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Enzyme Replacement Therapy

INZ-701 for Metabolic Bone Disease (ENERGY Trial)

Phase 1

Recruiting

Research Sponsored by Inozyme Pharma

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject must have a post-natal confirmed molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or equivalent.

Subject must be male or female ages ≥ 1 month to <1 year of age at Baseline

Must not have

Known malignancy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks (treatment period)

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a drug to see how safe and well-tolerated it is for infants with a rare metabolic disorder.

Who is the study for?

The ENERGY study is for infants weighing at least 0.5 kg with a genetic diagnosis of ENPP1 Deficiency, which can cause rickets and other related conditions. Infants must be between 1 month to less than 1 year old and able to complete the study procedures. Caregivers must consent and provide medical records access.

What is being tested?

This trial tests the safety and tolerability of a medication called INZ-701 in infants diagnosed with ENPP1 Deficiency, which affects bone development and can lead to rickets or vascular calcification.

What are the potential side effects?

While specific side effects are not listed, the trial aims to identify any adverse reactions that may occur when administering INZ-701 to infants with ENPP1 Deficiency.

Eligibility Criteria

Inclusion Criteria

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

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I have a confirmed genetic diagnosis of ENPP1 Deficiency.

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I am a baby aged between 1 month and less than 1 year.

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I weigh more than 0.5 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks (treatment period)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks (treatment period)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks (treatment period) 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)

Left Ventricular Ejection Fraction

Number of Treatment Emergent Adverse Events (TEAEs)

Secondary study objectives

Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701

Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels

ENPP1 Activity

+1 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 first 2 study participants will receive 1 dose of 0.2 mg/kg on Day 1 and start at Dose Level A (0.2 mg/kg twice weekly) on Day 8. The Data Review Committee (DRC) comprised of representatives of the Sponsor, the study Investigators, and a physician who is a subject matter expert not affiliated with the study or Sponsor, will review the safety data of the first study participant through Day 8. Contingent upon this review, the Sponsor will decide if additional study participants can begin receiving INZ-701. After the second study participant completes Day 32, the DRC will perform a cumulative review of safety and PK/PD data and will make dosing recommendations, for example, modifying the dose of the ongoing study participants and/or changing the starting dose for future participants. Dose Level A: 0.2 mg/kg twice weekly Dose Level B: 0.6 mg/kg twice weekly Dose Level C: 0.2 mg/kg once weekly Dose Level D: 0.6 mg/kg once weekly Dose Level E: 1.8 mg/kg once weekly

0 / 4Eligibility criteria met