A Phase 1b Study to Assess the Safety, Feasibility, Pharmacokinetics, and Activity of PTC299 Monotherapy or Combination Therapy With Hormonal Agents in Patients With Metastatic Breast Cancer
The study will be conducted in 2 stages. In Stage 1 of the study, successive groups of 3 to
6 patients will receive progressively higher PTC299 dose levels; in this stage, treatment
will be given in repeated 6-week cycles consisting of 4 weeks of oral PTC299 twice per day
followed by a 2-week, no-drug period. During Stage 2, study candidates must be women with
natural or induced suppression of ovarian function to post-menopausal levels who are
receiving or are candidates for hormonal therapy. These subjects will receive continuous
administration of PTC299, 100 mg/dose BID, in repeated 6-week cycles in combination with
continuous administration of one of 3 hormonal agents. All planned PTC299 dose levels in
all stages are expected to achieve circulating blood levels of PTC299 known to be active in
animal models of human cancer. Treatment for each patient can continue as long as the
therapy appears to be safely offering tumor control to that patient. Up to 36 evaluable
patients will be accrued across both stages.
Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum Tolerated Dose (MTD) within the tested dose range.
Jay Barth, MD
PTC Therapeutics, Inc.
United States: Food and Drug Administration
|Memorial Sloan-Kettering Cancer Center||New York, New York 10021|
|New York University Clinical Cancer Center||New York, New York 10016|
|Indiana University Simon Cancer Center||Indianapolis, Indiana 46202|