AdrenoleukodystrophyPhase 2/3AdvanceNCT03231878
A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients
A Phase 2/3 study testing Leriglitazone in people with Adrenoleukodystrophy, run by Minoryx Therapeutics, S.L. at 10 sites in 8 countries.
Trial summary
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the adrenomyeloneuropathy (AMN) phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Adrenoleukodystrophy
- What is being tested?
- Leriglitazone
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 116 participants
- Where
- 10 sitesin 8 countries
- Study dates
- 8 Dec 2017to 6 Mar 2025
- Phase
- Phase 2/3
- 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 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and between 18-65 years of age.
- Diagnosed with X-ALD based on genetic testing.
- Clinical evidence of spinal cord involvement.
- Additional Inclusion Criteria for Part 2 of the study:
- Completion of the entire 96-week double-blind period of the study (Part 1).
Who cannot join
- Any other chronic neurological disease with signs of spastic paraplegia, such as hereditary spastic paraplegia, multiple sclerosis, etc.
- Presence of inflammatory (Gd-enhancing) MRI lesions or any abnormality other than those mentioned in the inclusion criteria.
- Known type 1 or type 2 diabetes.
- Known intolerance to pioglitazone or any other thiazolidinedione.
- Taking or have taken honokiol, pioglitazone or other thiazolidinediones within the 6 months prior to screening.
- Previous bone marrow transplantation.
- Previous or current history of cancer (other than treated basal cell carcinoma).
- Previous or current history of congestive heart failure.
Trial locations and contacts
10 sites in 8 countries.
10 sites in 8 countries
United States3 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Massachusetts General Hospital
Boston, Massachusetts, 02114
Stanford University Medical Center
Stanford, California, 94304
Germany1 site
Universitat Leipzig Klinik and Poliklinik für Neurologie
Leipzig
United Kingdom1 site
National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)
London
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the adrenomyeloneuropathy (AMN) phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).
Read the detailed description
In Part 1 of the study, eligible patients were randomized in a 2:1 ratio to receive leriglitazone or placebo. For patients randomized to leriglitazone, dosing was adjusted on an individual-patient basis to achieve the target plasma leriglitazone exposure, with a maximum allowed range of ±20%. Patients randomized to placebo received the same amount of a matched placebo suspension. Blood samples to determine leriglitazone plasma concentrations were obtained at all visits after the baseline visit (V0). Dose-adjustment recommendations were made by a pharmacokinetic (PK) expert from the central laboratory who was unblinded to PK data. For patients not tolerating the target plasma exposure, the investigator could reduce the study medication dose at any timepoint by up to 50%. To preserve the blind, random dose adjustments were made for the patients receiving placebo.
Part 2 was an open-label treatment extension study which started immediately after the day of last treatment in Part 1 at V6, which, after 96 weeks of double-blind treatment in Part 1, was simultaneously the baseline visit of Part 2 of the study. All patients in Part 2 (including those randomized to placebo in Part 1) received a leriglitazone dose to achieve the predefined target plasma exposure. All patients who were taking doses of ≤ the starting dose in Part 1 at the end of Part 1 (V6) started Part 2 with the same dose. All other patients reverted to the Part 1 starting dose of leriglitazone oral suspension. Patients and investigators were advised that dose adjustments could occur in both groups, those previously assigned to leriglitazone and to placebo, since patients who were on a stable leriglitazone dose during Part 1 may no longer have been at their optimum exposure level at Week 96.
After the potential adjustment informed by PK results obtained at 100 weeks (V7) and 108 weeks (V8), the study medication dosage could not otherwise be changed except if required for safety/tolerability reasons. Downwards adjustments of the dose in this case could be introduced, but the minimum exposure had to be achieved. Patients who experienced study medication-related AEs and who could not tolerate the study medication, even at the minimum permitted exposure level, were discontinued from the study.
In Part 2 patients were evaluated with 144 weeks of daily administration of study medication and periodic on-site visits and follow-up phone calls. After 144 weeks of daily administration in Part 2, regular visits and safety visits or follow-up phone calls were to each take place at 6-month intervals for an unlimited time until either: (1) the patient withdrew consent; (2) significant safety or tolerability issues appeared and/or the patient's general health status, in the clinical judgment of the investigator, declined to an extent that the risks of treatment with leriglitazone outweighed the potential benefits; (3) the patient underwent hematopoietic stem-cell transplantation; (4) leriglitazone received marketing authorization for the treatment of AMN; or (5) Minoryx Therapeutics decided to terminate this study, whichever of these events occurred first.
