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

Minoryx Therapeutics, S.L.10 sites in 8 countries105 target enrollmentDecember 8, 2017

ConditionsAdrenoleukodystrophy

InterventionsMIN-102 Placebos

Overview

Phase: Phase 2
Intervention: MIN-102
Conditions: Adrenoleukodystrophy
Sponsor: Minoryx Therapeutics, S.L.
Enrollment: 105
Locations: 10
Primary Endpoint: To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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).

Registry

clinicaltrials.gov

Start Date

December 8, 2017

End Date

March 6, 2025

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

Male

Investigators

Sponsor

Minoryx Therapeutics, S.L.

Responsible Party

Sponsor

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.

Arms & Interventions

Active

Intervention: MIN-102

Placebo

Intervention: Placebos

Outcomes

Primary Outcomes

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 Outcomes

To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.(in 96 weeks)
SSPROM (Severity Score System for Progressive Myelopathy )(in 96 weeks)
Quality of life scales (Euroqol)(in 96 weeks)
Incidence of cerebral inflammatory lesions(in 96 weeks)
EDSS (Expanded Disability Status Scale )(in 96 weeks)