INZ-701 for Pseudoxanthoma Elasticum (PXE)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called INZ-701, an experimental therapy for Pseudoxanthoma Elasticum (PXE), a condition caused by ABCC6 deficiency that affects the skin, eyes, and blood vessels. The main goal is to assess the safety of INZ-701 and how the body processes it, while also identifying the best dose for future studies. Individuals with a confirmed diagnosis of PXE and symptoms impacting daily life may be suitable for this trial. Participants will receive varying doses to determine the most effective and safe dosage. As a Phase 1/Phase 2 trial, this study aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group.
Do I have to stop taking my current medications for the trial?
The trial does not specify if you must stop taking your current medications, but you cannot start new anti-lipid therapy within 6 months of screening. If you are on statins or PCSK9 inhibitors, you must have been on them for at least 6 months before screening.
Is there any evidence suggesting that INZ-701 is likely to be safe for humans?
Research shows that INZ-701 is under study to assess its safety and tolerability. Some studies have examined how this treatment functions in the body by restoring certain blood levels, which is crucial for its intended use. Early results indicated that the treatment is generally safe, with few serious side effects reported, suggesting that INZ-701 might be well-tolerated by patients.
However, this research remains ongoing. The trials are in the early stages, so scientists are still learning about the treatment's safety. Like any new treatment, there might be risks, but researchers are carefully monitoring participants' reactions to different doses. If considering joining a trial, these findings are promising, but discussing it with a healthcare provider is always wise.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Pseudoxanthoma Elasticum (PXE), which primarily focus on managing symptoms, INZ-701 offers a novel approach by targeting the underlying disease process. INZ-701 works by delivering an enzyme replacement that could potentially restore the mineral balance disrupted in PXE. Researchers are excited about this treatment because it not only addresses the symptoms but also aims to tackle the root cause, offering hope for more effective and long-lasting relief for patients.
What evidence suggests that INZ-701 might be an effective treatment for PXE?
Research shows that INZ-701, the investigational treatment in this trial, is a promising option for Pseudoxanthoma Elasticum (PXE). This treatment increases levels of a substance called PPi in the blood, helping to prevent unwanted calcium buildup in tissues. Previous studies have shown that INZ-701 can successfully raise PPi levels and reduce these harmful deposits. Early data from ongoing research reported positive effects on bone and mineral health in people with PXE. These findings suggest that INZ-701 could effectively manage PXE symptoms by addressing the underlying mineral imbalances.23678
Who Is on the Research Team?
Kurt Gunter, MD
Principal Investigator
Inozyme Pharma, Inc.
Are You a Good Fit for This Trial?
Adults aged 18-70 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
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 follow-up visits every 4 weeks until Week 48, then every 12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
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.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
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.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Inozyme Pharma
Lead Sponsor
IQVIA Biotech
Industry Sponsor
Citations
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in ...
This study is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701. The goal of ...
INZ-701, a recombinant ENPP1 enzyme, prevents ectopic ...
This study examined whether restoring plasma PPi levels by INZ-701, a recombinant human ENPP1 protein, the principal PPi-generating enzyme, prevents ectopic ...
Novel treatment for PXE: Recombinant ENPP1 enzyme ...
Our previous studies demonstrated that daily or every other day subcutaneous injections of INZ-701 increased plasma PPi levels and completely ...
Inozyme Pharma Announces Positive Topline Data from ...
Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 ...
5.
synapse.patsnap.com
synapse.patsnap.com/article/inozyme-to-present-phase-12-inz-701-data-for-enpp1-and-pxe-at-medical-conferencesInozyme to Present Phase 1/2 INZ-701 Data for ENPP1 ...
The first session, entitled "Impact of INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical Outcomes in Adults with ENPP1 Deficiency," ...
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in ...
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, ...
NCT05734196 | The ENERGY Study: Evaluation of Safety ...
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 ...
8.
investors.inozyme.com
investors.inozyme.com/news-releases/news-release-details/inozyme-pharma-announces-investor-and-analyst-event-andInozyme Pharma Announces Investor and Analyst Event ...
In April 2022, the Company reported positive preliminary PK/PD and safety data from its ongoing Phase 1/2 trial of INZ-701 in patients with ...
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