Interventional trials in ERN-RND – Leukodystrophies

DiseaseTreatmentCenterContactKey Information
Metachromatic LeukodystrophyTakeda/Shireintrathecal ERT (SHP611), MulticenterHospital Universitari Vall d'Hebron, Barcelona, Spain

">Contact

AdrenoleukodystrophyBluebirdLenti-D, MulticenterKremlin Bicêtre, FranceContact closed for inclusions
Vanishing White MatterAmsterdam UMCGuanabenz, open label, MonocenterAmsterdam Leukodystrophy CenterContact
Alexander diseaseIonisIntrathecal antisense oligonucleotide, MulticenterAmsterdam Leukodystrophy CenterContact
Pelizaeus-Merzbacher diseaseAmsterdam UMCDeferiprone, open label, MonocenterAmsterdam Leukodystrophy CenterContactNot yet started

Krabbe disease

PassageBiointrathecal gene therapy (PBKR03), MulticenterAmsterdam Leukodystrophy Center">Contact
AdrenoleukodystrophyMinoryxMIN-102, MulticenterKremlin Bicêtre, FranceContact
Metachromatic leukodystrophyTakeda/Shireintrathecal ERT (SHP611), MulticenterKremlin Bicêtre, FranceContactClosed for inclusions
Metachromatic LeukodystrophyTakeda/Shireintrathecal ERT (SHP611), MulticenterUniversity Children's Hospital TübingenContact
Metachromatic LeukodystrophyTakeda/Shireintrathecal ERT (SHP611), MulticenterAmsterdam Leukodystrophy CenterContactClosed for inclusions
AdrenoleukodystrophySwanBioAAV-9 gene therapy to stop progression of myelopathy, MulticenterAmsterdam Leukodystrophy CenterContact
AdrenoleukodystrophyMGHPramipexole, MulticenterAmsterdam Leukodystrophy CenterContact
AdrenoleukodystrophyPoxelPXL770, MulticenterAmsterdam Leukodystrophy CenterContact Not yet started