- Conditions
- Adrenoleukodystrophy
- Being tested
- Leriglitazone, Placebo
- Medical subject headings
- Adrenoleukodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LeriglitazoneDrug
Also called MIN-102
Administered once daily in the morning. Dosing was adjusted on an individual-patient basis to achieve target plasma leriglitazone exposure, with a maximum allowed range of ±20%.
PlaceboDrug
Administered once daily in the morning. Patients randomized to placebo received the same amount of a matched placebo suspension.
| Study group | Role | What they receive |
|---|---|---|
| Leriglitazone | Active comparator | Not described Receives: Drug: Leriglitazone |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Change from baseline to Week 96 in the total distance walked (meters) in the 6-minute walk test
Measured: Week 96
Part 2: Long-term safety and tolerability of leriglitazone, assessed in terms of adverse events (AEs) and serious adverse events (SAEs), vital signs, 12-lead electrocardiograms (ECGs), and clinical laboratory tests
The number of patients with AEs, SAEs, and SUSARs, and with abnormal vital signs (body weight, blood pressure, pulse rate), 12-lead ECGs (heart rate, PR interval, RR interval, QRS duration, QT interval, QTc interval [Bazzett's and Fridericia's] and investigator interpretation of the ECG profile), and clinical laboratory tests (hematology, blood chemistry including parameters for adrenal function, urinalysis and sample for cytology) will be presented.
Measured: Through study completion, up to 335 weeks
Secondary outcomes (26)
Part 1: Change from baseline to Week 96 in body sway amplitude - total (eyes closed/feet apart)
Total body sway was measured (mm) using a portable plantar pressure system. Change from baseline in total body sway amplitude under the eyes closed/feet apart condition is presented.
Measured: Week 96
Part 1: Percentage of patients reporting an improvement from baseline in the Patient Global Impression - Improvement scale (PGI-I) at Week 96
The PGI-I is a tool for assessing improvement as judged by the patient. It is a 1-7 point weighted scale, ranging from "very much improved" (1) to "very much worse" (7).
Measured: Week 96
Part 1: Change from baseline to Week 96 in Severity Score System for Progressive Myelopathy (SSPROM)
The SSPROM is a scale covering 5 domains (patient-reported motor disability, examined motor disability, spasticity and hyperreflexia, sensory loss, and patient information on sphincter dysfunction), ranging from 0 (total disability) to 100 (normal).
Measured: Week 96
Part 1: Change from baseline to Week 96 in the overall score of the Qualiveen Short Form Quality of Life Questionnaire for Urinary Disorders (Qualiveen-SF)
The Qualiveen-SF overall score ranges from 0 (not at all affected by the problem) to 4 (extremely affected by the problem).
Measured: Week 96
Part 1: Changes from baseline to Week 96 in body sway amplitude
Body sway was measured (mm) using a portable plantar pressure system, considering total sway, antero-posterior (A-P) sway, and mediolateral (M-L) sway. Body sway was measured under 4 conditions: eyes closed/feet apart; eyes closed/feet together; eyes open/feet apart; eyes open/feet together.
Changes from baseline in total body sway amplitude (eyes closed/feet together; eyes open/feet apart; eyes open/feet together), sway average amplitude A-P (all 4 conditions), and sway average amplitude M-L (all 4 conditions) are presented.
Measured: Week 96
Part 1: Change from baseline to Week 96 in the Expanded Disability Status Scale (EDSS) - ambulation score
The Kurtzke EDSS is a widely utilized method of quantifying disability and monitoring changes in the level of disability over time. It is based on a standard neurological examination. The ambulation score within EDSS ranges from 0 (unrestricted) to 12 (essentially restricted to bed or chair or perambulated in wheelchair).
Measured: Week 96
Part 1: Percentage of patients for whom an improvement from baseline was reported in the Clinical Global Impression - Improvement scale (CGI-I) at Week 96
The CGI-I is a tool for assessing total overall improvement as judged by the investigator that considers whether or not the improvement is entirely due to the study medication treatment. It is a 1-7 point weighted scale, ranging from "very much improved" (1) to "very much worse" (7).
Measured: Week 96
Part 1: Change from baseline to Week 96 in the European Quality of Life 5 Dimension 5 Level (EQ-5D-5L) index
The EQ-5D-5L consists of 5 individual domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression along with a health scale. The index value (health scale), which ranges from 0 to 1 (higher scores represent better health) was used for the evaluation of this outcome measure secondary endpoint.
