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

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05030831
(ClinicalTrials.gov)
April 11, 202226/8/2021Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXEA 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 InfancyDrug: INZ-701Inozyme PharmaIQVIA BiotechRecruiting18 Years69 YearsAll9Phase 1/Phase 2United States;United Kingdom
2EUCTR2020-004000-33-FR
(EUCTR)
10/06/202128/04/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 1;Phase 2France