A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants
Status:
Completed
Trial end date:
2013-11-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Onofre, Aurora Pujol, M.D.
Collaborators:
Fundacion Hesperia Ministerio de Sanidad, Servicios Sociales e Igualdad