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German Pilot-Study of Thalidomide

December 01, 2005

Charite University starts investigational therapy of ALS with oral administration of thalidomide. The rationale for this study is based on the anti-inflammatory properties of thalidomide through the modulation of inflammatory cytokines.

The primary aim of the drug trial is to determine whether treatment with thalidomide is safe and well tolerated in conjunction with riluzole and whether patients with ALS can tolerate daily doses of up to 400 mg.

Primary Goals:

to evaluate the long-term safety and tolerability of thalidomide
to compare the total number of adverse events, abnormal laboratory tests, and number of patients who completed the study between groups

Secondary Goals:

to evaluate the clinical effect of two oral doses of the thalidomide on the rate of functional decline in ALS patients measured by the ALS Functional Rating Scale-revised (ALS-FRS-R) over a 24 week treatment period
to investigate the effects of thalidomide on pulmonary function (forced vital capacity) over a 24 week treatment period
to evaluate the sleep quality and somnolence using the Epworth Sleeping Scale: ESS ≥ 18
to evaluate the frequency and severity of sensory neuropathy using the inflammatory neuropathy cause and treatment sensory sum score � ISS ≥ 4
to evaluate the frequency of thrombotic events
to determine the number of patients who require continuous non-invasive ventilation or invasive ventilation
to determine the number of patients who require percutanous endoscopic gastrostomy (PEG)
to evaluate the survival time or the time point until invasive ventilation is started

Enrollment: 40 patients with ALS
Expected completion: December 2006

Study Overview:
The study drug will provide 50 mg tablets. Patients will be instructed to take 2 tablets orally once a day during the evening at least 60 minutes after a meal. Thalidomide will be administered starting at 100 mg (Group 1) for 6 weeks. Thereafter, the dose will be increased every week by 50mg until reaching the dose of 400 mg/day. This treatment is continued for 12 weeks. Thalidomide is administered in conjunction with the standard treatment of riluzole (100mg/day).