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

Minoryx Therapeutics, S.L.105 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

105

Conditions

Adrenoleukodystrophy

Interventions

MIN-102DRUG

MIN-102 treatment

PlacebosDRUG

Placebo

Eligibility

Sex: MALEMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (10)

Stanford University Medical Center

Stanford, California, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.

Time frame: in 96 weeks

Secondary Outcomes

To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.

Time frame: in 96 weeks

SSPROM (Severity Score System for Progressive Myelopathy )

Time frame: in 96 weeks

EDSS (Expanded Disability Status Scale )

Time frame: in 96 weeks

Quality of life scales (Euroqol)

Time frame: in 96 weeks

Incidence of cerebral inflammatory lesions

Time frame: in 96 weeks

Data from ClinicalTrials.gov

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