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
17EUCTR2013-003771-35-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
18EUCTR2013-003771-35-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

256. 筋型糖原病


臨床試験数 : 180 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-004153-17-DK
(EUCTR)
03/05/201826/03/2018The effect of Triheptanoin on excercise in adults and adolescence with glycogenosesEvaluation 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 CenterNULLNot RecruitingFemale: yes
Male: yes
20Phase 2Denmark
2EUCTR2014-003644-12-DK
(EUCTR)
20/02/201504/11/2014The 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, RigshospitaletNULLNot RecruitingFemale: yes
Male: yes
28Phase 2Denmark

257. 肝型糖原病


臨床試験数 : 14 薬物数 : 27 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 8
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-004153-17-DK
(EUCTR)
03/05/201826/03/2018The effect of Triheptanoin on excercise in adults and adolescence with glycogenosesEvaluation 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 CenterNULLNot RecruitingFemale: yes
Male: yes
20Phase 2Denmark