Ignite Creation Date:
2024-05-06 @ 7:32 PM
Last Modification Date:
2024-10-26 @ 3:08 PM
Study NCT ID:
NCT06046820
Status:
RECRUITING
Last Update Posted:
2024-06-26
First Post:
2023-08-28
Brief Title:
The ENERGY 3 Study Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
Sponsor:
Inozyme Pharma
Organization:
Inozyme Pharma
Study Overview
Official Title:
The ENERGY 3 Study A Randomized Controlled Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children With Ectonucleotide PyrophosphatasePhosphodiesterase 1 ENPP1 Deficiency
Status:
RECRUITING
Status Verified Date:
2024-06
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 primary purpose of Study INZ701-106 The ENERGY 3 Study is to assess the efficacy and safety of INZ-701 in children with 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 INZ701-106 The ENERGY 3 Study is a multi-center randomized in a 21 ratio controlled open-label Phase 3 study to evaluate the efficacy and safety of INZ-701 in children with ENPP1 Deficiency
The study will consist of a Screening Period of up to 52 days including a washout period of up to 7 days for prohibited medications post-Randomization and a Randomized Treatment Period INZ-701 or control of 52 weeks followed by an Open-label Extension Period during which all study participants may receive INZ-701 and an End of Study EOS Safety visit 30 days after the last dose of INZ-701
Study INZ701-106 The ENERGY 3 Study is a multi-center randomized in a 21 ratio controlled open-label Phase 3 study to evaluate the efficacy and safety of INZ-701 in children with ENPP1 Deficiency
The study will consist of a Screening Period of up to 52 days including a washout period of up to 7 days for prohibited medications post-Randomization and a Randomized Treatment Period INZ-701 or control of 52 weeks followed by an Open-label Extension Period during which all study participants may receive INZ-701 and an End of Study EOS Safety visit 30 days after the 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