9 Participants Needed

INZ-701 for ENPP1 Deficiency

Recruiting at 7 trial locations
IC
Overseen ByInozyme Clinical Trial Information
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.

Who Is on the Research Team?

KG

Kurt Gunter, MD

Principal Investigator

Inozyme Pharma, Inc.

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)
In the opinion of the Investigator, must be willing and able to complete the Dose Evaluation Period
You have been diagnosed with ENPP1 Deficiency and have specific mutations in the ENPP1 gene.
See 5 more

Exclusion Criteria

Subjects who are pregnant, trying to become pregnant, or breastfeeding
Unable or unwilling to discontinue the use of any prohibited medication (examples include 1,25-dihydroxy vitamin D, phosphate, anti-FGF23 [eg, burosumab], calcimimetics, calcium-containing antacids, systemic corticosteroids, PTH suppressors). Discontinuation should be undertaken only if considered not detrimental and indicated by the subject's treating physician
You have had cancer, except for non-melanoma skin cancers or a specific type of early-stage cervical cancer, within the past 5 years.
See 6 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Dose Evaluation

Participants receive multiple ascending doses of INZ-701 to assess safety, tolerability, pharmacokinetics, and pharmacodynamics

32 days
Multiple visits for dose administration and monitoring

Extension

Participants continue to receive INZ-701 with regular follow-up visits to monitor long-term safety and efficacy

Until INZ-701 is approved or an alternative study is available
Visits every 4 weeks until Week 48, then every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks
1 visit (in-person) 30 days after last dose

What Are the Treatments Tested in This Trial?

Interventions

  • INZ-701
Trial Overview 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.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: INZ-701Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Inozyme Pharma

Lead Sponsor

Trials
10
Recruited
1,400+
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