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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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65 people
The number who actually took part.
- Started
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Jun 2021
- Finished
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May 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Adult males diagnosed with ALD (without cerebral disease) and symptoms of AMN who have no other major confounding comorbidities.
- Ages
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18 years and older
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Male adults aged ≥18 years 2. Diagnosed with ALD based on elevated VLCFA assay and pedigree analysis 3. Clinical evidence of spinal cord involvement with EDSS score between 1 and 6.5 Exclusion Criteria: 1. Diagnosed with cerebral inflammatory disease or has a history of diagnosis with cerebral inflammatory disease 2. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, or endocrine disease (other than adrenal insufficiency) or other abnormality, which may impact the ability to participate in the study or that may potentially confound the study results 3. Participant who, in the opinion of the Investigator, has any other medical or psychological condition or social circumstances which would impair their ability to participate reliably in the assessments, or who may increase the risk to themselves or others by participating
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Amsterdam UMC
Amsterdam, Netherlands
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Stanford Neuroscience Health Center
Stanford, California, 94304, United States
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University of Leipzig Medical Center
Leipzig, Germany
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University of Utah
Salt Lake City, Utah, 84112, United States
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Weill Medical College of Cornell University
New York, New York, 10065, United States
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