Working…
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    min-102 | Adrenoleukodystrophy
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03231878
Recruitment Status : Active, not recruiting
First Posted : July 27, 2017
Last Update Posted : March 25, 2019
Sponsor:
Information provided by (Responsible Party):
Minoryx Therapeutics, S.L.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
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 : October 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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: 96 weeks ]

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Layout table for location information
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for additonal information
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: March 25, 2019
Last Verified: March 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
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