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

Phase 2

Completed

• Conditions: Adrenoleukodystrophy
• Interventions: Drug: MIN-102; Drug: Placebos

• Registration Number: NCT03231878
• Lead Sponsor: Minoryx Therapeutics, S.L.

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-07-24
• Study First Submitted QC: 2017-07-26
• Study First Posted: 2017-07-27
• Study Start: 2017-12-08
• Primary Completion: 2021-06-25
• Status Verified: 2025-03
• Study Completion: 2025-03-06
• Last Update Submitted: 2025-03-17
• Last Update Posted: 2025-03-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 105

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Active	MIN-102	-
Placebo	Placebos	-

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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
Quality of life scales (Euroqol)	in 96 weeks
Incidence of cerebral inflammatory lesions	in 96 weeks
EDSS (Expanded Disability Status Scale )	in 96 weeks

Trial Locations

Locations (10)

Kennedy Krieger Institute; 🇺🇸 Baltimore, Maryland, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Hospital de la Pitié-Salpêtrière; 🇫🇷 Paris, France

Universitat Leipzig Klinik and Poliklinik für Neurologie; 🇩🇪 Leipzig, Germany

National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit); 🇬🇧 London, United Kingdom

Institute of Genomic Medicine and Rare Disorders; 🇭🇺 Budapest, Hungary

Academish Medisch Centrum; 🇳🇱 Amsterdam, Netherlands

Hospital Universitari Vall d'Hebrón; 🇪🇸 Barcelona, Spain

Instituto Neurologico Carlo Besta; 🇮🇹 Milano, Italy

Stanford University Medical Center; 🇺🇸 Stanford, California, United States