NCT06462547

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

The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: Inozyme Pharma · Industry
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The investigational product INZ-701 is being developed as a therapeutic protein for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency and adenosine triphosphate (ATP)-binding cassette subfamily C member 6 (ABCC6) Deficiency. INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin (Ig) G1 antibody. The ADAPT Study (INZ701-304) is an open-label study 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. The study will consist of a 30-day Screening Period, followed by an open-label Treatment Period during which all participants will receive once-weekly subcutaneous (SC) doses of INZ-701 and continue with treatment until INZ-701 is commercially available in the country/region of the participant's residence or until Inozyme chooses to discontinue development of INZ-701. Participants will complete an End of Study (EOS) safety follow-up visit approximately 30 days after their last designated study visit assigned by the Investigator and/or Sponsor.

Conditions

• Gene Mutations
• Pseudoxanthoma Elasticum
• Arterial Calcification
• Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
• Autosomal Recessive Hypophosphatemic Rickets Type 2

Interventions

Type	Name	Description
DRUG	INZ-701	INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).

Timeline

• Start date: 2024-06-19
• Primary completion: 2030-11-01
• Completion: 2030-12-01
• First posted: 2024-06-17
• Last updated: 2024-11-29

Locations

5 sites across 4 countries: United States, France, Germany, United Kingdom

Regulatory

• FDA-regulated drug study