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

NCT01495260 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2019-03-11

No results posted yet for this study

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.

Conditions

  • Adrenomyeloneuropathy

Interventions

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

Sponsors & Collaborators

  • Ministerio de Sanidad, Servicios Sociales e Igualdad

    collaborator OTHER_GOV

  • Fundacion Hesperia

    collaborator OTHER

  • Pujol, Aurora, M.D.

    lead INDIV

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-09-30
Primary Completion
2013-11-30
Completion
2013-11-30

Countries

  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01495260 on ClinicalTrials.gov