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Clinical Trial 19967

Cancer Type: Cutaneous
Study Type: Treatment
NCT#: NCT03897036

Phase: Phase I
Principal Investigator: Eroglu, Zeynep

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Study Title

A Phase I, Multi-Center, Open-Label, Treatment Duration Increment, Expansion, Safety, and Pharmacodynamic Study of CX-4945 Administered Orally Twice Daily to Patients with Advanced Basal Cell Carcinoma



Primary Objective The primary objective of this study is to determine the recommended phase II dose (RP2D) and schedule of CX-4945 when administered orally twice daily for 28 consecutive days, in a 4-week (28 days) cycle, in patients with locally advanced or metastatic basal cell carcinoma (BCC). Secondary Objective: To establish the safety and tolerability of CX-4945 in this patient population. To assess preliminary evidence of antitumor effects in this patient population by documentation of objective responses using standardized criteria. To evaluate the effect of CX-4945 treatment on the Hh signaling pathway using qRT-PCR in fresh-frozen tissue from patients with locally advanced BCC obtained at baseline and following CX-4945 treatment



Therapy (NOS)


CX-4945 ()

Inclusion Criteria

  • 18 years of age or older
  • For patients with metastatic basal cell carcinoma (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
  • Phase I Expansion: 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 patients should be considered as having mBCC and should be enrolled in the mBCC cohort
  • For patients with locally advanced BCC, histologically confirmed disease with at least one lesion that was 10 mm or more in at least 1 dimension by color photograph 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
  • Acceptable medical contraindications to surgery include: (1) BCC that has recurred in the same location after two or more surgical procedures and curative resection is deemed unlikely (2) 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) (3) Other conditions considered to be medically contraindicating must be discussed with the Medical Monitor before enrolling the patient.
  • For all patients, smoothened inhibitor must have been previously administered for their locally advanced or metastatic BCC, unless smoothened inhibitor is inappropriate (e.g., patient has received a smoothened inhibitor but became intolerant to the therapy). For patients whose BCC has been treated with smoothened inhibitor, disease must have progressed after treatment.
  • 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.
  • Previous Therapy: (1) Surgery: Previous surgery is permitted provided that a minimum of 28 days (4 weeks) have elapsed between any major surgery and date of registration, and that wound healing has occurred.(2) Cytotoxic Chemotherapy: There is no limit to the number of prior regimens received. (3) Other Systemic Therapy: Previous treatment with Hh pathway antagonists is not allowed (except for Smoothened inhibitors). There is no limit to the other prior therapies received
  • Patients must have recovered (to baseline or ≤ grade 1) from all reversible toxicity related to prior chemotherapy or systemic therapy and have adequate washout as outlined per protocol.
  • 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.
  • 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
  • Adequate hematopoietic capacity as defined per protocol.
  • Adequate hepatic function, as defined per protocol.
  • Adequate renal function, as defined per protocol.
  • Women/men of childbearing potential must have agreed to use two effective contraceptive methods while on study and for 6 months after the last dose of CX-4945.

  • Exclusion Criteria

  • Tumor histology consistent with basosquamous carcinoma (basal cell carcinoma with squamous differentiation or metatypical carcinoma).
  • Pregnant or nursing women.
  • 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. 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 CX-4945, surgery with curative intent may be permitted but must be discussed with the Medical Monitor prior to any surgical procedure.
  • History of other malignancies within 3 years of Day 1, exceptions apply.
  • Active or uncontrolled infections or with serious illnesses or medical conditions which would not permit the patient to be managed according to the protocol.
  • 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
  • Difficulty with swallowing oral medications
  • Chronic diarrhea (excess of 2-3 stools/day above normal frequency)

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