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Found 13 clinical trials
Gene Therapy Study for Children With CLN5 Batten Disease (CLN5-200)

This is a prospective, non-randomized, open-label study of a single dose administration of gene therapy in children who are 3 to 8 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype

  • 10 Mar, 2022
  • 1 location
Study for the Treatment for CLN7 Disease

This is a phase 1 open-label, single-administration of gene therapy agent AAV9/CLN7, administered intrathecally into the lumbar spinal cord region of pediatric patients with CLN7 Batten disease

  • 24 May, 2021
  • 1 location
An Open-label Safety, Pharmacokinetic, and Efficacy Study of the Combination of Trehalose and Miglustat for the Treatment of CLN3 Disease

This is an open label study in approximately 6 subjects in 2 centers to assess the safety, PK, and efficacy of the maximum tolerable dose (MTD) of intravenous (IV) trehalose (0.25, 0.50, or 0.75 g/kg administered once-weekly) and the MTD of oral miglustat (100 mg once daily [QD] to 200 …

  • 23 Mar, 2022
  • 1 location
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical …

tay sachs disease
niemann pick disease
inborn errors of metabolism
mutation analysis
  • 23 Dec, 2021
  • 1 location
Inherited Retinal Degenerative Disease Registry (MRTR)

The My Retina Tracker Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation. It is a patient-initiated registry accessible via a secure on-line portal at Affected individuals who register are guided to …

degenerative disease
genetic testing
Accepts healthy volunteers
other disease
  • 25 Jan, 2022
  • 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 disorders
cell transplantation
stem cell transplantation
neurodegenerative disorders
  • 13 Nov, 2021
  • 1 location
Genetic Characterization of Movement Disorders and Dementias

Background There are two basic types of movement disorders. Some cause excessive movement, some cause slowness or lack of movement. Some of these are caused by mutations in genes. On the other hand, dementia is a condition of declining mental abilities, especially memory. Dementia can occur at any age but …

movement disorder
cognitive impairment
Accepts healthy volunteers
  • 16 May, 2022
  • 1 location
Caregiving Networks Across Disease Context and the Life Course

Background In the U.S., about 53 million informal, unpaid caregivers provide care to a person who is ill, is disabled, or has age-related loss of function. These caregivers may be adult children, spouses, parents, or others. The stress of providing long-term care affects caregivers health and well-being. Researchers want to …

Accepts healthy volunteers
  • 14 May, 2022
  • 1 location
Clinical and Neuropsychological Investigations in Batten Disease

This study aims to assess the natural history of Batten disease (Neuronal Ceroid Lipofuscinosis) by obtaining information about the motor, behavioral, and functional capabilities of individuals

ceroid lipofuscinosis
  • 17 May, 2022
  • 1 location
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease the International DEM-CHILD Database

This is an observational study that aims at assessing the natural history of NCL diseases as part of the international DEM-CHILD Database. Patient data are collected from medical records, patient questionnaires and routine follow up clinical examinations with focus on assessing progression in key areas of disease such as motor, …

  • 03 Nov, 2021
  • 1 location