A Phase 2 interventional study of N-acetylcysteine and lipoic acid in Adrenomyeloneuropathy, sponsored by Pujol, Aurora, M.D.. Completed at 1 site in Spain. Open to participants aged 18 Years to 64 Years. Per ClinicalTrials.gov, last updated 2019-03-11.
Sponsored by Pujol, Aurora, M.D. · Phase 2, Interventional, and Treatment
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.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 13 is below the median of 31 across 40 interventional studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Pujol, Aurora, M.D. is the lead sponsor of 4 studies on the registry; none are open to participants now.
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Exclusion Criteria:
Two Dose titration design
Drug: N-acetylcysteine · Drug: lipoic acid · Drug: vitamin E
N-acetylcysteine, 800-2400 mg daily for 2 months
lipoic acid, 300-600 mg daily for 2 months
vitamin E, 150-300 mg daily for 2 months
oxidative lesion biomarkers
oxidative lesion biomarkers: protein, DNA and peroxidation biomarkers
Time frame: 12 months
clinical parameters
spasticity, disability,electroneurograms and evocated potentials. Cranial and spinal NMR will be done at the beginning and the end of the trial.
Time frame: 2, 6 and 12 months
This study is completed, as verified in Sep 2016. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Acetylcysteine · Thioctic Acid · Vitamin E
Pujol, Aurora, M.D.