The phase 1/2 ASCENT study is the first study with melflufen outside multiple myeloma.
The study is an open-label study with melflufen and dexamethasone for patients with AL-amyloidosis. The patients have undergone at least one previous line of therapy. Melflufen is administered every 28-days, in combination with dexamethasone. The first patient was enrolled in August 2020 and approximately 40 patients will be included, up to 18 of them should participate in the phase 1 trial.
The primary efficacy parameters in the phase 1 study are safety and tolerability. The study will also be used to define the optimal dosage for the phase 2 study.
The primary efficacy parameter in the phase 2 study will be the Overall Response Rate based on the percentage of patients who achieve partial or improved response after four treatment cycles. The patients continue treatment until the completion of cycle 8, unless they achieve a complete response rate after cycle 4, reach a plateau with partial or very good partial response after cycle 4, not respond after cycle 2, or experience unacceptable toxicity.