Interventional trials in ERN-RND

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