Measured: Week 96
Part 1: Percentage of patients for whom a worsening from baseline was reported in the Clinical Global Impression - Severity of Illness scale (CGI-S) at Week 96
The CGI-S is a rating for severity of illness based on a 1-7 point weighted scale that is filled in by the investigator at the start of treatment. It assesses "normal, not at all ill" (1) to "among the most extremely ill patients" (7).
Measured: Week 96
Part 1: Change from baseline to Week 96 in the dynamometry maximum value (kg) of the leg with the lowest average value at baseline
Dynamometry was performed using a hand-held device that was used to measure hip flexion strength in both legs. Measurements were repeated 3 times over the course of 5 minutes. The maximum value out of the 3 measurements of the leg with the lowest average value (kg) at baseline was used for analysis.
Measured: Week 96
Part 1: Change from baseline to Week 96 in the percentage out of 60 points of the Multiple Sclerosis Walking Scale, 12-item (MSWS-12)
The MSWS-12 is combined into a single score by summing the score for each of the 12 questions (maximum score of 60) and calculating a percentage, where a higher percentage means a greater impact on walking ability.
Measured: Week 96
Part 1: Change from baseline to Week 96 in the total score of the International Index of Erectile Function questionnaire (IIEF)
The IIEF was combined into a single score by creating 5 domains and summing the individual domain scores. The IIEF total score ranges from 0 (negative) to 75 (positive).
Measured: Week 96
Part 1: Progression of cerebral lesions in cerebral magnetic resonance imaging (MRI)
The progression of cerebral lesions in MRI was assessed by central readers blinded to treatment. Progression was defined as:
- Occurrence of inflammatory (gadolinium [Gd]-positive) lesions, or
- Growth of existing noninflammatory lesions since screening or baseline, or
- Occurrence of new noninflammatory lesions after screening or baseline
The number of patients with cerebral lesions present at baseline, and the number of patients showing progression of cerebral lesions at Weeks 48 and 96 is presented.
Measured: Weeks 48 and 96
Part 1: Change from baseline to Week 96 in Loes severity scores on the MRI
Loes severity score was assessed by central readers using standardized rating with severity scores for 9 brain regions and atrophy. Loes scores range from 0 (normal) to 34 (most severe). Note that increases in Loes severity score are expected in patients who showed cerebral progression.
Measured: Week 96
Part 2: Progression of cerebral lesions in cerebral MRI
The progression of cerebral lesions in MRI was assessed by central readers blinded to treatment. Progression was defined as:
- Occurrence of inflammatory lesions, or
- Growth of existing noninflammatory lesions since screening or baseline, or
- Occurrence of new noninflammatory lesions after screening or baseline
The following are presented:
Number of patients who entered Part 2 and did not develop progressive cALD in Part 1
Number of patients who entered Part 2 and had not presented progression of lesions during Part 1
Progression of lesions amongst patients who entered Part 2 and (1) did not develop progressive cALD in Part 1, and (2) had not presented progression of lesions in Part 1, measured by:
- Number of patients with progression at Weeks 144, 192, and 240
- Number of patients with progression between Week 144 and Week 192, and between Week 192 and Week 240
- Number of patients with progression per year
Measured: Weeks 144, 192, and 240
Part 2: Change from baseline to Week 240 in Loes severity scores on the MRI
Loes severity score was assessed by central readers using standardized rating with severity scores for 9 brain regions and atrophy. Loes scores range from 0 (normal) to 34 (most severe). Note that increases in Loes severity score are expected in patients who showed cerebral progression.
Measured: Week 240
Part 2: The number of SAE reports on diagnosis of clinically progressive cALD as judged by the investigator taking into account both MRI results and an overall clinical evaluation
The following are presented:
Number of patients who entered Part 2 and did not develop progressive cALD in Part 1
For patients who entered Part 2 and did not develop progressive cALD in Part 1
- Number of patients diagnosed with clinically progressive cALD at Weeks 144, 192, and 240
- Number of patients diagnosed with clinically progressive cALD between Week 144 and Week 192, and between Week 192 and Week 240
- Number of patients diagnosed with clinically progressive cALD per year
Measured: Weeks 144, 192, and 240
Part 2: Change from baseline to Week 240 in body sway amplitude - total (eyes closed/feet apart, eyes closed/feet together)
Body sway was measured (mm) using a portable plantar pressure system, considering total sway, A-P sway, and M-L sway. Body sway was measured under 2 conditions: eyes closed/feet apart; eyes closed/feet together.
