Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

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

The ENERGY study is investigating a new medicine called INZ-701 for infants (very young children) who have one of two rare genetic conditions. These conditions are sometimes called ENPP1 deficiency or ABCC6 deficiency. The main goal of this study is to find out if INZ-701 is safe for these children and to understand how their bodies react to the medicine. Researchers will be carefully checking for any side effects, observing vital signs like heart rate and weight, and performing blood and urine tests. They also want to see how the medicine moves through the body and assess its potential effects on the condition itself. This is an early-stage study, meaning it's one of the first times this medicine is being tested in humans.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Inozyme Pharma Inc.

Enrolment target

Start

11 Mar 2024

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

What is this study about?

The ENERGY study is a research project focusing on a new medicine, INZ-701, for infants diagnosed with very specific genetic conditions. These conditions are quite rare and can affect different parts of the body. The scientific names for these conditions are Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency and ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency. Because these names are long and complicated, it's easier to think of them as rare genetic problems that doctors are trying to understand and treat better.

In this study, doctors want to learn several important things about INZ-701. Firstly, they need to know if the medicine is safe for young children with these conditions. They will be looking very closely for any unwanted effects, often called side effects. Secondly, they want to understand how the medicine works in the body – for example, how much of it stays in the blood and for how long. This helps them figure out the best way to give the medicine in the future. They will also look at whether the medicine has any effect on certain markers related to the conditions.

This type of study is called a 'Phase I' study, which means it's one of the earliest steps in testing a new medicine in people. It's a key stage before a medicine can move on to larger studies to see how well it works. By carefully collecting information on safety, how the body handles the medicine, and early signs of its effects, researchers can decide if INZ-701 has the potential to help children with these rare conditions.

Key takeaways

This study is testing a new medicine (INZ-701) for infants with rare genetic conditions.
The main goals are to check the medicine's safety and how the body handles it.
It's an early-stage study, meaning it's one of the first times it's being tested in people.
Participation involves regular clinic visits, blood tests, and heart checks.
Both boys and girls with these specific conditions are eligible.
You can withdraw your child from the study at any time.

Who may be eligible?

This study is specifically for infants (babies) who have been diagnosed with one of two rare genetic conditions: ENPP1 deficiency or ABCC6 deficiency.

To be considered for the study, your baby's doctor would need to confirm that they have one of these specific conditions. There are no age limits within the 'infant' category, so babies of various ages could potentially be included.

Both boys and girls can take part in this study, assuming they meet the other requirements related to their specific medical condition.

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 your child an infant (baby)?
Has your child been diagnosed with ENPP1 deficiency?
OR has your child been diagnosed with ABCC6 deficiency?
Are you able to attend regular hospital or clinic visits?

Answer every question to see your result.

What does participation involve?

If your child takes part in the ENERGY study, they would receive the study medicine, INZ-701. Participation would involve regular visits to the clinic or hospital. During these visits, there would be various assessments to check your child's health and how they are reacting to the medicine. These would include checking their vital signs (like heart rate and breathing), taking their weight, and collecting blood and urine samples for tests. Doctors would also monitor their heart using specific scans (like an ultrasound of the heart called an echocardiogram and an electrocardiogram, which checks the electrical activity of the heart).

The study would also involve keeping track of any other medicines your child is taking and carefully noting any side effects or changes in their health. The information gathered, especially about how the medicine is absorbed and leaves the body, would be collected over regular intervals for up to a year (52 weeks). There will be a detailed schedule of visits and tests over this entire period.

Potential risks and benefits

As with any new medicine, there are potential benefits and potential risks to consider. The potential benefit is that INZ-701 might help manage or improve your child's rare genetic condition, although this is what the study aims to discover. Potential risks could include side effects from the medicine, which is why close monitoring is a key part of the study. There is also the possibility that the medicine might not help your child. You and your child have the right to withdraw from the study at any time, for any reason, without it affecting your child's future medical care.

Locations (1)

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

—
Unverified
Spain

Common questions

What is a 'genetic deficiency'?

A genetic deficiency means that there's a small change in a child's genes that causes their body not to make enough of a certain important protein or chemical.

Is this medicine available to everyone?

No, INZ-701 is an experimental medicine, which means it's still being studied and is not yet available as a regular treatment.

What does 'open-label' mean?

It means that both you and the doctors involved in the study will know that your child is receiving the study medicine, INZ-701.

What happens after the study ends?

At the end of the study, your child's doctors will discuss the next steps for their care. The study aims to gather information, not necessarily provide long-term treatment.

Will my child feel any pain?

Some procedures, like blood tests, involve minor discomfort. The study team will always try to make your child as comfortable as possible and explain everything beforehand.