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

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1+
SponsorInozyme Pharma

About this trial

The purpose of this study (Study INZ701-304 \[ADAPT\]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.

Eligibility criteria

Qualifiers

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)

Provide assent in accordance with local regulations, if <18 years of age

Male or female, greater than 1 year of age

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

Disqualifiers

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

Known intolerance to INZ-701 or any of its excipients

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

Pregnant, trying to become pregnant, or breastfeeding

Trial design

Treatments tested in this trial

  • INZ-701

Treatment groups

200 Participants
are divided into 1 treatment group

Sponsors and collaborators