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NCT Number: NCT01495260

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

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.

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Key information

Conditions

Adrenomyeloneuropathy Adrenal Gland Diseases Adrenal Insufficiency Adrenoleukodystrophy Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Diseases Endocrine System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hereditary Central Nervous System Demyelinating Diseases Heredodegenerative Disorders, Nervous System Intellectual Disability Leukoencephalopathies Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Peroxisomal Disorders X-Linked Intellectual Disability

Age range

18 year–64 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hospital Universitari de Bellvitge

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

N-acetylcysteine

Drug

N-acetylcysteine, 800-2400 mg daily for 2 months

lipoic acid

Drug

lipoic acid, 300-600 mg daily for 2 months

Vitamin E

Drug

vitamin E, 150-300 mg daily for 2 months

Primary outcomes

  1. oxidative lesion biomarkers

    Time frame: 12 months

    oxidative lesion biomarkers: protein, DNA and peroxidation biomarkers

Secondary outcomes

  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.

Sponsors and collaborators

Lead sponsor

Pujol, Aurora, M.D.

Indiv

Collaborators

  • Fundacion Hesperia
  • Ministerio de Sanidad, Servicios Sociales e Igualdad

Registry information

Official study 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

Important dates

Study start
2011
Primary completion
2013
Study completion
2013
First posted
Dec 19, 2011
Registry last updated
Mar 11, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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