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

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

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 Overview

• Status: Active, not recruiting
• Conditions: Adrenoleukodystrophy
• Intervention / Treatment: Drug: Placebos; Drug: MIN-102

• Study Type: Interventional
• Enrollment (Estimated): 105
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• France
  • Paris, France
    • Hospital de la Pitie-Salpetriere
• Germany
  • Leipzig, Germany
    • Universitat Leipzig Klinik and Poliklinik für Neurologie
• Hungary
  • Budapest, Hungary
    • Institute of Genomic Medicine and Rare Disorders
• Italy
  • Milano, Italy
    • Instituto Neurologico Carlo Besta
• Netherlands
  • Amsterdam, Netherlands
    • Academish Medisch Centrum
• Spain
  • Barcelona, Spain
    • Hospital Universitari Vall d'Hebron
• United Kingdom
  • London, United Kingdom
    • National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)
• United States
  • California
    • Stanford, California, United States, 94304
      • Stanford University Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Kennedy Krieger Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebos; Placebo
Active Comparator: Active	Drug: MIN-102; MIN-102 treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Minoryx Therapeutics, S.L.

Study record dates

Study Major Dates

• Study Start (Actual): December 8, 2017
• Primary Completion (Actual): June 25, 2021
• Study Completion (Estimated): June 1, 2025

Study Registration Dates

• First Submitted: July 24, 2017
• First Submitted That Met QC Criteria: July 26, 2017
• First Posted (Actual): July 27, 2017

Study Record Updates

• Last Update Posted (Actual): November 29, 2023
• Last Update Submitted That Met QC Criteria: November 28, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Demyelinating Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Endocrine System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Leukoencephalopathies; Adrenal Gland Diseases; Hereditary Central Nervous System Demyelinating Diseases; Peroxisomal Disorders; Adrenal Insufficiency; Adrenoleukodystrophy
• Other Study ID Numbers: MT-2-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No