:Triheptanoin    (DrugBank: Triheptanoin)

9 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
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17
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:508,薬物数:530(DrugBank:146),標的遺伝子数:170,標的パスウェイ数:221
Searched query = "Amyotrophic lateral sclerosis", "ALS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 508 trial found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT03506425June 21, 201813/4/2018A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)ALSDrug: TriheptanoinRichard Bedlack, M.D., Ph.D.Ultragenyx Pharmaceutical IncCompleted18 YearsN/AAll15Phase 1/Phase 2United States

8. ハンチントン病 [臨床試験数:197,薬物数:171(DrugBank:54),標的遺伝子数:82,標的パスウェイ数:144
Searched query = "Huntington disease", "Huntington chorea"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
4 / 197 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1EUCTR2014-005112-42-NL11/12/201507/08/2015A 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: 20.0Level: PTClassification code 10070668Term: Huntington's diseaseSystem 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
2NCT02453061June 29, 201512/5/2015A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's DiseaseA Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's DiseaseHuntington DiseaseDrug: Triheptanoin oil;Drug: PlaceboInstitut National de la Santé Et de la Recherche Médicale, FranceUltragenyx Pharmaceutical IncActive, not recruiting18 YearsN/AAll100Phase 2France;Netherlands
3EUCTR2014-005112-42-FR22/06/201522/06/2015A 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: 18.0Level: PTClassification code 10070668Term: Huntington's diseaseSystem 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
4NCT01882062May 201318/6/2013Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington DiseaseHuntington DiseaseDrug: Triheptanoin 1g/kg/dayInstitut National de la Santé Et de la Recherche Médicale, FranceNULLCompleted18 YearsN/AAll10Phase 2France

149. 片側痙攣・片麻痺・てんかん症候群 [臨床試験数:22,薬物数:33(DrugBank:12),標的遺伝子数:13,標的パスウェイ数:17
Searched query = "Hemiconvulsion hemiplegia epilepsy syndrome", "One side convulsions", "Hemiplegia", "Epilepsy syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 22 trial found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1NCT02408354April 15, 201524/3/2015Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of ChildhoodEtude 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 HEMIHEPAlternating Hemiplegia of ChildhoodDrug: Triheptanoin;Drug: PlaceboInstitut National de la Santé Et de la Recherche Médicale, FranceNULLCompleted15 YearsN/AAll10Phase 2France

