Neuronal Ceroid Lipofuscinoses (NCL) Clinical Trials

A listing of Neuronal Ceroid Lipofuscinoses (NCL) 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 14 clinical trials
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 …

seizure
cortical atrophy
sandhoff disease
umbilical cord blood transplantation
metabolic diseases
  • 153 views
  • 28 Jan, 2021
  • 1 location
Using NPT to Evaluate Providing PPC as ELNEC-PPC WBT for Nurses

The purpose of this study is to explain the provision of palliative care at the end of life by the implementation of the ELNEC course, as WBT Program using the Normalization Process Theory, that focus attention on how complex interventions become routinely embedded in practice. In addition to, identify the …

maternity
  • 0 views
  • 25 Jan, 2021
  • 1 location
Inherited Retinal Degenerative Disease Registry

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 www.MyRetinaTracker.org. Affected individuals who register are guided to …

degenerative disease
other disease
genetic analysis
blindness
genetic testing
  • 426 views
  • 10 May, 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
blood tests
heart disease
cognitive impairment
anemia
  • 572 views
  • 11 Jul, 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.

cell transplantation
stem cell transplantation
palliative care
genetic disorders
neurodegenerative disorders
  • 359 views
  • 26 Jan, 2021
  • 1 location
Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

Neuronal Ceroid Lipofuscinoses (NCL) or Batten's disease are the most common juvenile neurodegenerative disease, characterized by early blindness, movement disorders, cognitive and behavioral

  • 0 views
  • 27 May, 2021
  • 1 location
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis

Background CLN3, or Batten disease, is a genetic disorder. This deadly disease leads to decline of brain and nervous system functions. Symptoms of CLN3 typically occur between 4 and 7 years of age. They include changes in how a person sees, thinks, and moves. CLN3 can also cause seizures. No …

seizure
ceroid lipofuscinosis
genetic disorder
batten
  • 93 views
  • 16 Jun, 2021
  • 1 location
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501.

gene therapy
cln6
ceroid lipofuscinosis
batten
  • 46 views
  • 22 Apr, 2021
  • 1 location
Natural History of Neuronal Ceroid Lipofuscinosis Batten's CLN6 Diseae

CLN6 is a rare, neurodegenerative disease that causes progressive loss of acquired skills with motor delay, visual loss, seizures and ataxia. The investigators propose a natural history study of this rare disorder since it is currently unknown. It is important to understand disease progression in CLN6 disease to be able …

seizure
visual loss
gene therapy
batten
ceroid lipofuscinosis
  • 57 views
  • 29 Jan, 2021
  • 1 location
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

The investigators propose a study to assess cognitive and developmental outcomes of patients with CLN2 that are untreated and receiving cerliponase alfa. This study aims to validate standardized assessment measures to establish a standard of care. The secondary aim is to compare cognitive and developmental outcomes of patients with CLN2 …

  • 0 views
  • 23 Jan, 2021
  • 1 location