This subproject is one of many research subprojects utilizing theresources provided by a Center grant funded by NIH/NCRR. The subproject andinvestigator (PI) may have received primary funding from another NIH source,and thus could be represented in other CRISP entries. The institution listed isfor the Center, which is not necessarily the institution for the investigator.OBJECTIVE: The primary study objective is to identify whether CoQ10 reduces functional decline in patients with ALS. This will be done in two stages. Stage 1 will identify which of two (1800 mg or 2700 mg) doses of CoQ10 is preferable and Stage 2 compares the selected dose against placebo to assess efficacy.RESEARCH PLAN: This is a two-stage phase II, randomized, placebo-controlled, double-blind, multicenter clinical trial of high dose, solubilized CoQ10 against placebo.METHODS: The study consists of two stages. In Stage 1, a total of 105 patients will be randomized to one of three groups: CoQ10 2700 mg, CoQ10 1800 mg or placebo. Patients will be seen every other month for nine months following baseline and screening visits. Safety labs, vital signs, breathing and functional tests will be done at each visit. Following Stage 1, there will be a close out, data and statistical analysis of three months. Stage 2 begins following analysis of the selected CoQ10 dose. During Stage 2, 80 patients will be placed in one of two groups: Selected CoQ10 dose and placebo. This stage will follow the format of Stage 1 with patient visits every other month for nine months and utilizing the same test and evaluation format. In addition, the data from 70 patients in Stage 1 will be utilized in analyzing Stage 2 results.
To date the only registered pharmacological treatment for ALS is riluzole, which extends patients survival on average by approximately three months. Any treatment which is effective in reducing functional decline in ALS or improves on current therapies by counteracting the degeneration of motor neurons and consequently slowing the progression of ALS would provide a significant advance in the understanding and treatment of ALS.
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