ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY
Conditions
Sponsors
Minoryx Therapeutics S.L.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Main Study: 1. Male patients aged =18 to =65 years. 2. Diagnosis of ALD based on elevated very long-chain fatty acids (VLCFA) and genetic testing 3. Clinical evidence of spinal cord involvement, with an EDSS score between 2 and 6 4. Ability to walk for 6 minutes, without or with rest, with usual walking aids (cane or walker) 5. Ability to stand on a force plate with closed eyes and with feet apart for a minimum of 20 seconds 6. Either a normal brain MRI or a type-3 pattern MRI abnormality in which the abnormality is considered to represent the centripetal extension of the distal axonopathy 7. Normal adrenal function or appropriate steroid replacement if adrenal insufficiency is present. Extension Study: 1. Consent to participate in the extension study part 2. Completion of the entire 96-weeks double-blind period of the study (Part 1) and consent to participate in the extension study part 3. Normal adrenal function or appropriate steroid replacement Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 105 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Main Study & Extension Study: 1. Any other chronic neurological disease with signs of spastic paraplegia 2. Known type 1 or type 2 diabetes. 3. Known intolerance to pioglitazone or any other thiazolidinedione. 4. Previous or current use of honokiol, pioglitazone or other thiazolidinediones, biotin (MD-1003), unstable dose of Lorenzo's oil 5. Current treatment with immunosuppressant medication, except for corticosteroids. 6. Previous or current history of cancer, bone marrow transplantation, Congestive heart failure, significant liver and renal disorders, anemia, pulmonary or cardiac diseases, Cognitive or behavioral abnormalities, alcohol/drug abuse etc.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Main Study: Efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by the change from baseline in Six-Minute Walk Test (6MWT) compared with placebo after 96 weeks of treatment. Extension Study: To assess the safety and tolerability of MIN-102 upon long-term treatment.;Secondary Objective: Main Study: Efficacy objectives: To evaluate the effects of MIN-102 after 96 weeks of treatment on: • Change from baseline in body sway amplitude • Comprehensive clinical rating scales (SSPROM and EDSS) • Clinician and patient global impression of symptom severity and change • Muscle strength • Quality of life • Incidence of cerebral inflammatory lesions. Safety objectives: To evaluate the safety and tolerability of MIN-102 compared with placebo Exploratory objectives- To evaluate the effects of MIN-102 on various biochemical markers in plasma and cerebrospinal fluid (CSF), and spinal cord imaging parameters. Extension Study: To evaluate the long-term effects of MIN-102 on: • Change from baseline in 6MWT and body sway • Comprehensive clinical rating scales (SSPROM and EDSS) • Quality of life • Incidence of cerebral inflammatory lesions. Exploratory objectives- To assess the long-term effects of MIN-102 on various biochemical markers in plasma.;Primary end point(s): Main Study: 1. Efficacy: Change from baseline to week 96 in the total walking distance in the 6MWT Extension Study: 1. Long-term safety and tolerability assessed in terms of AE, SAE, SUSARs, Vitals signs, ECG, Clinical Laboratory tests;Timepoint(s) of evaluation of this end point: During treatment period | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Main Study: 1. Changes from baseline to week 96 in the following: • Body sway amplitude • Comprehensive clinical rating scales (SSPROM, EDSS) • Clinician and patient global impression Scales (CGI-S, CGI-I, PGI-I) • Dynamometry • Quality of Life Assessments (EQ-5D-5L, MSWS-12, Qualiveen-SF and IIEF) • Cerebral MRI (incidence of inflammatory lesions) 2. Safety : Assessed in terms of AE, SAE, SUSARs, Vitals signs, ECG, Clinical Laboratory tests Extension Study: Changes from the baseline to the last scheduled on-study assessment for the following variables: • 6MWT • Body sway amplitude • Comprehensive clinical rating scales (SSPROM, EDSS) • Quality of Life Assessments (EQ-5D-5L, MSWS-12, Qualiveen-SF and IIEF) • Cerebral MRI (incidence of inflammatory lesions);Timepoint(s) of evaluation of this end point: During treatment period | — |
Countries
France, Germany, Hungary, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Contacts
Public ContactSílvia Pascual
Minoryx Therapeutics S.L.
Outcome results
None listed