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

NCT ID: NCT03231878

Last Updated: 2025-03-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

105 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-08

Study Completion Date

2025-03-06

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).

Detailed Description

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Conditions

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Adrenoleukodystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Active

Group Type ACTIVE_COMPARATOR

MIN-102

Intervention Type DRUG

MIN-102 treatment

Placebo

Group Type PLACEBO_COMPARATOR

Placebos

Intervention Type DRUG

Placebo

Interventions

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MIN-102

MIN-102 treatment

Intervention Type DRUG

Placebos

Placebo

Intervention Type DRUG

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.

* 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.

Exclusion Criteria

* Any other chronic neurological disease with signs of spastic paraplegia, such as hereditary spastic paraplegia, multiple sclerosis, etc.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Minoryx Therapeutics, S.L.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Stanford University Medical Center

Stanford, California, United States

Site Status

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Hospital de la Pitié-Salpêtrière

Paris, , France

Site Status

Universitat Leipzig Klinik and Poliklinik für Neurologie

Leipzig, , Germany

Site Status

Institute of Genomic Medicine and Rare Disorders

Budapest, , Hungary

Site Status

Instituto Neurologico Carlo Besta

Milan, , Italy

Site Status

Academish Medisch Centrum

Amsterdam, , Netherlands

Site Status

Hospital Universitari Vall d'Hebrón

Barcelona, , Spain

Site Status

National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)

London, , United Kingdom

Site Status

Countries

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United States France Germany Hungary Italy Netherlands Spain United Kingdom

References

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Kohler W, Engelen M, Eichler F, Lachmann R, Fatemi A, Sampson J, Salsano E, Gamez J, Molnar MJ, Pascual S, Rovira M, Vila A, Pina G, Martin-Ugarte I, Mantilla A, Pizcueta P, Rodriguez-Pascau L, Traver E, Vilalta A, Pascual M, Martinell M, Meya U, Mochel F; ADVANCE Study Group. Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial. Lancet Neurol. 2023 Feb;22(2):127-136. doi: 10.1016/S1474-4422(22)00495-1.

Reference Type DERIVED
PMID: 36681445 (View on PubMed)

Other Identifiers

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MT-2-01

Identifier Type: -

Identifier Source: org_study_id

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