Adrenomyeloneuropathy Without Cerebral InvolvementPhase 2/3NCT06513533
Dimethyl Fumarate in Adrenomyeloneuropathy
A Phase 2/3 study testing Dimethyl fumarate in people with Adrenomyeloneuropathy Without Cerebral Involvement, run by Pujol, Aurora, M.D. at 3 sites in Spain.
Trial summary
The goal of this clinical trial is to determine if dimethyl fumarate is effective in treating motor problems in adults with Adrenomyeloneuropathy. The trial will also assess the safety of dimethyl fumarate and explore the molecular mechanisms underlying the disease. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Adrenomyeloneuropathy Without Cerebral Involvement
- What is being tested?
- Dimethyl fumarate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 3 sitesSpain
- Study dates
- 23 Apr 2024to 30 Jun 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and women of 18 to 65 years old at the time of the inclusion, suffering from AMN with:
- elevated plasma VLCFA
- ABCD1 gene mutation identified
- Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk (EDSS score ≥ 2.0 and ≤ 6.5). EDSS score will also be re-evaluated at M12, M24 and M36.
- Normal brain MRI or brain MRI showing:
- abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4
Show all 12Show fewer
- and/or stable (≥ 6 months) cerebral demyelination without gadolinium enhancement with a Loes score ≤ 12
- Appropriate steroid replacement if adrenal insufficiency is present
- Potential childbearing women should use an adequate method of contraception to avoid pregnancy throughout the study to minimize the risk of pregnancy. If oral contraceptives are used, the use of an alternative barrier method is recommended.
- Likely to be able to participate in all scheduled evaluations and complete all required study procedures
- Signed and dated written informed consent to participate in the study in accordance with local regulations
- Not easily contactable by the investigator in case of emergency or not able to call the investigator
Who cannot join
- Any progressive neurological disease other than AMN
- Leukopenia below 3.0x109/L, lymphopenia below 0.5x109/L or other pathological results in the complete blood count
- Suspected or confirmed progressive multifocal leukoencephalopathy (PML)
- Severe gastrointestinal disease
- Uncontrolled hepatic, renal or cardiovascular disease, or any evolutive malignancy
- Pregnancy and breast-feeding in woman and potential childbearing woman unable or unwilling to use an acceptable contraceptive method during the study
- Any new medication for AMN initiated less than three months prior to inclusion
- Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body as aneurysm clips, pacemakers, intraocular metal or cochlear implants
Trial locations and contacts
3 sites in 1 country.
Spain3 sites
Donostia University Hospital
Donostia / San Sebastian, Donostia, 20014
Bellvitge University Hospital
L'Hospitalet de Llobregat, Barcelona, 08907
University Hospital 12 de Octubre
Madrid, Madrid, 28041
About this study
Official title: Effect of Dimethyl Fumarate Administered to Patients With Adrenomyeloneuropathy: a Multicenter, Placebo Controlled, Phase IIb/III Trial
The goal of this clinical trial is to determine if dimethyl fumarate is effective in treating motor problems in adults with Adrenomyeloneuropathy. The trial will also assess the safety of dimethyl fumarate and explore the molecular mechanisms underlying the disease. The primary questions it aims to answer are:
- Does dimethyl fumarate improve motor problems in participants?
- What medical issues do participants experience while taking dimethyl fumarate? Researchers will compare the effects of dimethyl fumarate to a placebo (a substance that looks like the drug but contains no active ingredients) to evaluate its effectiveness in treating Adrenomyeloneuropathy.
Read more
Participants will:
- Take either dimethyl fumarate or a placebo daily for 36 months.
- Visit the clinic at the start of the trial, then at 3 months, 6 months, and every 6 months thereafter for checkups and tests.
Read the detailed description
Adrenoleukodystrophy (X-ALD) is the most prevalent rare genetic disorder affecting the brain's white matter. It is caused by mutations in the ABCD1 gene, which encodes a transporter involved in the degradation of very long-chain fatty acids (VLCFA). As a result, VLCFA accumulate in tissues and plasma, serving as a pathognomonic biomarker for diagnosis. The disease manifests in two main forms: i) adrenomyeloneuropathy (AMN), characterized by chronic progressive spastic paraplegia due to distal axonopathy, and ii) cerebral ALD (cALD), a rapidly progressing and fatal demyelinating leukodystrophy. Current therapeutic options are inadequate, limited to bone marrow transplants and gene therapy for patients with cerebral inflammation. No treatment is available for AMN, which affects 60% of patients.
