INZ-701 for Pseudoxanthoma Elasticum (PXE)

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 older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks (day 1 through safety follow-up visit)

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing INZ-701, a specially designed protein, to treat adults with a rare genetic disorder called ABCC6 Deficiency. The disorder can cause problems like Pseudoxanthoma elasticum (PXE). INZ-701 works by helping to manage the buildup of certain substances in the body. The study aims to find the best dose and schedule for this treatment.

Who is the study for?

Adults aged 18-70 with a clinical diagnosis of pseudoxanthoma elasticum (PXE) due to ABCC6 deficiency can join. They must agree to use effective contraception and not donate eggs or sperm during the trial. Excluded are those with recent malignancies, intolerance to INZ-701, significant unrelated diseases, active infections including COVID-19, or using prohibited medications.

What is being tested?

The trial is testing multiple doses of INZ-701 for safety and effectiveness in treating ABCC6 Deficiency causing PXE. It aims to find the right dose for future studies by looking at how the body processes it and its effects on disease markers.

What are the potential side effects?

Specific side effects aren't listed but generally could include reactions related to immune response, infusion-related discomforts, potential organ-specific inflammation due to protein nature of treatment, and any unforeseen intolerances.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks (day 1 through safety follow-up visit)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks (day 1 through safety follow-up visit)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks (day 1 through safety follow-up visit) for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of Treatment Emergent Adverse Events (TEAEs)

Secondary 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 (ADAs)

+2 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. 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, with a twice weekly dose regimen. During the Extension Period, INZ-701 administration will initially be at the dose and dose schedule assigned in the Dose Evaluation Period; however, the Sponsor may modify the assigned dose and/or dosing regimen in the Extension Period post-Week 48 based on cumulative review of safety and PK/PD data, including population PK. Study visits will be in-clinic every 4 weeks until Week 48 of the Extension Period and then every 12 weeks (remote or in-clinic) 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.

Do I qualify?Apply below to find out