Treatment of Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumors in First Line With Nilotinib

NCT00756509 | Completed Phase 4 Results

• 2 other identifiers: CAMN107DDE062008-000358-11
• Study Type: interventional
• Target: 34
• Locations: 4 countries
• Sites: 7

Brief Summary

The purpose of this multicenter, single-arm, phase II trial is to evaluate the efficacy of Nilotinib in patients with unresectable or metastatic gastrointestinal stromal tumors (GIST).

Conditions

Gastrointestinal Stromal Tumors

Keywords

unresectable or metastatic GIST; 1st. line treatment

Outcome Measures

Primary Outcomes (1)

• Proportion of Participants With Best Overall Response at Month 6 (Core Phase) Determined According to the RECIST v1.0.

  The primary efficacy measure is the proportion of patients reaching Complete Response (CR), Partial Response (PR), or Stable Disease (SD) by Month 6, as per RECIST v1.0. Definitions are as follows: CR requires at least two CRs at least four weeks apart before any progression; PR requires two or more PRs at least four weeks apart, without qualifying for CR; SD is at least one SD more than six weeks after treatment start, not qualifying for CR or PR. Progressive Disease (PD) is defined as progression or cancer-related death within 12 weeks of starting treatment, not qualifying for CR, PR, or SD. UNK refers to cases not meeting these criteria, such as absence of confirmed CR/PR, no SD after six weeks, or early progression. The percentage of patients with CR, PR, or SD will be presented with a one-sided exact 90% (or 80% two-sided) confidence interval for the ITT\_F group.

  from baseline to month 6, core phase

Secondary Outcomes (6)

• Proportion of Participants With Objective Response Rate (ORR) at Month 6 (Core Phase) Observed According to RECIST

  from baseline to month 6, core phase

• Time to Response (TTR) at Month 6 (Core Phase)

  from baseline to month 6, core phase

• Duration of Response (CR or PR) for Complete Study (Core and Follow-up Phases)

  from date of first response (CR or PR) until progression or death, up to data cut off (up to approximately 16 years)

• Progression-free Survival (PFS) for Complete Study (Core and Follow-up Phases)

  from date of first response (CR or PR) until progression or death, up to data cut off (up to approximately 16 years)

• Overall Survival for Complete Study (Core and Follow-up Phases)

  from date of first response (CR or PR) until progression or death, up to data cut off (up to approximately 16 years)

Study Arms (1)

nilotinib

EXPERIMENTAL

nilotinib

Drug: Nilotinib

Interventions

Nilotinib DRUG

800 mg/d orally

nilotinib

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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. The scans should be at maximum 2 weeks old. New scans are only required as baseline scans if they are older than 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 medication.):
• Potassium ≥ LLN,
• Magnesium ≥ LLN,
• Phosphorus ≥ LLN,
• Total calcium (corrected for serum albumin) ≥ LLN
• Patients must have normal organ, electrolyte, and marrow function as defined below:
• Absolute Neutrophil Count (ANC) ≥ 1.5x 109/L;
• Platelets ≥ 100 x 109/L;
• ALT and AST ≤ 2.5 x upper limit of normal (ULN) or ≤ 5.0 x ULN if considered due to tumor;
• Alkaline phosphatase ≤ 2.5 x ULN unless considered due to tumor;
• Serum bilirubin ≤ 1.5 x ULN;

You may not qualify if:

• 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 or imatinib in first line treatment for maximum of 4 weeks.
• 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:
• LVEF \< 45% or below the institutional LLN range (whichever is higher) as determined by echocardiogram at Visit 1
• Complete left bundle branch block
• Use of a ventricular paced cardiac pacemaker
• Congenital long QT syndrome or family history of long QT syndrome
• History of or presence of significant ventricular or atrial tachyarrhythmias
• Clinically significant resting bradycardia (\< 50 beats per minute)
• 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.
• Right bundle branch block plus left anterior hemiblock, bifascicular block
• Myocardial infarction within 12 months prior to Visit 1
• 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 diabetes

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (7)

Novartis Investigative Site

Helsinki, FIN-00029, Finland

Location

Novartis Investigative Site

Lyon, 69373, France

Location

Novartis Investigative Site

Munich, Bavaria, 81377, Germany

Location

Novartis Investigative Site

Bad Saarow, 15526, Germany

Location

Novartis Investigative Site

Essen, 45147, Germany

Location

Novartis Investigative Site

Hanover, 30625, Germany

Location

Novartis Investigative Site

Milan, MI, 20133, Italy

Location

MeSH Terms

Conditions

Gastrointestinal Stromal Tumors

Interventions

nilotinib

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

862 778 8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2008
• First Posted: September 22, 2008
• Study Start: August 29, 2008
• Primary Completion: December 18, 2024
• Study Completion: December 18, 2024
• Last Updated: January 20, 2026
• Results First Posted: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

FR (1); FI (1); IT (1); DE (4)