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

Launched by INOZYME PHARMA · Jun 14, 2024

Keywords

ClinConnect Summary

The ADAPT Study is a clinical trial looking at the long-term safety of a treatment called INZ-701 for patients with specific genetic conditions, particularly ENPP1 or ABCC6 Deficiency. These conditions can lead to issues like arterial calcification and related health problems. The study is designed for people who have previously participated in another study with INZ-701 and want to continue receiving this treatment to see how it affects their health over time. Currently, the study is recruiting participants aged 1 year and older, including both males and females.

To be eligible for this trial, participants must have completed the earlier parts of the INZ-701 study and provide consent. If participants are under 18, they will need to give assent, which is a way of agreeing to participate that is appropriate for their age. Additionally, there are some requirements related to contraception for sexually active participants to ensure safety during the study. Those interested should not be pregnant, trying to become pregnant, or currently breastfeeding. Participants can expect to receive ongoing treatment and monitoring to help the researchers understand how INZ-701 works over a longer period.

Gender

ALL

Eligibility criteria

• Individuals eligible to participate must meet all of the following 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, greater than 1 year of age
• • 4. Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor
• • 5. Female participants of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701
• • 6. Male participants who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701; participants must agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701
• • 7. In the opinion of the Investigator, able to complete all aspects of the study
• Individuals who meet any of the following exclusion criteria will not be eligible to participate:
• • 1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results
• • 2. Known intolerance to INZ-701 or any of its excipients
• • 3. Concurrent participation in another interventional clinical study and/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device
• • 4. Pregnant, trying to become pregnant, or breastfeeding
• • 5. Male participants trying to father a child