ENERGY 2
Research type
Research Study
Full title
The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
IRAS ID
1010123
Contact name
Elizabeth Alessi
Contact email
Sponsor organisation
Inozyme Pharma, Inc.
Research summary
The purpose of this study is to assess the efficacy and safety of the study medication, INZ-701 in infants with ENPP1 Deficiency.
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 1 year 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 12 children with ENPP1 Deficiency at about 10 study sites globally will participate in this study.
This study has a Screening Period of up to 60 days during which it will be confirmed if the child is eligible for the study. If eligibility is confirmed, the child will be enrolled in a 52-week Study Treatment Period followed by a 52-week Extension period as well as by an End of Treatment (EOT) Visit 30 days after the last dose of INZ-701. The Sponsor is planning a long-term extension study that will allow patients to receive continued treatment until INZ-701 is approved and commercially available in the country where the study participant resides or until an alternative study of INZ-701 is available.
Participants will be expected to attend clinic visits whereby study assessments will take place e.g., blood and urine collection, vital signs, ECG, Chest CT Scan, Echocardiogram, monitoring and completion of questionnaires.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
24/NW/0217
Date of REC Opinion
8 Aug 2024
REC opinion
Further Information Favourable Opinion