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

Running, not enrolling · Phase 3

Conditions studied: Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy

In brief

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.

Key facts

Study ID

NCT06046820

Run by

Inozyme Pharma

People needed

27

Starts

2023-11-05

Expected to finish

2026-02-01

Last updated by the study team

2025-05-01

Who can join

Age: 1 and older, up to 12. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Study participants must meet all of the following inclusion criteria:
• 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)
• Study participant's assent in accordance with local regulations
• 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
• Males and females ≥1 year and <13 years of age at Study Day 1
• Open growth plates of the distal femur and proximal tibia in both legs
• Plasma PPi concentration of <1400 nM at Screening
• 25-hydroxyvitamin D (25[OH]D) levels of ≥12 ng/mL at Screening
• Radiographic evidence of skeletal abnormalities based on an RSS ≥2
• Female participants of childbearing potential must have a negative serum pregnancy test at Screening and must not be breastfeeding
• 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
• In the opinion of the Investigator, able to complete all aspects of the study

You may not qualify if…

• Study participants meeting any of the following exclusion criteria will not be eligible to participate in the study:
• 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
• 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
• 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
• Planned orthopedic surgery that may confound the interpretation of study results during the 52-week Randomized Treatment Period
• Known intolerance to INZ-701 or any of its excipients
• A positive COVID-19 test within 5 days prior to Randomization, only if required as per local regulations or institutional policy
• Previous treatment with INZ-701
• 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

Where it is running

• 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
• Hospital San Joan de Deu — Barcelona, Spain
• Umraniye Training and Research Hospital — Istanbul, Turkey (Türkiye)
• Cukurova Universitesi Tip Fakultesi — Sarıçam, Turkey (Türkiye)
• Al Jalila Children's Specialty Hospital — Dubai, United Arab Emirates
• Royal Manchester Children's Hospital — Manchester, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.