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A Phase II Trial of Nilotinib in the Treatment of Patients With c-KIT Mutated Advanced Acral and Mucosal Melanoma

Phase 2
18 Years
Open (Enrolling)
Mucosal Lentiginous Melanoma, Acral Lentiginous Malignant Melanoma

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Trial Information

A Phase II Trial of Nilotinib in the Treatment of Patients With c-KIT Mutated Advanced Acral and Mucosal Melanoma

NICAM has a two step consent process. Patients diagnosed with advanced acral or mucosal
melanoma first consent for study registration and undergo screening tests including testing
samples of melanoma tissue for the c-KIT mutation.

Following confirmation of the c-KIT mutation, patients are asked to consent to study entry
with continuation of screening. Eligible patients then enter the study and commence taking
nilotinib tablets twice a day for as long as clinical benefit is maintained.

Inclusion Criteria:

1. Patients with c-KIT mutated histologically proven advanced mucosal or acral melanoma
in which the mutation is not known to be associated with nilotinib resistance.

2. Advanced mucosal and acral melanoma defined as unresectable locally advanced or
metastatic disease

3. The presence of one or more clinically or radiologically measurable lesions at least
10mm in size

4. Age 18 or greater

5. ECOG performance status 0, 1 or 2

6. Life expectancy greater than 12 weeks

7. At least 14 days since any major surgery

8. The capacity to understand the patient information sheet and ability to provide
written informed consent

9. Willingness and ability to comply with scheduled visits, treatment plans, laboratory
tests and other study procedures

10. Women must not be pregnant or lactating with no intention of pregnancy during study
treatment. Women of child bearing potential must have a negative serum pregnancy
test prior to study entry (even if surgically sterilised). Men and women of
childbearing potential must use adequate birth control measures (e.g. abstinence,
oral contraceptives, intrauterine device, barrier method with spermicide, implantable
or injectable contraceptives or surgical sterilisation) for the duration of the study
and should continue such precautions for 6 months after receiving the last study

11. Serum alanine transaminase (ALT) or serum aspartate aminotransferase ≤2.5 x upper
limit of normal (ULN) and total serum bilirubin ≤1.5 x ULN

12. Serum creatinine ≤1.5 x ULN

13. Serum lipase and amylase <1.5 x ULN

14. Haemoglobin ≥9.0 g/dL, absolute neutrophil count ≥1.5 x 109/L, platelets ≥100 x 109/L

15. Prothrombin time (PT) ≤1.5 x ULN

16. Able to swallow and retain oral medication.

Exclusion Criteria:

1. Intracranial disease, unless there has been radiological evidence of stable
intracranial disease > 6 months. In the case of a solitary brain metastasis, evidence
of a disease-free interval of at least 3 months post surgery. All patients
previously treated for brain metastases must be stable off corticosteroid therapy for
at least 28 days

2. Women who are pregnant, nursing, or planning to become pregnant during the course of
the trial

3. Men who plan to father a child during the course of the trial

4. Use of any investigational drug within 30 days prior to screening (both cancer and
non cancer treatments)

5. Use of herbal or chinese medication

6. Use of therapeutic coumarin derivatives (ie warfarin, acenocoumarol, phenprocoumon)

7. Significant cardiac disease including patients who have or who are at significant
risk of developing prolongation of QTc

8. Severe and/or uncontrolled medical disease

9. Known chronic liver disease

10. Past medical history of chronic pancreatitis

11. Known HIV infection

12. Previous radiotherapy to 25% or more of the bone marrow

13. Radiation therapy in the 4 weeks prior to study entry

14. Prior exposure to a tyrosine kinase inhibitor

15. Known lactose intolerance

16. Any malabsorption syndrome (i.e. partial gastrectomy, small bowel resection, Crohn's
disease or ulcerative colitis).

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Proportion of participants with the c-KIT mutation who remain progression free at 6 months.

Outcome Description:

Progression free survival times will be measured from the date of enrolment into the treatment phase until the first date (following start of treatment) of either death or confirmed progressive disease according to RECIST.

Outcome Time Frame:

6 months

Safety Issue:


Principal Investigator

James Larkin, MA BM BCh MRCP PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Royal Marsden NHS Foundation


United Kingdom: Medicines and Healthcare Products Regulatory Agency

Study ID:




Start Date:

December 2009

Completion Date:

Related Keywords:

  • Mucosal Lentiginous Melanoma
  • Acral Lentiginous Malignant Melanoma
  • c-KIT mutation
  • advanced disease
  • tyrosine kinase inhibitors
  • Melanoma