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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 13, 2020
Sponsor:
Information provided by (Responsible Party):
Minoryx Therapeutics, S.L.

Study Description
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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).

Condition or disease Intervention/treatment Phase
Adrenoleukodystrophy Drug: MIN-102 Drug: Placebos Phase 2 Phase 3

Study Design
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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
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Active Drug: MIN-102
MIN-102 treatment
Placebo Comparator: Placebo Drug: Placebos
Placebo


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Eligibility Criteria
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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
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Minoryx Therapeutics, S.L.
More Information
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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 13, 2020
Last Verified: November 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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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