Ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Sat, 13 Jun 2026 04:03:02 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on INZ-701 for Improving Survival in Infants with ENPP1 Deficiency https://clinicaltrials.eu/trial/study-on-inz-701-for-improving-survival-in-infants-with-enpp1-deficiency/ Wed, 29 Apr 2026 14:59:09 +0000 https://clinicaltrials.eu/trial/study-on-inz-701-for-improving-survival-in-infants-with-enpp1-deficiency/ The study focuses on a rare condition called Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency, which affects infants. This condition can lead to serious health issues, including problems with the heart and the hardening of arteries. The treatment being tested is a medication known as INZ-701, which is a special type of protein designed to help manage this deficiency. The purpose of the study is to see if INZ-701 can increase the levels of a substance called inorganic pyrophosphate (PPi) in the body and improve the overall survival of infants with this condition.

During the study, infants will receive INZ-701 through a subcutaneous injection, which means it is given under the skin. The study will observe the infants over a period of time to see how their bodies respond to the treatment. Researchers will monitor changes in the infants’ health, including heart function and growth, to determine the effectiveness and safety of the treatment. The study will also compare the results with those of infants who do not receive the treatment to understand its impact better.

The study aims to provide valuable information on whether INZ-701 can help prevent heart dysfunction and the calcification of arteries in infants with ENPP1 Deficiency. By increasing the levels of PPi, the treatment may offer a new way to manage this challenging condition and improve the quality of life for affected infants. The study will continue for a set period, allowing researchers to gather comprehensive data on the treatment’s long-term effects.

]]> Long-term Safety Study of INZ-701 for Patients with ENPP1 and ABCC6 Deficiencies, Including Pseudoxanthoma Elasticum and Generalized Arterial Calcification of Infancy https://clinicaltrials.eu/trial/long-term-safety-study-of-inz-701-for-patients-with-enpp1-and-abcc6-deficiencies-including-pseudoxanthoma-elasticum-and-generalized-arterial-calcification-of-infancy/ Wed, 29 Apr 2026 14:58:53 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-inz-701-for-patients-with-enpp1-and-abcc6-deficiencies-including-pseudoxanthoma-elasticum-and-generalized-arterial-calcification-of-infancy/ 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.

]]> Study on the Effects and Safety of INZ-701 for Children with ENPP1 Deficiency https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-inz-701-for-children-with-enpp1-deficiency/ Wed, 29 Apr 2026 14:47:59 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-inz-701-for-children-with-enpp1-deficiency-2/ This clinical trial is focused on studying a rare condition known as Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency. This condition affects the body’s ability to manage certain minerals, which can lead to problems with bone development. The study will use a treatment called INZ-701, which is a special type of protein designed to help improve the symptoms of this deficiency. INZ-701 is given as an injection under the skin.

The purpose of the study is to see if INZ-701 can increase the levels of a substance called PPi in the blood and improve bone health in children with ENPP1 Deficiency. The study will last for about a year, during which participants will receive regular injections of INZ-701. Throughout the study, doctors will monitor the participants’ health and check for any changes in their bone condition and overall well-being.

Participants in the study will be children who have been diagnosed with ENPP1 Deficiency. The study will involve regular visits to the clinic for check-ups and assessments. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of INZ-701. The study aims to provide valuable information on the safety and effectiveness of INZ-701 in treating this rare condition.

]]> Study on the Safety and Effects of INZ-701 for Infants with ENPP1 or ABCC6 Deficiency https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-inz-701-for-infants-with-enpp1-or-abcc6-deficiency/ Wed, 29 Apr 2026 14:47:55 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-inz-701-for-infants-with-enpp1-or-abcc6-deficiency/ The ENERGY Study is focused on understanding the safety and effects of a treatment called INZ-701 in infants who have a rare condition known as ENPP1 Deficiency or ABCC6 Deficiency. These conditions can lead to serious health issues, including problems with calcium buildup in the body, which can affect the heart and other organs. The treatment being studied, INZ-701, is a special type of protein designed to help manage these conditions.

The purpose of the study is to evaluate how safe and tolerable INZ-701 is for infants with these deficiencies. The study will involve giving the treatment as an injection under the skin. Participants will be closely monitored to see how their bodies respond to the treatment, including any side effects they might experience. The study will also look at how the treatment moves through the body and how it affects certain biological markers related to the conditions.

Throughout the study, various health checks will be conducted, such as monitoring vital signs, weight, and conducting laboratory tests. These tests will help researchers understand the impact of INZ-701 on the participants’ health. The study aims to gather important information that could lead to better treatments for infants with ENPP1 Deficiency or ABCC6 Deficiency in the future.

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