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Completed

NCT Number: NCT03231878

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

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).

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Key information

Conditions

Adrenoleukodystrophy Adrenal Gland Diseases Adrenal Insufficiency Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Diseases Endocrine System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hereditary Central Nervous System Demyelinating Diseases Heredodegenerative Disorders, Nervous System Intellectual Disability Leukoencephalopathies Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Peroxisomal Disorders X-Linked Intellectual Disability

Age range

18 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Hospital de la Pitié-Salpêtrière, Paris, France

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

MIN-102

Drug

MIN-102 treatment

Placebos

Drug

Placebo

Primary outcomes

  1. 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

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

    Time frame: in 96 weeks

  2. SSPROM (Severity Score System for Progressive Myelopathy )

    Time frame: in 96 weeks

  3. EDSS (Expanded Disability Status Scale )

    Time frame: in 96 weeks

  4. Quality of life scales (Euroqol)

    Time frame: in 96 weeks

  5. Incidence of cerebral inflammatory lesions

    Time frame: in 96 weeks

Sponsors and collaborators

Lead sponsor

Minoryx Therapeutics, S.L.

Industry

Registry information

Official study title

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

Acronym: Advance

Important dates

Study start
2017
Primary completion
2021
Study completion
2025
First posted
Jul 27, 2017
Registry last updated
Mar 18, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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