Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency research hub
4 indexed studies · 0 currently recruiting
- active not recruitingPHASE3
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
The ENERGY 3 Study is looking at a new treatment called INZ-701 for children aged 1-12 with a rare genetic condition called ENPP1 Deficiency. This study wants to see how well INZ-701 works and if it's safe compared to usual care.
United States · Australia · Canada - active not recruitingPHASE2
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
This study looks at the long-term safety of a treatment called INZ-701 for people with rare conditions like ENPP1 Deficiency or ABCC6 Deficiency. It's for patients who have already tried INZ-701 in a previous study and wish to continue.
United States · France · Germany - active not recruitingPHASE3
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
This study, called ENERGY 2, is looking at a new medicine called INZ-701 for babies with a rare genetic condition called ENPP1 Deficiency. Researchers want to see if the medicine is safe and helps improve the health of these babies.
Brazil · France · Hungary - active not recruitingPHASE1
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
The ENERGY Study is looking into a new medicine called INZ-701 for babies under one year old with two rare genetic conditions: ENPP1 Deficiency or ABCC6 Deficiency. The main goal is to check how safe the medicine is and if babies can tolerate it well.
United States · Spain · United Kingdom