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Study Summary
No. 2005-0949:.......Melanoma......Kevin B. Kim......Melanoma Medical Oncology
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Study Summary Title
Study Summary
Number:
2005-0949
Study Title:A Phase I/II, Open-label, Dose-escalation Trial to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of RAF265 (CHIR-265) Administered Orally to Subjects with Locally Advanced or Metastatic Melanoma
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Physician New Patient Referral
Name:Kevin B. KimPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:Melanoma Medical OncologyReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-2921
Contact us about clinical trials
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General Information
Disease Group:MelanomaSupported By:N/A
Phase of Study:Phase I/Phase IIReturn
Visit:
Once a week for cycle 1 and then once every 2 weeks for cycle 2 and every 4
weeks for subsequent cycles
Treatment
Agents:
RAF265Home Care:N/A
Treatment Loc:Independent Multicenter Arrangements
Estimated
Length of Stay
in Houston:
N/A
Description/
Intervention:
The goal of this clinical research study is to find the highest tolerable dose
of RAF265 that can be given to patients with melanoma that has spread to other
parts of their body. The safety and effectiveness of this drug will also be
studied.

RAF265 is a drug that has been shown to prevent or slow the growth of melanoma
cells in tumors in animals by blocking certain receptors that are important in
melanoma cell growth and survival.
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Study Objectives / Outcomes
The primary objectives of the study are to:
    1. Determine the MTD and/or OBD, DLTs, and the safety profile of RAF265 when administered orally to patients with locally advanced or metastatic melanoma.
    2. Determine the plasma PK of orally administered RAF265 (including but not limited to area under the AUC, half-life [t1/2], observed maximum plasma concentration [Cmax], time at which maximum concentration is observed [tmax])
    3. Evaluate the potential pharmacodynamic effects of RAF265 using tumor biopsies, peripheral blood samples, and imaging:
      • Tumor biopsies: signaling molecules, apoptosis, proliferation, microvessel density. Peripheral blood: levels of soluble growth factors and melanoma-related markers
      • Tumor imaging: Tumor metabolic activity relative to baseline as measured by FDG-PET and tumor volume measured relative to baseline will be evaluated. Changes in tumor vascular perfusion and permeability relative to baseline as measured by DCE MRI (at the dose expansion).
4. The primary objective of Arm 4 is to confirm the safety and tolerability of the tablet formulation of RAF265 when dosed daily at its MTD, and to confirm that exposure with the tablet is comparable to that of the liquid formulation under steady-state conditions.

Secondary objectives of the study are to:
    1. Evaluate the effect of somatic BRAF and N-RAS mutations on modulation of pharmacodynamic markers and with clinical response.
    2. Determine the preliminary response rate at the MTD and/or optimal biologic dose (OBD) in patients with or without BRAF mutations
    3. Determine the OBD based on its safety, PK, and pharmacodynamic data.
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Study Status Information
Study Activation / Registration Date:05/04/2007
IRB Review and Approval Date:06/09/2006
Study Type:Phase I
Recruitment Status:Open
Projected Accrual:A total of about 162 patients: approximately 126 to 132 patients in Arm 2 will be enrolled. Arm 4 will require approximately 12 patients.
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:1) Age >/= 18 years.

2) Histologically confirmed diagnosis of locally advanced or metastatic melanoma (for cutaneous melanoma American Joint Committee on Cancer [AJCC] Stage IIIB to IV, pathologic Stage III and IV for noncutaneous melanoma).

3) Must have either archival tumor tissue or tumor that can be biopsied in order to determine whether it contains mutated or wild-type BRAF.

4) Evidence of measurable disease, defined as at least one lesion that can accurately be measured in at least one dimension as >/= 20 mm with conventional techniques or >/= 10 mm with spiral CT scan; cutaneous lesions must have clearly defined margins and measure >/= 5 mm in at least one diameter.

5) Patients scheduled for FDG-PET should have uptake of the tracer in at least one lesion (tumor-to-muscle ration>2) in the baseline PET/CT scan in order to be eligible for the follow-up FDG PET scans. Patients who do not have uptake will be excluded from having additional FDG-PET scans, but can continue in the trial.

