The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

Inclusion Criteria

• •1. Caregiver's written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)
• •2. Study participant's assent in accordance with local regulations
• •3. A confirmed postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or regional equivalent
• •4. Males and females ≥1 year and \<13 years of age at Study Day 1
• •5. Open growth plates of the distal femur and proximal tibia in both legs
• •6. Plasma PPi concentration of \<1400 nM at Screening
• •7. 25-hydroxyvitamin D (25\[OH\]D) levels of ≥12 ng/mL at Screening
• •8. Radiographic evidence of skeletal abnormalities based on an RSS ≥2
• •9. Female participants of childbearing potential must have a negative serum pregnancy test at Screening and must not be breastfeeding
• •10. Study participants of childbearing potential who are sexually active must agree to use a highly effective form of contraception in accordance with Clinical Trials Facilitation and Coordination Group (CTFG) guidance and local guidelines for the duration of the study
• •11. In the opinion of the Investigator, able to complete all aspects of the study