OTX-2002-101

About this trial

Last updated 2 years ago

Study ID

OTX-2002-101

Status

Recruiting

Type

Interventional

Phase

Phase 1/Phase 2

Placebo

Accepting

18+ Years

All

Not accepting

Healthy Volunteers

Trial Timing

Started 3 years ago

This is a Phase 1/2 open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a single agent and in combination with standard of care in patients with hepatocellular carcinoma (HCC) and other solid tumor types known for association with the MYC oncogene. The study consists of Part 1 (OTX-2002 monotherapy) and Part 2 (OTX-2002 combined with standard of care in hepatocellular carcinoma). Part 1 consists of escalation and expansion, and Part 2 consists of safety run-in and expansion. The objective of Part 1 escalation and Part 2 safety run-in will be safety and tolerability, while anti-tumor activity will be evaluated as the primary endpoint in Part 1 and Part 2 expansion.

Key inclusion

- Participants with metastatic, advanced (non-resectable), or recurrent solid tumor who
progressed on, relapsed after, are refractory to, or intolerant of standard of care
(only applicable to Part 1 escalation)

- Participants with BCLC Stage B (intermediate stage) or C (advanced stage), Child-Pugh
A hepatocellular carcinoma who is not amenable to locoregional therapy, refractory to
locoregional therapy or not amenable to curative treatment approach

- Adult participants age ≥ 18 years at the time of signing informed consent

- Participant must have progressed on, have relapsed after, be refractory to, or be
intolerant of at least 1 prior systemic therapy, and without available subsequent
standard of care

- Participants with chronic hepatitis B must have received antiviral therapy for
hepatitis B virus (HBV) for at least 12 weeks and HBV viral load must be < 500 IU/mL
prior to first dose of study drug.

- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1

Key exclusion

- Mixed histology cholangiocarcinoma and HCC, or fibrolamellar variant HCC

- Hepatocellular carcinoma with ≥ 50% liver occupation

- Clear invasion into the bile duct

- Portal vein invasion with Vp4

- Active/untreated CNS metastases or carcinomatous meningitis

- History of ascites requiring paracentesis within the past 3 months

- Esophageal or gastric variceal bleeding in the past 3 months

- History of hepatic encephalopathy in the past 3 months.