We are investigating whether the drug INZ-701 can help infants with ENPP1 deficiency by improving survival and preventing heart and respiratory problems. The study aims to see if it can also support physical growth and reduce artery issues.
Health conditions and diseases that the clinical trial is designed to study and treat.
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency is a rare genetic disorder that affects the metabolism of certain nucleotides, leading to symptoms such as bone abnormalities, cardiovascular issues, and potential developmental delays.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.
