Studies and Trials – Leukodystrophies – ERN-RND | European Reference Network on Rare Neurological Diseases

Ongoing

List of all Trials and Studies to which ERN-RND expert centers contribute
Type of study	Acronym	Title	NCT ID	Other study IDs
Clnical trial	Orbit Study	Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)	NCT06150716
Clnical trial		A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)	NCT04849741
Clnical trial	Rocket Study	Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease	NCT05659901
Clnical trial	CALICO	An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease	NCT05757141	M23-523; EU CT: 2023-505704-30-00
Observational study	MLDinitiative	MLDinitiative - European disease registry for metachromatic leukodystrophy
Observational study		A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease	NCT05659901

A Study of VGL101 in Patients with Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (NCT05677659)
EMBOLDEN – A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy (NCT03771898)