A Trial Looking at Nilotinib to Treat Acral and Mucosal Melanoma Skin Cancer That Has Spread (NICAM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01395121
Recruitment Status : Completed
First Posted : July 15, 2011
Last Update Posted : June 8, 2017
Royal Marsden NHS Foundation Trust
Information provided by (Responsible Party):
Institute of Cancer Research, United Kingdom

Brief Summary:
The aim of this study is to see if a drug called nilotinib (Tasigna®) is effective in the treatment of patients with a rare group of acral and mucosal melanomas that have a change (mutation) in a protein called cKIT. Nilotinib interferes with signalling inside cells with this mutation and it is believed that this may lead to shrinkage of tumours. Acral melanomas are found on the palms and soles and mucosal melanomas start inside body cavities rather than on the skin.

Condition or disease Intervention/treatment Phase
Mucosal Lentiginous Melanoma Acral Lentiginous Malignant Melanoma Drug: nilotinib Phase 2

Detailed Description:

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Trial of Nilotinib in the Treatment of Patients With c-KIT Mutated Advanced Acral and Mucosal Melanoma
Actual Study Start Date : December 2009
Actual Primary Completion Date : December 12, 2016
Actual Study Completion Date : December 12, 2016

Arm Intervention/treatment
Experimental: nilotinib
nilotinib 400mgs oral tablets
Drug: nilotinib
nilotinib 400 mgs orally twice daily until disease progression or withdrawal from treatment
Other Name: Tasigna

Primary Outcome Measures :
  1. Proportion of participants with the c-KIT mutation who remain progression free at 6 months. [ Time Frame: 6 months ]
    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.

Secondary Outcome Measures :
  1. toxicity of treatment [ Time Frame: evaluated every 4 weeks whilst the patient is on treatment (on average estimated to be between 4 and 52 weeks) ]
    Treatment related toxicity will be assessed at each clinic visit approximately every 4 weeks whilst the patient continues on study treatment. Study treatment will continue until the patient relapses or is withdrawn from study therapy (on average estimated to be between 4 and 52 weeks).

  2. response at 12 weeks [ Time Frame: tumours measured at 12 weeks from start of treatment ]
    Lesions must be measured and or evaluated at 12 weeks in accordance with the Response evaluation criteria in solid tumours (RECIST)

  3. overall survival [ Time Frame: Expected to be 6 - 12 months (Measured from commencement of treatment until time of death) ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 treatment
  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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01395121

United Kingdom
Royal Marsden NHS Foundation Trust
London, United Kingdom, SW3 6JJ
Sponsors and Collaborators
Institute of Cancer Research, United Kingdom
Royal Marsden NHS Foundation Trust
Principal Investigator: James Larkin, MA BM BCh MRCP PhD Royal Marsden NHS Foundation

Responsible Party: Institute of Cancer Research, United Kingdom Identifier: NCT01395121     History of Changes
Other Study ID Numbers: ICR-CTSU/2009/10020
2009-012945-49 ( EudraCT Number )
Oxfordshire C 09/H0606/103 ( Other Identifier: Main REC reference )
CRUK/09/028 ( Other Identifier: Cancer Research UK )
CTA 15983/0226/001 ( Other Identifier: MHRA )
ISRCTN 39058880 ( Other Identifier: ISRCTN )
First Posted: July 15, 2011    Key Record Dates
Last Update Posted: June 8, 2017
Last Verified: June 2017

Keywords provided by Institute of Cancer Research, United Kingdom:
c-KIT mutation
advanced disease
tyrosine kinase inhibitors

Additional relevant MeSH terms:
Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Nevi and Melanomas