We have discovered that excess VLCFA leads to mitochondrial reactive oxygen species (ROS) production and oxidative damage, a major factor driving pathogenesis. More recently, we found that the main endogenous response to oxidative damage (the NRF-2 pathway) is impaired in X-ALD. Preclinical tests with an NRF2 activator, specifically the current treatment for multiple sclerosis, dimethyl fumarate (DMF/Tecfidera), showed promising results. All major molecular and cellular pathogenic mechanisms were restored, including: i) mitochondrial function and biogenesis, ii) redox homeostasis, iii) bioenergetic failure, iv) neuroinflammation, along with axonal damage and clinical signs of the disease such as locomotor disability. Consequently, we obtained an international patent for repurposing DMF for X-ALD (US15/957,601) and Orphan Drug Designation by the EMA in 2020 (EMA/OD/0000010028).
Now we are translating this knowledge into a randomized phase IIb/III double-blind placebo-controlled study over 36 months for 40 AMN patients, to determine if DMF is effective in these patients. For the first 24 months, patients will be divided into two groups (placebo and active treatment) in a ratio of 1:2. A 12-month extension phase will follow, during which all patients will receive treatment. Furthermore, we aim to elucidate the molecular mechanisms driving the disease and dissect the redox-inflammatory effects of DMF using an integrative multi-omics approach, which will involve single-cell RNA sequencing in PBMC, and lipidomics in plasma. The clinical and molecular data from historical national and international AMN and cALD cohorts will be pooled to identify markers of severity and progression. Our goal is to address unmet needs in AMN while generating novel fundamental knowledge that will be useful for this and other common axonopathies.
- Being tested
- Placebo, Dimethyl fumarate
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboOther
1 tablet twice daily (one in the morning and one in the evening) for the first 7 days as a starter dose followed by 2 tablets (240 mg) twice daily
Dimethyl fumarateDrug
Also called Skilarence, Tecfidera
1 tablet twice daily (one in the morning and one in the evening) for the first 7 days as a starter dose followed by 2 tablets (240 mg) twice daily
| Study group | Role | What they receive |
|---|---|---|
| DMF arm | Experimental | Oral administration of dimethyl fumarate, 480 mg/day, for 36 months Receives: Drug: Dimethyl fumarate |
| Placebo arm | Placebo comparator | Oral administration of placebo for 24 months, followed by oral dimethyl fumarate, 480 mg/day for 12 months Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Postural sway test
Balance is assessed with static posturography by measuring the extent of postural sway in four conditions, each for two 20-second trials: (1) eyes open feet shoulder width apart (EOFA), (2) eyes closed feet shoulder width apart (ECFA), (3) eyes open feet together (EOFT), and (4) eyes closed feet together (ECFT)
Measured: Interim analysis 1 (at 12 months of treatment)
Postural sway test
Balance is assessed with static posturography by measuring the extent of postural sway in four conditions, each for two 20-second trials: (1) eyes open feet shoulder width apart (EOFA), (2) eyes closed feet shoulder width apart (ECFA), (3) eyes open feet together (EOFT), and (4) eyes closed feet together (ECFT)
Measured: Final analysis 1 (at 24 months of treatment)
Postural sway test
Balance is assessed with static posturography by measuring the extent of postural sway in four conditions, each for two 20-second trials: (1) eyes open feet shoulder width apart (EOFA), (2) eyes closed feet shoulder width apart (ECFA), (3) eyes open feet together (EOFT), and (4) eyes closed feet together (ECFT)
Measured: Final analysis 2 (at 36 months of treatment)
Secondary outcomes (27)
2 Minute Walk Test (2MWT)
It measures the distance an individual is able to walk over a total of two minutes on a hard, flat surface.
