A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants

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Brief Title

A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants

Official Title

A Clinical Trial for Adrenomyeloneuropathy (AMN): Validation of Biomarkers of Oxidative Stress, and Efficacy, Tolerance and Safety of a Mixture of the Antioxidants N-acetylcysteine, Lipoic Acid and Vitamin E

Brief Summary

      X-linked adrenoleukodystrophy is a rare, demyelinating and neurodegenerative disorder, due to
      a loss of function of a fatty acid transporter, the peroxisomal ABCD1protein. Its more
      frequent phenotype, the adrenomyeloneuropathy in adults, is characterized by axonal
      degeneration in spinal cord, spastic paraparesis and a disabling peripheral neuropathy.
      Actually, there is no efficient treatment for the disease. Our work in the last twelve years
      dissecting the physiopathological basis of the disorder has uncovered an involvement of the
      oxidative stress early in the neurodegenerative cascade. In a preclinical trial we have
      identified an antioxidant cocktail that efficiently reverse the clinical symptoms and the
      axonal degeneration in the mouse model for the disease. We propose the translation of the
      results to an open trial to test the tolerance and effectiveness of these drugs in the
      correction of the previously identified oxidative lesion biomarkers, as a first step to a
      randomized versus placebo, multicentric and international trial. You will be clinically
      explored and assessed in the Hospital Universitari of Bellvitge (HUB) using clinical scales
      for spasticity, disability, electroneurogram and cranial and spinal Nuclear Magnetic
      resonance (NMR). The information will be collected in a data base that will be of great value
      to improve the present attention and the future follow-up to facilitate your inclusion in
      therapeutic randomized, double blind, against placebo clinical trials.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

oxidative lesion biomarkers

Secondary Outcome

 clinical parameters

Condition

Adrenomyeloneuropathy

Intervention

N-acetylcysteine

Study Arms / Comparison Groups

 N-acetylcysteine, lipoic acid and vitamin E
Description:  Two Dose titration design

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

13

Start Date

September 2011

Completion Date

November 2013

Primary Completion Date

November 2013

Eligibility Criteria

        Inclusion Criteria:

          -  Symptomatic AMN patients,

          -  18-64 years old,

          -  male and female,

          -  clinically and biochemically diagnosed;

          -  females must be obligated heterozygotes or must have gene mutation identified.

        Exclusion Criteria:

          -  Pregnant and lactation in females,

          -  Cerebral inflammatory disease with cognitive disorder, and/or

          -  need the help of two walking sticks,

          -  epilepsy,

          -  hypersensibility to cysteine related compounds,

          -  transaminases 2 fold up normal values.
      

Gender

All

Ages

18 Years - 64 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Spain

Location Countries

Spain

Administrative Informations


NCT ID

NCT01495260

Organization ID

XAMNANTIOXAP2010


Responsible Party

Sponsor

Study Sponsor

Onofre, Aurora Pujol, M.D.

Collaborators

 Ministerio de Sanidad, Servicios Sociales e Igualdad

Study Sponsor

, , 


Verification Date

September 2016