Not recruitingRare disease

Long-term Safety Study of INZ-701 for Patients with ENPP1 and ABCC6 Deficiencies, Including Pseudoxanthoma Elasticum and Generalized Arterial Calcification of Infancy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the long-term safety of a treatment called INZ-701 for patients with certain rare conditions. These conditions include Pseudoxanthoma Elasticum (PXE), Generalized Arterial Calcification of Infancy (GACI), and deficiencies in Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) and ATP-binding cassette sub-family C member 6 (ABCC6). These are genetic disorders that can cause problems with the skin, eyes, and blood vessels, among other symptoms.

The treatment being studied, INZ-701, is a type of medication that is given as an injection under the skin. It is a specially designed protein that aims to help manage these conditions. The study will follow participants who have already received INZ-701 in a previous clinical study to see how they respond to the treatment over a longer period.

Participants in this study will receive regular injections of INZ-701 and will be monitored for any side effects or changes in their health. The study will look at how often and how severe any side effects are, as well as any changes in laboratory tests. This information will help researchers understand the long-term safety of INZ-701 for people with these rare conditions.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the patient must have completed the required safety and efficacy period of a previous INZ-701 clinical study for ENPP1 or ABCC6 deficiency.
The patient must provide written or electronic informed consent after the study's nature has been explained.
Step 2
Medication administration
The patient will receive INZ-701, a lyophilized powder prepared for injection.
The medication is administered through subcutaneous injection.
The dosage and frequency of administration will be determined by the study protocol.
Step 3
Monitoring and assessments
The study aims to assess the long-term safety of INZ-701.
The patient will be monitored for any adverse events, including treatment-emergent adverse events and serious adverse events.
Regular clinical laboratory tests, including chemistry, hematology, and urinalysis, will be conducted to identify any changes resulting in adverse events.
Step 4
Additional evaluations
The study will measure INZ-701 plasma pharmacokinetic parameters using immunoassay and enzymatic activity.
Plasma inorganic pyrophosphate (PPi) concentrations will be assessed based on a validated assay.
Step 5
Completion of the study
The study is estimated to conclude by December 31, 2030.
The patient will continue to be monitored for safety and efficacy throughout the study duration.

Who can join the trial?

7 criteria

Provide written or electronic informed consent after the study has been explained, before any research-related procedures. This means you agree to participate after understanding the study.
If under 18 years of age, provide assent according to local rules. Assent is a way for younger participants to agree to join the study.
Be male or female and older than 1 year of age.
Must have completed the required safety and effectiveness periods of a previous INZ-701 clinical study for ENPP1 or ABCC6 Deficiency, as confirmed by the study sponsor. This means you have already participated in a related study and finished certain parts of it.
Female participants who can have children and are sexually active must use or agree to use one highly effective form of birth control from at least one month before the first dose of INZ-701 through 30 days after the last dose. They must also agree not to donate eggs during this time.
Male participants who are sexually active must agree to use condoms from the first dose of INZ-701 through 30 days after the last dose. They must also agree not to donate sperm during this time.
In the opinion of the study doctor, be able to complete all parts of the study. This means the doctor believes you can participate in all study activities.

Who cannot join the trial?

4 criteria

Patients with **Pseudoxanthoma elasticum (PXE)** cannot participate. PXE is a rare genetic disorder that affects the skin, eyes, and blood vessels.
Patients with **Generalized Arterial Calcification of Infancy (GACI)** are excluded. GACI is a condition where calcium builds up in the arteries of infants.
Patients with **Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency** cannot join. This deficiency affects the body's ability to process certain molecules.
Patients with **ATP-binding cassette sub-family C member 6 (ABCC6) Deficiency** are not eligible. This deficiency is related to a gene that affects the transport of molecules in the body.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Hopital Necker Enfants Malades
Paris, France
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PAREXEL International GmbH
Berlin, Germany
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University Medical Center Hamburg-Eppendorf
Hamburg, Germany

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Investigated drugs

INZ-701 is a medication being studied for its long-term safety in patients with ENPP1 Deficiency and ABCC6 Deficiency. This medication is designed to help manage these specific genetic conditions, which can affect the body's ability to regulate certain minerals and lead to complications. The study aims to ensure that INZ-701 is safe for patients to use over an extended period.

Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1

What is already known about the treatment

INZ-701 – INZ-701 is an investigational medication administered through injection, currently being studied for its long-term safety in treating conditions like Pseudoxanthoma elasticum (PXE), Generalized Arterial Calcification of Infancy (GACI), and deficiencies in ENPP1 and ABCC6. It is not yet approved for general medical use, as it is still undergoing clinical trials. The medication is designed to address these rare genetic disorders by potentially correcting enzyme deficiencies that lead to abnormal calcification in the body. INZ-701 falls under the category of enzyme replacement therapies, aiming to restore normal function at the molecular level.

Investigated diseases

Pseudoxanthoma Elasticum (PXE)
This is a genetic disorder that affects the connective tissue in the body, primarily impacting the skin, eyes, and blood vessels. It is characterized by the progressive calcification and fragmentation of elastic fibers, leading to changes in skin texture, vision problems, and cardiovascular issues. The skin may develop yellowish papules, especially on the neck and flexural areas. Over time, vision can be affected due to changes in the retina, potentially leading to vision loss. The disease progresses at varying rates among individuals, with symptoms often appearing in childhood or early adulthood.
Generalized Arterial Calcification of Infancy (GACI)
This rare genetic disorder is characterized by the abnormal accumulation of calcium in the walls of large and medium-sized arteries. It typically presents in infancy, leading to the hardening and narrowing of the arteries, which can affect blood flow. The condition may cause high blood pressure and heart problems due to the increased workload on the heart. As the disease progresses, it can lead to complications in various organs due to reduced blood supply. The severity and progression of symptoms can vary widely among affected individuals.
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency and ATP-binding cassette sub-family C member 6 (ABCC6) Deficiency
These are genetic conditions that affect the body's ability to regulate mineralization, leading to abnormal calcification in tissues. ENPP1 deficiency is associated with GACI, while ABCC6 deficiency is linked to PXE. Both conditions result in the improper deposition of calcium and other minerals in the body, affecting the skin, eyes, and cardiovascular system. Symptoms can include skin changes, vision problems, and cardiovascular complications. The progression of these conditions varies, with symptoms often appearing in early life.