RecruitingPHASE3INTERVENTIONAL

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

The ENERGY 2 study is a research project focusing on a rare genetic condition known as ENPP1 Deficiency, which can cause problems like brittle bones (rickets) and calcium build-up in the arteries. This study aims to find out how well a new medicine, INZ-701, works and if it's safe for infants with this condition. It's a Phase 3 study, meaning it's one of the final stages of testing before a medicine might become widely available. Babies taking part will receive the medicine for a year, with follow-up appointments after that. The goal is to see if INZ-701 can make a positive difference for these very young patients.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Inozyme Pharma

Enrolment target

Start

26 Mar 2025

Estimated completion

07 Nov 2028

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy 1

What is this study about?

ENPP1 Deficiency is a very rare genetic condition that some babies are born with. It happens because a specific gene isn't working properly, which means the body doesn't have enough of an important enzyme. This shortage can lead to several serious health problems. One common issue is a severe form of rickets, which makes bones weak and soft. Another concern is that calcium can build up in the blood vessels, particularly in the heart and surrounding areas, a condition known as GACI.

Researchers are always looking for new ways to help people with rare diseases. The ENERGY 2 study is specifically investigating a new medicine called INZ-701. The main purpose of this study is to see how well INZ-701 works and if it's safe for babies with ENPP1 Deficiency. This type of research is vital because it helps doctors understand if new treatments can improve the lives of those affected by these challenging conditions.

By taking part in this study, families are contributing valuable information that could lead to a better future for other children with ENPP1 Deficiency. The study carefully monitors the babies to see if the medicine helps their condition and ensures their safety throughout the process.

Key takeaways

This study is testing a new medicine (INZ-701) for a rare genetic condition called ENPP1 Deficiency.
It focuses on babies aged 1 year or younger with this condition.
The main goals are to see if the medicine is safe and effective.
Participation involves about two years of treatment and monitoring.
Families are carefully guided and supported throughout the study.
This research helps doctors find better treatments for future patients.

Who may be eligible?

This study is looking for very specific participants. To be considered, a baby must be 1 year old or younger when they join the study. They need to have a confirmed diagnosis of ENPP1 Deficiency, which would have been identified through genetic testing. Also, they should be showing signs of GACI, which means calcium build-up in their blood vessels or problems with their heart.

It's important that the baby is generally well enough to take part in a year-long treatment study, and a parent or legal guardian must agree to their participation by signing a consent form.

There are also some reasons why a baby wouldn't be able to join. For example, if they are receiving end-of-life care, or if they've already received INZ-701 (unless it was through a special programme). Babies also can't be in another medical study at the same time or have major surgery planned during the study period.

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.

Is my baby 1 year old or younger?
Does my baby have a confirmed diagnosis of ENPP1 Deficiency from a genetic test?
Does my baby have signs of GACI (calcium in blood vessels or heart problems)?
Is my baby generally well enough for a year-long medical study?
Is my baby currently in any other medical research studies?
Does my baby have major surgery planned in the near future?

Answer every question to see your result.

What does participation involve?

If your baby joins the ENERGY 2 study, it will involve several stages. First, there's a screening period, which can last up to 60 days. During this time, doctors will do tests and checks to make sure your baby is suitable for the study.

Once accepted, your baby will enter the treatment period, which lasts for 52 weeks (about one year). During this time, they will receive the INZ-701 medicine. After the treatment period, there's an extension period, also lasting 52 weeks, where your baby will continue to be monitored.

Finally, about 30 days after the last dose of INZ-701, there will be a final check-up visit. The total duration of active involvement for your baby would be around two years, plus the initial screening time.

Potential risks and benefits

Taking part in any clinical study has potential benefits and risks. A potential benefit of this study is that INZ-701 might help to improve your baby's condition by addressing the underlying issues of ENPP1 Deficiency. However, it's also possible that the medicine may not work or could cause side effects. Researchers will be carefully monitoring your baby for any changes. You have the right to withdraw your baby from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (8)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Hospital Universitario Pedro Ernesto/Rio de Janeiro
Verified postcode
Rio de Janeiro, Brazil· Recruiting
Hopital Necker - Enfants Malades
Verified postcode
Paris, France· Recruiting
Gyermekgyogyaszat, DE
Verified postcode
Debrecen, Hungary· Recruiting
Azienda Ospedaliera Universitaria Meyer
Verified postcode
Florence, Italy· Recruiting
King Faisal Specialist Hospital and Research Center
Verified postcode
Riyadh, Saudi Arabia· Recruiting
Hospital Sant Joan de Deu Edificio Consultas Externas. Unitat de Recerca
Verified postcode
Barcelona, Spain· Recruiting
Umraniye Traiing and Research Hospital
Verified postcode
Istanbul, Turkey (Türkiye)· Recruiting
Royal Manchester Children's Hospital
Verified postcode
Manchester, United Kingdom· Recruiting

Common questions

What is ENPP1 Deficiency?

It's a rare genetic condition where a crucial gene doesn't work properly, leading to weak bones and calcium build-up in blood vessels.

What is INZ-701?

INZ-701 is a new medicine being tested in this study to see if it can help babies with ENPP1 Deficiency.

How long will the study last for my baby?

Your baby's involvement will be about two years, including one year of treatment and one year of monitoring, plus an initial screening period.

What is a Phase 3 study?

Phase 3 is typically one of the last stages of testing a new medicine in a larger group of patients before it might be approved for wider use.

Can my baby stop participating at any time?

Yes, you have the right to take your baby out of the study at any point, and it won't affect their future medical treatment.

How to find out more

Trial Specialist

medinfo@bmrn.com +1 800-983-4587 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.