Motor Neuron Disease Clinical Trial
Official title:
Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that
selectively affects motor neurons in the brain and spinal cord, leading to bulbar,
respiratory, and limb weakness. There is no effective treatment, and the disease usually
progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells
(MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates
for cellular therapy in ALS.
Design—A phase 1 open-safety clinical trial. 4 patients will be selected according to a
restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there
will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal
autologous stem cells infusions escalated in two intrathecal administrations in patients with
ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response,
laboratorial and magnetic resonance imaging of patients submitted to cellular escalating
doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale
and functional scales.
Conclusion: This study is a primary step before a large randomized double-blind clinical
trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS
patients, initial data of efficacy in addition to improved quality of life.
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