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Study Summary
No. 2011-0757:.......Melanoma......Wen-Jen Hwu......Melanoma Medical Oncology
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Study Summary Title
Study Summary
Number:
2011-0757
Study Title:Phase I Study of Single Agent MK-3475 in Patients with Progressive
Locally Advanced or Metastatic Carcinomas, Melanoma and Non-Small Cell Lung Carcinoma
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Physician New Patient Referral
Name:Wen-Jen HwuPatients 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:Merck
Phase of Study:Phase IReturn
Visit:
Treatment
Agents:
MK-3475Home Care:
Treatment Loc:Both at MDACC & and Other Sites
Estimated
Length of Stay
in Houston:
Description/
Intervention:
There are 6 parts to this clinical research study. MD Anderson will be
taking part in Parts B, D, and F. This consent form will cover Parts B, D, and
F.

The goal of Part B is to further test the highest tolerable dose that was found
in Part A when given to patients with melanoma. The safety of this drug will
also be studied.

The goal of Part D is to study 2 dose levels of MK-3475 when given to patients
with melanoma.

The goal of Part F is to study 2 dose levels of MK-3475 when given to patients
with lung cancer.

MK-3475 is a drug that includes a protein naturally created by living cells.
It is designed to help the body's natural defense system react against tumors
by blocking proteins that cancers cells create to "turn off" the body's immune
(defense) system.

This is the first study using MK-3475 in humans.
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Study Objectives / Outcomes
Primary Objectives:

1) To evaluate and characterize the tolerability and safety profile of single agent MK-3475 in adult patients with unresectable advanced carcinoma including non-small cell lung cancer (NSCLC or melanoma (MEL).

2) To evaluate anti-tumor activity of MK-3475 in MEL and NSCLC.

3) To evaluate the extent of tumor response that correlates with the degree of biomarker positivity in the tumors of ipilimumab naive patients treated with MK-3475 with the intent that the cut point for the PD-L1 assay willb e explored and refined with tumor samples from ipilimumab-naive MEL.

4) To evaluate anti-tumor activity of MK-3475 in unselected MEL refractory to ipilimumab patients and MEL patients refractory to ipilimumab with PD-L1 expressing tumors.

Secondary Objectives:

1) To characterize the PK profile of single agent MK-3475.

2) To evaluate target engagement and modulation in peripheral blood program cell death-1 (PD-1) receptor occupancy and modulation of receptor activity.

3) To investigate the relationship between candidate efficacy biomarkers and antitumor activity of MK-3475:
- To evaluate the correlation between program cell death-ligand 1 (PD-L1) expression levels and anti-tumor activity of MK-3475, excluding ipi-refractory patients as stated in the primary objectives.
- To investigate other biomarkers (e.g., tumor infiltrating lymphocytes, program death-ligand 2 (PD-L2), PD-1; ribonucleic acid (RNA) signature profiles) that may correlate with tumor responses.
- To evaluate differences in tumor tissue characteristics in biopsies taken during or post-treatment with MK-3475 versus baseline.

4) To evaluate response duration, progression-free-survival and overall survival of patients who are treated with MK-3475.

5) To evaluate response duration, progression-free survival and overall survival of
NSCLC patients who are treated with MK-3475.

Tertiary Objectives:

To examine concordance between archival tumor tissues, formalin-fixed, paraffin embedded tissue (FFPET) and fresh frozen tumor tissue with respect to PD-L1 expression and other candidate efficacy biomarkers.
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Study Status Information
Study Activation / Registration Date:03/06/2012
IRB Review and Approval Date:03/06/2012
Study Type:Phase I
Recruitment Status:Open
Projected Accrual:693
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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) In Part B of the study, patients must have a histological or cytological diagnosis of MEL with progressive locally advanced or metastatic disease that is not amenable to definitive local therapy with curative intent. Ipilimumab-naïve Patients: Patients naive to ipilimumab may not have received more than 2 prior systemic treatment regimens for treatment of MEL. Ipilimumab-treated Patients: After the first 13 patients are enrolled, patients who have had ipilimumab may be enrolled, provided the following requirements are met: Full resolution of ipilimumab related adverse effects (including immunerelated adverse effects) and no treatment for these adverse events (AEs) for at least 4 weeks prior to the time of enrollment. Minimum of 12 weeks from the first dose of ipilimumab and >6 weeks from the last dose.

