Procaspase Activating Compound-1 (PAC-1) in the Treatment of Advanced Malignancies - Component 1

Completed · Phase 1

Conditions studied: Solid Tumor, Pancreatic Neuroendocrine Tumor, Neuroendocrine Tumors

In brief

This Phase I dose escalation study will evaluate Procaspase Activating Compound-1 (PAC-1), a small molecule that activates procaspase -3 to caspase-3, resulting in apoptosis of cancer cells, in patients with advanced malignancies. As of March 1, 2019, only patients with neuroendocrine tumors will be enrolled in Component 1 of this study. PAC-1 is taken orally on days 1-21 of a 28-day cycle. The maximum tolerated dose (MTD) of PAC-1 (5 dose levels) will be determined using a modified-Fibonacci dose-escalation 3+3 design. Treatment continues until disease progression, unacceptable toxicity, physician discretion, or patient refusal.

Key facts

Study ID: NCT02355535
Run by: Vanquish Oncology, Inc.
People needed: 48
Starts: 2015-02-01
Expected to finish: 2020-05-18
Last updated by the study team: 2020-09-24

Who can join

Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Male or female ≥ 18 years of age
Diagnosis of advanced solid tumor or hematologic malignancy (limited to lymphoma) that has failed or become intolerant to standard therapy
Has measurable disease, defined as at least 1 tumor that fulfills the criteria for a target lesion according to RECIST 1.1, or lymphoma that fulfills the Deauville PET Criteria
Has an ECOG PS of 0, 1, or 2
Has total bilirubin < 1.5 mg/dL, serum albumin > 3.0 gm/dL, AST and ALT < 1.5 ULN or < 3 x ULN for subjects with known hepatic metastases
Has serum creatinine < 1.5 × ULN
Has hemoglobin ≥ 10 g/dL, ANC ≥ 1.5 × 109/L, and platelet count ≥ 100 × 109/L
Must be able to take oral medication and to maintain a fast as required for 2 hours before and 1 hour after capsule(s) administration
Must be willing and able to comply with study
Has read, understood, and signed the ICF
Women of childbearing potential must not be pregnant or breast-feeding. In addition, a medically acceptable method of birth control must be used or total abstinence. Women who are postmenopausal for at least 1 year or surgically sterile (bilateral tubal ligation, bilateral oophorectomy, or hysterectomy) are not considered to be WOCP
Men who are not surgically or medically sterile must agree to use an acceptable method of contraception. Male patients with female sexual partners who are pregnant, possibly pregnant, or who could become pregnant during the study must agree to use condoms at least one month after the last dose of study drug. Total abstinence for the same study period is an acceptable alternative
Prior systemic treatments for metastatic disease are permitted but may not be ongoing, including targeted therapies, biologic response modifiers, chemotherapy, hormonal therapy, or investigational therapy
Willingness to donate blood for biomarker studies related to the type of therapies used in this trial and the tumor types being treated

You may not qualify if…

Had surgery within 4 weeks prior to study treatment except for minor procedures (hepatic biliary stent placement is allowed)
Gliomas are excluded, as well as any history of brain metastases, seizures or underlying brain injury
May not have received cytotoxic chemotherapy, targeted therapies, biologic response modifiers, chemotherapy, and hormonal therapy within the last 3 weeks, or nitrosureas within the last 6 weeks prior to study treatment.
Has a history of blood clots, pulmonary embolism, or DVT unless controlled by anticoagulant treatment
Has a history of an arterial thromboembolic event within the prior six months including CVA, TIA, MI, or unstable angina
Has uncontrolled HIV or hepatitis B or C
Has any clinically significant infection
Has any other severe, uncontrolled medical condition, including uncontrolled DM or unstable CHF
Radiation therapy to more than 25% of the bone marrow
Prior allogeneic bone marrow or organ transplantation
> Grade 1 peripheral neuropathy within 14 days before enrollment.
Patient has received other investigational drugs with 14 days before enrollment
Other severe acute or chronic medical or psychiatric conditions or laboratory abnormality that may increase the risk associated with study participation
Abnormalities on 12-lead electrocardiogram (ECG) considered by the investigator to be clinically significant (such as acute ischemia, left bundle branch block, ventricular arrhythmias) or baseline prolongation of the rate-corrected QT interval (e.g., repeated demonstration of QTc interval > 480 milliseconds)
Presence of any non-healing wound, fracture, or ulcer
Has any condition that, in the opinion of the investigator, might jeopardize the safety of the patient or interfere with protocol compliance
Has any mental or medical condition that prevents the patient from giving informed consent

Where it is running

University of Illinois at Chicago — Chicago, Illinois, United States
Johns Hopkins Kimmel Cancer Center — Baltimore, Maryland, United States
Regions Hospital — Saint Paul, Minnesota, United States

Full record on ClinicalTrials.gov

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