Clinical Trials

Inclusion Criteria:

• Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures.
• Clinical diagnosis of Autosomal Recessive Hypophosphatemic Rickets 2 (ARHR2) supported by prior or concurrent genetic testing of the ENPP1 gene.
• Male or female, 18 to 64 years of age.
• Plasma PPi < lower limit of normal (LLN) at screening.
• Willing to withhold treatment with oral phosphate and vitamin D analogs (e.g., calcitriol) beginning at least 14 days prior to first dose of INZ-701 and throughout the Treatment Period.
• If using, discontinue use of bisphosphonates for 6 months prior to initiation of the study.
• Women of child-bearing potential (WOCBP as defined in Clinical Trial Facilitation Group (CTFG) 2014) must have a negative urine pregnancy test at Screening and at all additional pregnancy tests during the study.
• Fertile males with partners of child-bearing potential and WOCBP must agree to use 2 highly effective forms of contraception (per CTFG 2014) from the period following the first dose of INZ-701 through 25 days after last dose of INZ-701.
• Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 25 days after the last dose of INZ-701.
• In the opinion of the Investigator, must be willing and able to complete all aspects of the study.
• Agree to provide access to relevant medical records.

Exclusion Criteria:

• In the opinion of the Investigator and/or Sponsor, presence of any clinically significant disease (outside of those considered associated with the diagnosis of ENPP1 Deficiency) that precludes study participation or may confound interpretation of study results.
• Clinically significant abnormal laboratory result at screening, including but not limited to, elevations of aspartate aminotransferase, alanine aminotransferase, bilirubin, or creatinine greater than 2 times the upper limit of normal.
• Known active fungal, bacterial, and/or viral infection including human immunodeficiency virus, hepatitis B virus, or hepatitis C virus. If status is unknown, a local test should be completed prior to enrollment.
• Known intolerance to INZ-701 or any of its excipients.
• Unable or unwilling to discontinue the use of any prohibited medication (bisphosphonates, anti-fibroblast growth factor 23 (FGF23) [e.g., burosumab], calcimimetics, antacids, systemic corticosteroids, parathyroid hormone suppressors) or procedures.
• Receipt of any other investigational new drug or use of an investigational device within 4 weeks prior to the first dose of INZ-701.