The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency

Inclusion Criteria

• •1. Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)
• •2. Provide assent in accordance with local regulations, if \<18 years of age
• •3. Male or female, ≥1 year of age
• •4. A confirmed postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic variants (ie, homozygous or compound heterozygous) performed using assays that meet CE-marked requirements, or from a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory, or regional equivalent
• •5. Must have at least one of the following clinical signs and/or symptoms consistent with ENPP1 Deficiency:
• •1. ≥1 atraumatic vertebral fracture
• •2. ≥2 fractures as an adult (eg, long bones, digits, vertebrae)
• •3. For participants \<55 years of age, low bone mineral density measured at any of the following sites: lumbar spine, radius, and hip (DXA Z-score less than -1.5)
• •4. History of myocardial infarction, unstable angina, transient ischemic attack, or low cardiac output before the age of 40 years
• •5. Renal vascular hypertension or other evidence for vascular insufficiency/stenosis
• •6. History of rickets or bone deformity
• •7. Diagnosis of ossification of the posterior longitudinal ligament
• •8. Other clinical signs/symptoms, with approval by Inozyme
• •6. Fasting plasma PPi concentration of \<1400 nM at Screening
• •7. Serum level of 25-hydroxyvitamin D (25\[OH\]D) ≥12 ng/mL at Screening (Participants may be rescreened after receiving cholecalciferol treatment.)
• •8. Women of childbearing potential (WOCBP, as defined in Clinical Trials Coordination Group \[CTCG 2024\]) must have a negative serum pregnancy test at Screening and must not be breastfeeding
• •9. Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701
• •10. WOCBP and partners of fertile males who are WOCBP must be using or agree to use a highly effective form of contraception (as per CTCG 2024) from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ701 (greater than 5 half-lives of INZ-701)
• •11. In the opinion of the Investigator, able to complete all aspects of the study