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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks (treatment period)
Awards & highlights

ENERGY Trial 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.Check my eligibility
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.See study design
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.

ENERGY Trial Eligibility Criteria

Inclusion Criteria

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

ENERGY Trial Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Anti-Drug Antibodies (ADA)
Left Ventricular Ejection Fraction
Number of Treatment Emergent Adverse Events (TEAEs)
Secondary outcome measures
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

ENERGY Trial Design

1Treatment groups
Experimental Treatment
Group I: INZ-701Experimental Treatment1 Intervention
The first 2 study participants will receive a single 0.2 mg/kg dose of INZ-701 on Day 1. On Day 8, they will commence receiving Dose Level A (0.2 mg/kg twice weekly). 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 to Dose Levels B, C, D, E, or F. Each study participant's safety and PK/PD data will also be reviewed by the DRC during its quarterly review, based upon which the participant's dose may be modified to Dose Levels B, C, D, E, or F as specified in the protocol. 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 Dose Level F: 3.0 mg/kg once weekly

Find a Location

Who is running the clinical trial?

Inozyme PharmaLead Sponsor
7 Previous Clinical Trials
1,130 Total Patients Enrolled
1 Trials studying Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
33 Patients Enrolled for Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
Alex Lai, MDStudy DirectorInozyme Pharma
1 Previous Clinical Trials
33 Total Patients Enrolled
1 Trials studying Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency
33 Patients Enrolled for Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency

Media Library

Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency Clinical Trial 2023: INZ-701 Highlights & Side Effects. Trial Name: NCT05734196 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this clinical trial permit participation from individuals under 45 years old?

"This trial has an age requirement of between 1 month and 1 year for its participants."

Answered by AI

What are the primary objectives of this clinical investigation?

"This trial will span the course of 52 weeks and its primary outcome measure is Treatment Emergent Adverse Events (TEAE). Other secondary outcomes to be monitored include Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701, Maximum Plasma Concentration (Cmax) of INZ-701, and ENPP1 Activity as a reflection of the efficacy of INZ 701 treatment."

Answered by AI

Has the INZ-701 drug been given a green light from the FDA?

"The safety of INZ-701 is rated 1 out of 3 when evaluated by Power, as this trial is still in the Phase 1 stage and only has limited data affirming its efficacy."

Answered by AI

Are participants sought at this time for the clinical research?

"Indeed, according to information on clinicaltrials.gov, this medical trial is still recruiting participants as of April 27th 2023. This research was originally posted on May 1st and requires 8 individuals from 2 different centres to take part."

Answered by AI

What is the uppermost threshold of participants for this experiment?

"Affirmative. The clinical trial data posted on clinicaltrials.gov reveals that recruitment for this medical study is still ongoing, having first been made available to the public on May 1st 2023 and updated most recently on April 27th 2023. As of now, 8 patients are needed from 2 distinct locations."

Answered by AI

Is my profile suitable for participating in this experiment?

"This clinical trial is open to 8 individuals with ectonucleotide pyrophosphatase/phosphodiesterase1 deficiencies aged between one month and twelve months. Applicants need to fulfill the following conditions: written or electronic consent from caregivers prior to any study-related procedures in accordance with International Conference on Harmonisation (ICH) Good Clinical Practice (GCP), a postnatal confirmed molecular genetic diagnosis of ENPP1 Deficiency, weight greater than 0.5 kg at time of first INZ-701 dose, ability for subject to complete all aspects of study and access by caregiver(s) to child's relevant medical records"

Answered by AI
Recent research and studies
clinicaltrials.govStudy
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency
2023. "The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05734196.
~4 spots leftby Mar 2025
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