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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-09-30

Study Completion Date

2013-11-30

Brief Summary

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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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Detailed Description

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Conditions

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Adrenomyeloneuropathy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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N-acetylcysteine, lipoic acid and vitamin E

Two Dose titration design

Group Type EXPERIMENTAL

N-acetylcysteine

Intervention Type DRUG

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

lipoic acid

Intervention Type DRUG

lipoic acid, 300-600 mg daily for 2 months

vitamin E

Intervention Type DRUG

vitamin E, 150-300 mg daily for 2 months

Interventions

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N-acetylcysteine

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

Intervention Type DRUG

lipoic acid

lipoic acid, 300-600 mg daily for 2 months

Intervention Type DRUG

vitamin E

vitamin E, 150-300 mg daily for 2 months

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

64 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No