Phase II Study for the Determination of Efficacy and Tolerability of the Combination of Valproic Acid and Lenalidomide in the Treatment of Patients With Myelodysplastic Syndrome With Favorable Risk Profile
Treatment will be administered as continuous therapy, i.e. it should be taken on each day as
described below without treatment interruption as long as no criteria for termination of
treatment are met. After two years the primary endpoint will be evaluated. Non-responders
will be taken off study after 4 months of therapy. Patients who relapse after an initial
response to study treatment can receive one attempt to re-start therapy after a short
duration of discontinuation.
Treatment with Valproic Acid starts at day 1. The dose of Valproic Acid is slowly increased.
In the morning of day 13 trough level of Valproic Acid will be checked. The target range
will be 50-110 µg/l. The dose of Valproic Acid will be adjusted depending on the trough
In the first eight weeks of therapy weekly controls of Valproic Acid levels are required.
Thereafter, Valproic Acid levels will be checked every four weeks.
The planned dose of lenalidomide is 10 mg/day, orally as continuous therapy. Dosing will be
in the morning at approximately the same time each day. Capsules may be taken before or
after a meal. In the course of the study the dose will be adjusted to the results of the
Only one cycle of study drug (28 days) will be supplied to the patient every four weeks.
Patients experiencing adverse events may need study treatment modifications.
During treatment with study medication weekly control visits for the detection of adverse
events are required during the first eight weeks, thereafter the patient must be seen every
Therapeutic success is evaluated in 4-weekly intervals. Bone marrow will be examined after
12 weeks and after 48 weeks or in case of premature study termination
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Norbert Gattermann, Professor
Department of Hematology, Oncology and Clinical Immunology
Germany: Federal Institute for Drugs and Medical Devices