A Phase 2/3 interventional study of Leriglitazone and Placebo in Adrenoleukodystrophy, sponsored by Minoryx Therapeutics, S.L.. Completed at 10 sites in 8 countries. Open to male participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-10-28.
Sponsored by Minoryx Therapeutics, S.L. · Phase 2/3, Interventional, and Treatment
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).
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.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 116 is above the median of 31 across 40 interventional studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Minoryx Therapeutics, S.L. is the lead sponsor of 5 studies on the registry; 1 is open to participants now.
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The criteria were revised on the registry on Oct 28, 2026. See what changed
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Additional Inclusion Criteria for Part 2 of the study:
- Completion of the entire 96-week double-blind period of the study (Part 1).
Exclusion Criteria for both parts of the study:
Drug: Leriglitazone
Drug: Placebo
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%.
Also known as: MIN-102
Administered once daily in the morning. Patients randomized to placebo received the same amount of a matched placebo suspension.
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
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.
Time frame: Through study completion, up to 335 weeks
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.
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
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).
Time frame: 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).
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)
The Qualiveen-SF overall score ranges from 0 (not at all affected by the problem) to 4 (extremely affected by the problem).
Time frame: 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.
Time frame: 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).
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
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).
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
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.
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
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).
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
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.
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)
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.
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)
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).
Time frame: 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.
Time frame: 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.
Time frame: 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
Time frame: 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.
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
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
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)
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).
Time frame: 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).
Time frame: 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).
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
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.
Time frame: 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.
Time frame: 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).
Time frame: 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.
Time frame: 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).
Time frame: Week 240
Ran from Dec 2017 to primary completion in Oct 2020, 34 months.
Primary completion moved once since we started tracking on Sep 25, 2026: Jun 2021 → Oct 2020, on Oct 28, 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is completed, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Leriglitazone
Minoryx Therapeutics, S.L.