An observational study in AMN, AMN Gene Mutation and X-ALD, sponsored by SwanBio Therapeutics, Inc.. Terminated at 6 sites in 3 countries. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-10-30.
Sponsored by SwanBio Therapeutics, Inc. · Observational
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments. This study will help obtain a better understanding of the progression of disease with AMN and facilitate efficient clinical development of future interventional medications.
Progressive weakness and spasticity of the legs are characteristics of numerous disorders and conditions, including those that are inherited neurological disorders.
Adrenomyeloneuropathy (AMN) is an example of an inherited form of spastic paraplegia.
Adrenoleukodystrophy (ALD) is a progressive neurodegenerative disorder caused by a mutation in the ABCD1 gene localized to the X-chromosome (Xq28). The ABCD1 gene encodes a peroxisomal adenosine triphosphate (ATP) binding cassette transporter responsible for transport of very long chain fatty acids (VLCFA) from the cytosol into the peroxisome for degradation. A mutation in ABCD1 results in reduction in the degradation of the VLCFA by peroxisomal β-oxidation, and saturated VLCFA, in particular C26:0, accumulate in tissues and body fluids (i.e., brain, nervous system, adrenal glands). One of the key clinical symptoms during aging of ALD patients is a slowly progressive axonopathy affecting sensory ascending and motor descending spinal cord tracts with 100% penetrance in men, an ALD phenotype known as AMN. There are no treatment options available, which leaves AMN patients with a progressive disorder that leads to lifelong physical disability. The progressive dying-back axonopathy represents the core clinical feature of AMN, with onset usually between 20 and 30 years of age in male participants. The initial symptoms include progressive stiffness and weakness of the legs, impaired vibration and position senses in the lower limbs, falls, sphincter disturbances and impotence, as well as scarce scalp hair (alopecia). About 66% of male AMN patients have adrenocortical insufficiency (Addison disease).
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments. This study will help obtain a better understanding of the progression of disease with AMN and facilitate efficient clinical development of future SwanBio interventional medications.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 65 is below the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →SwanBio Therapeutics, Inc. is the lead sponsor of 2 studies on the registry; none are open to participants now.
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Adult males diagnosed with ALD (without cerebral disease) and symptoms of AMN who have no other major confounding comorbidities.
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Exclusion Criteria:
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Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Adult males with confirmed diagnosis of ALD and symptoms of AMN.
Other: Natural History Observation
Data collection on progression of disease
Disease progression
Characterize disease progression in adults diagnosed with AMN in serial clinical evaluations of walking
Time frame: 5 years
Change in Quality of Life
Characterize the Change in multiple Quality of Life (QoL) parameters over time
Time frame: 5 years
Plan to share: Undecided
No publications or documents are linked to this record.
Ran from Jun 2021 to primary completion in May 2025, 46 months.
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This study is terminated, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.
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SwanBio Therapeutics, Inc.