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

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Brief Title

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

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).
    


Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.

Secondary Outcome

 To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.

Condition

Adrenoleukodystrophy

Intervention

MIN-102

Study Arms / Comparison Groups

 Active
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

105

Start Date

December 8, 2017

Completion Date

June 2021

Primary Completion Date

June 2021

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.
      

Gender

Male

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT03231878

Organization ID

MT-2-01


Responsible Party

Sponsor

Study Sponsor

Minoryx Therapeutics, S.L.


Study Sponsor

, , 


Verification Date

November 2020