RecruitingPHASE2INTERVENTIONAL

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

The ADAPT study aims to check the long-term safety of a medicine called INZ-701. This medicine is for people with rare genetic conditions called ENPP1 Deficiency or ABCC6 Deficiency. These conditions can cause issues like hardening of the arteries or bone problems. This study is specifically for patients who have already been taking INZ-701 in a different clinical trial and want to keep receiving the treatment. Participants will be given INZ-701 once a week by an injection under the skin and will continue treatment until the medicine is widely available or its development stops. The main goal is to understand if INZ-701 is safe over a longer period.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Inozyme Pharma

Enrolment target

200

Start

19 Jun 2024

Estimated completion

01 Dec 2030

Gene Mutations Pseudoxanthoma Elasticum Arterial Calcification Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Type 2

What is this study about?

This study, called ADAPT, is designed to look closely at the long-term safety of a new medication named INZ-701. This treatment is being developed for people living with very rare conditions called ENPP1 Deficiency and ABCC6 Deficiency. These conditions are caused by changes in specific genes and can lead to serious health problems, such as hardening of the arteries (when blood vessels become stiff) or difficulties with bone development.

INZ-701 is a special protein designed to help address the underlying cause of these conditions. It's a type of 'replacement' therapy, meaning it aims to do the job of the missing or faulty protein in the body. The 'ADAPT' part of the name helps remind us that it’s about adapting to long-term use and understanding its effects over time.

This study is for a very specific group of people: those who have already taken INZ-701 in a previous clinical trial and found it helpful or are keen to continue its potential benefits. By studying these patients over a longer period, researchers can learn more about how safe the medicine is and if there are any long-term side effects. This information is crucial for deciding if INZ-701 can become a widely available treatment in the future.

Key takeaways

This study looks at the long-term safety of INZ-701.
It's for people with ENPP1 Deficiency or ABCC6 Deficiency who've already had INZ-701.
Treatment involves weekly injections under the skin.
The study aims to provide ongoing access to INZ-701.
You'll have regular check-ups to monitor your health and safety.

Who may be eligible?

To join this study, you must have already taken part in a previous INZ-701 study for ENPP1 Deficiency or ABCC6 Deficiency and successfully completed the safety and effectiveness checks in that study. You need to be at least 1 year old.

If you are able to get pregnant and are sexually active, you must agree to use effective birth control throughout the study and for 30 days after your last dose of INZ-701. Similarly, if you are a male participant who is sexually active, you will need to use a condom during treatment and for 30 days after your last dose.

You cannot join if you have any other significant health problems that are not related to your ENPP1 or ABCC6 Deficiency, if you've had a bad reaction to INZ-701 before, or if you're already participating in another study that involves a new treatment.

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.

Have I taken part in a previous INZ-701 study for ENPP1 or ABCC6 Deficiency?
Am I at least 1 year old?
Am I willing to use effective birth control/condoms if sexually active?
Do I have any other major health issues not related to ENPP1 or ABCC6 Deficiency?
Have I had a bad reaction to INZ-701 before?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll first have a screening period lasting about 30 days. During this time, the study team will carry out checks to make sure you're suitable for the study. After screening, all participants will receive the study medicine, INZ-701. This will be given once a week as an injection just under your skin.

You will continue to receive INZ-701 for an extended period, until it becomes widely available for purchase in your country or until the company decides to stop its development. Throughout this time, you'll have regular visits to the clinic so the study team can monitor your health and how you're responding to the treatment. After your last dose of INZ-701, you'll have one final safety check-up about 30 days later to make sure all is well.

Potential risks and benefits

The main benefit of joining this study is the chance to continue receiving INZ-701, which could potentially help manage your condition, especially if you've already experienced benefits in a previous study. However, like all medicines, INZ-701 can have side effects, which the study aims to monitor closely over the long term. There may also be inconveniences from regular clinic visits and injections. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (5)

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

Mayo Clinic
Verified postcode
Rochester, United States· Recruiting
Clinilabs Drug Development Corporation
Verified postcode
Eatontown, United States· Recruiting
Necker-Enfants Malades Hospital
Verified postcode
Paris, France· Recruiting
Universitätsklinikum Hamburg-Eppendorf (UKE)
Verified postcode
Hamburg, Germany· Recruiting
VCTC
Verified postcode
Oxford, United Kingdom· Recruiting

Common questions

What is INZ-701?

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

Who is this study for?

This study is for people who have already taken part in a previous study of INZ-701 for ENPP1 or ABCC6 Deficiency and want to continue the treatment.

How is INZ-701 given?

INZ-701 is given as an injection under the skin once a week.

How long will I be in the study?

You'll continue treatment until INZ-701 is generally available or its development stops, followed by a final safety check-up.

What are the main risks?

The main risks include potential side effects from INZ-701 and the time commitment for clinic visits and injections. All risks will be fully explained.

How to find out more

Inozyme Clinical Trial Information

clinicaltrials@inozyme.com +1 857 330 4340 Official trial record

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