Trial Information

Recombinant Human Erythropoietin (r-HuEPO) in the Prevention of Neurologic Sequelae From Malignant Spinal Cord Compression: a Multi-Center, Placebo-Controlled, Phase 2 Randomized Study

For patients with malignant spinal cord compression (MSCC) who are paraparetic or paraplegic
before initiating treatment, the current treatment options provide a meager to poor chance
of neurologic recovery and the prognosis is guarded. Improving the chance of ambulation
after treatment for MSCC may dramatically improve patients' quality of life, decrease days
spent in hospital and improve survival. Steroids appear to prevent neurologic damage from
MSCC and increasing doses appear to have an increasingly protective effect, however, higher
doses are limited by an increasing incidence of serious toxicity.

Recombinant human erythropoetin has been shown to improve quality of life in patients with
anemia of chronic disease and animal models suggest that r-HuEPO may have a neuroprotective
effect. Human studies have demonstrated increased CSF concentrations of r-HuEPO after
intravenous administration, including patients with MESCC. Furthermore, there is a
suggestion that patients treated with intravenous r-HuEPO, steroids and RT may recover
ambulatory function to a greater degree and faster than patients not treated with r-HuEPO.

Ultimately the effect of r-HuEPO in improving neurologic, functional and quality of life
outcomes will need to be tested in a properly designed, large, randomized control trial.
However, in order to successfully complete this study in a timely manner, a multicenter
study will need to be performed. There are logistical issues that need to be addressed when
setting up a r-HuEPO infusion program.

Therefore, a multicenter, randomized phase 2 study of intravenous r-HuEPO, steroids and RT
will allow the investigators to address three issues: i) confirm that the logistical issues
at each center can be addressed; ii) confirm in a larger cohort of patients whether the
encouraging neurologic outcomes seen in the preliminary study can be replicated across
different settings when compared with a randomized control group; iii) ensure the safety of
EPO in this population including overall survival and incidence of subsequent TVEs with and
without EPO.