Scientists launch study to unravel RNA's role in rare brain diseases

NCT ID NCT04394871

Recruiting now Knowledge-focused Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions, and healthy volunteers. Participants undergo tests like MRI, blood draws, and muscle strength assessments over up to 3 years, but no experimental treatment is given.

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Conditions

The condition(s) this trial relates to.

Aicardi-Goutieres syndrome amyotrophic lateral sclerosis type 4 progressive external ophthalmoplegia with mitochondrial DNA deletions, autosomal dominant 2 spinocerebellar ataxia, autosomal recessive, with axonal neuropathy 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AMYOTROPHIC LATERAL SCLEROSIS TYPE 4 INHERITED NEUROLOGICAL DISORDERS OF RNA PROCESSING

Contacts and locations

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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