Interventional trials in ERN-RND – Leukodystrophies

Disease	Sponsor	Treatment	Center	Contact	Key Information
Metachromatic Leukodystrophy	Takeda/Shire	intrathecal ERT (SHP611), Multicenter	Hospital Universitari Vall d'Hebron, Barcelona, Spain	Contact
Adrenoleukodystrophy	Bluebird	Lenti-D, Multicenter	Kremlin Bicêtre, France	Contact	closed for inclusions
Vanishing White Matter	Amsterdam UMC	Guanabenz, open label, Monocenter	Amsterdam Leukodystrophy Center	Contact
Alexander disease	Ionis	Intrathecal antisense oligonucleotide, Multicenter	Amsterdam Leukodystrophy Center	Contact
Pelizaeus-Merzbacher disease	Amsterdam UMC	Deferiprone, open label, Monocenter	Amsterdam Leukodystrophy Center	Contact	Not yet started
Krabbe disease	PassageBio	intrathecal gene therapy (PBKR03), Multicenter	Amsterdam Leukodystrophy Center	Contact
Adrenoleukodystrophy	Minoryx	MIN-102, Multicenter	Kremlin Bicêtre, France	Contact
Metachromatic leukodystrophy	Takeda/Shire	intrathecal ERT (SHP611), Multicenter	Kremlin Bicêtre, France	Contact	Closed for inclusions
Metachromatic Leukodystrophy	Takeda/Shire	intrathecal ERT (SHP611), Multicenter	University Children's Hospital Tübingen	Contact
Metachromatic Leukodystrophy	Takeda/Shire	intrathecal ERT (SHP611), Multicenter	Amsterdam Leukodystrophy Center	Contact	Closed for inclusions
Adrenoleukodystrophy	SwanBio	AAV-9 gene therapy to stop progression of myelopathy, Multicenter	Amsterdam Leukodystrophy Center	Contact
Adrenoleukodystrophy	MGH	Pramipexole, Multicenter	Amsterdam Leukodystrophy Center	Contact
Adrenoleukodystrophy	Poxel	PXL770, Multicenter	Amsterdam Leukodystrophy Center	Contact	Not yet started