Phase II Multicentric Uncontrolled National Trial Assessing the Efficacy of Nilotinib in First or Second Line Treatment of Primary Melanomas , Stage III Unresectable Melanomas, or Stage IV Melanomas With c-KIT Mutation or Amplification.
NILOMEL is a phase II multicentric uncontrolled open national trial assessing the efficacy
of Nilotinib in first or second line treatment of primary melanomas , stage III
unresectable melanomas, or Stage IV melanomas with c-KIT mutation or amplification (in case
of c-KIT amplification, no B-RAF nor N-Ras mutation should be detected). The primary
objective is overall response rate (partial and complete response) according to RECIST 1.1
criteria, assessed using CT-SCAN (stage IV melanoma) or MRI (unresectable melanoma) after 6
months therapy with Nilotinib 800 mg/d. Secondary objectives include:
- Disease control rate (complete, partial response and stable disease) according to
- Metabolic response rate (TEP-SCAN)
- Tolerance NCI CTCAE Version 3.0
- Biomarkers associated to response and disease control (evaluated at M0, M1 and M6).
Protein analysis of c-KIT, PI3K, MAPK and STAT signalling pathways as well as PDGFR and
Ephrin signalling pathways.
Patients with progressive disease after 3 months therapy will be withdrawn. Patient with
stable disease after 3 months will continue Nilotinib until evaluation at 6 months. Patients
with stable disease or progressive disease at 6 months will continue Nilotinib until
The trial has been planned using a one-stage design (Fleming TR) . We considered that a
response rate under 7.5% would define the null hypothesis of no efficacy . To detect a
response rate of 30% or more with power 90% using a one-sided test at the 0.05 level, 25
patients have to be recruited.
Accrual for 2.5 years total study duration: 3 years
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Partial or complete response per Response Evaluation Criteria in Solid Tumors (RECIST).
Celeste Lebbe, MD, PhD
Hôpital Saint-Louis, Paris, France
France: Ministry of Health