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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
Clinical diagnosis of pseudoxanthoma elasticum (PXE) supported by prior genetic identification of biallelic ABCC6 mutations (ie, homozygous or compound heterozygous)
Male or female, ages 18 to <70 years of age at Screening
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks (day 1 through safety follow-up visit)
Awards & highlights
No Placebo-Only Group

Study Summary

This trial will study the safety and tolerability of a new drug, INZ-701, for the treatment of ABCC6 Deficiency, which can cause calcification of arteries and other tissues.

Eligible Conditions
  • Pseudoxanthoma Elasticum
  • ABCC6 Deficiency
  • Generalized Arterial Calcification

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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.
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.


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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Treatment Emergent Adverse Events (TEAEs)
Secondary outcome measures
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 during the Dose Evaluation Period is a MAD 3+3 with 3 dose cohorts. The planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg administered via subcutaneous injection twice weekly. During the Extension Period, subjects will be administered INZ-701 at the dose and dose schedule assigned in the Dose Evaluation Period. However, the administered dose and dose schedule for a subject may change once the selected dosing regimen has been determined upon completion of the Dose Evaluation Period, at which time all subjects will be assigned to the selected dosing regimen.

Find a Location

Who is running the clinical trial?

IQVIA BiotechIndustry Sponsor
18 Previous Clinical Trials
4,898 Total Patients Enrolled
Inozyme PharmaLead Sponsor
5 Previous Clinical Trials
118 Total Patients Enrolled
1 Trials studying Pseudoxanthoma Elasticum
30 Patients Enrolled for Pseudoxanthoma Elasticum
Borut Cizman, MDStudy DirectorInozyme Pharma, Inc.
1 Previous Clinical Trials
9 Total Patients Enrolled

Media Library

INZ-701 (Recombinant Fusion Protein) Clinical Trial Eligibility Overview. Trial Name: NCT05030831 — Phase 1 & 2
Pseudoxanthoma Elasticum Research Study Groups: INZ-701
Pseudoxanthoma Elasticum Clinical Trial 2023: INZ-701 Highlights & Side Effects. Trial Name: NCT05030831 — Phase 1 & 2
INZ-701 (Recombinant Fusion Protein) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05030831 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any vacancies for those wishing to partake in the clinical trial?

"Correct. According to the clinicaltrials.gov database, this medical study is at present attempting to recruit its participants - with a recruitment start date of September 22nd 2021 and last updated on January 12th 2022 - from one location for a total of nine individuals."

Answered by AI

Are there specific criteria that make a person an ideal candidate for this research?

"This clinical trial is searching for 9 participants who suffer from pseudoxanthoma elasticum and are between 18 to 64 years of age."

Answered by AI

What is the cumulative enrollment of this clinical trial?

"Affirmative. According to clinicaltrials.gov, this trial is currently recruiting patients and was initially posted on September 22nd 2021 before being updated as recently as January 12th 2022. Only 9 participants are needed across the sole site involved in the experiment."

Answered by AI

Is the inclusion criteria for this trial limited to individuals of a certain age or can geriatric patients participate?

"This study is interested in participants who are of legal adult age and under 64 years old."

Answered by AI
~4 spots leftby Feb 2025