Measured: Interim analysis 1 (at 6 months of treatment)
2 Minute Walk Test (2MWT)
It measures the distance an individual is able to walk over a total of two minutes on a hard, flat surface.
Measured: Interim analysis 2 (at 12 months of treatment)
2 Minute Walk Test (2MWT)
It measures the distance an individual is able to walk over a total of two minutes on a hard, flat surface.
Measured: Final analysis 1 (at 24 months of treatment)
2 Minute Walk Test (2MWT)
It measures the distance an individual is able to walk over a total of two minutes on a hard, flat surface.
Measured: Final analysis 2 (at 36 months of treatment)
6 Minute Walk Test (6MWT)
It measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface
Measured: Interim analysis 1 (at 6 months of treatment)
6 Minute Walk Test (6MWT)
It measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface
Measured: Interim analysis 2 (at 12 months of treatment)
6 Minute Walk Test (6MWT)
It measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface
Measured: Final analysis 1 (at 24 months of treatment)
6 Minute Walk Test (6MWT)
It measures the distance an individual is able to walk over a total of six minutes on a hard, flat surface
Measured: Final analysis 2 (at 36 months of treatment)
Time to walk 25 Feet (TW25)
The TW25 (time to walk 25 feet) is a widely used outcome measure of Leg function/ambulation
Measured: Interim analysis 1 (at 6 months of treatment)
Time to walk 25 Feet (TW25)
The TW25 (time to walk 25 feet) is a widely used outcome measure of Leg function/ambulation
Measured: Interim analysis 2 (at 12 months of treatment)
Time to walk 25 Feet (TW25)
The TW25 (time to walk 25 feet) is a widely used outcome measure of Leg function/ambulation
Measured: Final analysis 1 (at 24 months of treatment)
Time to walk 25 Feet (TW25)
The TW25 (time to walk 25 feet) is a widely used outcome measure of Leg function/ambulation
Measured: Final analysis 2 (at 36 months of treatment)
Stair-climbing
Participants are timed using a hand-held stopwatch as they ascended one flight of 4 steps as quickly and safely as possible with handrail use allowed as needed
Measured: Interim analysis 1 (at 12 months of treatment)
Stair-climbing
Participants are timed using a hand-held stopwatch as they ascended one flight of 4 steps as quickly and safely as possible with handrail use allowed as needed
Measured: Final analysis 1 (at 24 months of treatment)
Stair-climbing
Participants are timed using a hand-held stopwatch as they ascended one flight of 4 steps as quickly and safely as possible with handrail use allowed as needed
Measured: Final analysis 2 (at 36 months of treatment)
Strength
The average of two maximal effort break test trials for each side and muscle group is measured using a dynamometer and documented in Kg
Measured: Interim analysis 1 (at 12 months of treatment)
Strength
The average of two maximal effort break test trials for each side and muscle group is measured using a dynamometer and documented in Kg
Measured: Final analysis 1 (at 24 months of treatment)
Strength
The average of two maximal effort break test trials for each side and muscle group is measured using a dynamometer and documented in Kg
Measured: Final analysis 2 (at 36 months of treatment)
SF-Qualiveen (Short-Form Qualiveen)
The Qualiveen is a specific patients' health-related quality of life developed to assess the impact of urinary disorders in patients with neurological conditions.
Measured: Interim analysis 1 (at 12 months of treatment)
SF-Qualiveen (Short-Form Qualiveen)
The Qualiveen is a specific patients' health-related quality of life developed to assess the impact of urinary disorders in patients with neurological conditions.
Measured: Final analysis 1 (at 24 months of treatment)
SF-Qualiveen (Short-Form Qualiveen)
The Qualiveen is a specific patients' health-related quality of life developed to assess the impact of urinary disorders in patients with neurological conditions.
Measured: Final analysis 2 (at 36 months of treatment)
Revised Fecal Incontinence Scale (RFIS)
The RFIS is a short, reliable, and valid five item scale that can be used to assess fecal incontinence and to monitor patient outcomes following treatment.
Measured: Interim analysis 1 (at 12 months of treatment)
Revised Fecal Incontinence Scale (RFIS)
The RFIS is a short, reliable, and valid five item scale that can be used to assess fecal incontinence and to monitor patient outcomes following treatment.