Changes from baseline in total body sway amplitude, sway average amplitude A-P, and sway average amplitude M-L for both conditions).
Measured: Week 240
Part 2: Change from baseline to Week 240 in SSPROM
The SSPROM is a scale covering 5 domains (patient-reported motor disability, examined motor disability, spasticity and hyperreflexia, sensory loss, and patient information on sphincter dysfunction), ranging from 0 (total disability) to 100 (normal).
Measured: Week 240
Part 2: Change from baseline to Week 240 in the EDSS - ambulation score
The Kurtzke EDSS is a widely utilized method of quantifying disability and monitoring changes in the level of disability over time. It is based on a standard neurological examination. The ambulation score within EDSS ranges from 0 (unrestricted) to 12 (essentially restricted to bed or chair or perambulated in wheelchair).
Measured: Week 240
Part 2: Change from baseline to Week 240 in the total distance walked (meters) in the 6-minute walk test
Measured: Week 240
Part 2: Change from baseline to Week 240 in the EDSS - step score
The Kurtzke EDSS is a widely utilized method of quantifying disability and monitoring changes in the level of disability over time. It is based on a standard neurological examination. The EDSS step score ranges from 0 to 10 in 0.5-unit increments that represent higher levels of disability.
Measured: Week 240
Part 2: Change from baseline to Week 240 in the EQ-5D-5L index
The EQ-5D-5L consists of 5 individual domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression along with a health scale. The index value (health scale), which ranges from 0 to 1 (higher scores represent better health) was used for the evaluation of this outcome measure secondary endpoint.
Measured: Week 240
Part 2: Change from baseline to Week 240 in the overall score of the Qualiveen-SF
The Qualiveen-SF overall score ranges from 0 (not at all affected by the problem) to 4 (extremely affected by the problem).
Measured: Week 240
Part 2: Change from baseline to Week 240 in the percentage out of 60 points of the MSWS-12
The MSWS-12 is combined into a single score by summing the score for each of the 12 questions (maximum score of 60) and calculating a percentage, where a higher percentage means a greater impact on walking ability.
Measured: Week 240
Part 2: Change from baseline to Week 240 in the total score of the IIEF
The IIEF was combined into a single score by creating 5 domains and summing the individual domain scores. The IIEF total score ranges from 0 (negative) to 75 (positive).
Measured: Week 240
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2017
- Study start8 Dec 2017
- Primary completion13 Oct 2020
- Study completion6 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AdrenoleukodystrophyStudies 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
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 116 participants (actual)
Sponsor and investigators
- Lead sponsor
- Minoryx Therapeutics, S.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03231878
- Sponsor's protocol ID
- MT-2-01
- Acronym
- Advance
- Status last verified
- Oct 2026
- First submitted
- 24 Jul 2017
- Last updated
- 28 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Kohler W, Engelen M, Eichler F, Lachmann R, Fatemi A, Sampson J, Salsano E, Gamez J, Molnar MJ, Pascual S, Rovira M, Vila A, Pina G, Martin-Ugarte I, Mantilla A, Pizcueta P, Rodriguez-Pascau L, Traver E, Vilalta A, Pascual M, Martinell M, Meya U, Mochel F; ADVANCE Study Group. Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial. Lancet Neurol. 2023 Feb;22(2):127-136. doi: 10.1016/S1474-4422(22)00495-1.
PubMed 36681445
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy
- ClinicalTrials.gov ID
- NCT03231878
- Acronym / sponsor ID
- Advance / MT-2-01
- Conditions
- Adrenoleukodystrophy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 116 participants (actual)
- Lead sponsor
- Minoryx Therapeutics, S.L.Industry (pharma, biotech, devices)
- Study start
- 8 Dec 2017 (actual)
- Primary completion
- 13 Oct 2020 (actual)
- Study completion
- 6 Mar 2025 (actual)
- First posted
- 27 Jul 2017 (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.
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the adrenomyeloneuropathy (AMN) phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).
