INZ-701 for Pseudoxanthoma Elasticum and Arterial Calcification

Phase 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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 years (long term safety assessment)

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to evaluate the safety of a drug called INZ-701 in patients with specific genetic conditions who have previously taken the drug in another study and wish to continue treatment.

Who is the study for?

The ADAPT study is for males and females over 1 year old with ENPP1 or ABCC6 Deficiency who have previously taken INZ-701 in a clinical trial. They must be able to complete the study, provide consent (or assent if under 18), and use effective contraception.

What is being tested?

This trial tests the long-term safety of INZ-701, a potential treatment for conditions like Pseudoxanthoma Elasticum and various forms of Hypophosphatemic Rickets associated with ENPP1 or ABCC6 genetic mutations.

What are the potential side effects?

Specific side effects are not listed here, but this study focuses on understanding the long-term safety profile of INZ-701 by monitoring any adverse reactions in participants continuing its use from previous trials.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 years (long term safety assessment)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 years (long term safety assessment)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 years (long term safety assessment) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of Anti-Drug Antibodies (ADA)

Number of Treatment Emergent Adverse Events (TEAEs)

Secondary study objectives

Maximum Plasma Concentration (Cmax) of INZ-701

Mean Change from Baseline in Plasma PPi Concentration

Time to maximum serum concentration (Tmax)

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

INZ-701 will be administered by subcutaneous injection on a once-weekly basis as follows: * Study participants from 1 year to \<13 years of age will receive a dose of 2.4 mg/kg * Study participants ≥13 years will either receive a 1.8 mg/kg dose or a 150 mg flat dose of INZ-701

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