A Single Agent Phase II Study of Romidepsin (Depsipeptide, FK228) in the Treatment of Cutaneous T-cell Lymphoma (CTCL)

GPI-04-0001 was a Phase II, non-randomized, open label, single arm study that was conducted at approximately 30 sites, primarily in the United States, Europe and Russia. It assessed the efficacy, safety, and tolerability of romidepsin as a treatment for cutaneous T-cell lymphoma (CTCL). Study patients (pts) received romidepsin in a dose of 14 mg/m\^2 intravenously over 4 hours on Days 1, 8 and 15 of each 28-day cycle. The duration of study treatment was 6 cycles although pts who showed an objective response or stable disease could continue to receive therapy, at the discretion of the investigator, until disease progression or another withdrawal criterion was met.

Responses were evaluated according to a composite assessment (Objective Primary Disease Response Evaluation Criteria \[OPDREC\]) that included cutaneous manifestations of disease, lymph node involvement, and circulating malignant T-cells (Sézary cells). Skin involvement was measured using a weighted body surface area skin assessment tool (WBSA/SWAT) or an erythroderma score, depending upon the pt's disease. Disease response was assessed by the Investigators and an Independent Response Review Committee (IRRC) with the IRRC assessment considered supportive of the Investigator's evaluations using the following criteria: Complete Response (CR): * Complete resolution of skin patches, skin plaques, and skin tumors, or erythroderma * No evidence of abnormal lymph nodes * Absence of circulating Sézary cells. * No evidence of new tumors (cutaneous or non-cutaneous) * Findings confirmed by skin biopsy Clinical complete response (CCR): \- Same as CR but without skin biopsy Partial Response (PR): * ≥50% improvement in the summation of (change in Skin + change in Lymph Node + change in Peripheral Blood) with * At least \>30% improvement in Skin and * No worsening in Lymph Node or Sézary cells. * No evidence of new tumors (cutaneous/non-cutaneous) Stable Disease (SD): * Not enough improvement or worsening in the summation of (change in Skin + change in Lymph Node + change in Peripheral Blood to qualify as PR or PD * No evidence of new tumors (cutaneous/non-cutaneous) SD90: \- SD90 was defined as documented evidence of SD for at least 90 Days Duration Progressive Disease (PD): * Evidence of new tumor (cutaneous or non-cutaneous), OR * \>25% worsening in the summation of (change in Skin + change in Lymph Node + change in Peripheral Blood) with \>15% worsening in change in Skin.