Detailed description
In Part 1 of the study, eligible patients were randomized in a 2:1 ratio to receive leriglitazone or placebo. For patients randomized to leriglitazone, dosing was adjusted on an individual-patient basis to achieve the target plasma leriglitazone exposure, with a maximum allowed range of ±20%. Patients randomized to placebo received the same amount of a matched placebo suspension. Blood samples to determine leriglitazone plasma concentrations were obtained at all visits after the baseline visit (V0). Dose-adjustment recommendations were made by a pharmacokinetic (PK) expert from the central laboratory who was unblinded to PK data. For patients not tolerating the target plasma exposure, the investigator could reduce the study medication dose at any timepoint by up to 50%. To preserve the blind, random dose adjustments were made for the patients receiving placebo.
Part 2 was an open-label treatment extension study which started immediately after the day of last treatment in Part 1 at V6, which, after 96 weeks of double-blind treatment in Part 1, was simultaneously the baseline visit of Part 2 of the study. All patients in Part 2 (including those randomized to placebo in Part 1) received a leriglitazone dose to achieve the predefined target plasma exposure. All patients who were taking doses of ≤ the starting dose in Part 1 at the end of Part 1 (V6) started Part 2 with the same dose. All other patients reverted to the Part 1 starting dose of leriglitazone oral suspension. Patients and investigators were advised that dose adjustments could occur in both groups, those previously assigned to leriglitazone and to placebo, since patients who were on a stable leriglitazone dose during Part 1 may no longer have been at their optimum exposure level at Week 96.
After the potential adjustment informed by PK results obtained at 100 weeks (V7) and 108 weeks (V8), the study medication dosage could not otherwise be changed except if required for safety/tolerability reasons. Downwards adjustments of the dose in this case could be introduced, but the minimum exposure had to be achieved. Patients who experienced study medication-related AEs and who could not tolerate the study medication, even at the minimum permitted exposure level, were discontinued from the study.
In Part 2 patients were evaluated with 144 weeks of daily administration of study medication and periodic on-site visits and follow-up phone calls. After 144 weeks of daily administration in Part 2, regular visits and safety visits or follow-up phone calls were to each take place at 6-month intervals for an unlimited time until either: (1) the patient withdrew consent; (2) significant safety or tolerability issues appeared and/or the patient's general health status, in the clinical judgment of the investigator, declined to an extent that the risks of treatment with leriglitazone outweighed the potential benefits; (3) the patient underwent hematopoietic stem-cell transplantation; (4) leriglitazone received marketing authorization for the treatment of AMN; or (5) Minoryx Therapeutics decided to terminate this study, whichever of these events occurred first.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Leriglitazone | Active comparator | Drug: Leriglitazone |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male and between 18-65 years of age.
- Diagnosed with X-ALD based on genetic testing.
- Clinical evidence of spinal cord involvement.
- Completion of the entire 96-week double-blind period of the study (Part 1).
Key exclusion criteria (8 listed)
- Any other chronic neurological disease with signs of spastic paraplegia, such as hereditary spastic paraplegia, multiple sclerosis, etc.
- Presence of inflammatory (Gd-enhancing) MRI lesions or any abnormality other than those mentioned in the inclusion criteria.
- Known type 1 or type 2 diabetes.
- Known intolerance to pioglitazone or any other thiazolidinedione.
- Taking or have taken honokiol, pioglitazone or other thiazolidinediones within the 6 months prior to screening.
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 |
|---|---|---|
| LeriglitazoneAlso: MIN-102Administered once daily in the morning. Dosing was adjusted on an individual-patient basis to achieve target plasma leriglitazone exposure, with a maximum allowed range of ±20%. | Drug | Leriglitazone |
| PlaceboAdministered once daily in the morning. Patients randomized to placebo received the same amount of a matched placebo suspension. | Drug | Placebo |
Primary outcome
Part 1: Change from baseline to Week 96 in the total distance walked (meters) in the 6-minute walk test
Time frame: Week 96
Part 2: Long-term safety and tolerability of leriglitazone, assessed in terms of adverse events (AEs) and serious adverse events (SAEs), vital signs, 12-lead electrocardiograms (ECGs), and clinical laboratory tests
Time frame: Through study completion, up to 335 weeks
The number of patients with AEs, SAEs, and SUSARs, and with abnormal vital signs (body weight, blood pressure, pulse rate), 12-lead ECGs (heart rate, PR interval, RR interval, QRS duration, QT interval, QTc interval [Bazzett's and Fridericia's] and investigator interpretation of the ECG profile), and clinical laboratory tests (hematology, blood chemistry including parameters for adrenal function, urinalysis and sample for cytology) will be presented.
