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The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

The ENERGY 3 Study: A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

NCT06046820•Inozyme Pharma

View on ClinicalTrials.gov

Summary

The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.

Detailed Description

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy (ERT) in development for the treatment of ENPP1 Deficiency, an ultra-rare genetic disorder with an incidence of 1 in 64,000 pregnancies. Study INZ701-106 (The ENERGY 3 Study) is a multi-center, randomized in a 2:1 ratio, controlled, open-label Phase 3 study to evaluate the efficacy and safety of INZ-701 in children with ENPP1 Deficiency. The study will consist of a Screening Period of up to 52 days (including a washout period of up to 7 days for prohibited medications post-Randomization) and a Randomized Treatment Period (INZ-701 or control) of 52 weeks, followed by an Open-label Extension Period during which all study participants may receive INZ-701, and an End of Study (EOS) Safety visit 30 days after the last dose of INZ-701.

Eligibility

Age

1 - 12 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Caregiver's written or electronic informed 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)
•2. Study participant's assent in accordance with local regulations
•3. A confirmed postnatal 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 regional equivalent
•4. Males and females ≥1 year and \<13 years of age at Study Day 1
•5. Open growth plates of the distal femur and proximal tibia in both legs
•6. Plasma PPi concentration of \<1400 nM at Screening
•7. 25-hydroxyvitamin D (25\[OH\]D) levels of ≥12 ng/mL at Screening
•8. Radiographic evidence of skeletal abnormalities based on an RSS ≥2
•9. Female participants of childbearing potential must have a negative serum pregnancy test at Screening and must not be breastfeeding
•10. Study participants of childbearing potential who are sexually active must agree to use a highly effective form of contraception in accordance with Clinical Trials Facilitation and Coordination Group (CTFG) guidance and local guidelines for the duration of the study
+1 more criteria

Exclusion Criteria

•1. In the opinion of the Investigator, has clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency that will preclude study participation and/or may confound the interpretation of study results
•2. If receiving any of the following prohibited medications as indicated in the protocol: systemic corticosteroids (\>5 mg prednisone equivalent per day), anti-fibroblast growth factor 23 (FGF23), and oral and/or IV bisphosphonates
•3. Unable or unwilling to discontinue calcitriol or other active forms of vitamin D3 (or analogs) within 7 days prior to Study Day 1 and/or oral phosphate supplements within 36 hours prior to Study Day 1 if randomized to the INZ-701 arm
•4. Planned orthopedic surgery that may confound the interpretation of study results during the 52-week Randomized Treatment Period
•5. Known intolerance to INZ-701 or any of its excipients
•6. A positive COVID-19 test within 5 days prior to Randomization, only if required as per local regulations or institutional policy
•7. Previous treatment with INZ-701
•8. Concurrent participation in another interventional clinical study and/or has received an investigational drug within 5 half-lives of the last dose or within 4 weeks prior to Randomization, whichever is longer, or use of an investigational device

Locations (15)

Children's Hospital of Colorado

Aurora, Colorado, United States

Ann & Robert H. Lurie Children's Hospital

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

Queensland Children's Hospital

South Brisbane, Australia

Centre Hospitalier Universitaire (CHU) Sainte-Justine

Montreal, Canada

Hôpital Bicêtre, Service d'endocrinologie et diabète de l'enfant (Childhood Endocrinology and Diabetes Department)

Le Kremlin-Bicêtre, France

King Faisal Specialist Hospital and Research Centre

Riyadh, Saudi Arabia

Key Dates

Start Date

November 5, 2023

Primary Completion Date

January 1, 2026

Completion Date

February 1, 2026

Last Updated

May 1, 2025

Enrollment

ACTUAL participants

Conditions

Ectonucleotide Pyrophosphatase/Phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy

Interventions

INZ-701

DRUG

Control Arm (Conventional Therapy)

DRUG

The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency

NCT06739980

Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic Rickets+1 more

View study

UNKNOWN

Natural History of GACI With or Without ARHR2 or PXE

NCT03758534

Generalized Arterial Calcification in InfancyAutosomal Recessive Hypophosphatemic Rickets+1 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 1, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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