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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01495260
Recruitment Status : Completed
First Posted : December 19, 2011
Last Update Posted : March 11, 2019
Ministerio de Sanidad, Servicios Sociales e Igualdad
Fundacion Hesperia
Information provided by (Responsible Party):
Onofre, Aurora Pujol, M.D.

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.

Condition or disease Intervention/treatment Phase
Adrenomyeloneuropathy Drug: N-acetylcysteine Drug: lipoic acid Drug: vitamin E Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : September 2011
Actual Primary Completion Date : November 2013
Actual Study Completion Date : November 2013

Arm Intervention/treatment
Experimental: N-acetylcysteine, lipoic acid and vitamin E
Two Dose titration design
Drug: N-acetylcysteine
N-acetylcysteine, 800-2400 mg daily for 2 months

Drug: lipoic acid
lipoic acid, 300-600 mg daily for 2 months

Drug: vitamin E
vitamin E, 150-300 mg daily for 2 months

Primary Outcome Measures :
  1. oxidative lesion biomarkers [ Time Frame: 12 months ]
    oxidative lesion biomarkers: protein, DNA and peroxidation biomarkers

Secondary Outcome Measures :
  1. clinical parameters [ Time Frame: 2, 6 and 12 months ]
    spasticity, disability,electroneurograms and evocated potentials. Cranial and spinal NMR will be done at the beginning and the end of the trial.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01495260

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Hospital Universitari de Bellvitge
L'Hospitalet de LLobregat, Barcelona, Spain, 08907
Sponsors and Collaborators
Onofre, Aurora Pujol, M.D.
Ministerio de Sanidad, Servicios Sociales e Igualdad
Fundacion Hesperia
Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Onofre, Aurora Pujol, M.D. Identifier: NCT01495260    
Other Study ID Numbers: XAMNANTIOXAP2010
First Posted: December 19, 2011    Key Record Dates
Last Update Posted: March 11, 2019
Last Verified: September 2016
Keywords provided by Onofre, Aurora Pujol, M.D.:
Additional relevant MeSH terms:
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Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases
Vitamin E
Thioctic Acid
Growth Substances