Table 2.
Recent interventional clinical trials of pharmacological agents for the treatment of breast cancer-related lymphedema
| Trial | Agent | Phase | N | Design | Primary outcome | Results |
|---|---|---|---|---|---|---|
| NCT02257970 [136] | Ketoprofen | 4 | 89 | Open label exploratory, then randomized, double-blind, placebo-controlled trial of pts with arm or leg LE |
Exploratory Phase: Derm. score Randomized Phase: Skin thickness |
Exploratory Phase: Score improvement − 3.4 (p < 0.0001) Randomized Phase: Skin thickness reduced (P = 0.01), but no change in limb volume |
| NCT04243837 [137] | LYT-100 | 1/2 | 100 |
Part 1: Dose escalation Part 2: Food effect study Part 3: Randomized, double-blind, placebo-controlled trial in pts with Stage 1 or 2 BCRL |
Part 1: safety, MTD Part 2: PK, food effect Part 3: Efficacy signals |
Part 1: well tolerated, MTD not reached at 100–1000 mg BID Part 2: 19% lower exposure in fed vs. fasting Part 3: Results not yet reported |
| NCT02994771 [138] | Lymfactin® | 1 | 15 | Single-arm trial of adenoviral VEGF-C combined with VLNT in pts with BCRL | Safety | Well tolerated, no DLT at maximum dose |
| NCT03658967 [139] | Lymfactin® | 2 | 39 | Double-blind, randomized, placebo-controlled trial of adenoviral VEGF-C combined with VLNT in pts with BCRL | Arm volume; Lymphatic flow by lymphoscintigraphy; QoL per LQOLI | Company press release states that results were "inconclusive" [140] |
| NCT04390685 | Tacrolimus, topical | 1/2 | 60 | Single-arm study of tacrolimus ointment following ALND for BCRL prevention | Arm volume change by water displacement | Not yet reported |
| NCT04541290 | Tacrolimus, topical | 1/2 | 20 | Single-arm study of tacrolimus ointment in pts with existing BCRL | Arm volume change by water displacement | Not yet reported |
| NCT02494206 [141] | QBX258 | NS | 9 | Single-arm study of anti-IL4/ anti-IL13 blockade in Stage 1 or 2 BCRL | Arm volume change by perometry | Arm volume significantly increased relative to baseline (P = 0.046) |
ALND axillary lymph node dissection, BCRL breast cancer-related lymphedema, BID twice-daily, LE lymphedema, LQLOLI lymphedema quality of life inventory, MTD maximum tolerated dose, NS not specified, QoL quality of life, VEGF-C vascular endothelial growth factor C, VLNT vascularized lymph node transfer