A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. (Advance)
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ClinicalTrials.gov Identifier: NCT03231878 |
Recruitment Status :
Active, not recruiting
First Posted : July 27, 2017
Last Update Posted : November 1, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Adrenoleukodystrophy | Drug: MIN-102 Drug: Placebos | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 105 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | December 8, 2017 |
Actual Primary Completion Date : | June 25, 2021 |
Estimated Study Completion Date : | June 2023 |

Arm | Intervention/treatment |
---|---|
Active Comparator: Active |
Drug: MIN-102
MIN-102 treatment |
Placebo Comparator: Placebo |
Drug: Placebos
Placebo |
- 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 ]
- To evaluate the efficacy of MIN-102 in terms of patient reported outcomes. [ Time Frame: in 96 weeks ]
- SSPROM (Severity Score System for Progressive Myelopathy ) [ Time Frame: in 96 weeks ]
- EDSS (Expanded Disability Status Scale ) [ Time Frame: in 96 weeks ]
- Quality of life scales (Euroqol) [ Time Frame: in 96 weeks ]
- Incidence of cerebral inflammatory lesions [ Time Frame: in 96 weeks ]

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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | 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:
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03231878
United States, California | |
Stanford University Medical Center | |
Stanford, California, United States, 94304 | |
United States, Maryland | |
Kennedy Krieger Institute | |
Baltimore, Maryland, United States, 21205 | |
United States, Massachusetts | |
Massachusetts General Hospital | |
Boston, Massachusetts, United States, 02114 | |
France | |
Hospital de la Pitié-Salpêtrière | |
Paris, France | |
Germany | |
Universitat Leipzig Klinik and Poliklinik für Neurologie | |
Leipzig, Germany | |
Hungary | |
Institute of Genomic Medicine and Rare Disorders | |
Budapest, Hungary | |
Italy | |
Instituto Neurologico Carlo Besta | |
Milano, Italy | |
Netherlands | |
Academish Medisch Centrum | |
Amsterdam, Netherlands | |
Spain | |
Hospital Universitari Vall d'Hebrón | |
Barcelona, Spain | |
United Kingdom | |
National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit) | |
London, United Kingdom |
Responsible Party: | Minoryx Therapeutics, S.L. |
ClinicalTrials.gov Identifier: | NCT03231878 |
Other Study ID Numbers: |
MT-2-01 |
First Posted: | July 27, 2017 Key Record Dates |
Last Update Posted: | November 1, 2021 |
Last Verified: | November 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Adrenoleukodystrophy Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Hereditary Central Nervous System Demyelinating Diseases Leukoencephalopathies Demyelinating Diseases Mental Retardation, X-Linked Intellectual Disability |
Neurobehavioral Manifestations Neurologic Manifestations Genetic Diseases, X-Linked Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolism, Inborn Errors Peroxisomal Disorders Metabolic Diseases Adrenal Insufficiency Adrenal Gland Diseases Endocrine System Diseases |