INZ-701 for ENPP1 Deficiency

Phase 1 & 2

Waitlist Available

Research Sponsored by Inozyme Pharma

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 32 days (dose evaluation period)

Awards & highlights

No Placebo-Only Group

Summary

This trial will test a new therapy for two rare genetic disorders that cause calcium buildup in arteries and bones.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions related to immune response or infusion processes typical in enzyme replacement therapies such as allergic reactions, discomfort at injection site, fever, chills or nausea.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 32 days (dose evaluation period)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~32 days (dose evaluation period)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 32 days (dose evaluation period) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701

Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels

Incidence of Anti-Drug Antibodies (ADA)

+3 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: INZ-701Experimental Treatment1 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.

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