The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

Title

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

Lead sponsor

Children's Hospital of Philadelphia

Phase

Observational

Overall status

Recruiting

Start date

December 8, 2016

Completion date

December 8, 2030

Enrollment

12,000 (estimated)

Sites

23

4h syndrome, adld, adrenoleukodystrophy, adrenomyeloneuropathy, ags, aicardi goutieres syndrome, ald, ald (adrenoleukodystrophy), ald gene mutation, alexander disease, alexanders leukodystrophy, allan-herndon-dudley syndrome, alsp, amn, axd, bpan, cadasil, canavan disease, cerebrotendinous xanthomatoses, charcot-marie-tooth, cmt, cockayne syndrome, csf1r gene mutation, ctx, galc deficiency, gangliosidoses, globoid leukodystrophy, gm2 gangliosidosis, h-abc - hypomyelination, atrophy of basal ganglia and cerebellum, hbsl, hbsl - hypomyelination, brain stem, spinal cord, leg spasticity, hcc - hypomyelination and congenital cataract, krabbe disease, labrune syndrome, lbsl, lcc, leukodystrophy, leukoencephalopathies, leukoencephalopathy with brain stem and spinal cord involvement and high lactate syndrome (disorder), leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation, mct8 (slc16a2)-specific thyroid hormone cell transporter deficiency, megalencephalic leukoencephalopathy with subcortical cysts, metachromatic leukodystrophy, mlc1, mld, mucopolysaccharidoses, multiple sulfatase deficiency, pelizaeus merzbacher like disease, pelizaeus-merzbacher disease, peroxisomal biogenesis disorder, plp1 gene duplication | blood or tissue | mutations, plp1 null syndrome, pmd, refsum disease, salla disease, sialic storage disease, sjogren-larsson syndrome, sjögren, tbck-related intellectual disability syndrome, tubb4a-related leukodystrophy, van der knapp disease, vanishing white matter disease, white matter disease, x-ald, x-linked adrenoleukodystrophy, zellweger syndrome