The ENERGY 3 Study
Research type
Research Study
Full 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 Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
IRAS ID
1008125
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, is safe and works like the missing ENPP1 enzyme to manage
the signs and symptoms of ENPP1 Deficiency in children 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 between 1 and 12 years old. The parent or legal guardian will be asked to give permission for their child, who has ENPP1 Deficiency, to take part in this research study.
Approximately 33 children with ENPP1 Deficiency at about 15 study sites globally will participate in this study.
This study has 3 parts: A Screening Period (52 days) , a study Treatment Period (52 Weeks) and an Extension Period. The child will be randomly assigned to one of two groups:
Study Medication Group: Participants assigned to this group will receive INZ-701 once weekly for the full duration of the study. During the 52-week Study Treatment Period, participants will return to the clinic 12 times for study tests and assessments.
Control Group: Participants assigned to this group will continue to receive their current treatment until Week 52. During the 52-week Study Treatment Period participants will return to the clinic 9 times for study tests and assessments.
Participants will be expected to attend clinic visits whereby study assessments will take place e.g., blood and urine collection, vital signs , ECG, X-rays, monitoring and completion of questionnaires.
After Week 52 the participant may enter an Extension Period where they will 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. The study doctor will explain the child’s options to the parents at the end of the 52-week Study Treatment Period and at the end of their child’s participation in the study.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
23/NW/0342
Date of REC Opinion
12 Dec 2023
REC opinion
Further Information Favourable Opinion