Ignite Creation Date:
2024-05-06 @ 3:36 PM
Last Modification Date:
2024-10-26 @ 1:52 PM
Study NCT ID:
NCT04686175
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-30
First Post:
2020-12-22
Brief Title:
Evaluation of Safety Tolerability and Efficacy of INZ-701 in Adults With ENPP1 Deficiency
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 ENPP1 Deficiency
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 enzyme replacement therapy for the treatment of ENPP1 Deficiency The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency
Detailed Description:
INZ-701 is an ectonucleotide pyrophosphatasephosphodiesterase 1 ENPP1 enzyme replacement therapy ERT in development for the treatment of ENPP1 Deficiency an ultra-rare genetic disorder with an incidence of 1 in 64000 pregnancies
Study INZ701-101 is a Phase 12 multicenter open-label FIH MAD dose-finding study followed by a long-term open-label Extension Period conducted in adults with ENPP1 Deficiency 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 No placebo will be used in the study Subjects will be 18 to 65 years of age with a confirmed genetic diagnosis of ENPP1 Deficiency and biochemical evidence of hypopyrophosphatemia ie PPi 1300 nM Exploratory endpoints for the Extension Period of the study include evaluations of skeletal assessment X-ray and DEXA arterial and organ calcification either Na18F-PETCT or low dose CT full body without contrast echocardiogram and renal ultrasound and cardiovascular function echocardiogram as well as patient reported outcomes
Subject participation consists of a Screening Period of up to 30 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 followed by every 12 weeks until the subject leaves the study
Subjects will complete an End of Study EOS Visit Safety Follow-Up Visit 30 days after their last dose of INZ-701
Study INZ701-101 is a Phase 12 multicenter open-label FIH MAD dose-finding study followed by a long-term open-label Extension Period conducted in adults with ENPP1 Deficiency 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 No placebo will be used in the study Subjects will be 18 to 65 years of age with a confirmed genetic diagnosis of ENPP1 Deficiency and biochemical evidence of hypopyrophosphatemia ie PPi 1300 nM Exploratory endpoints for the Extension Period of the study include evaluations of skeletal assessment X-ray and DEXA arterial and organ calcification either Na18F-PETCT or low dose CT full body without contrast echocardiogram and renal ultrasound and cardiovascular function echocardiogram as well as patient reported outcomes
Subject participation consists of a Screening Period of up to 30 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 followed by every 12 weeks until the subject leaves the study
Subjects will complete an End of Study EOS Visit Safety 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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2020-003716-27 | EUDRACT_NUMBER | None | None |