SAFETY AND PHARMACOKINETICS STUDY OF ARIMOCLOMOL IN ALS

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is a phase IIA double-blind study to assess the safety and CSF penetration of 3 doses of Arimoclomol. Eighty subjects from multiple centers will be randomized to 1 of 4 groups: placebo or Arimoclomol at 25, 50, or 100mg t.i.d., and receive medication for 12 weeks. After 4 weeks of treatment, subjects will be admitted to the GCRC for a blood and CSF pharmacokinetic (PK) study. Each dose will be assessed based on the safety and the PK data and the best two dosages for the phase IIb study will be selected.