PTC299 for Treatment of Advanced Cancer

Inclusion Criteria

• •1. Age ≥18 years.
• •2. Body weight 40-100 kg.
• •3. Capable of swallowing oral medication.
• •4. The Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
• •5. Life expectancy \>3 months.
• •6. Histologically or cytologically confirmed diagnosis of a solid tumor. Note: Participants with lymphomas may be enrolled. Participants with leukemia should not be included.
• •7. Presence of locally advanced or metastatic disease that is not amenable to surgery, radiation therapy, or chemotherapy with curative intent.
• •8. Cancer progression on or after standard therapy or cancer for which no standard therapy is available.
• •9. Discontinuation of all anticancer therapies ≥3 weeks before initiation of study treatment. Note: Prior treatment with antiangiogenic therapies (for example, bevacizumab, sunitinib, sorafenib, or investigational antiangiogenic agents) is allowed.
• •10. Acute toxic effects (as evaluated by CTCAE, Version 3.0) of any prior therapy resolved as shown below:
• •* Neuropathy - Grade ≤2 (Stage 1 and 2)
• •* Neuropathy - Grade ≤1 (Stage 3)
• •* Alopecia - Grade ≤2 (all stages)
• •* Fatigue - Grade ≤2 (all stages)
• •* All others - Grade ≤1 (all stages)
• •11. Required baseline laboratory data:
• •* Absolute neutrophil count ≥1,500/cubic millimeter (mm\^3)
• •* Platelets ≥100,000/mm\^3
• •* Hemoglobin ≥9.0 grams/deciliter (g/dL)
• •* Serum total bilirubin \<ULN; ≤1.5x ULN is acceptable if there is liver involvement secondary to tumor
• •* Serum Alanine transaminase and Aspartate transaminase \<ULN; ≤2.5x ULN is acceptable if there is liver involvement secondary to tumor
• •* Serum alkaline phosphatase ≤2.5x ULN regardless of liver involvement with tumor
• •* Serum albumin ≥3.0 g/dL
• •* Serum creatinine ≤2.0 milligrams/liter (mg/L)
• •* Urine protein \<2+ by dipstick (or spot urinary protein: creatinine ratio \<1.0 mg/dL:mg/dL, if quantitative method used)
• •* Prothrombin time and Activated partial thromboplastin time ≤ULN
• •* Serum beta-human chorionic gonadotropin (HCG) negative
• •12. Willingness, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ an effective method of contraception during the study periods.
• •13. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions.
• •14. Ability to provide written informed consent.
• •15. Evidence of a personally signed informed consent indicating that the participant is aware of the neoplastic nature of his or her disease and has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, possible side effects, potential risks and discomforts, and other pertinent aspects of study participation.
• •16. An interval of \>2 weeks from corticosteroid dose stabilization prior to obtaining the baseline MRI scan in participants with CNS malignancy.