Metachromatic Leukodystrophy - MLD Clinical Trials

A listing of Metachromatic Leukodystrophy - MLD medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 5 clinical trials
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination …

  • 90 views
  • 28 Feb, 2022
  • 1 location
Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

Evaluating the safety and efficacy of Lentiviral Hematopoietic Stem Cell Gene Therapy for advanced stage of Metachromatic Leukodystrophy and adrenoleukodystrophy.

abcd1
fatty acids
gene therapy
ACTH
metachromatic leukodystrophy
  • 133 views
  • 19 Jun, 2022
  • 1 location
Manual Lymphatic Drainage Before and After Total Knee Replacement a Single-center Observer-blinded Randomized Controlled Trial

The aim of this observer-blinded randomized controlled trial is the evaluation of the influence of manual lymphatic drainage (MLD) on the outcome of patients receiving total knee replacement (TKA).

joint reconstruction
total knee replacement
  • 0 views
  • 24 Nov, 2021
  • 1 location
Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

palliative care
genetic disorder
neurodegenerative disorders
genetic disorders
cell transplantation
  • 363 views
  • 13 Nov, 2021
  • 1 location
MATRIX - "Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia" (MATRIX)

), juvenile/adult metachromatic leukodystrophy (MLD) and adults with leukoencephalopathy and axonal spheroids and pigmented glia (ALSP). 49 subjects will be enrolled with one blood sample collection during

Accepts healthy volunteers
  • 0 views
  • 27 Oct, 2022
  • 1 location