Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: examining a more efficient trial design.

ABSTRACT:

Objective

To use a historical placebo control design to determine whether lithium carbonate slows progression of amyotrophic lateral sclerosis (ALS).

Methods

A phase II trial was conducted at 10 sites in the Western ALS Study Group using similar dosages (300-450 mg/day), target blood levels (0.3-0.8 mEq/L), outcome measures, and trial duration (13 months) as the positive trial. However, taking riluzole was not a requirement for study entry. Placebo outcomes in patients matched for baseline features from a large database of recent clinical trials, showing stable rates of decline over the past 9 years, were used as historical controls.

Results

The mean rate of decline of the ALS Functional Rating Scale-Revised was greater in 107 patients taking lithium carbonate (-1.20/

SUBMITTER: Miller RG 

PROVIDER: S-EPMC3171956 | biostudies-literature | 2011 Sep

REPOSITORIES: biostudies-literature