INZ-701 for ENPP1 Deficiency

Recruiting at 7 trial locations

Overseen By Inozyme Clinical Trial Information

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Inozyme Pharma

No Placebo Group

Trial Summary

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.

Research Team

Kurt Gunter, MD

Principal Investigator

Inozyme Pharma, Inc.

Eligibility Criteria

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

Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)

In the opinion of the Investigator, must be willing and able to complete the Dose Evaluation Period

You have been diagnosed with ENPP1 Deficiency and have specific mutations in the ENPP1 gene.

Exclusion Criteria

Subjects who are pregnant, trying to become pregnant, or breastfeeding

Unable or unwilling to discontinue the use of any prohibited medication (examples include 1,25-dihydroxy vitamin D, phosphate, anti-FGF23 [eg, burosumab], calcimimetics, calcium-containing antacids, systemic corticosteroids, PTH suppressors). Discontinuation should be undertaken only if considered not detrimental and indicated by the subject's treating physician

You have had cancer, except for non-melanoma skin cancers or a specific type of early-stage cervical cancer, within the past 5 years.

Treatment Details

Interventions

• INZ-701 (Enzyme Replacement Therapy)

Trial Overview The trial is testing multiple doses of INZ-701, an enzyme replacement therapy for treating ENPP1 Deficiency. It aims to determine the safety, how well it's tolerated, its effects on the body (PK/PD), and find a suitable dose for further studies.

Participant Groups

Group I: INZ-701 Experimental Treatment 1 Intervention

The study design of the Dose Evaluation Period is a MAD 3 + 3 with 3 dose cohorts. Additional cohorts may be added to evaluate an intermediate dose and/or an alternative dosing regimen of an existing dose level. Based on nonclinical findings and nonclinical pharmacology modeling, the initial planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg all twice weekly, not to exceed 3.6 mg/kg weekly. During the Extension Period, visits will be every 4 weeks until Week 48 and then every 12 weeks until the subject leaves the study. Subjects will complete an End of Study (EOS) Visit (Safety Follow-up Visit) 30 days after their last dose of INZ-701 (greater than 5 half-lives of INZ-701) for all subjects.

Who Is Running the Clinical Trial?

Inozyme Pharma

Lead Sponsor

Trials

10

Recruited

1,400+