Measured: Final analysis 1 (at 24 months of treatment)
Revised Fecal Incontinence Scale (RFIS)
The RFIS is a short, reliable, and valid five item scale that can be used to assess fecal incontinence and to monitor patient outcomes following treatment.
Measured: Final analysis 2 (at 36 months of treatment)
Brain neuroimaging: Loes
The Loes score is a 34-point imaging-based severity scale-based scoring system for patients with X-ALD based on the neuroanatomical involvement and the presence or absence of total and/or global atrophy. This 34-point imaging-based severity scale was based on the location and extent of central nervous system involvement and presence of either focal or global atrophy.
Measured: Interim analysis 1 (at 12 months of treatment)
Brain neuroimaging: Loes
The Loes score is a 34-point imaging-based severity scale-based scoring system for patients with X-ALD based on the neuroanatomical involvement and the presence or absence of total and/or global atrophy. This 34-point imaging-based severity scale was based on the location and extent of central nervous system involvement and presence of either focal or global atrophy.
Measured: Final analysis 1 (at 24 months of treatment)
Brain neuroimaging: Loes
The Loes score is a 34-point imaging-based severity scale-based scoring system for patients with X-ALD based on the neuroanatomical involvement and the presence or absence of total and/or global atrophy. This 34-point imaging-based severity scale was based on the location and extent of central nervous system involvement and presence of either focal or global atrophy.
Measured: Final analysis 2 (at 36 months of treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2024
- Study start23 Apr 2024
- Primary completion31 Mar 2028 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adrenomyeloneuropathy Without Cerebral InvolvementStudies 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
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double-arm, randomized, placebo-controlled, multicentric trial
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pujol, Aurora, M.D.
- Collaborators
- Instituto de Salud Carlos III, Spanish Clinical Research Network - SCReN, Institut d'Investigació Biomèdica de Bellvitge
- Responsible party
- Sponsor
- Investigators
- Carlos Casasnovas, MD, PhD, Principal investigator, Hospital Universitari de Bellvitge
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06513533
- Sponsor's protocol ID
- XAMNDMFAP2022
- Other IDs
- 2021-003826-65
- Status last verified
- May 2026
- First submitted
- 17 Jul 2024
- Last updated
- 17 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Dimethyl Fumarate Administered to Patients With Adrenomyeloneuropathy: a Multicenter, Placebo Controlled, Phase IIb/III Trial
- ClinicalTrials.gov ID
- NCT06513533
- Acronym / sponsor ID
- XAMNDMFAP2022
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Pujol, Aurora, M.D.
- Collaborators
- Instituto de Salud Carlos III, Spanish Clinical Research Network - SCReN, Institut d'Investigació Biomèdica de Bellvitge
- Study start
- 23 Apr 2024 (actual)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 22 Jul 2024 (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 goal of this clinical trial is to determine if dimethyl fumarate is effective in treating motor problems in adults with Adrenomyeloneuropathy. The trial will also assess the safety of dimethyl fumarate and explore the molecular mechanisms underlying the disease. The primary questions it aims to answer are:
- Does dimethyl fumarate improve motor problems in participants?
- What medical issues do participants experience while taking dimethyl fumarate? Researchers will compare the effects of dimethyl fumarate to a placebo (a substance that looks like the drug but contains no active ingredients) to evaluate its effectiveness in treating Adrenomyeloneuropathy.
Participants will:
- Take either dimethyl fumarate or a placebo daily for 36 months.
- Visit the clinic at the start of the trial, then at 3 months, 6 months, and every 6 months thereafter for checkups and tests.
Detailed description
Adrenoleukodystrophy (X-ALD) is the most prevalent rare genetic disorder affecting the brain's white matter. It is caused by mutations in the ABCD1 gene, which encodes a transporter involved in the degradation of very long-chain fatty acids (VLCFA). As a result, VLCFA accumulate in tissues and plasma, serving as a pathognomonic biomarker for diagnosis. The disease manifests in two main forms: i) adrenomyeloneuropathy (AMN), characterized by chronic progressive spastic paraplegia due to distal axonopathy, and ii) cerebral ALD (cALD), a rapidly progressing and fatal demyelinating leukodystrophy. Current therapeutic options are inadequate, limited to bone marrow transplants and gene therapy for patients with cerebral inflammation. No treatment is available for AMN, which affects 60% of patients.
