INZ-701 for Pseudoxanthoma Elasticum (PXE)

Recruiting in Eatontown (>99 mi)

+2 other locations

Overseen By Inozyme Clinical Trial Information

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Inozyme Pharma

Must be taking: Statins, PCSK9 inhibitors

Disqualifiers: Uncontrolled thyroid, Active infections, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 5

10 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Kurt Gunter, MD

Principal Investigator

Inozyme Pharma, Inc.

Eligibility Criteria

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.

Inclusion Criteria

WOCBP and partners of fertile males who are WOCBP must be using or agree to use one highly effective form of contraception (per CTFG 2020) and a barrier method from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ-701 (greater than 5 half-lives of INZ-701). WOCBP and partners of fertile males who are WOCBP must also agree to not donate ova from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701.

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

Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701. Males must also agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701.

Exclusion Criteria

You had any type of cancer, other than non-melanoma skin cancers or early stage cervical cancer, in the last 5 years.

You have had a bad reaction to INZ-701 or any of the ingredients in it.

In the opinion of the Investigator, presence of any clinically significant disease (outside of those considered associated with the diagnosis of ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid, or unrelated connective tissue, bone, mineral, lipid, ophthalmologic, or muscle disease

Timeline

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Dose Evaluation

Participants receive multiple ascending doses of INZ-701 to assess safety, tolerability, pharmacokinetics, and pharmacodynamics

4 weeks

In-clinic visits as per dose schedule

Extension

Participants continue to receive INZ-701 with follow-up visits every 4 weeks until Week 48, then every 12 weeks

Long-term until drug approval or alternative study availability

In-clinic every 4 weeks until Week 48, then every 12 weeks (remote or in-clinic)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person) 30 days after last dose

Treatment Details

Interventions

• INZ-701

Trial Overview 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.

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. 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.

Who Is Running the Clinical Trial?

Inozyme Pharma

Lead Sponsor

Trials

10

Recruited

1,400+

IQVIA Biotech

Industry Sponsor

Trials

22

Recruited

5,900+