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Study Summary
No. 2008-0585:.......Skin......Michael R. Migden......Dermatology
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Study Summary Title
Study Summary
Number:
2008-0585
Study Title:A PIVOTAL PHASE II, MULTICENTER, SINGLE-ARM, TWO-COHORT TRIAL EVALUATING THE EFFICACY AND SAFETY OF GDC-0449 IN PATIENTS WITH ADVANCED BASAL CELL CARCINOMA
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Physician New Patient Referral
Name:Michael R. MigdenPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:DermatologyReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-794-1450
Contact us about clinical trials
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General Information
Disease Group:SkinSupported By:Genentech
Phase of Study:Phase IIReturn
Visit:
I screening visit; 12 treatment cycles(Day 1, weeks 4, 8, 12, 16, 20, 24, 28,
32, 36, 40, 44; 1 End of Study; 1 F/U visit
Treatment
Agents:
GDC-0449Home Care: n/a
Treatment Loc:Both at MDACC & outside MDACC at one or more Collaborating Sites or Institutions
Estimated
Length of Stay
in Houston:
n/a
Description/
Intervention:
The goal of this clinical research study is to learn if GDC-0449 (also called
vismodegib) can help to control the disease in patients with advanced basal
cell carcinoma. The safety of this drug will also be studied.
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Study Objectives / Outcomes
The primary objective of this study is to estimate the clinical benefit of GDC-0449 given as therapy for patients with locally advanced or metastatic BCC (basal cell carcinoma), as measured by overall response rate (ORR).

The secondary objectives of this study are the following:
  • To estimate the duration of response, progression-free survival (PFS), and overall survival (OS)
  • To assess the safety and tolerability of GDC-0449 in this patient population
  • To assess the pharmacokinetics of GDC-0449 in this population (at participating sites only)
  • To assess patient-reported outcomes
  • To assess the histopathologic effect of GDC-0449 in tumor biopsies obtained at baseline and following GDC-0449 treatment in patients with locally advanced BCC
  • To evaluate the status of the Hh (Hedgehog) signaling pathway using quantitative reverse transcriptase polymerase chain reaction (qRT-PCR) in archival tissue

Exploratory objectives
The exploratory objectives of this study are the following:
  • To evaluate the effect of GDC-0449 treatment on the Hh signaling pathway using qRT-PCR and/or other approaches in tissue obtained at baseline and/or following GDC-0449 treatment
  • To evaluate the relationship between the effects of GDC-0449 treatment on the Hh signaling pathway and efficacy

Description of Drug GDC-0449
Hh pathway activation has been implicated in several types of cancer. Hh signaling regulates epithelial and mesenchymal interactions in a variety of tissues during mammalian embryogenesis. Mutations in the Hh receptor components, PTCH1 (patched) or SMO (smoothened), result in constitutive pathway activation & have been identified in BCC and medulloblastoma. GDC-0449 is a small-molecule antagonist of the Hh signal pathway. GDC-0449 binds to and inhibits SMO that results in blocking of the Hh signal. Furthermore, GDC-0449 has proven to be efficacious in nonclinical tumor models of both mutated & ligand-overexpressing tumors. The molecule to be evaluated in this pivotal Phase II study, has oral bioavailability and potent anti-tumor activity in a variety of primary human tumor xenografts & tumor cell line xenograft models.

Standard therapy are surgical modalities. If further surgical resection is not possible, there is no approved therapy and no standard of care exists. Although palliative radiation therapy may be used, its use
may be limited by the location of the tumor and involved structures as well as prior
cumulative radiation dosage.
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Study Status Information
Study Activation / Registration Date:09/03/2009
IRB Review and Approval Date:04/01/2009
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Closed
Projected Accrual:100
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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) Signed informed consent.

2) Men and women age >/= 18 years

3) ECOG performance status 0, 1, or 2

4) For patients with mBCC, histologic confirmation of distant BCC metastasis (e.g., lung, liver, lymph nodes, or bone), with metastatic disease that is RECIST measurable using CT or MRI. If a patient with locally advanced BCC also has a tumor that is not contiguous with cutaneous BCC, e.g., regional lymph nodes (if confirmed on biopsy as BCC and RECIST measurable), the patient should be considered as having mBCC and should be enrolled in the mBCC cohort.

