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

Status:	Active, not recruiting
Conditions:	Endocrine
Therapuetic Areas:	Endocrinology
Healthy:	No
Age Range:	18 - 65
Updated:	3/27/2019
Start Date:	December 8, 2017
End Date:	October 2020

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

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).

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.

We found this trial at

sites

Hospital de la Pitié-Salpêtrière

Paris,

from

Paris,