ACT001: This phase I trial is evaluating the study drug ACT001 in patients with advanced (solid) cancersA phase 1 dose-escalation study to evaluate the safety, tolerability and pharmacokinetics of ACT001 in patients with advanced solid tumors

Inclusion

1. Histologically or cytologically confirmed advanced or metastatic solid tumor, including glioblastoma, with no standard treatment options.
2. Patients with recurrent glioblastoma who satisfy all of the following may be enrolled:
- Progressive tumor despite prior treatment with radiation therapy and temozolomide.
- Measurable disease by Response Assessment in Neuro-Oncology (RANO) criteria using gadolinium-enhanced MRI scanning.
- Either on no glucocorticoids or on a stable dose for 7 days prior to the first dose of study therapy.
- No evidence of intracranial hemorrhage (except for stable grade 1 hemorrhage), not receiving therapeutic anticoagulation or anti-platelet therapy, and have a normal INR and APTT.
- No prior treatment with bevacizumab or other antiangiogenic drugs.
- No radiation in the past 3 months.
3. Male or female and at least 18 years of age.
4. Adequate organ function (ANC more or equal to 1.5 x 10 to the power of 9 /L, platelets more or equal to 75 x 10 to the power of 9 /L, Hb more or equal to 10 g/dl; total bilirubin less or equal to 1.5 times the institutional upper limit of normal (ULN); ALT and AST less or equal to 2.5 times ULN (less or equal to 5.0 times ULN if liver metastasis); plasma creatinine less or equal to 1.5 times ULN;QTc less than 450 ms (male), less than 470 ms (female).
5. A life expectancy of at least 12 weeks.
6. Eastern Cooperative Oncology Group (ECOG) performance status 0-1.
7. Female subjects are eligible if they are of:
a) Non-childbearing potential, defined as
- Previous hysterectomy or bilateral oophorectomy.
- Previous bilateral tubal ligation.
- Post-menopausal (total cessation of menses for at least 1 year).
b) Childbearing potential with a negative serum pregnancy test at screening (within 7 days of the first investigational product administration), and uses adequate contraception before study entry and throughout the study until 28 days after the last investigational product administration. Adequate contraception, when used consistently and in accordance with both the product label and the instructions of the physician, are defined as follows:
- Vasectomized partner who is sterile prior to the female patient’s enrolment and is her sole sexual partner.
- An intrauterine device with a documented failure rate of less than 1% per year.
- Double barrier contraception defined as condom with a female diaphragm.
8. Male patients, if sexually active, must agree to use a highly-effective method of contraception (< 1% failure rate per year) with their female partners.
9. Provided written informed consent prior to enrollment.