A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

Study Overview
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Brief Summary
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This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the 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).

Official Title
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A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 (IMP) on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy

Conditions
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Adrenoleukodystrophy

Intervention / Treatment
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* Drug: MIN-102
* Drug: Placebos


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and between 18-65 years of age. Diagnosed with X-linked adrenoleukodystrophy (X-ALD) based on elevated VLCFA and genetic testing. Clinical evidence of spinal cord involvement. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Active<br> | Drug: MIN-102<br>* MIN-102 treatment<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebos<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test. |  | in 96 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the efficacy of MIN-102 in terms of patient reported outcomes. |  | in 96 weeks | 
| SSPROM (Severity Score System for Progressive Myelopathy ) |  | in 96 weeks | 
| EDSS (Expanded Disability Status Scale ) |  | in 96 weeks | 
| Quality of life scales (Euroqol) |  | in 96 weeks | 
| Incidence of cerebral inflammatory lesions |  | in 96 weeks |