Phase I/II Study of PDR001 in Patients With Advanced Malignancies
The purpose of this "first-in-human" study of PDR001 is to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of PDR001 administered i.v. as a single agent to adult patients with solid tumors. By blocking the interaction between PD-1 and its ligands, PD-L1 and PD-L2, PDR001 inhibits the PD-1 immune checkpoint, resulting in activation of an antitumor immune response by activating effector T-cells and inhibiting regulatory T-cells. This study has been designed as a phase I/II, multi-center, open-label study starting with a phase I dose escalation part followed by a phase II part. PDR001 will be administered every 2 weeks until patient experiences unacceptable toxicity, progressive disease per immune related Response Criteria (irRC) and/or treatment is discontinued at the discretion of the investigator or the patient.
Study Start Date
Estimated Completion Date
- Biological: PDR001
- Oncology: Dermatologic Oncology
- Pharmacy: Chemotherapy/Oncology
- Dermatology: Dermatologic Oncology
Novartis -- CPDR001X2101
Phase 1/Phase 2
- Written informed consent must be obtained prior to any screening procedures
- Phase I part: Patients with advanced/metastatic solid tumors, with measurable or non-measurable disease as determined by RECIST version 1.1 (refer to Appendix 1), who have progressed despite standard therapy or are intolerant of standard therapy, or for whom no standard therapy exists.
- Phase II part: Patients with advanced/metastatic solid tumors, with at least one measurable lesion as determined by RECIST version 1.1, who have received standard therapy or are intolerant of standard therapy, have progressed following their last prior therapy, and fit into one of the following groups:
- Group 1: NSCLC (no selection for PD-L1)
- Group 2: Melanoma (no selection for PD-L1)
- Group 3: Gastric cancer and esophageal adenocarcinoma (including tumors involving the gastro-esophageal junction) positive for PD-L1 expression.
- Group 4: CRC that is MSI-High (MSI-H), positive for PD-L1 expression
- Group 5: Anal cancer positive for PD-L1 expression
- ECOG Performance Status ? 2.
- Patients must have a site of disease amenable to biopsy, and be a candidate for tumor biopsy. Patient must be willing to undergo a new tumor biopsy at baseline, and during therapy on this study.
- History of severe hypersensitivity reactions to other mAbs
- Active autoimmune disease or a documented history of autoimmune disease, except vitiligo or resolved childhood asthma/atopy.
- Active infection requiring systemic antibiotic therapy.
- Known history of HIV infection.
- Active HBV or HCV infection.
- Systemic anti-cancer therapy within 2 weeks of the first dose of study treatment. For cytotoxic agents that have major delayed toxicity, e.g. mitomycin C and nitrosoureas, 4 weeks washout period.
- Prior PD-1
- or PD-L1-directed therapy.
- Patients receiving treatment with systemic steroid therapy, other than replacement-dose steroids in the setting of adrenal insufficiency. Topical, inhaled, nasal and ophthalmic steroids are not prohibited.
- Patients receiving systemic treatment with any immunosuppressive medication.
- Use of any vaccines against infectious diseases (e.g. influenza, varicella, pneumococcus) within 4 weeks of initiation of study treatment.
- Presence of ? CTCAE grade 2 toxicity (except alopecia, peripheral neuropathy and ototoxicity, which are excluded if ? CTCAE grade 3) due to prior cancer therapy Other protocol defined Inclusion/Exclusion may apply.
18 Years and older
Accepts Healthy Volunteers
Study Locations and Contact Information (3)
|Massachusetts General Hospital - Boston, Massachusetts||2.8 miles||Carrie Quadrinofirstname.lastname@example.org|
|Sarah Cannon Research Institute SCRI RC - Nashville, Tennessee||942.8 miles||Leah Flynnemail@example.com|
|Oregon Health Science University SC10 - Portland, Oregon||2,538.3 miles||Cassie Thompkinsfirstname.lastname@example.org|