Adrenoleukodystrophy Clinical Trial
— AdvanceOfficial 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
Verified date | November 2023 |
Source | Minoryx Therapeutics, S.L. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Active, not recruiting |
Enrollment | 105 |
Est. completion date | June 2025 |
Est. primary completion date | June 25, 2021 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 18 Years to 65 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
France | Hospital de la Pitié-Salpêtrière | Paris | |
Germany | Universitat Leipzig Klinik and Poliklinik für Neurologie | Leipzig | |
Hungary | Institute of Genomic Medicine and Rare Disorders | Budapest | |
Italy | Instituto Neurologico Carlo Besta | Milano | |
Netherlands | Academish Medisch Centrum | Amsterdam | |
Spain | Hospital Universitari Vall d'Hebrón | Barcelona | |
United Kingdom | National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit) | London | |
United States | Kennedy Krieger Institute | Baltimore | Maryland |
United States | Massachusetts General Hospital | Boston | Massachusetts |
United States | Stanford University Medical Center | Stanford | California |
Lead Sponsor | Collaborator |
---|---|
Minoryx Therapeutics, S.L. |
United States, France, Germany, Hungary, Italy, Netherlands, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | To evaluate the efficacy of MIN-102 in terms of patient reported outcomes. | in 96 weeks | ||
Secondary | SSPROM (Severity Score System for Progressive Myelopathy ) | in 96 weeks | ||
Secondary | EDSS (Expanded Disability Status Scale ) | in 96 weeks | ||
Secondary | Quality of life scales (Euroqol) | in 96 weeks | ||
Secondary | Incidence of cerebral inflammatory lesions | in 96 weeks |
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