Interventional trials in ERN-RND DiseaseCenterSponsorTreatmentCenterContactKey Information Metachromatic LeukodystrophyHospital Universitari Vall d'HebronTakeda/Shireintrathecal ERT (SHP611), MulticenterHospital Universitari Vall d'Hebron, Barcelona, SpainContact AdrenoleukodystrophyKremlin Bicêtre, FranceBluebirdLenti-D, MulticenterKremlin Bicêtre, FranceContact closed for inclusions Vanishing White MatterAmsterdam Leukodystrophy CenterAmsterdam UMCGuanabenz, open label, MonocenterAmsterdam Leukodystrophy CenterContact Alexander diseaseAmsterdam Leukodystrophy CenterIonisIntrathecal antisense oligonucleotide, MulticenterAmsterdam Leukodystrophy CenterContact Pelizaeus-Merzbacher diseaseAmsterdam Leukodystrophy CenterAmsterdam UMCDeferiprone, open label, MonocenterAmsterdam Leukodystrophy CenterContact Not yet started Krabbe diseaseAmsterdam Leukodystrophy CenterPassageBiointrathecal gene therapy (PBKR03), MulticenterAmsterdam Leukodystrophy CenterContact AdrenoleukodystrophyKremlin Bicêtre, FranceMinoryxMIN-102, MulticenterKremlin Bicêtre, FranceContact Metachromatic leukodystrophyKremlin Bicêtre, FranceTakeda/Shireintrathecal ERT (SHP611), MulticenterKremlin Bicêtre, FranceContact closed for inclusions Metachromatic LeukodystrophyUniversity Children's Hospital TübingenTakeda/Shireintrathecal ERT (SHP611), MulticenterUniversity Children's Hospital TübingenContact Metachromatic LeukodystrophyAmsterdam Leukodystrophy CenterTakeda/Shireintrathecal ERT (SHP611), MulticenterAmsterdam Leukodystrophy CenterContact closed for inclusions AdrenoleukodystrophyAmsterdam Leukodystrophy CenterSwanBioAAV-9 gene therapy to stop progression of myelopathy, MulticenterAmsterdam Leukodystrophy CenterContact AdrenoleukodystrophyAmsterdam Leukodystrophy CenterMGHPramipexole, MulticenterAmsterdam Leukodystrophy CenterContact AdrenoleukodystrophyAmsterdam Leukodystrophy CenterPoxelPXL770, MulticenterAmsterdam Leukodystrophy CenterContact Not yet started