156. レット症候群 [臨床試験数:36,薬物数:49(DrugBank:19),標的遺伝子数:77,標的パスウェイ数:112
Searched query = "Rett syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 36 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
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Inclusion_
agemin
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PhaseCountries
1NCT03059160April 1, 20172/2/2017Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.Rett SyndromeDrug: Tridecanoic AcidSheba Medical CenterUltragenyx Pharmaceutical IncNot yet recruiting5 Years18 YearsFemale10Phase 2NULL
2NCT02696044June 201625/2/2016Treatment of Mitochondrial Dysfunction in Rett Syndrome With TriheptanoinTreatment 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 EpilepsyRett SyndromeDrug: triheptanoinCenter for Rare Neurological Diseases, Norcross, GAUltragenyx Pharmaceutical Inc;Rett Syndrome Research TrustRecruiting2 YearsN/AFemale12Phase 2United 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]
Searched query = "Glucose transporter type 1 deficiency", "GLUT1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
17 / 30 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1NCT03773770December 27, 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
2NCT03301532June 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 YearsAll15Phase 2United States
3NCT03181399April 18, 201825/4/2017Diet 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 SyndromeDrug: TriheptanoinUniversity of Texas Southwestern Medical CenterNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting24 Months35 YearsAll45Phase 2United States
4EUCTR2013-003771-35-DK07/06/201711/04/2017A 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: 20.0Level: LLTClassification code 10061032Term: Carbohydrate transport disorderSystem 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
5NCT03041363March 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: TriheptanoinUniversity of Texas Southwestern Medical CenterNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting2 Years35 YearsAll36Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02968953March 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
7EUCTR2013-003771-35-HU13/04/201610/08/2015A 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: 18.1Level: HLGTClassification code 10039911Term: 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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40France;United States;Hungary;Spain;Denmark;Australia;Israel;Italy;United Kingdom
8NCT02021526December 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
9EUCTR2013-003771-35-ES19/11/201510/08/2015A 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: 18.0Level: HLGTClassification code 10039911Term: 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
10NCT02000960April 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
11NCT01993186February 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
12EUCTR2013-003771-35-IT11/02/201411/12/2013A 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: 14.1Level: HLGTClassification code 10039911Term: 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
13NCT02036853January 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 IncRecruiting1 Year50 YearsAll50Phase 2United States
14NCT02014883December 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 IncRecruiting3 YearsN/ABoth20Phase 2France
15NCT02018302October 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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PhaseCountries
16NCT02018315January 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
17EUCTR2013-003771-35-FR17/06/2015A 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: 18.0Level: HLGTClassification code 10039911Term: 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:161,薬物数:114(DrugBank:23),標的遺伝子数:26,標的パスウェイ数:106
Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
5 / 161 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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gender
Target_
size
PhaseCountries
1NCT03642860August 15, 201820/8/2018The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With GlycogenosesTriheptanoin'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 StudyTarui Disease;Debrancher Deficiency;GYG1 DEFICIENCYDrug: Triheptanoin;Drug: Placebo OilRigshospitalet, DenmarkUltragenyx Pharmaceutical IncUnknown status15 Years85 YearsAll20Phase 2Denmark
2EUCTR2017-004153-17-DK03/05/201826/03/2018The 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: 20.1Level: PTClassification code 10053241Term: Glycogen storage disease type VIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1Level: PTClassification code 10053250Term: Glycogen storage disease type IIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10053255Term: Tarui diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10016983Term: Forbes' diseaseSystem 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
3NCT02919631October 201628/9/2016Triheptanoin in Mc ArdleThe Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V)Glycogen Storage Disease Type VDrug: Triheptanoin;Drug: Placebo oilInstitut National de la Santé Et de la Recherche Médicale, FranceRigshospitalet, Denmark;Rigshospitalet, DenmarkNot yet recruiting18 Years65 YearsBoth12Phase 2NULL
4NCT02432768April 20154/11/2014The 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)Glycogen Storage Disease Type VDrug: Triheptanoin;Other: Placebo oilRigshospitalet, DenmarkGroupe Hospitalier Pitie-Salpetriere;University of Texas Southwestern Medical Center;Ultragenyx Pharmaceutical IncCompleted18 Years65 YearsAll22Phase 2Denmark
5EUCTR2014-003644-12-DK20/02/201504/11/2014The 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: 19.0Level: LLTClassification code 10026970Term: McArdles diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0Level: LLTClassification code 10026969Term: McArdle's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0Level: PTClassification code 10018462Term: Glycogen storage disease type VSystem 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:10,薬物数:19(DrugBank:6),標的遺伝子数:1,標的パスウェイ数:6
Searched query = "Hepatic glycogenosis", "Liver glycogenosis", "Hepatic glycogen storage disease", "Liver glycogen storage disease", "Glycogen storage disease type I", "GSDI", "von Gierke disease", "Glucose-6-phosphatase deficiency", "G6Pase deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Adult polyglucosan body disease", "Glycogen storage disease type VI", "GSDVI", "Hers disease", "Hepatic phosphorylase deficiency", "Liver phosphorylase deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 10 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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gender
Target_
size
PhaseCountries
1NCT03665636October 16, 202023/8/2018Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type IAnaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type IGlycogen Storage Disease Type IDrug: TriheptanoinAreeg El-GharbawyUltragenyx Pharmaceutical IncRecruiting1 Month65 YearsAll5Early Phase 1United States
2EUCTR2017-004153-17-DK03/05/201826/03/2018The 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: 20.1Level: PTClassification code 10053241Term: Glycogen storage disease type VIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1Level: PTClassification code 10053250Term: Glycogen storage disease type IIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10053255Term: Tarui diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: LLTClassification code 10016983Term: Forbes' diseaseSystem 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
3NCT00947960June 200924/7/2009Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body DiseaseA Treatment Trial of Triheptanoin in Patients With Adult Polyglucosan Body Disease - A Randomized Controlled StudyAdult Polyglucosan Body Disease;Glycogen Brancher Enzyme Deficiency;Glycogen Storage Disease Type IVDrug: Triheptanoin;Other: Vegetable OilBaylor Research InstituteUltragenyx Pharmaceutical IncCompleted18 Years75 YearsAll23Phase 2France;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
Searched query = "Trifunctional protein deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 3 trial found
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1NCT01461304November 30, 201125/10/2011Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy MetabolismDietary Therapy for Inherited Disorders of Energy MetabolismVery 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 SyndromeDrug: triheptanoinJerry Vockley, MD, PhDUltragenyx Pharmaceutical IncAvailable1 MonthN/AAllUnited States

324. メチルグルタコン酸尿症 [臨床試験数:3,薬物数:4(DrugBank:3),標的遺伝子数:1,標的パスウェイ数:8
Searched query = "Methylglutaconic aciduria", "3-methylglutaconyl-CoA hydratase deficiency", "3-MGA", "Barth syndrome", "Costeff syndrome", "Mitochondrial respiratory chain disorder", "Dilated cardiomyopathy with ataxia syndrome", "DCMA syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 3 trial found
No.TrialIDDate_
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agemin
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1NCT01461304November 30, 201125/10/2011Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy MetabolismDietary Therapy for Inherited Disorders of Energy MetabolismVery 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 SyndromeDrug: triheptanoinJerry Vockley, MD, PhDUltragenyx Pharmaceutical IncAvailable1 MonthN/AAllUnited States