Interventional trials in ERN-RND

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


Contact

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 disease

Amsterdam 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