Multiple Sclerosis (MS) - Relapsing-remitting Clinical Trial
Official title:
A Phase I/II, Open-Label Pilot Trial to Evaluate the Safety of Rapamune (Sirolimus) in Patients With Multiple Sclerosis
The purpose of this study is to determine the safety and tolerability of the drug sirolimus in patients with multiple sclerosis (MS) who have failed other treatments.
MS is a chronic autoimmune disease of the central nervous system in which myelin, the
protein sheath that protects nerve cells, is degraded by T cells and macrophages, leading to
an eventual loss of neurologic function. MS can be classified as either relapsing-remitting,
in which patients experience worsening in symptoms followed by partial or complete recovery
of function; or progressive, in which patients have a gradual increase in symptoms, with or
without relapses. Standard treatments used to treat relapsing-remitting MS are only modestly
effective and may be associated with significant toxicity. There is a need to develop
therapies with lower toxicities that can be administered early during the course of disease
and have the potential to stop disease progression altogether. Sirolimus has been
demonstrated to provide potent immunosuppression in recent clinical trials involving kidney
transplantation, and may help people with autoimmune diseases like MS. This study will
determine the benefit of sirolimus in MS patients.
Blood and urine collection will occur at screening. Participants will take daily doses of
sirolimus for 6 months. There will be nine study visits; they will occur at Days 14, 28, 42,
56, 90, 120, 150, 180, and 225. Medication adverse events, concomitant medications, and
vital signs will be recorded at Visits 1 through 8. At all visits, patient compliance to the
sirolimus regimen will be measured, and blood and urine collection will occur. Physical and
neurological exams, magnetic resonance imaging (MRI) brain scans, MS status tests, and a
chest x-ray will be conducted at selected times throughout the study.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment