Efficacy and Safety of Imatinib in Chordoma

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: NCT00150072
Recruitment Status : Completed
First Posted : September 8, 2005
Last Update Posted : February 23, 2017
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
Preliminary response data, observed by Casali (Cancer, 2004) with imatinib 800 mg/day in patients affected by chordoma, need to be confirmed by a Phase II study, whose primary endpoint will be the formal assessment of clinical and pathological response. Aim of the study will be to explore treatment's activity, but also the potential impact of tumor response, the feasibility and outcome of subsequent surgery and radiotherapy. In addition, patterns of tumour response need to be investigated as well, given the peculiar patterns of response shown with molecular-targeted therapy in solid tumors.

Condition or disease Intervention/treatment Phase
Chordoma Drug: imatinib Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 55 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of Imatinib Mesylate in Chordoma
Study Start Date : October 2004
Actual Primary Completion Date : April 2008
Actual Study Completion Date : April 2008

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: imatinib Drug: imatinib

Primary Outcome Measures :
  1. Tumor response [ Time Frame: Every 3 months for 2 years ]
    objective response according to RECIST and clinical response

Secondary Outcome Measures :
  1. Overall survival [ Time Frame: 2 years ]
    from the first day of sudy treatment to the day of death for any cause

  2. Progression free survival [ Time Frame: 2 years ]
    from the first day of sudy treatment to the day of death for any cause or documented progression

  3. Safety and tolerability [ Time Frame: 2 years ]
    frequency of adverse events, abnormal lab values, bone pain, use of analgesic medication

  4. proportion of patients undergoing complete surgery [ Time Frame: 2 years ]
    number of pts undergoing complete surgery vs the one of pts not amenable to complete surgery at enrolment

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Histological diagnosis of chordoma.
  2. Biomolecular or immunohistochemical evidence of Imatinib mesylate target (PDGFRβ activation and/or presence of PDGFB). Biomolecular assessment of PDGFRβ activation should be made whenever possible. To this end, if frozen material is not available, obtaining of, fresh material is encouraged, if it should be obtained with no major distress for the patient, preferably through an incisional biopsy (to allow immunoprecipitation) or, if this is not feasible, a Trucut biopsy (to allow Western Blot assessment). However, if frozen or fresh material cannot be obtained, paraffined material is also acceptable.

    The biomolecular assessment will be centralized to the reference centers (to be defined).

  3. Measurable or evaluable disease
  4. Surgical resection of local disease unfeasible radically, or unaccepted by the patient, or amenable to become less demolitive, or easier, or likely more feasible, after cytoreduction, and/or metastatic disease. Debulking surgery before enrolment is allowed. In this case, enrolment should occur at least one month after surgery
  5. Performance status 0, 1, 2 or 3 (ECOG) (see § 8.1).
  6. Adequate end organ function, defined as the following: total bilirubin <1.5 x ULN, SGOT and SGPT <2.5 x UNL (or <5 x ULN if hepatic metastases are present), creatinine <1.5 x ULN.
  7. Adequate bone marrow function, defined as the following: ANC >1.5 x 10^9/L, platelets >100 x 10^9/L, Hb >9 g/dL. Blood transfusions are allowed to reach the baseline requested Hb level.
  8. Female patients of child-bearing potential must have negative pregnancy test within 7 days before initiation of study drug dosing. Post menopausal women must be amenorrheic for at least 12 months to be considered of non-childbearing potential. Male and female patients of reproductive potential must agree to employ an effective method of birth control throughout the study and for up to 3 months following discontinuation of study drug.
  9. Written, voluntary, informed consent.

Exclusion Criteria:

  1. Previous treatment with any other investigational or not investigational agents within 28 days of first day of study drug dosing.
  2. Other primary malignancy with <5 years clinically assessed disease-free interval, except basal cell skin cancer, cervical carcinoma in situ, or other neoplasms judged to entail a low risk of relapse.
  3. Grade III/IV cardiac problems as defined by the New York Heart Association Criteria (i.e., congestive heart failure, myocardial infarction within 6 months of study)
  4. Severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
  5. Known brain metastasis.
  6. Known chronic liver disease (i.e., chronic active hepatitis, and cirrhosis).
  7. Known diagnosis of human immunodeficiency virus (HIV) infection.
  8. Previous radiotherapy to >=25 % of the bone marrow.
  9. Major surgery within 2 weeks prior to study entry.
  10. Expected non-compliance to medical regimens.

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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): NCT00150072

Novartis Investigative Site
Aviano, Italy
Novartis Investigative Site
Bologna, Italy
Novartis Investigative Site
Candiolo, Italy
Novartis Investigative Site
Firenze, Italy
Novartis Investigative Site
Milano, Italy
Novartis Investigative Site
Napoli, Italy
Novartis Investigative Site
Padova, Italy
Novartis Investigative Site
Pisa, Italy
Novartis Investigative Site
Roma, Italy
Novartis Investigative Site
Rozzano, Italy
Novartis Investigative Site
Torino, Italy
Novartis Investigative Site
Lausanne, Switzerland
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

Additional Information:
Publications of Results:
Responsible Party: Novartis Pharmaceuticals Identifier: NCT00150072     History of Changes
Other Study ID Numbers: CSTI571BIT15
First Posted: September 8, 2005    Key Record Dates
Last Update Posted: February 23, 2017
Last Verified: February 2017

Keywords provided by Novartis ( Novartis Pharmaceuticals ):

Additional relevant MeSH terms:
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Imatinib Mesylate
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action