Clinical trial NCT03231878: A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Adrenoleukodystrophy
Lead sponsor: Minoryx Therapeutics, S.L.
Sponsor class: INDUSTRY
First posted: 2017-07-27
Enrollment: 105
Sex: MALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility 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.

Primary outcomes:
measure: To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.
timeFrame: in 96 weeks
Secondary outcomes:
measure: To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.
timeFrame: in 96 weeks
measure: SSPROM (Severity Score System for Progressive Myelopathy )
timeFrame: in 96 weeks
measure: EDSS (Expanded Disability Status Scale )
timeFrame: in 96 weeks
measure: Quality of life scales (Euroqol)
timeFrame: in 96 weeks
measure: Incidence of cerebral inflammatory lesions
timeFrame: in 96 weeks
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University Medical Center
state: California
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Paris
country: France
facility: Hospital de la Pitié-Salpêtrière
city: Leipzig
country: Germany
facility: Universitat Leipzig Klinik and Poliklinik für Neurologie
city: Budapest
country: Hungary
facility: Institute of Genomic Medicine and Rare Disorders
city: Milano
country: Italy
facility: Instituto Neurologico Carlo Besta
city: Amsterdam
country: Netherlands
facility: Academish Medisch Centrum
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebrón
city: London
country: United Kingdom
facility: National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)
Study officials:
References: