Ignite Creation Date:
2024-05-06 @ 4:36 PM
Last Modification Date:
2024-10-26 @ 2:12 PM
Study NCT ID:
NCT05030831
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-30
First Post:
2021-08-26
Brief Title:
Evaluation of Safety Tolerability and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
Sponsor:
Inozyme Pharma
Organization:
Inozyme Pharma
Study Overview
Official Title:
A Phase 12 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
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to assess the safety tolerability pharmacokinetics PK and pharmacodynamics PD of multiple ascending doses of INZ-701 an ectonucleotide pyrophosphatasephosphodiesterase 1 ENPP1 recombinant fusion protein for the treatment of ABCC6 Deficiency The goal of the study is to identify a dose regimen for further clinical development in the treatment of ABCC6 Deficiency
Detailed Description:
INZ-701 is an ectonucleotide pyrophosphatasephosphodiesterase 1 ENPP1 recombinant fusion protein in development for the treatment of ABCC6 Deficiency a rare genetic disorder
Study INZ701-201 is a Phase 12 multicenter open-label multiple ascending dose MAD dose-finding study followed by a long-term open-label Extension Period conducted in adults with ABCC6 Deficiency manifesting as Pseudoxanthoma elasticum PXE This study is designed to assess the safety tolerability pharmacokinetics PK and pharmacodynamics PD of multiple ascending doses of INZ-701 The goal of the study is to identify a dose and dose schedule number of doses per week for further clinical development in ABCC6 Deficiency No placebo will be used in this study
Exploratory endpoints for the Extension Period of the study include evaluations of arterial and organ calcification ophthalmologic cardiac and renal parameters and physical function as well as patient reported outcomes
Subject participation consists of a Screening Period of up to 60 days a 32-day Dose Evaluation Period and an Extension Period during which subjects may continue to receive INZ-701 with options for self- caregiver- or healthcare provider administration until INZ-701 is approved and available in the country where the subject resides or until an alternative study for subjects to continue receiving study drug is available During the Extension Period follow-up visits will be conducted every 4 weeks until Week 48 of the Extension Period and then every 12 weeks remote or in-clinic until the subject leaves the study
Subjects will complete a follow up visit 30 days after their last dose of INZ-701
Study INZ701-201 is a Phase 12 multicenter open-label multiple ascending dose MAD dose-finding study followed by a long-term open-label Extension Period conducted in adults with ABCC6 Deficiency manifesting as Pseudoxanthoma elasticum PXE This study is designed to assess the safety tolerability pharmacokinetics PK and pharmacodynamics PD of multiple ascending doses of INZ-701 The goal of the study is to identify a dose and dose schedule number of doses per week for further clinical development in ABCC6 Deficiency No placebo will be used in this study
Exploratory endpoints for the Extension Period of the study include evaluations of arterial and organ calcification ophthalmologic cardiac and renal parameters and physical function as well as patient reported outcomes
Subject participation consists of a Screening Period of up to 60 days a 32-day Dose Evaluation Period and an Extension Period during which subjects may continue to receive INZ-701 with options for self- caregiver- or healthcare provider administration until INZ-701 is approved and available in the country where the subject resides or until an alternative study for subjects to continue receiving study drug is available During the Extension Period follow-up visits will be conducted every 4 weeks until Week 48 of the Extension Period and then every 12 weeks remote or in-clinic until the subject leaves the study
Subjects will complete a follow up visit 30 days after their last dose of INZ-701
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None