5) For patients with locally advanced BCC, histologically confirmed disease that is considered to be inoperable or medical contraindication to surgery, in the opinion of a Mohs dermatologic surgeon, head and neck surgeon, or plastic surgeon

6) Acceptable medical contraindications to surgery include: a.) BCC that has recurred in the same location after two or more surgical procedures and curative resection is deemed unlikely; b.) anticipated substantial morbidity and/or deformity from surgery (e.g., removal of all or part of a facial structure, such as nose, ear, eyelid, eye; or requirement for limb amputation); c.) other conditions considered to be medically contraindicating must be discussed with the Medical Monitor before enrolling the patient.

7) For patients with locally advanced BCC, radiotherapy must have been previously administered for their locally advanced BCC, unless radiotherapy is contraindicated or inappropriate (e.g., hypersensitivity to radiation due to genetic syndrome such as Gorlin syndrome, limitations because of location of tumor, or cumulative prior radiotherapy dose). For patients whose locally advanced BCC has been irradiated, disease must have progressed after radiation.

8) Patients with nevoid BCC syndrome (Gorlin syndrome) may enroll in this study but must meet the criteria for locally advanced or metastatic disease listed above.

9) For patients with locally advanced BCC, willingness to consent to biopsy of tumor(s) at baseline and during the study, as mandated by the protocol

10) For all patients, representative tumor specimens in paraffin blocks (preferred) or at least 15 unstained slides, with an associated pathology report, obtained at any time prior to entry of study

11) Adequate hematopoietic capacity, as defined by the following: Hemoglobin > 8.5 g/dL and not transfusion dependent Granulocyte count >/= 1000/microL Platelet count >/= 75,000/microL

12) Adequate hepatic function, as defined by the following: AST (aspartate aminotransferase) and ALT(alanine transaminase) </= 3 × the upper limit of normal (ULN) Total bilirubin </= 1.5 × the ULN or within 3 × the ULN for patients with Gilbert disease

13) Adequate renal function, as defined by the following: Serum creatinine </= 2.0 mg/dL or calculated creatinine clearance > 50 mL/min

14) For women of childbearing potential, agreement to the use of two acceptable methods of contraception, including one barrier method, during the study and for 7 months after discontinuation of GDC-0449.

15) For men with female partners of childbearing potential, agreement to use a condom, and to advise their female partner to use an additional method of contraception during the study and for 7 months after discontinuation of GDC-0449

16) Agreement not to donate blood or blood products during the study and for 7 months after discontinuation of GDC-0449; for male patients, agreement not to donate sperm during the study and for 7 months after discontinuation of GDC-0449

Exclusion Criteria:1) Prior treatment with GDC-0449 or other antagonists of the Hh pathway

2) Pregnancy or lactation.

3) Life expectancy of < 12 weeks.

4) Patients with superficial multifocal BCC who may be considered unresectable due to breadth of involvement

5) Concurrent non-protocol-specified anti-tumor therapy (e.g., chemotherapy, other targeted therapy, radiation therapy, or photodynamic therapy). For patients with multiple cutaneous BCCs at baseline that are not designated by the investigator as target lesions, treatment of these non-target BCCs with surgery may be permitted but must be discussed with the Medical Monitor prior to any surgical procedure. (eligibility continues in #5).

6) (continues from exclusion #6) For patients with locally advanced BCC whose target lesion(s) is/are inoperable at baseline but is/are later deemed potentially operable because of tumor response to GDC-0449, surgery with curative intent may be permitted but must be discussed with the Medical Monitor prior to any surgical procedure.

7) Recent (within 4 weeks of Day 1), current, or planned participation in an experimental drug study.

8) History of other malignancies within 3 years of Day 1, except for tumors with a negligible risk for metastasis or death, such as adequately treated squamous-cell carcinoma of the skin, ductal carcinoma in situ of the breast, or carcinoma in situ of the cervix.

9) Uncontrolled medical illnesses such as infection requiring treatment with intravenous antibiotics.

10) History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or renders the patient at high risk from treatment complications.

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Links
Registration Number: NCT00833417
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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