NCT03231878

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).

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
105

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Dec 2017

Longer than P75 for phase_2

Geographic Reach
8 countries

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 24, 2017

Completed
3 days until next milestone

First Posted

Study publicly available on registry

July 27, 2017

Completed
4 months until next milestone

Study Start

First participant enrolled

December 8, 2017

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 25, 2021

Completed
3.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 6, 2025

Completed
Last Updated

March 18, 2025

Status Verified

March 1, 2025

Enrollment Period

3.5 years

First QC Date

July 24, 2017

Last Update Submit

March 17, 2025

Conditions

Adrenoleukodystrophy

Outcome Measures

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.

    in 96 weeks

Secondary Outcomes (5)

  • 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

Study Arms (2)

Active

ACTIVE COMPARATOR
Drug: MIN-102

Placebo

PLACEBO COMPARATOR
Drug: Placebos

Interventions

MIN-102DRUG

MIN-102 treatment

Active
PlacebosDRUG

Placebo

Placebo

Eligibility Criteria

Age18 Years - 65 Years
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • Any other chronic neurological disease with signs of spastic paraplegia, such as hereditary spastic paraplegia, multiple sclerosis, etc.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Stanford University Medical Center

Stanford, California, 94304, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Hospital de la Pitié-Salpêtrière

Paris, France

Location

Universitat Leipzig Klinik and Poliklinik für Neurologie

Leipzig, Germany

Location

Institute of Genomic Medicine and Rare Disorders

Budapest, Hungary

Location

Instituto Neurologico Carlo Besta

Milan, Italy

Location

Academish Medisch Centrum

Amsterdam, Netherlands

Location

Hospital Universitari Vall d'Hebrón

Barcelona, Spain

Location

National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)

London, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 36681445DERIVED

MeSH Terms

Conditions

Adrenoleukodystrophy

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHereditary Central Nervous System Demyelinating DiseasesLeukoencephalopathiesDemyelinating DiseasesX-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous SystemMetabolism, Inborn ErrorsPeroxisomal DisordersMetabolic DiseasesNutritional and Metabolic DiseasesAdrenal InsufficiencyAdrenal Gland DiseasesEndocrine System Diseases

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 24, 2017

First Posted

July 27, 2017

Study Start

December 8, 2017

Primary Completion

June 25, 2021

Study Completion

March 6, 2025

Last Updated

March 18, 2025

Record last verified: 2025-03

Locations

GB(1)US(3)ES(1)FR(1)NL(1)DE(1)HU(1)IT(1)