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Recombinant Fusion Protein
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)
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 moreAwards & 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.
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Who is running the clinical trial?
IQVIA BiotechIndustry Sponsor
20 Previous Clinical Trials
5,725 Total Patients Enrolled
Inozyme PharmaLead Sponsor
8 Previous Clinical Trials
1,323 Total Patients Enrolled
4 Trials studying Pseudoxanthoma Elasticum
1,228 Patients Enrolled for Pseudoxanthoma Elasticum
Borut Cizman, MDStudy DirectorInozyme Pharma, Inc.
1 Previous Clinical Trials
9 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility 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.You have ongoing bleeding in both of your eyes.You have a current fungal, bacterial, or viral infection, including HIV, hepatitis B, hepatitis C, or COVID-19.Your blood level of a specific substance is less than 1300 nM during the screening.You have been diagnosed with pseudoxanthoma elasticum (PXE) and have specific genetic mutations in the ABCC6 gene.You are between 18 and 69 years old.Your kidney function or vitamin D levels are not within the normal range.If you could become pregnant, you need to have a negative pregnancy test when you are screened.If you take statins or PCSK9 inhibitors for high cholesterol, you must have been taking them for at least 6 months before the screening, and you cannot start any new cholesterol-lowering medication for 6 months before the screening.
Research Study Groups:
This trial has the following groups:- Group 1: INZ-701
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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