Leucovorin for the Treatment of 5 q Minus Syndrome
Status:
Completed
Trial end date:
2002-07-01
Target enrollment:
Participant gender:
Summary
The 5 q minus syndrome is a condition that occurs due to a missing segment of chromosome 5 in
the normal genetic make-up of the cells responsible for forming blood cells. The condition
causes patients to have the inability to make blood normally. Many patients with this
syndrome need transfusions of red blood cells, platelets, and/or white blood cells. Low
levels of platelets may cause the patient to bleed easily and low levels of whit blood cells
make the patient susceptible to infections. A small number of patients with 5 q minus
syndrome develop leukemia, which is often untreatable with chemotherapy.
Researchers believe that one of the genes missing in 5 q minus syndrome is the gene
responsible for making folic acid active in the body. Folic acid is a vitamin required for
normal blood production.
The purpose of this study to test the effectiveness of a drug called leucovorin for the
treatment of 5 q minus syndrome. Leucovorin is an active form of the vitamin folic acid that
does not require the missing genes to activate it.
Patients participating in this study may or may not improve with leucovorin treatment.
However, the study will improve researchers understanding of the disease and may lead other
potential therapies for the disease.
Phase:
Phase 2
Details
Lead Sponsor:
National Institutes of Health Clinical Center (CC)