26 secondary outcomes
Part 1: Change from baseline to Week 96 in body sway amplitude - total (eyes closed/feet apart)
Time frame: Week 96
Part 1: Percentage of patients reporting an improvement from baseline in the Patient Global Impression - Improvement scale (PGI-I) at Week 96
Time frame: Week 96
Part 1: Change from baseline to Week 96 in Severity Score System for Progressive Myelopathy (SSPROM)
Time frame: Week 96
Part 1: Change from baseline to Week 96 in the overall score of the Qualiveen Short Form Quality of Life Questionnaire for Urinary Disorders (Qualiveen-SF)
Time frame: Week 96
Part 1: Changes from baseline to Week 96 in body sway amplitude
Time frame: Week 96
Part 1: Change from baseline to Week 96 in the Expanded Disability Status Scale (EDSS) - ambulation score
Time frame: Week 96
Part 1: Percentage of patients for whom an improvement from baseline was reported in the Clinical Global Impression - Improvement scale (CGI-I) at Week 96
Time frame: Week 96
Part 1: Change from baseline to Week 96 in the European Quality of Life 5 Dimension 5 Level (EQ-5D-5L) index
Time frame: Week 96
Part 1: Percentage of patients for whom a worsening from baseline was reported in the Clinical Global Impression - Severity of Illness scale (CGI-S) at Week 96
Time frame: Week 96
Part 1: Change from baseline to Week 96 in the dynamometry maximum value (kg) of the leg with the lowest average value at baseline
Time frame: Week 96
Part 1: Change from baseline to Week 96 in the percentage out of 60 points of the Multiple Sclerosis Walking Scale, 12-item (MSWS-12)
Time frame: Week 96
Part 1: Change from baseline to Week 96 in the total score of the International Index of Erectile Function questionnaire (IIEF)
Time frame: Week 96
Part 1: Progression of cerebral lesions in cerebral magnetic resonance imaging (MRI)
Time frame: Weeks 48 and 96
Part 1: Change from baseline to Week 96 in Loes severity scores on the MRI
Time frame: Week 96
Part 2: Progression of cerebral lesions in cerebral MRI
Time frame: Weeks 144, 192, and 240
Part 2: Change from baseline to Week 240 in Loes severity scores on the MRI
Time frame: Week 240
Part 2: The number of SAE reports on diagnosis of clinically progressive cALD as judged by the investigator taking into account both MRI results and an overall clinical evaluation
Time frame: Weeks 144, 192, and 240
Part 2: Change from baseline to Week 240 in body sway amplitude - total (eyes closed/feet apart, eyes closed/feet together)
Time frame: Week 240
Part 2: Change from baseline to Week 240 in SSPROM
Time frame: Week 240
Part 2: Change from baseline to Week 240 in the EDSS - ambulation score
Time frame: Week 240
Part 2: Change from baseline to Week 240 in the total distance walked (meters) in the 6-minute walk test
Time frame: Week 240
Part 2: Change from baseline to Week 240 in the EDSS - step score
Time frame: Week 240
Part 2: Change from baseline to Week 240 in the EQ-5D-5L index
Time frame: Week 240
Part 2: Change from baseline to Week 240 in the overall score of the Qualiveen-SF
Time frame: Week 240
Part 2: Change from baseline to Week 240 in the percentage out of 60 points of the MSWS-12
Time frame: Week 240
Part 2: Change from baseline to Week 240 in the total score of the IIEF
Time frame: Week 240
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
- Derived from this study: PubMed 36681445Kohler W, Engelen M, Eichler F, Lachmann R, Fatemi A, Sampson J, Salsano E, Gamez J, Molnar MJ, Pascual S, Rovira M, Vila A, Pina G, Martin-Ugarte I, Mantilla A, Pizcueta P, Rodriguez-Pascau L, Traver E, Vilalta A, Pascual M, Martinell M, Meya U, Mochel F; ADVANCE Study Group. Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial. Lancet Neurol. 2023 Feb;22(2):127-136. doi: 10.1016/S1474-4422(22)00495-1.
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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.
10 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Leriglitazone in people with Adrenoleukodystrophy, run by Minoryx Therapeutics, S.L. at 10 sites in 8 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 5 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: finished, as of the sponsor's last update on 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, France, Germany, Hungary, Italy and 3 more countries. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Part 1: Change from baseline to Week 96 in the total distance walked (meters) in the 6-minute walk test (time frame: Week 96). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03231878, which also lists 1 linked publication.
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 28 Oct 2026; AzTrials last refreshed its copy on 1 Nov 2026.