INZ-701 for ENPP1 Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.
Who Is on the Research Team?
Kurt Gunter, MD
Principal Investigator
Inozyme Pharma, Inc.
Are You a Good Fit for This Trial?
Adults aged 18 to <65 with a clinical diagnosis of ENPP1 Deficiency, confirmed by genetic testing, can join this trial. They must be able to complete the study and provide medical records. Women who can have children and men must agree to use contraception during the study.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Evaluation
Participants receive multiple ascending doses of INZ-701 to assess safety, tolerability, pharmacokinetics, and pharmacodynamics
Extension
Participants continue to receive INZ-701 with regular follow-up visits to monitor long-term safety and efficacy
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- INZ-701
Find a Clinic Near You
Who Is Running the Clinical Trial?
Inozyme Pharma
Lead Sponsor