inical Studies
Multiple Myeloma
Trial 1 was a Phase 2, multicenter, randomized open label study in patients with relapsed multiple myeloma who were refractory to their last myeloma therapy and had received lenalidomide and bortezomib. Patients were considered relapsed if they had achieved at least stable disease for at least one cycle of treatment to at least one prior regimen and then developed progressive disease. Patients were considered refractory if they experienced disease progression on or within 60 days of their last therapy. A total of 221 patients were randomized to receive Pomalyst alone or Pomalyst with Low dose Dex. In Trial 1, the safety and efficacy of Pomalyst 4 mg, once daily for 21 of 28 days, until disease progression, were eva luated alone and in combination with Low dose Dex (40 mg per day given only on Days 1, 8, 15 and 22 of each 28-day cycle for patients 75 years or younger, or 20mg per day given only on Days 1, 8, 15 and 22 of each 28-day cycle for patients greater than 75 years of age). Patients in the Pomalyst alone arm were allowed to add Low dose Dex upon disease progression.
Table 5 summarizes the baseline patient and disease characteristics in study Trial 1. The baseline demographics and disease characteristics were balanced and comparable between the study arms.
Table 5: Baseline Demographic and Disease-Related Characteristics – Trial 1 Pomalyst
(N=108) Pomalyst/Low
dose Dex
(N=113)
Patient Characteristics
Median Age, years (range)
61 (37, 88) 64 (34, 88)
Age Distribution n (%)
< 65 years
greater than or equal to 65
years 65 (60.2)
43 (39.8) 60 (53.1)
53 (46.9)
Sex n (%)
Male
Female 57 (52.8)
51 (47.2) 62 (54.9)
51 (45.1)
Race/Ethnicity n (%)
White
Black or African American
All Other Race 86 (79.6)
16 (14.8)
6 (5.6) 92 (81.4)
17 (15)
4 (3.6)
ECOG Performance n (%)
Status 0-1 95 (87.9) 100 (88.5)
Disease Characteristics
Number of Prior Therapies
Median, (Min, Max) 5 (2, 12) 5 (2,13)
Prior transplant n (%) 82 (75.9) 84 (74.3)
Refractory to bortezomib
and lenalidomide n (%) 64 (59.3) 69 (61.1)
Table 6 summarizes the analysis results of overall response rate (ORR) and duration of response (DOR), based on assessments by the Independent Review Adjudication Committee for the treatment arms in Study 1. Overall response rate did not differ based on type of prior anti-myeloma therapy.
Table 6: Trial 1 Results 1. ORR =PR+CR per EBMT criteria,
2. Results are prior to the addition of dexamethasone
NE = not established (the median has not yet been reached), CI: confidence interval
Pomalyst 2
(N=108) Pomalyst/
Low dose Dex
(N = 113)
Response
Overall Response Rate
(ORR)1, n (%) 8 (7.4) 33 (29.2)
95% CI for ORR (%) (3.3, 14.1) |
