Randomized Controlled Phase II Clinical Trial on mRNA Electroporated Autologous Dendritic Cells for Stage III/IV Melanoma Patients Who Are Disease-free Following the Local Treatment of Macrometastases
This is an open label, 2-arm, 1-stage, randomized controlled phase II study in patients with
AJCC stage IIIB/C & -IV melanoma. At baseline tumor assessment (using total body
FDG-PET/CT), patients should be free from measurable tumor lesions (according to RECISTv1.1
definitions) following prior local therapy (e.g. following surgical resection, isolated limb
perfusion, radiofrequency ablation, cryotherapy, radiotherapy, electrochemotherapy, …).
Patients should not have symptomatic non-measurable tumor lesions (e.g. bone metastasis, or
pleural effusion), and lesions treated by prior local therapy should be free from
progression. Patients should not have received any prior systemic therapy (non-experimental
- Patients will be randomized between two treatment arms (Arm-A and -B). In study Arm-A,
patients will receive DC-administrations during one year following randomization.
Salvage treatment by local therapies will be allowed during the study treatment in
Arm-A. In study Arm-B, patients will initiate DC-administrations only after documented
recurrence of the melanoma that cannot be salvaged by local therapy.
- The primary endpoint of this clinical trial is to determine the rate (%) of patients
who are free from macrometastases (: measurable tumor lesions and symptomatic
non-measurable tumor lesions) at 1-year (= 52 weeks) after randomization.
Patients treated on Arm-B will serve as a contemporary control-arm to help interpreting the
outcome of patients treated in Arm-A. By design (phase II) this trial will not be powered to
statistically prove a predefined difference between the two study arms (this would require a
phase III design). Patients treated in Arm-B will be able to initiate immunotherapy with
autologous DC at the time of recurrence that can not be salvaged by local therapy.
Documentation of the anti-tumor activity and survival following DC-treatment at recurrence
in Arm-B patients will be a secondary objective of this clinical trial.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
1-year disease free survival percentage
Patients will be evaluated 52 weeks following randomization for their melanoma disease status (= macro-metastases present or absent)
1-year following recruitment date
Bart Neyns, Phd Md
Belgium: Federal Agency for Medicinal Products and Health Products