RecruitingNCT04880356

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Clinical, Instrumental and Laboratory Data Collection of Subjects with Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases

Sponsor

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Enrollment

100 participants

Start Date

Mar 1, 2021

Study Type

OBSERVATIONAL

Conditions

Metabolic Disease Neurologic Disorder Rare Diseases Neuro-Degenerative Disease Inherited Disease Undiagnosed Disease

Summary

General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less than 5:100,000) in adulthood through the systematic longitudinal collection of clinical, laboratory and instrumental data.

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This is a long-term observational study that tracks patients with ultra-rare inherited neurological diseases — conditions affecting the nervous system that are passed down through families. Because these diseases are so rare, this study aims to build a detailed picture of how they progress over time to support future treatment development. **You may be eligible if...** - You are 18 or older - You have been diagnosed with a rare inherited degenerative or metabolic neurological disease - You have an undiagnosed neurological condition believed to be inherited **You may NOT be eligible if...** - (There are no exclusion criteria for this study) Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERcollection of data

collection of retrospective and prospective data from adult patients with ultra-rare inherited neurological diseases

Locations(1)

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Milano, Italy

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NCT04880356

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