We have discovered that excess VLCFA leads to mitochondrial reactive oxygen species (ROS) production and oxidative damage, a major factor driving pathogenesis. More recently, we found that the main endogenous response to oxidative damage (the NRF-2 pathway) is impaired in X-ALD. Preclinical tests with an NRF2 activator, specifically the current treatment for multiple sclerosis, dimethyl fumarate (DMF/Tecfidera), showed promising results. All major molecular and cellular pathogenic mechanisms were restored, including: i) mitochondrial function and biogenesis, ii) redox homeostasis, iii) bioenergetic failure, iv) neuroinflammation, along with axonal damage and clinical signs of the disease such as locomotor disability. Consequently, we obtained an international patent for repurposing DMF for X-ALD (US15/957,601) and Orphan Drug Designation by the EMA in 2020 (EMA/OD/0000010028).
Now we are translating this knowledge into a randomized phase IIb/III double-blind placebo-controlled study over 36 months for 40 AMN patients, to determine if DMF is effective in these patients. For the first 24 months, patients will be divided into two groups (placebo and active treatment) in a ratio of 1:2. A 12-month extension phase will follow, during which all patients will receive treatment. Furthermore, we aim to elucidate the molecular mechanisms driving the disease and dissect the redox-inflammatory effects of DMF using an integrative multi-omics approach, which will involve single-cell RNA sequencing in PBMC, and lipidomics in plasma. The clinical and molecular data from historical national and international AMN and cALD cohorts will be pooled to identify markers of severity and progression. Our goal is to address unmet needs in AMN while generating novel fundamental knowledge that will be useful for this and other common axonopathies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- Double-arm, randomized, placebo-controlled, multicentric trial
| Arm | Type | Receives |
|---|---|---|
| DMF arm | Experimental | Drug: Dimethyl fumarateOral administration of dimethyl fumarate, 480 mg/day, for 36 months |
| Placebo arm | Placebo comparator | Other: PlaceboOral administration of placebo for 24 months, followed by oral dimethyl fumarate, 480 mg/day for 12 months |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Men and women of 18 to 65 years old at the time of the inclusion, suffering from AMN with:
- Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk (EDSS score ≥ 2.0 and ≤ 6.5). EDSS score will also be re-evaluated at M12, M24 and M36.
- Normal brain MRI or brain MRI showing:
- Appropriate steroid replacement if adrenal insufficiency is present
- Potential childbearing women should use an adequate method of contraception to avoid pregnancy throughout the study to minimize the risk of pregnancy. If oral contraceptives are used, the use of an alternative barrier method is recommended.
Key exclusion criteria (8 listed)
- Any progressive neurological disease other than AMN
- Leukopenia below 3.0x109/L, lymphopenia below 0.5x109/L or other pathological results in the complete blood count
- Suspected or confirmed progressive multifocal leukoencephalopathy (PML)
- Severe gastrointestinal disease
- Uncontrolled hepatic, renal or cardiovascular disease, or any evolutive malignancy
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 |
|---|---|---|
| Placebo1 tablet twice daily (one in the morning and one in the evening) for the first 7 days as a starter dose followed by 2 tablets (240 mg) twice daily | Other | Placebo arm |
| Dimethyl fumarateAlso: Skilarence, Tecfidera1 tablet twice daily (one in the morning and one in the evening) for the first 7 days as a starter dose followed by 2 tablets (240 mg) twice daily | Drug | DMF arm |
Primary outcome
Postural sway test
Time frame: Interim analysis 1 (at 12 months of treatment)
Balance is assessed with static posturography by measuring the extent of postural sway in four conditions, each for two 20-second trials: (1) eyes open feet shoulder width apart (EOFA), (2) eyes closed feet shoulder width apart (ECFA), (3) eyes open feet together (EOFT), and (4) eyes closed feet together (ECFT)
