RecruitingPHASE1INTERVENTIONAL

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

The ENERGY Study (also known as INZ701-104) is a clinical trial exploring a new medication called INZ-701. This medicine is being developed for babies under one year of age who have certain rare genetic conditions: ENPP1 Deficiency or ABCC6 Deficiency. These conditions can cause serious health problems like hardening of the arteries. The study is in its early stages (Phase 1), meaning researchers are primarily focused on understanding if INZ-701 is safe for these infants and if they can tolerate it without too many side effects. Participants will receive the medicine and be closely monitored over a long period to see how their bodies react and to gather important information about this potential new treatment.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Inozyme Pharma

Enrolment target

Start

25 Jun 2023

Estimated completion

11 Nov 2027

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy ATP-Binding Cassette Subfamily C Member 6 Deficiency Pseudoxanthoma Elasticum

What is this study about?

The ENERGY Study is looking into a new medicine called INZ-701 for very young babies. This medicine is designed for infants who have one of two rare genetic conditions: ENPP1 Deficiency or ABCC6 Deficiency. These conditions can lead to serious health issues, including problems with their arteries (the blood vessels that carry blood from the heart). At the moment, there aren't many specific treatments for these illnesses, so researchers are hoping INZ-701 could make a difference.

This study, also known as INZ701-104, is in its first phase (Phase 1). This means the main focus is on making sure the new medicine, INZ-701, is safe for babies and that they can take it without too many problems. Researchers will be carefully checking for any side effects and how the babies' bodies handle the medicine. They will also look at how the medicine works in the body and if it has any positive effects.

The study aims to gather important information about INZ-701. If it proves to be safe and manageable for these very ill babies, it could potentially move on to larger studies to see if it's an effective treatment. This research is a crucial step in finding new ways to help infants with these challenging genetic conditions.

Key takeaways

The ENERGY Study is for babies under one year old with rare genetic conditions (ENPP1 or ABCC6 Deficiency).
It's testing a new medicine called INZ-701 to see if it's safe and well-tolerated.
The treatment period lasts for about one year, with potential for extension.
Babies must have certain health signs, including 'generalized arterial calcification of infancy' (GACI).
Close medical monitoring is a key part of participating.
Your decision to join or leave the study is entirely voluntary and won't affect care.

Who may be eligible?

This study is specifically designed for very young babies. To be considered, your baby must be less than one year old when they join the study. They also need to have a confirmed diagnosis of either ENPP1 Deficiency or ABCC6 Deficiency through genetic testing.

Additionally, your baby must be showing signs of a condition called 'generalized arterial calcification of infancy' (often shortened to GACI or GACI-2). This could include things like unusual calcium buildup in their body, heart problems (like heart failure or an enlarged heart), breathing difficulties, swelling, blue skin (cyanosis), or high blood pressure. Your baby also needs to weigh at least half a kilogram (about 1.1 pounds) when they receive their first dose of the study medicine. A parent or legal guardian must be able to give written permission for their baby to join.

There are also some reasons why a baby might not be able to join. For example, if the doctor believes other significant health problems could interfere with the study results, or if the baby has cancer, or is already receiving end-of-life care. Also, babies who are already taking part in another clinical trial unrelated to this study or receiving other experimental treatments would not be eligible.

Quick self-check

Is my baby less than one year old?
Does my baby have a confirmed genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency?
Is my baby showing signs of GACI, such as heart problems or unusual calcium buildup?
Does my baby weigh at least 0.5 kg (about 1.1 lbs)?
Is my baby currently not in another experimental drug study?
Am I, as a parent or guardian, able to give written consent?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your baby joins the study, the first step is a 'screening period' that can last up to 60 days. During this time, doctors will do checks to make sure your baby is suitable for the study. After screening, your baby will enter a 'treatment period' for 52 weeks (about a year), where they will regularly receive the study medicine, INZ-701.

After this year, there's a possibility of an 'extension period.' This means your baby might continue to receive INZ-701 until it's available for everyone or until another study for INZ-701 starts. About a month after your baby's last dose of INZ-701, there will be a final check-up visit. Even after treatment stops, the study team will keep in touch to check on your baby's health and survival outcome, usually every three months until the study officially ends.

Potential risks and benefits

Participating in a clinical trial can offer potential benefits, such as access to a new medicine that isn't yet available and close medical monitoring for your baby. However, there are also potential risks, as INZ-701 is a new medicine and its full effects, including side effects, are not yet completely known. The study team will closely watch your baby for any reactions and make sure their safety is the top priority. Remember, 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 (7)

Rady Children's Hospital
San Diego, United States· Recruiting
Boston Children's Hospital
Boston, United States· Withdrawn
Nationwide Children's Hospital
Columbus, United States· Recruiting
The Children's Hospital of Philadelphia
Philadelphia, United States· Recruiting
The University of Utah
Salt Lake City, United States· Withdrawn
Hospital Sant Joan de Déu
Barcelona, Spain· Terminated
Royal Manchester Children's Hospital
Manchester, United Kingdom· Terminated

Common questions

What is the main purpose of this study?

The main purpose is to check how safe a new medicine called INZ-701 is for babies with ENPP1 Deficiency or ABCC6 Deficiency, and if they can tolerate it well.

What kind of babies can join the study?

Babies under one year old with a confirmed diagnosis of ENPP1 or ABCC6 Deficiency, who are showing signs of GACI, and weigh at least 0.5 kg.

How long will the study last for my baby?

Your baby will receive treatment for about 52 weeks (one year), and there might be an option to continue treatment after that. There will also be follow-up checks for some time after treatment stops.

What is INZ-701?

INZ-701 is a new medicine being developed to treat rare genetic conditions called ENPP1 Deficiency or ABCC6 Deficiency.

Can I take my baby out of the study at any time?

Yes, you have the right to withdraw your baby from the study at any point, and it won't affect their regular medical care.

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.