Ornithine ( DrugBank: Ornithine )
2 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 245 | プロピオン酸血症 | 2 |
| 251 | 尿素サイクル異常症 | 34 |
245. プロピオン酸血症
臨床試験数 : 13 / 薬物数 : 16 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 6
Showing 1 to 2 of 2 diseases
| 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 | NCT01599286 (ClinicalTrials.gov) | September 2012 | 11/5/2012 | Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia | Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia | Propionic Acidemia, Type I and/or Type II;Methylmalonic Acidemia;Carbamoyl-Phosphate Synthase I Deficiency Disease;Ornithine Carbamoyltransferase Deficiency Propionic Acidemia, Type I and/or Type II;Methylmalonic Acidemia;Carbamoyl-Phosphate Synthase I Defi ... | Drug: Carbaglu;Drug: Placebo;Drug: Standard of Care Treatment | Mendel Tuchman | Children's National Research Institute;Boston Children's Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Stanford University;Icahn School of Medicine at Mount Sinai;University of Pittsburgh;Children's Hospital Colorado Children's National Research Institute;Boston Children's Hospital;University Hospitals Cleveland Med ... | Active, not recruiting | N/A | 99 Years | All | 114 | Phase 2 | United States |
| 2 | NCT00645879 (ClinicalTrials.gov) | July 2008 | 25/3/2008 | Anaplerotic Therapy in Propionic Acidemia | Safety & Efficacy of Investigational Products: Ornithine Alpha-ketoglutarate, Glutamine, or Disodium Citrate on Hyperammonemia in Propionic Acidemia. Safety & Efficacy of Investigational Products: OrnithineAlpha-ketoglutarate, Glutamine, or Disodium ... | Propionic Acidemia | Drug: ornithine alpha ketoglutarate;Drug: glutamine;Drug: disodium citrate | Nicola Longo | National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases ... | Completed | 5 Years | 12 Years | Both | 3 | Phase 1 | United States |
251. 尿素サイクル異常症
臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
Showing 1 to 10 of 34 diseases
| 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 | EUCTR2020-003384-25-FR (EUCTR) | 16/12/2022 | 26/11/2021 | A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency A clinical study of a virus transferring the gene for human OrnithineTranscarbamylase (OTC) in patie ... | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (A ... | Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Late-onset Ornithinetranscarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification ... | Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Acetate INN or Proposed INN: Sodium Ethanoate (1- 13 C) Other descriptive name: Sodium acetate (1-13C) Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Aceta ... | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Portugal;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan United States;Portugal;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;G ... | ||
| 2 | NCT05345171 (ClinicalTrials.gov) | October 18, 2022 | 18/4/2022 | Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency | A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Seroty ... | OTC Deficiency | Genetic: DTX301;Other: Placebo;Drug: Oral Corticosteroids;Drug: Placebo for oral corticosteroids;Drug: Sodium Acetate Genetic: DTX301;Other: Placebo;Drug: Oral Corticosteroids;Drug: Placebo for oral corticosteroids;Dru ... | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 12 Years | N/A | All | 50 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom United States;Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spai ... |
| 3 | EUCTR2020-003384-25-DE (EUCTR) | 02/08/2022 | 17/12/2021 | A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency A clinical study of a virus transferring the gene for human OrnithineTranscarbamylase (OTC) in patie ... | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (A ... | Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Late-onset Ornithinetranscarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification ... | Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Acetate INN or Proposed INN: Sodium Ethanoate (1- 13 C) Other descriptive name: Sodium acetate (1-13C) Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Aceta ... | Ultragenyx Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 3 | United States;Portugal;Spain;United Kingdom;Italy;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan United States;Portugal;Spain;United Kingdom;Italy;France;Canada;Argentina;Brazil;Croatia;Australia;G ... | ||
