🏛️ Verified NIH Record • Updated 2026-08-05

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency (NCT05734196)

The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

🔵 Active, Not Recruiting Phase1 💊 Investigational Drug / Biologic Sponsor: Inozyme Pharma

🩺 Plain-English Summary (8th-Grade Level)

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.

Who can join: Review the eligibility criteria below.

Location: Rady Children's Hospital — San Diego, California

Age range: Not specified to 1 Year

🧪 Interventions in This Study

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (5 Sites)

  • Rady Children's Hospital — San Diego, California
  • Nationwide Children's Hospital — Columbus, Ohio
  • The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
  • Hospital Sant Joan de Déu — Barcelona,
  • Royal Manchester Children's Hospital — Manchester,

💰 Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