2) #1 Contd. No history of severe immune related adverse effects from ipilimumab (CTCAE Grade 4; CTCAE Grade 3 requiring treatment >4 weeks). Unequivocal PD within 6 months of the first dose of ipilimumab Ipilimumab

3) #1 Contd.Ipilimumab-refractory Patients: With Amendment 05 and 06, patients who have had ipilimumab may be enrolled, provided the following requirements are met (these patients are considered ipilimumab-refractory): Received at least two doses of ipilimumab. Progressive disease will be defined as increase in tumor burden >25% relative to nadir (minimum recorded tumor burden) which is confirmed by repeat assessment (investigator determination based on site radiology reading; SPONSOR will collect CT scans for retrospective analysis) no less than four weeks from the date of the first documented PD. Once PD is confirmed, initial date of PD documentation will be considered as the date of disease progression. Tumor burden is defined by irRC

4) #1 Contd: Documented disease progression within 24 weeks of the last dose of ipilimumab. Patients who were re-treated with ipilimumab and patients who were on maintenance ipilimumab will be allowed to enter the trial as long as there is documented PD within 24 weeks of the last treatment date (with ipilimumab). Full resolution of ipilimumab related AEs (including irAEs) back to baseline and <10 mg/day prednisone or equivalent dose for irAEs for at least two weeks prior to first dose of study drug.

5) contd #1: No history of severe irAEs from ipilimumab CTCAE Grade 4 requiring steroid treatment. o No history of CTCAE Grade 3 irAEs from ipilimumab requiring steroid treatment (>10 mg/day prednisone or equivalent dose) >12 weeks. o Minimum of four weeks (wash out period) from the last dose of ipilimumab. Patients with BRAF V600mutant melanoma must had a prior treatment regimen that included a BRAF and/or MEK inhibitor. Patient must have progressive disease after the most recent treatment regimen.

6) #1 contd. In Part D of the study, patients must have a histological or cytological diagnosis of MEL with progressive locally advanced or metastatic disease that is not amenable to definitive local therapy with curative intent. Patients must be naive to ipilimumab and may not have received more than 2 prior systemic treatment regimens for treatment of MEL. One of them may have included a BRAF or MEK inhibitor.

7) #1 contd. In Part F of the study, patients must have a histologically-confirmed or cytologicallyconfirmed diagnosis of non-small cell lung cancer, Patient has non-squamous NSCLC Patients' tumors must express PD-L1 as confirmed by a central vendor. Patients have tumor(s) amenable to biopsy. Patients in F-1 must be naive to systemic treatment for NSCLC (adjuvant therapy may not have been administered within 1 year of the relapse). Patients in F-2 have experienced progression of locally advanced or metastatic NSCLC after two or more prior systemic antineoplastic regimens (adjuvant therapy will count as a regimen if administered within 1 year before the relapse). Patient has an estimated life expectancy of at least 12 weeks.

8) #2. Measurable disease:In Part B, C, D, E and F of the study, patients must have measurable disease as defined per irRC (Appendix 6.5): i. Tumor mass: Must be accurately measurable in 2 perpendicular diameters, with both its longest diameter and its longest perpendicular must be greater than or equal to 10 mm or 2 times the axial slice thickness. Clinical lesions will only be considered measurable when they are superficial, such as skin or palpable lymph node. For patients who are being screened for enrollment in Part B, the ipilimumab-refractory cohort, clinical lesions alone will not be considered as sufficient for enrollment; there must be measurable disease evident on CT imaging. ii. Malignant lymph nodes: Must be measurable in 2 perpendicular diameters, with both its longest diameter and its longest perpendicular, must be greater than or equal to 15 mm or 2 times the axial slice thickness.

9) #3: Patient is male or female and ≥18 years of age on day of signing informed consent.

10) #4. Patient must have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) Performance Scale

11) #5. Patient must have adequate organ function as indicated by the following laboratory values: Hematological: Absolute neutrophil count (ANC) >/=1,500 /mcL; Platelets >/=100,000 mcL; Hemoglobin >/=9 g/dL or >/=5.6 mmol/L. Renal: Serum creatinine </=1.5 X upper limit of normal (ULN). Hepatic: Serum total bilirubin </= 1.5 X ULN OR Direct bilirubin </= ULN for patients with total bilirubin levels > 1.5 ULN. AST (SGOT) and ALT (SGPT) </= 2.5 X ULN OR </= 5 X ULN for patients with liver metastases. Coagulation: International Normalized Ratio (INR) or Prothrombin Time (PT) </=1.5 X ULN. Activated Partial Thromboplastin Time (aPTT) </=1.5 X ULN

12) #6. Patient (Parts A, B, C, D, E and F) has voluntarily agreed to participate by giving written informed consent. For Parts B, C, D, E and F, patient has agreed to a fresh biopsy of tumor (that can be biopsied based on investigator's assessment) and to providing the acquired tissue for biomarker analysis. Tissue obtained for the biopsy must not be previously irradiated. No systemic antineoplastic therapy may be received by the patient between the time of the biopsy and the first administration of MK-3475.