| 4 | NCT05526066 (ClinicalTrials.gov) | July 6, 2022 | 8/8/2022 | Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 Study for Adolescent and Adult Participants With OrnithineTranscarbamylase Deficiency to Evaluate Sa ... | Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Stu ... | Ornithine Transcarbamylase Deficiency;OTC Deficiency;OTCD | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 12 Years | 65 Years | All | 24 | Phase 2 | Belgium;Spain;United Kingdom |
| 5 | EUCTR2020-003384-25-ES (EUCTR) | 04/04/2022 | 17/12/2021 | A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency A clinical study of a virus transferring the gene for human OrnithineTranscarbamylase (OTC) in patie ... | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (A ... | Late-onset Ornithine transcarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Late-onset Ornithinetranscarbamylase (OTC) deficiency MedDRA version: 20.0;Level: LLT;Classification ... | Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Acetate INN or Proposed INN: Sodium Ethanoate (1-13C) Other descriptive name: Sodium acetate (1-13C) Product Name: DTX301 INN or Proposed INN: avalotcagene ontaparvovec Product Name: (1-13C) Sodium Aceta ... | Ultragenyx Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 3 | Portugal;United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;Germany;Netherlands;Japan Portugal;United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Croatia;Australia;G ... | ||
| 6 | NCT05092685 (ClinicalTrials.gov) | February 1, 2022 | 28/9/2021 | Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn | Phase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for Paediatric Patients With Ornithine Transcarbamylase Deficiency. Phase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for P ... | Ornithine Transcarbamylase Deficiency | Genetic: AAVLK03hOTC | University College, London | NULL | Not yet recruiting | N/A | 16 Years | All | 12 | Phase 1/Phase 2 | NULL |
| 7 | EUCTR2021-001081-38-ES (EUCTR) | 12/01/2022 | 31/08/2021 | A Multi-dose Study of an Investigational Treatment to Evaluate Safety, Tolerability, and Ability to Improve Urea Cycle Function in Adolescents and Adults with Ornithine Transcarbamylase Deficiency A Multi-dose Study of an Investigational Treatment to Evaluate Safety, Tolerability, and Ability to ... | Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants with Ornithine Transcarbamylase Deficiency - Phase 2 nested single/multiple ascending dose study of ARCT-810 in Participants with OTC Deficiency Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Stu ... | Ornithine transcarbamylase deficiency MedDRA version: 21.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism;System Organ Class: 200000003094;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Ornithinetranscarbamylase deficiency MedDRA version: 21.1;Level: LLT;Classification code 10013373;Te ... | Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: mRNA encoding modified Ornithine transcarbamylase Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: ... | Arcturus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | Belgium;Spain;United Kingdom | ||
| 8 | EUCTR2018-000156-18-FR (EUCTR) | 02/11/2020 | 31/01/2020 | A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term A clinical study to learn about the effects of a virus that transfers the gene for human OrnithineTr ... | A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Sero ... | Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Ornithinetranscarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Te ... | Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301 | Ultragenyx Pharmaceutical, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Phase 1;Phase 2 | United States;France;Canada;Spain;United Kingdom | ||
| 9 | NCT03767270 (ClinicalTrials.gov) | December 2019 | 28/11/2018 | Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With ... | A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase Deficiency A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and P ... | Ornithine Transcarbamylase Deficiency | Biological: MRT5201;Other: Placebo | Translate Bio, Inc. | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 1/Phase 2 | NULL |
| 10 | JPRN-jRCTs041190055 | 24/07/2019 | 24/07/2019 | Carglumic Acid Administration for OTC Deficienscy | A Clinical Study of Carglumic Acid in Patients with Late-Onset Ornithine Transcarbamylase Deficiency A Clinical Study of Carglumic Acid in Patients with Late-Onset OrnithineTranscarbamylase Deficiency ... | ornitine transcarbamylase deficiency patients | administraton of carglumic acid | Ito Tetsuya | NULL | Complete | Not applicable | < 75age old | Both | 2 | Phase 4 | Japan |