The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
The ENERGY 2 study is a Phase 3 clinical trial looking at an experimental treatment called INZ-701 for babies born with ENPP1 deficiency. ENPP1 deficiency is a very rare genetic condition where the body lacks an important enzyme. Without this enzyme, levels of a natural protective substance drop, causing calcium to settle in blood vessels and vital organs. This can lead to serious heart complications, breathing problems, and poor growth. In this study, all babies receive INZ-701. Researchers will track their progress over 52 weeks (one year) to find out if the treatment safely restores key chemical balances, protects heart and blood vessel health, and helps babies gain weight and grow normally. Doctors will also check whether the medicine reduces the need for breathing machines and lowers the risk of heart failure.
At a glance
What is this study about?
ENPP1 deficiency is a rare inherited condition that affects how the body manages minerals. Normally, an enzyme called ENPP1 helps create a substance that stops calcium crystals from forming in soft tissues. When babies do not have enough of this enzyme, calcium can quickly build up inside their blood vessels and heart. This can make blood vessels narrow and stiff, putting heavy strain on a young baby's heart and lungs.
The ENERGY 2 study is looking at a new medicine called INZ-701. It is designed to act like the missing enzyme, helping the body produce the protective substances it needs. By doing this, doctors hope to stop or reverse dangerous calcium deposits, protect the heart, and support normal feeding, weight gain, and physical development.
This trial is an 'open-label' Phase 3 study, which means every baby who joins will receive the actual medicine rather than a dummy treatment (placebo). Because ENPP1 deficiency can be serious in early life, research like this is vital to finding safe, effective treatments that could give affected infants the best possible start.
Key takeaways
- ENERGY 2 is testing a new medicine, INZ-701, for babies with ENPP1 deficiency.
- The study aims to prevent calcium build-up in blood vessels and protect heart health.
- This is an open-label trial, so every enrolled baby receives the study medicine.
- Babies are followed closely by specialist medical teams over 52 weeks.
- Taking part is completely voluntary and you can withdraw at any time.
Who may be eligible?
This trial is designed specifically for babies who have been diagnosed with ENPP1 deficiency. Both boys and girls can take part.
To join, babies must be able to attend the required hospital visits with their parents or guardians. Doctors will review the baby's overall health and medical history to ensure that taking part in the study is appropriate and safe.
Because this is a study for young infants, parents or legal guardians must provide full informed consent before any tests or treatments begin. Your hospital specialist can confirm if your baby meets all the exact medical criteria.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- My child is an infant (baby).
- My child has a confirmed or suspected diagnosis of ENPP1 deficiency.
- I am able to bring my baby to regular hospital appointments for check-ups and scans.
- I am the baby's parent or legal guardian and able to give consent.
What does participation involve?
Taking part in the ENERGY 2 study lasts for about 52 weeks (one year). During this time, your baby will receive the study medicine, INZ-701, and will be monitored closely by a team of specialist doctors and nurses.
Participation involves regular hospital visits. At these visits, the medical team will carry out health checks to see how well the medicine is working and check for any side effects. These assessments include simple blood tests to measure enzyme and mineral levels, heart scans (ultrasounds called echocardiograms), and low-dose imaging scans (CT scans) to check blood vessels.
The team will also track your baby's day-to-day progress, including their length, weight, feeding, and whether they need any breathing support. You will have direct support from the study team throughout the year.
Potential risks and benefits
Locations (5)
- —UnverifiedHungary
- —UnverifiedFrance
- —UnverifiedSweden
- —UnverifiedItaly
- —UnverifiedSpain
Common questions
What does an 'open-label' study mean?
An open-label study means that both you and the doctors will know that your baby is receiving the active study medicine. No dummy treatments (placebos) are used in this trial.
How long will my baby be in the study?
The main part of the study lasts for 52 weeks, which is about one year of regular check-ups and treatment.
Will taking part cost our family anything?
No. The study medicine and all trial-related appointments, tests, and scans are provided at no cost to your family.
Can the study cure my baby's condition?
The trial is testing whether INZ-701 can effectively control the condition and prevent complications, but it is not guaranteed to be a permanent cure.
Can we leave the study if we change our minds?
Yes. You have the right to withdraw your baby at any time, for any reason, and your baby's standard medical care will continue as normal.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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