166. 弾性線維性仮性黄色腫 Pseudoxanthoma elasticum Clinical trials / Disease details
臨床試験数 : 16 / 薬物数 : 27 - (DrugBank : 5) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 28
| 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 | NCT05030831 (ClinicalTrials.gov) | April 11, 2022 | 26/8/2021 | Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE | A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults With ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum (PXE) | ATP-Binding Cassette Subfamily C Member 6 Deficiency;Pseudoxanthoma Elasticum;Generalized Arterial Calcification of Infancy | Drug: INZ-701 | Inozyme Pharma | IQVIA Biotech | Recruiting | 18 Years | 69 Years | All | 9 | Phase 1/Phase 2 | United States;United Kingdom |
| 2 | EUCTR2020-004000-33-FR (EUCTR) | 10/06/2021 | 28/04/2021 | A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of INZ-701 in Adults with ABCC6 Deficiency causing Pseudoxanthoma elasticum (PXE) | A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE) | Treatment of patients with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE) MedDRA version: 20.0;Level: PT;Classification code 10037150;Term: Pseudoxanthoma elasticum;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: INZ-701 Product Code: INZ-701 INN or Proposed INN: RECOMBINANT HUMAN ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE 1 FUSED TO THE FC FRAGMENT OF IGG1 Other descriptive name: RECOMBINANT HUMAN ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE 1 FUSED TO THE FC FRAGMENT OF IGG1 | Inozyme Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 1;Phase 2 | France |