The ADAPT Study: An Open-Label, Long-term Safety Study of INZ-701 in Patients with ENPP1 Deficiency and ABCC6 Deficiency
The ADAPT study is testing an experimental medicine called INZ-701 in adults diagnosed with rare genetic conditions known as ENPP1 deficiency or ABCC6 deficiency. People with these conditions have low levels of a substance that stops calcium from building up where it should not, such as in blood vessels and the skin. INZ-701 is designed to help restore this balance. This is an 'open-label' study, which means every participant will receive the study medicine, with no dummy drug (placebo) used. The main goal is to find out if the treatment is safe to take over a longer period. Doctors will closely track any side effects, carry out regular blood and urine tests, and see how the body processes the drug. The results will help researchers understand if INZ-701 could become a safe treatment option in the future.
At a glance
What is this study about?
ENPP1 deficiency and ABCC6 deficiency are rare genetic conditions that affect how the body manages minerals. Normally, our bodies make a natural compound called pyrophosphate, which stops minerals like calcium from sticking to blood vessels, eyes, and joints. In people with ENPP1 or ABCC6 deficiency, pyrophosphate levels are far too low. Over time, this can lead to calcium deposits that harden blood vessels and cause other long-term health problems.
INZ-701 is a new medicine designed to work like the missing enzyme in the body. Its goal is to raise pyrophosphate levels back toward a healthy range, which may help prevent or reduce harmful mineral buildup. While earlier research has looked at how the drug works in the short term, doctors now need to see how safe it is when taken over a longer period.
The ADAPT study is an 'open-label' trial, meaning both the doctors and the participants know that everyone taking part receives INZ-701. Researchers will track side effects very closely. They will also check whether the immune system reacts to the drug and measure the levels of the medicine in the blood.
Taking part in this study helps researchers gather vital safety details about INZ-701. For rare conditions with few or no approved treatments, studies like this are an important step toward finding safe and helpful therapies.
Key takeaways
- The ADAPT study is testing the long-term safety of an experimental drug called INZ-701.
- The study is for adults aged 18 and older with ENPP1 deficiency or ABCC6 deficiency.
- All participants receive the study medicine; there is no dummy drug (placebo).
- Doctors will use regular blood tests, urine checks, and clinic visits to watch for side effects.
- Taking part is voluntary, and you can stop at any time without affecting your normal care.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with either ENPP1 deficiency or ABCC6 deficiency. Both men and women can take part.
Because this is a specialised safety study, participants must be able to attend regular clinic visits for health checks and tests. The study doctors will carefully review your full medical history, your current health, and any other medicines you take to ensure it is safe for you to join.
You may not be eligible if you have certain other serious medical conditions or if you are taking medications that could interfere with the study drug. The trial team will discuss the complete list of entry rules with you before you agree to take part.
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.
- I am aged 18 or older.
- I have a confirmed diagnosis of ENPP1 deficiency or ABCC6 deficiency.
- I am able and willing to attend regular hospital or clinic visits for monitoring.
- I am open to receiving an experimental medicine and having regular blood and urine tests.
What does participation involve?
If you take part, you will receive the study medicine, INZ-701, and visit the clinic regularly so the medical team can monitor your health. During visits, you will have routine physical exams, blood tests, and urine tests. The team will check your blood pressure, ask how you are feeling, and record any other medicines you are taking. Blood tests will also measure drug levels and check for any immune reactions. The study runs over an extended period to assess long-term safety, and the trial team will explain the exact visit schedule and expected duration before you join.
Potential risks and benefits
Locations (2)
- —UnverifiedGermany
- —UnverifiedFrance
Common questions
Will I receive a placebo (dummy drug)?
No. This is an open-label study, which means every person who joins will receive the actual study medicine, INZ-701.
Does it cost anything to take part?
No. The study medicine and all trial-related tests are provided free of charge. Travel expenses to and from the clinic are often covered as well.
Can I leave the study early if I change my mind?
Yes. You have the right to withdraw from the trial at any point, and your standard medical care will not be affected in any way.
Will this medicine cure my condition?
INZ-701 is an experimental treatment and is not proven to be a cure. The main purpose of this study is to see if it is safe to use over time.
Can I keep taking my usual medicines?
The study team will review all your current medicines to make sure they can be safely taken alongside INZ-701.
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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