6) ECOG Performance Status of 0 or 1.

7) Required baseline laboratory data include: Absolute neutrophil count (ANC) >/= 1,500/mm^3 (SI units 10^9;L); Platelets >/= 100,000/mm^3 (SI units 10^9/L); Hemoglobin >/= 9.0 gm/dL (SI units gm/l); Serum creatinine </= 1.5 x upper limit of normal (ULN); Bilirubin </= 1.5 x ULN; Lipase/Amylase </= ULN; Asparate transaminase (AST) </= 2.5 x ULN, except for patients with tumor Alanine trasaminase (ALT) involvement of the liver who must have AST and ALT </=5 x ULN

8) At least 4 weeks must have elapsed since any major surgery.

9) For women of childbearing potential, negative serum pregnancy test within 72 hours of treatment and use of physician-approved method of birth control throughout the study.

10) No concurrent anticancer or investigational therapy for at least 4 weeks before study entry, with full recovery (NCI CTCAE grade 1) from the toxic effects of that treatment.

11) Written informed consent, willingness, and ability to comply with all study procedures.

Exclusion Criteria:1) Previous therapy with the following molecularly-targeted agents: (1) MEK inhibitors (such as PD0325901); (2) VEGF or VEGFR inhibitors (such as Avastin and small molecule inhibitors with activity against any of the VEGF receptor isoforms); (3) Raf inhibitors (such as sorafenib)

2) Patients with a history of primary central nervous system tumors or brain metastases or who have singns/symptoms attributable to brain metastases and have not been assessed wtih radiologic imaging to rule out the presence of brain metastases.

3) Clinically significant cardiac disease including congestive heart failure (New York Heart Association Class III or IV), arrhythmia or conduction abnormality requiring medication, or cardiomyopathy; or clinically uncontrolled hypertension (Blood pressure > 160/110 mmHg). Impaired cardiac function or clinically significant cardiac diseases, including any one of the following: 1). LVEF < 45% as determined by MUGA scan or echocardiogram 2) Complete left bundle branch block 3) Obligate use of cardiac pacemaker 4). Congenital long QT syndrome 5) History or presence of ventricular tachyarrhythmia.

4) (Cont'd from #3) - Presence of unstable atrial fibrillation (ventricular resonse > 100 bpm). Patients with stable atrial fibrillation are eligible, provided they do not meet any of the other exclusion criteria. 7) Clinically significant resting bradycardia (<50 bmp) 8) QTc > 480 msec on screening ECG 9) Right bundle branch block + left anterior hemiblock (bifasicular block) 10) Angina pectoria </= 3 months prior to starting study drug. 11) Acute MI </= 3 months prior to starting study drug.

5) Previous or concurrent malignancy. Exceptions to this exclusion criteria include: adequately treated basal cell or squamous cell skin cancer; in situ carcinoma of the cervix treated curatively and without evidence of recurrence for at least 3 years prior to study entry; or other solid tumor treated curatively, and without evidence of recurrence for at least 3 years prior to study entry.

6) Active, and uncontrolled clinically significant infection.

7) Breastfeeding women.

8) Chronic anticoagulation therapy with full strength acetylsalicylic acid (ASA), warfarin sodium, or heparin (low dose ASA </= 100 mg, or low dose warfarin </= 1 mg to maintain indwelling venous access device patency is allowed).

9) History of thromboembolic or cerebrovascular events within the last 12 months, including transient ischemic attack, cerebrovascular accident, deep vein thrombosis, or pulmonary embolism.

10) History of melena, hematemesis, or hemoptysis within the last 3 months.

11) Prior acute or chronic pancreatitis of any etiology.

12) Prior intra or extrahepatic biliary obstruction within the previous 12 months, or history of malignant obstruction requiring a biliary stent, unless stably treated with no prior obstruction or blockage of the stent.

13) Other severe, acute, or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration or that may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for the study.

14) Blood pressure > 140/90.

15) Current evidence of retinal disease, confirmed by ophthalmologic examination

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Links
Registration Number: NCT00304525
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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