- authorisedTherapeutic confirmatory (Phase III)
A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, βS/βC Genotype
This study looks at a new gene therapy, exa-cel, for people with severe sickle cell disease. It aims to see if a single treatment can stop painful sickle cell crises for a year or more. Researchers will check if it's safe and how well it helps patients.
Italy · France - authorisedTherapeutic confirmatory (Phase III)
The Effect of Mitapivat on Cerebral Perfusion and Oxygen Metabolism in Sickle Cell Disease
This study looks at a new medicine called mitapivat for people with sickle cell anaemia. It wants to see how this medicine affects blood flow to the brain and how the brain uses oxygen, using special MRI scans over 12 months.
Netherlands - authorisedTherapeutic use (Phase IV)
Morphine PCA vs Morphine and Lidocaine PCA for the treatment of vaso-occlusive crisis-related pain in sickle-cell anaemia patients: a prospective, randomized, double blind monocentric trial.
This study looks at treating pain from sickle cell crises. Researchers are comparing morphine alone with a combination of morphine and lidocaine to see if the combined treatment helps reduce the amount of morphine needed to ease the pain.
Belgium