Long-term Safety Study of INZ-701 for Patients With ENPP1 and ABCC6 Deficiencies Open-label Phase 2 Study of Weekly INZ-701 Treatment

What this Trial Is About

Researchers are evaluating the long-term safety of INZ-701, an investigational protein treatment for patients with ENPP1 Deficiency or ABCC6 Deficiency. These rare genetic conditions are linked to gene mutations causing arterial calcification and other complications. The study focuses on patients who have previously received INZ-701 in earlier trials and are continuing treatment to assess ongoing safety. INZ-701 is given as a once-weekly subcutaneous injection. Children aged 1 to under 13 years receive a dose based on their weight 2.4 mgkg, while participants 13 years and older receive either a 1.8 mgkg dose or a flat 150 mg dose. The study includes a 30-day screening period followed by an open-label treatment phase. Participants will continue treatment until the drug becomes commercially available or the sponsor stops development, with a safety follow-up visit about 30 days after their last treatment. Participants will be monitored closely throughout the study with safety assessments including tracking treatment-emergent adverse events and the presence of anti-drug antibodies over six years. Additional measures include blood tests for drug concentration and plasma markers related to the condition. The study ensures long-term observation of participants receiving INZ-701 to gather important safety data while supporting their ongoing care.

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency