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An Open-label, Multi-center, Single-arm Study to Evaluate the Efficacy of Nilotinib in Adult Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumors in First Line Treatment

Phase 4
18 Years
Open (Enrolling)
Gastrointestinal Stromal Tumors

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Trial Information

An Open-label, Multi-center, Single-arm Study to Evaluate the Efficacy of Nilotinib in Adult Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumors in First Line Treatment

Inclusion Criteria:

- Age ≥18 years

- Histologically confirmed diagnosis of GIST that is unresectable and/or metastatic and
therefore not amenable to surgery or combined modality with curative intent prior to
or at Visit 1

- At least one measurable site of disease on CT/MRI scan at Visit 1, as defined by
RECIST criteria (see Post Text Suppl 3 for details) The scans should be at maximum 2
weeks old. New scans are only required as baseline scans if they are older then
approx. 2 weeks.

- WHO Performance Status of 0, 1 or 2

- Patients must have the following laboratory values (≥ LLN (lower limit of normal) or
corrected to within normal limits with supplements prior to the first dose of study

1. Potassium ≥ LLN,

2. Magnesium ≥ LLN,

3. Phosphorus ≥ LLN,

4. Total calcium (corrected for serum albumin) ≥ LLN

- Patients must have normal organ, electrolyte, and marrow function as defined below:

1. Absolute Neutrophil Count (ANC) ≥ 1.5x 109/L;

2. Platelets ≥ 100 x 109/L;

3. ALT and AST ≤ 2.5 x upper limit of normal (ULN) or ≤ 5.0 x ULN if considered due
to tumor;

4. Alkaline phosphatase ≤ 2.5 x ULN unless considered due to tumor;

5. Serum bilirubin ≤ 1.5 x ULN;

6. Serum lipase and amylase ≤ 1.5 x ULN;

7. Serum creatinine ≤ 1.5 x ULN or 24-hour creatinine clearance ≥ 50 ml/min.
(calculated creatinine clearance using Cockroft formula is acceptable)

- Ability to understand and willingness to sign a written informed consent

Exclusion Criteria:

- Prior treatment with nilotinib

- Treatment with any cytotoxic and/or investigational cytotoxic drug ≤ 4 weeks (6 weeks
for nitrosurea or mitomycin C) prior to Visit 1 with the exception of imatinib
targeted therapy as an adjuvant therapy

- Prior or concomitant malignancies requiring active treatment other than GIST with the
exception of previous or concomitant basal cell skin cancer, previous cervical
carcinoma in situ

- Impaired cardiac function at including any one of the following:

1. LVEF < 45% or below the institutional LLN range (whichever is higher) as
determined by echocardiogram at Visit 1

2. Complete left bundle branch block

3. Use of a ventricular paced cardiac pacemaker

4. Congenital long QT syndrome or family history of long QT syndrome

5. History of or presence of significant ventricular or atrial tachyarrhythmias

6. Clinically significant resting bradycardia (< 50 beats per minute)

7. QTc > 450 msec on screening ECG (using the QTcF formula). If QTc > 450 msec and
electrolytes are not within normal ranges (electrolytes should be corrected and
then the patient rescreened for QTc.

8. Right bundle branch block plus left anterior hemiblock, bifascicular block

9. Myocardial infarction within 12 months prior to Visit 1

10. Other clinically significant heart disease (e.g., unstable angina, congestive
heart failure or uncontrolled hypertension,)

- Patients with severe and/or uncontrolled concurrent medical disease that in the
opinion of the investigator could cause unacceptable safety risks or compromise
compliance with the protocol e.g. impairment of gastrointestinal (GI) function, or GI
disease that may significantly alter the absorption of the study drugs, uncontrolled

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:

To evaluate the efficacy of Nilotinib in patients with unresectable or metastatic gastrointestinal stromal tumors. Efficacy is defined as the proportion of patients showing stable disease (SD), partial response (PR) or complete response (CR) during the

Outcome Time Frame:

6 months

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

August 2008

Completion Date:

December 2013

Related Keywords:

  • Gastrointestinal Stromal Tumors
  • unresectable or metastatic GIST
  • 1st. line treatment
  • Gastrointestinal Stromal Tumors