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CompletedNCT03231878AdvanceUpdated Mar 18, 2025

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

A Phase 2/3 interventional study of MIN-102 and Placebos in Adrenoleukodystrophy, sponsored by Minoryx Therapeutics, S.L.. Completed at 10 sites in 8 countries. Open to male participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2025-03-18.

Sponsored by Minoryx Therapeutics, S.L. · Phase 2/3, Interventional, and Treatment

Phase
Phase 2/3
Study type
Interventional
Enrollment
105
Allocation
Randomized
Ages
18 Years to 65 Years
Sex
Male
01

Study 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).

02

Conditions studied

  • Adrenoleukodystrophy

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03

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
Male
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 2 / Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
105 participants (actual)

Study arms

  • Active comparator
    Active

    Drug: MIN-102

  • Placebo comparator
    Placebo

    Drug: Placebos

Interventions

  • DrugMIN-102

    MIN-102 treatment

  • DrugPlacebos

    Placebo

05

What researchers measure

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

06

Study locations

10 sites
  • Stanford University Medical Center
    Stanford, California 94304, United States
  • Kennedy Krieger Institute
    Baltimore, Maryland 21205, United States
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • Hospital de la Pitié-Salpêtrière
    Paris, France
  • Universitat Leipzig Klinik and Poliklinik für Neurologie
    Leipzig, Germany
  • Institute of Genomic Medicine and Rare Disorders
    Budapest, Hungary
  • Instituto Neurologico Carlo Besta
    Milano, Italy
  • Academish Medisch Centrum
    Amsterdam, Netherlands
  • Hospital Universitari Vall d'Hebrón
    Barcelona, Spain
  • National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)
    London, United Kingdom
07

Registry details

Key details

Study ID
NCT03231878
Lead sponsor
Minoryx Therapeutics, S.L.
Responsible party
Sponsor
First posted
Jul 27, 2017
Start date
Dec 8, 2017
Primary completion
Jun 25, 2021
Completion
Mar 6, 2025
Last update
Mar 18, 2025

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
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