Postural sway test
Time frame: Final analysis 1 (at 24 months of treatment)
Balance is assessed with static posturography by measuring the extent of postural sway in four conditions, each for two 20-second trials: (1) eyes open feet shoulder width apart (EOFA), (2) eyes closed feet shoulder width apart (ECFA), (3) eyes open feet together (EOFT), and (4) eyes closed feet together (ECFT)
Postural sway test
Time frame: Final analysis 2 (at 36 months of treatment)
Balance is assessed with static posturography by measuring the extent of postural sway in four conditions, each for two 20-second trials: (1) eyes open feet shoulder width apart (EOFA), (2) eyes closed feet shoulder width apart (ECFA), (3) eyes open feet together (EOFT), and (4) eyes closed feet together (ECFT)
27 secondary outcomes
2 Minute Walk Test (2MWT)
Time frame: Interim analysis 1 (at 6 months of treatment)
2 Minute Walk Test (2MWT)
Time frame: Interim analysis 2 (at 12 months of treatment)
2 Minute Walk Test (2MWT)
Time frame: Final analysis 1 (at 24 months of treatment)
2 Minute Walk Test (2MWT)
Time frame: Final analysis 2 (at 36 months of treatment)
6 Minute Walk Test (6MWT)
Time frame: Interim analysis 1 (at 6 months of treatment)
6 Minute Walk Test (6MWT)
Time frame: Interim analysis 2 (at 12 months of treatment)
6 Minute Walk Test (6MWT)
Time frame: Final analysis 1 (at 24 months of treatment)
6 Minute Walk Test (6MWT)
Time frame: Final analysis 2 (at 36 months of treatment)
Time to walk 25 Feet (TW25)
Time frame: Interim analysis 1 (at 6 months of treatment)
Time to walk 25 Feet (TW25)
Time frame: Interim analysis 2 (at 12 months of treatment)
Time to walk 25 Feet (TW25)
Time frame: Final analysis 1 (at 24 months of treatment)
Time to walk 25 Feet (TW25)
Time frame: Final analysis 2 (at 36 months of treatment)
Stair-climbing
Time frame: Interim analysis 1 (at 12 months of treatment)
Stair-climbing
Time frame: Final analysis 1 (at 24 months of treatment)
Stair-climbing
Time frame: Final analysis 2 (at 36 months of treatment)
Strength
Time frame: Interim analysis 1 (at 12 months of treatment)
Strength
Time frame: Final analysis 1 (at 24 months of treatment)
Strength
Time frame: Final analysis 2 (at 36 months of treatment)
SF-Qualiveen (Short-Form Qualiveen)
Time frame: Interim analysis 1 (at 12 months of treatment)
SF-Qualiveen (Short-Form Qualiveen)
Time frame: Final analysis 1 (at 24 months of treatment)
SF-Qualiveen (Short-Form Qualiveen)
Time frame: Final analysis 2 (at 36 months of treatment)
Revised Fecal Incontinence Scale (RFIS)
Time frame: Interim analysis 1 (at 12 months of treatment)
Revised Fecal Incontinence Scale (RFIS)
Time frame: Final analysis 1 (at 24 months of treatment)
Revised Fecal Incontinence Scale (RFIS)
Time frame: Final analysis 2 (at 36 months of treatment)
Brain neuroimaging: Loes
Time frame: Interim analysis 1 (at 12 months of treatment)
Brain neuroimaging: Loes
Time frame: Final analysis 1 (at 24 months of treatment)
Brain neuroimaging: Loes
Time frame: Final analysis 2 (at 36 months of treatment)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2026.
- Individual participant dataSharing planned: No
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Adrenomyeloneuropathy Without Cerebral Involvement
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.
Carlos Casasnovas, MD, PhD principal investigator
Hospital Universitari de Bellvitge
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Dimethyl fumarate in people with Adrenomyeloneuropathy Without Cerebral Involvement, run by Pujol, Aurora, M.D. at 3 sites in Spain.
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 12 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Spain. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Postural sway test (time frame: Interim analysis 1 (at 12 months of treatment)). The study lists 3 primary outcomes in all. 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 17 Jun 2026.