The ENERGY Study - INZ701-104
Research type
Research Study
Full title
The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infant Subjects with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
IRAS ID
1008844
Contact name
Elizabeth Alessi
Contact email
Sponsor organisation
Inozyme Pharma, Inc.
Clinicaltrials.gov Identifier
Research summary
The purpose of this study is to see if the study medication, INZ-701 can replace the missing ENPP1 enzyme and safe with ENPP1 Deficiency in participant with this condition.
This research study is testing an investigational drug, INZ-701, (“study medication”) for the potential treatment of ENPP1 Deficiency
(ectonucleotide pyrophosphatase/ phosphodiesterase 1) in children from birth to less than 1year of age. The parents/legal guardians will be asked to give permission for their child, who has ENPP1 Deficiency, to take part in this research study.
Approximately 8 children with ENPP1 Deficiency at about 5 study sites globally will participate in this study.
This study has a Screening Period of up to 60 days, and then 2 main parts: a 52-week Study Treatment Period and subsequently an optional Extension Period. During the Screening Period if the child is eligible for the study, they will be enrolled in the Study Treatment Period. If the child completes the Study Treatment Period, parents/legal guardians will discuss the child’s options with the study doctor, including whether or not they want the child to participate in an Extension Period.
The Extension Period is where participants may be allowed to continue to receive the study medication INZ-701 until it is approved for use in the country where they reside or until another study with the study medication becomes available.
Participants will be expected to attend clinic visits whereby study assessments will take place e.g., blood and urine collection, vital
signs , ECG, Echocardiogram, monitoring and completion of questionnaires.REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
24/YH/0001
Date of REC Opinion
4 Mar 2024
REC opinion
Further Information Favourable Opinion