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Enzyme Replacement Therapy

INZ-701 for ENPP1 Deficiency

Phase 1 & 2

Recruiting

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.

Eligible Conditions

Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
Generalized Arterial Calcification
Hypophosphatemic Rickets

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)

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

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 more

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.

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Who is running the clinical trial?

Inozyme PharmaLead Sponsor

7 Previous Clinical Trials

1,129 Total Patients Enrolled

1 Trials studying Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency

33 Patients Enrolled for Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency

Deborah Wenkert, MDStudy DirectorInozyme Pharma, Inc.

2 Previous Clinical Trials

40 Total Patients Enrolled

Borut Cizman, MDStudy DirectorInozyme Pharma, Inc.

1 Previous Clinical Trials

10 Total Patients Enrolled

Media Library

INZ-701 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04686175 — Phase 1 & 2

Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency Research Study Groups: INZ-701

Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency Clinical Trial 2023: INZ-701 Highlights & Side Effects. Trial Name: NCT04686175 — Phase 1 & 2

INZ-701 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04686175 — 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 participants being accepted for this trial at the moment?

"Affirmative, the details found on clinicaltrials.gov indicate that this medical trial has opened up enrollment and is currently looking for 9 candidates to be recruited from 3 different sites. This inquiry first went online in June 18th 2021 and was most recently updated on January 4th 2022."

Answered by AI

What is the current subject pool for this research?

"Affirmative. According to information on clinicaltrials.gov, recruitment for this research endeavor is ongoing since its initial posting date of June 18th 2021 and most recent edit of January 4th 2022. 9 participants are sought after from 3 medical centres."

Answered by AI

Is the age requirement for enrolment in this trial limited to those under 45 years old?

"According to the parameters documented in this trial's inclusion criteria, eligible patients must fall between 18 and 64 years of age."

Answered by AI

Am I eligible to participate in this research program?

"For this trial, nine persons of hypophosphatemic rickets who are aged between 18 and 64 may be enrolled. The essential criteria for eligibility involve providing written or digital consent after being briefed on the clinical study's purpose in accordance with ICH GCP regulations as well as agreeing to present access to related medical documents. Additionally, participants must identify themselves either male or female and demonstrate their willingness and capability to complete the Dose Evaluation Period."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

2021. "Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04686175.