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Batten Disease Clinical Trials

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

RESULTS

Found (15) clinical trials

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

The inherited metabolic disorders (IMD) are a heterogeneous group of genetic diseases, most of which involve a single gene mutation resulting in an enzyme defect. In the majority of cases, the enzyme defect leads to the accumulation of substrates that are toxic and/or interfere with normal cellular function. Often times, ...

Phase

Batten CLN6 Gene Therapy

The proposed clinical trial is the first human, open-label, single dose study of self-complementary AAV9 carrying the CLN6 gene under the control of a hybrid CMV enhancer/chicken--actin promoter (scAAV9.CB.CLN6) delivered one-time through an intrathecal catheter inserted by a lumbar puncture into the interspinous into the subarachnoid space of the lumbar ...

Phase

Longitudinal Study of Neurodegenerative Disorders

Patients would be evaluated by a multidisciplinary team at specific time points every 3 months the first year, every 6 months the second year and once a year thereafter.

Phase N/A

Clinical and Neuropsychological Investigations in Batten Disease

Batten Disease is an inherited disorder that causes progressive cognitive and behavioral decline in children. There have been no systematic clinical studies of Batten Disease using standardized rating instruments with known inter-rater reliability and validity. The Batten Study Group developed the Unified Batten Disease Rating Scale (UBDRS), a clinical rating ...

Phase N/A

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

The National Registry of DM and FSHD Patients and Family Members was developed to create a link between patients and scientists in order to promote research on these rare illnesses. The Registry is sponsored by the National Institutes of Health. Patients that are interested in joining the Registry can email ...

Phase N/A

Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy

Congenital Myotonic Dystrophy (CDM) is a multi-systemic, dominantly inherited disorder caused by a trinucleotide repeat expansion (CTGn) in the DMPK gene. CDM occurs when the CTGn increases between the adult myotonic dystrophy type-1 (DM1) parent and the child. Children with CDM present at birth with respiratory insufficiency, talipes equinovarus, feeding ...

Phase N/A

Myotonic Dystrophy Family Registry

The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) such as disease symptoms and demographic information to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials. The Registry supports trials and studies, making ...

Phase N/A

Natural History of Neuronal Ceroid Lipofuscinosis Batten's CLN6 Diseae

Neuronal Ceroid Lipofuscinosis (NCL) is the most common childhood neurodegenerative disorder characterized by accumulation of autofluorescent waxy lipopigments in the brain and other tissues. The symptoms manifest as blindness, seizures, ataxia, myoclonus and loss of milestones or dementia. This group of disorders caused by an intracellular accumulation of lipopigment (ceroid ...

Phase N/A

Sleep Breathing Disorders a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ?

Obstructive sleep apnea (OSA) and central sleep apnea (CSA), the most common form of sleep disordered breathing (SDB), are prevalent in patients with myotonic dystrophy type 1 (DM1). Among the serious complications from sleep apnea, the most alarming are cardiovascular, including arrhythmias and sudden cardiac death (SCD). Diagnosis of SDB ...

Phase N/A

Study of Ranolazine in Myotonia Congenita Paramyotonia Congenita and Myotonic Dystrophy Type 1

Recent advances in the understanding of myotonia congenita have identified potential areas that could possibly respond to treatment in a drug study. The drug ranolazine (trade name Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease. Ranolazine has been studied in mice with myotonia congenita. ...

Phase