Phase 1 Study of Leukemic Dendritic Cell Vaccination in Patients With Acute Myeloid Leukemia
DCPrime is testing an allogeneic (non-self cells, standardised product) DC-based
immunotherapy in cancer patients. The technology consists of sustainable dendritic
progenitor cells (named "DCOne™") and a proprietary method to expand these and to create
functional mature dendritic cells (DCP-001).
AML is a fast growing form of leukemia that particularly in the elderly (>60) is life
threatening. As age is an important factor in determining the success of AML treatment,
overall, AML has a bad prognosis as only 24% of the patients are alive 5 years after
Without treatment AML is fatal, usually within months. Chemotherapy can cure patients and
prolong survival in responders; however, chemotherapy is also quite toxic and can cause
substantial morbidity and mortality. The most commonly prescribed first line therapy for
patients with AML is a combination of an anthracycline and cytarabine; in the Western world
the anthracycline is either daunorubicin or idarubicin. Post remission therapy
(consolidation therapy) is usually given.
There is therefore substantial medical need for new treatment modalities. One of the major
difficulties regarding development of new agents is that relatively low response rates and
toxicity issues have been in the way of approval of new agents. Immunotherapy, in particular
with the therapeutic vaccines, is expected to have potential in prevention of recurrence of
disease after cytoreductive therapy. Any drug that could prevent or reduce minimal residual
disease in the population is likely to meet a strong medical need for this population of
high risk patients.
In this phase 1 trial consecutive eligible patients will be treated until 12 patients have
completed the study. Patients will be started with the vaccination program within 2 months
after having achieved complete remission or in patients who have stable disease over at
least a 2 month period.
The first cohort (n=3) will receive 4 bi-weekly vaccinations of 1x10E7 DCP-001, the second
cohort (n=3) will receive 4 bi-weekly vaccinations of 2.5x10E7 DCP-001, and the last cohort
(n=3) will receive 4 bi-weekly vaccinations of 5x10E7 DCP-001. The Dose Limiting Toxicity
(DTL) is defined as non-hematological toxicity of ≥ 3 according to common toxicity criteria
v3.0. The 4th cohort (matched for HLA-A2) will receive 4 vaccinations of the highest dose
(5x10E7 DCP-001) or, in case this turned out to be toxic (as determined by the vaccination
profile of cohort 1, 2 and 3), this group will receive the Maximum Tolerated Dose (MTD).
DCP-001 vaccine is presented as a direct injectable sterile cell suspension consisting of
irradiated mature dendritic cells in cryopreservation solution packed in vials. The vaccine
will be administered intradermally.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label
Safety/Feasibility of DCP-001 vaccination
The primary endpoint of this study will be the safety and feasibility of DCP-001 vaccination in AML patients. Safety will be assessed by means of laboratory evalulations, lhysical examination, vital sign assessments and adverse events recording. Clinical efficacy is assessed by the presence of leukemic cells in blood and bonemarrow and physical examination at baseline, during and after vaccination
three months and follow up after study completion
The Netherlands: Central Committee on Research involving Human Subjects