Dimethyl Fumarate in Adrenomyeloneuropathy
NCT Number: NCT06513533
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2|PHASE3
Conditions: Adrenomyeloneuropathy Without Cerebral Involvement
Interventions: OTHER: Placebo|DRUG: Dimethyl fumarate
Sponsor: Pujol, Aurora, M.D.
Collaborators: Instituto de Salud Carlos III|Spanish Clinical Research Network - SCReN|Institut d'Investigació Biomèdica de Bellvitge
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 40
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: TRIPLE (PARTICIPANT, CARE_PROVIDER, INVESTIGATOR)|Primary Purpose: TREATMENT
Start Date: 2024-04-23
Completion Date: 2028-06-30
Locations: Bellvitge University Hospital, L'Hospitalet De Llobregat, Barcelona, 08907, Spain|Donostia University Hospital, Donostia, 20014, Spain|University Hospital 12 de Octubre, Madrid, 28041, Spain

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. 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.

Primary Outcome Measures: 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), 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), 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), Final analysis 2 (at 36 months of treatment)
Secondary Outcome Measures: 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., 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., 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., 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., 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, 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, 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, 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, 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, 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, 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, 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, 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, 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, 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, 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, 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, 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, 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., 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., 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., 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., 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., 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., 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., 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., 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., Final analysis 2 (at 36 months of treatment)