AMNCYGNETNCT05008874
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
An observational study of AMN and AMN Gene Mutation, run by SwanBio Therapeutics, Inc. at 6 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- AMN, AMN Gene Mutation, X-ALD
- What is being tested?
- Natural History Observation
- How many people
- 65 participants
- Where
- 6 sitesin 3 countries
- Study dates
- 21 Jun 2021to 16 May 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Adult males diagnosed with ALD (without cerebral disease) and symptoms of AMN who have no other major confounding comorbidities.
Who may be able to join
- Male adults aged ≥18 years
- Diagnosed with ALD based on elevated VLCFA assay and pedigree analysis
- Clinical evidence of spinal cord involvement with EDSS score between 1 and 6.5
Who cannot join
- Diagnosed with cerebral inflammatory disease or has a history of diagnosis with cerebral inflammatory disease
- 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
- 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
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
United States4 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Weill Medical College of Cornell University
New York, New York, 10065
University of Utah
Salt Lake City, Utah, 84112
Stanford Neuroscience Health Center
Stanford, California, 94304
About this study
Official title: Prospective, Retrospective, Multicenter, Observational Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
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.
Read the detailed description
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.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Being tested
- Natural History Observation
- Keywords
- AMN, X-ALD, Natural History, Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Adrenomyeloneuropathy, Myeloneuropathy, Spastic paraplegia, Hereditary Spastic Paraplegia, HSP, ALD, ABCD1, ALDP, CALD, CCALD, Brain Diseases, Metabolic, Inborn, Brain Diseases, Metabolic, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Demyelinating Diseases
- Medical subject headings
- Adrenoleukodystrophy, Paraplegia, Spastic Paraplegia, Hereditary, Brain Diseases, Metabolic, Inborn, Brain Diseases, Metabolic, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Demyelinating Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Natural History ObservationOther
Data collection on progression of disease
| Study group | Role | What they receive |
|---|---|---|
| Males with AMN | Adult males with confirmed diagnosis of ALD and symptoms of AMN. Receives: Other: Natural History Observation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease progression
Characterize disease progression in adults diagnosed with AMN in serial clinical evaluations of walking
Measured: 5 years
Secondary outcomes (1)
Change in Quality of Life
Characterize the Change in multiple Quality of Life (QoL) parameters over time
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2021
- Study start21 Jun 2021
- Primary completion16 May 2025
- Study completion16 May 2025
Why it stopped: The AMN program requires significantly more time, patients, and resources, including the manufacture of additional investigational product, than what is currently available to Spur.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AMNStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- SwanBio Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05008874
- Sponsor's protocol ID
- SBTNHX-CT901
- Acronym
- CYGNET
- Status last verified
- Jun 2025
- First submitted
- 12 Aug 2021
- Last updated
- 30 Oct 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Retrospective, Multicenter, Observational Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
- ClinicalTrials.gov ID
- NCT05008874
- Acronym / sponsor ID
- CYGNET / SBTNHX-CT901
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Keywords
- AMN, X-ALD, Natural History, Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Adrenomyeloneuropathy, Myeloneuropathy, Spastic paraplegia
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: The AMN program requires significantly more time, patients, and resources, including the manufacture of additional investigational product, than what is currently available to Spur.
- Enrolment
- 65 participants (actual)
- Lead sponsor
- SwanBio Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Jun 2021 (actual)
- Primary completion
- 16 May 2025 (actual)
- Study completion
- 16 May 2025 (actual)
- First posted
- 17 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Males with AMN | – | Other: Natural History ObservationAdult males with confirmed diagnosis of ALD and symptoms of AMN. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult males diagnosed with ALD (without cerebral disease) and symptoms of AMN who have no other major confounding comorbidities.
Key inclusion criteria (3 listed)
- Male adults aged ≥18 years
- Diagnosed with ALD based on elevated VLCFA assay and pedigree analysis
- Clinical evidence of spinal cord involvement with EDSS score between 1 and 6.5
Key exclusion criteria (3 listed)
- Diagnosed with cerebral inflammatory disease or has a history of diagnosis with cerebral inflammatory disease
- 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
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Natural History ObservationData collection on progression of disease | Other | Males with AMN |
Primary outcome
Disease progression
Time frame: 5 years
Characterize disease progression in adults diagnosed with AMN in serial clinical evaluations of walking
1 secondary outcome
Change in Quality of Life
Time frame: 5 years
Results and safety
The study was stopped early (reason given: The AMN program requires significantly more time, patients, and resources, including the manufacture of additional investigational product, than what is currently available to Spur.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AMN and AMN Gene Mutation, run by SwanBio Therapeutics, Inc. at 6 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Germany, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: The AMN program requires significantly more time, patients, and resources, including the manufacture of additional investigational product, than what is currently available to Spur.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease progression (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2025.