Browse Studies — Arctuva

Phase 1

Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard …

AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more

Estimated

~$10 – $7,500

Joanne Kurtzberg, MDNCT02254863

Adrenoleukodystrophy National Registry Study

The aim of this registry to understand the natural history and disease progression in ALD and potentially develop bio-markers using the biospecimens collected …

ALD (Adrenoleukodystrophy)AdrenoleukodystrophyCerebral Adrenoleukodystrophy

Estimated

~$10 – $750

Masonic Cancer Center, University of MinnesotaNCT03789721

A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)

The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in…

Cerebral Adrenoleukodystrophy (CALD)

Estimated

~$10 – $1,050

Genetix Biotherapeutics Inc.NCT06224413

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

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…

LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more