13) #7. Female patient of childbearing potential has a negative urine or serum pregnancy test. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. The serum pregnancy test must be negative for the patient to be eligible.

14) #8. Female patients enrolled in the study, who are not free from menses for >2 years, post hysterectomy/oophorectomy, or surgically sterilized, must be willing to use either 2 adequate barrier methods or a barrier method plus a hormonal method of contraception to prevent pregnancy or to abstain from heterosexual activity throughout the study, starting with Visit 1 through 90 days after the last dose of study therapy. Approved contraceptive methods include for example; intra uterine device, diaphragm with spermicide, cervical cap with spermicide, male condoms, or female condom with spermicide. Spermicides alone are not an acceptable method of contraception. Male patients must agree to use an adequate method of contraception starting with the first dose of study drug through 90 days after the last dose of study therapy.

15) #9. Subject may also provide consent/assent for Future Biomedical Research. However, the subject may participate in the main trial without participating in Future Biomedical Research.

Exclusion Criteria:1) Patient who has had chemotherapy, radioactive, or biological cancer therapy within 4 weeks prior to the first dose of study therapy, or who has not recovered to CTCAE grade 1 or better from the adverse events due to cancer therapeutics administered more than 4 weeks earlier. - Patient who has had ipilimumab therapy may be enrolled in Part B or C of the study (after 13 ipilimumab naïve patients are enrolled in Part B) if the requirements specified in Inclusion Criterion) are met.

2) Patient is currently participating or has participated in a study of an investigational agent or using an investigational device within 30 days of administration of MK-3475.

3) Patient is expected to require any other form of antineoplastic therapy while on study (including maintenance therapy with another agent for NSCLC).

4) Patient is on chronic systemic steroid therapy or on any other form of immunosuppressive medication.

5) Patient has a history of acute diverticulitis, intra-abdominal abscess, GI obstruction, abdominal carcinomatosis which are known risk factors for bowel perforation.

6) Patient has a known history of a hematologic malignancy, primary brain tumor or sarcoma, or of another primary solid tumor, unless the patient has undergone potentially curative therapy with no evidence of that disease for 5 years. Note: The time requirement for no evidence of disease for 5 years does not apply to the tumor for which a patient is enrolled in the study. The time requirement does also not apply to patients who underwent successful definitive resection of basal cell carcinoma of the skin, superficial bladder cancer, squamous cell carcinoma of the skin, in situ cervical cancer, or other in situ cancers.

7) Patient has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Patients with previously treated brain metastases may participate provided they are clinically stable for at least 8 weeks prior to study entry, have no evidence of new or enlarging brain metastases and are off steroids.

8) Patient previously had a severe hypersensitivity reaction to treatment with another mAb.

9) Patient has an active autoimmune disease or a documented history of autoimmune disease or syndrome that requires systemic steroids or immunosuppressive agents. Patients with vitiligo or resolved childhood asthma/atopy would be exception to this rule. Patients that require intermittent use of bronchodilators or local steroid injections would not be excluded from the study. Patients with hypothyroidism that is stable on hormone replacement will not be excluded from the study.

10) Patient had prior treatment with any other anti-PD-1, or PD-L1 or PD-L2 agent or an antibody targeting other immuno-regulatory receptors or mechanisms (with exception of ipilimumab in study Part B and Part C).

11) Patient has an active infection requiring therapy.

12) Patient is positive for Human Immunodeficiency Virus (HIV) (HIV 1/2 antibodies), Hepatitis B (HBsAg reactive) or Hepatitis C (HCV RNA (qualitative) is detected).

13) Patient has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the patient's participation for the full duration of the study, or is not in the best interest of the patient to participate, in the opinion of the treating Investigator.

14) Patient has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.

15) Patient is, at the time of signing informed consent, a regular user (including "recreational use") of any illicit drugs or had a recent history (within the last year) of substance abuse (including alcohol).

16) Patients with symptomatic ascites or pleural effusion. A patient who is clinically stable following treatment for these conditions (including therapeutic thoraco- or paracentesis) is eligible.

17) Patient is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the study.

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Links
Registration Number: NCT01295827
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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