251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details
臨床試験数 : 48 / 薬物数 : 52 - (DrugBank : 13) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 29
| 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 | 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 | 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 | 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] | Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: mRNA encoding modified Ornithine transcarbamylase | Arcturus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | Belgium;Spain;United Kingdom | ||
| 2 | NCT04442347 (ClinicalTrials.gov) | November 3, 2020 | 19/6/2020 | Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency | A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 18 Years | N/A | All | 12 | Phase 1 | United States |
| 3 | NCT04416126 (ClinicalTrials.gov) | June 1, 2020 | 28/5/2020 | Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects | A Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Healthy Adult Subjects | Ornithine Transcarbamylase Deficiency | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 18 Years | 65 Years | All | 30 | Phase 1 | New Zealand |
| 4 | EUCTR2021-001081-38-BE (EUCTR) | 26/08/2021 | 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 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 | 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] | Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: mRNA encoding modified Ornithine transcarbamylase | Arcturus Therapeutics, Inc. | NULL | NA | Female: yes Male: yes | 24 | Phase 2 | Belgium;United Kingdom |