Showing 16 of 16 recruiting trials for “metachromatic-leukodystrophy-adult-form”
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
Direct Lentiviral Injection Gene Therapy for MLD
👨⚕️ Lung-Ji Chang, Ph.D, Shenzhen Geno-Immune Medical Institute📍 1 site📅 Started May 2025View details ↗
Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
RecruitingNCT06190834 ↗
Relationship Between High-Density Lipoprotein Subtypes and Coronary Heart Disease Prognosis.
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
An Intervention to Increase Genetic Testing in Families Who May Share a Gene Mutation Related to Cancer Risk and An Intervention to Help Patients and Their Primary Care Providers Stay Up-to-date About Uncertain Genetic Test Results
👨⚕️ Kenneth Offit, MD, MPH, Memorial Sloan Kettering Cancer Center📍 8 sites📅 Started Dec 2022View details ↗
RecruitingNCT05103969 ↗
Cohort of Tumors With POLE/D1 Mutation
👨⚕️ Rosine GUIMBAUD, PhD.MD, Fédération francophone de Cancérologie digestive📍 1 site📅 Started Oct 2021View details ↗
RecruitingNCT04925349 ↗
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
👨⚕️ Fanny MOCHEL, MCU-PH, Institut du Cerveau et de la Moëlle épinière📍 2 sites📅 Started Aug 2021View details ↗
Enrolling by InvitationNCT04503213 ↗
A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation
Pembrolizumab in Treating Participants With Metastatic, Recurrent or Locally Advanced Cancer and Genomic Instability
👨⚕️ Eugenia Girda, MD, Rutgers Cancer Institute of New Jersey📍 2 sites📅 Started Mar 2018View details ↗
The Myelin Disorders Biorepository Project
👨⚕️ Adeline Vanderver, MD, Children's Hospital of Philadelphia📍 20 sites📅 Started Dec 2016View details ↗
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
RecruitingNCT03333200 ↗
Longitudinal Study of Neurodegenerative Disorders
RecruitingNCT03639285 ↗
Natural History, Diagnosis, and Outcomes for Leukodystrophies
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