Rare disease mystery: scientists watch AMN progress in hopes of finding a cure

NCT ID NCT05008874

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study followed 65 adult men with a rare inherited nerve disease called AMN (a form of spastic paraplegia) to understand how their symptoms change over time. Researchers collected data on walking ability and quality of life. The goal was to fill gaps in knowledge about the disease's progression, which could help design better treatments in the future. However, the study was stopped early, so results may be limited.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to

If successful, this study could provide crucial data to help design future treatments for AMN, a rare and poorly understood disease.

What could go wrong

This is an observational study, not a treatment trial. It was terminated early, so the data may be incomplete and not lead to clear conclusions.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

adrenocortical insufficiency adrenoleukodystrophy adrenomyeloneuropathy Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn demyelinating disease Hereditary Central Nervous System Demyelinating Diseases hereditary spastic paraplegia inborn errors of metabolism inherited neurodegenerative disorder Leukoencephalopathies metabolic disease paraplegia Peroxisomal Disorders

As listed by the trial registrant

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

Contacts and locations

Locations

  • Amsterdam UMC

    Amsterdam, Netherlands

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Stanford Neuroscience Health Center

    Stanford, California, 94304, United States

  • University of Leipzig Medical Center

    Leipzig, Germany

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • Weill Medical College of Cornell University

    New York, New York, 10065, United States

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