Ignite Creation Date:
2024-05-06 @ 6:38 PM
Last Modification Date:
2024-10-26 @ 2:52 PM
Study NCT ID:
NCT05734196
Status:
RECRUITING
Last Update Posted:
2024-06-20
First Post:
2023-01-26
Brief Title:
The ENERGY Study Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Sponsor:
Inozyme Pharma
Organization:
Inozyme Pharma
Study Overview
Official Title:
The ENERGY Study An Open-Label Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase Phosphodiesterase 1 ENPP1 Deficiency or ATP-binding Cassette Sub-family C Member 6 ABCC6 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:
ENERGY
Brief Summary:
The primary purpose of Study INZ701-104 the ENERGY study is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency
Detailed Description:
INZ-701 is an ectonucleotide pyrophosphatasephosphodiesterase 1 ENPP1 enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder ENPP1 Deficiency or with ABCC6 Deficiency
Study INZ701-104 the ENERGY study is a Phase 1b open-label study to assess the safety tolerability pharmacokinetics PK and pharmacodynamics PD of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency
The study will consist of up to a 60-day Screening Period a 52-week Treatment Period during which study participants will receive INZ-701 an Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides or until an alternative study of INZ-701 is available and an End of Treatment EOT visit 30 days after the last dose of INZ-701 Upon treatment discontinuation participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study
Study INZ701-104 the ENERGY study is a Phase 1b open-label study to assess the safety tolerability pharmacokinetics PK and pharmacodynamics PD of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency
The study will consist of up to a 60-day Screening Period a 52-week Treatment Period during which study participants will receive INZ-701 an Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides or until an alternative study of INZ-701 is available and an End of Treatment EOT visit 30 days after the last dose of INZ-701 Upon treatment discontinuation participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study
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