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Enzyme Replacement Therapy
INZ-701 for ENPP1 Deficiency
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 between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks (day 1 through safety follow-up visit)
Awards & highlights
Study Summary
This trial will test a new therapy for two rare genetic disorders that cause calcium buildup in arteries and bones.
Who is the study for?
Adults aged 18 to <65 with a clinical diagnosis of ENPP1 Deficiency, confirmed by genetic testing, can join this trial. They must be able to complete the study and provide medical records. Women who can have children and men must agree to use contraception during the study.Check my eligibility
What is being tested?
The trial is testing multiple doses of INZ-701, an enzyme replacement therapy for treating ENPP1 Deficiency. It aims to determine the safety, how well it's tolerated, its effects on the body (PK/PD), and find a suitable dose for further studies.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions related to immune response or infusion processes typical in enzyme replacement therapies such as allergic reactions, discomfort at injection site, fever, chills or nausea.
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
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary 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 (ADA)
+3 moreTrial 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. Additional cohorts may be added to evaluate an intermediate dose and/or an alternative dosing regimen of an existing dose level.
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 all twice weekly, not to exceed 3.6 mg/kg weekly.
During the Extension Period, visits will be every 4 weeks until Week 48 and then every 12 weeks 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 (greater than 5 half-lives of INZ-701) for all subjects.
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Who is running the clinical trial?
Inozyme PharmaLead Sponsor
7 Previous Clinical Trials
1,134 Total Patients Enrolled
1 Trials studying Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
33 Patients Enrolled for Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
Kurt Gunter, MDStudy DirectorInozyme Pharma, Inc.
7 Previous Clinical Trials
1,111 Total Patients Enrolled
Deborah Wenkert, MDStudy DirectorInozyme Pharma, Inc.
2 Previous Clinical Trials
40 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- You have been diagnosed with ENPP1 Deficiency and have specific mutations in the ENPP1 gene.You have had cancer, except for non-melanoma skin cancers or a specific type of early-stage cervical cancer, within the past 5 years.You are allergic to INZ-701 or any of the other ingredients in the medication.You have a current fungal, bacterial, or viral infection, including HIV, hepatitis B, hepatitis C, or COVID-19.Your PPI level is less than 1300 nM at the time of screening.Women who could become pregnant must have a negative pregnancy test before starting the study.Men who are trying to have a baby.Your recent lab test results show significant issues, such as low kidney function, vitamin D levels, or high parathyroid hormone levels.
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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