Trial Title:
A Phase 1/2 Study to Evaluate OTX-2002 in Patients with Hepatocellular Carcinoma and Other Solid Tumor Types Known for Association with the MYC Oncogene
NCT ID:
NCT05497453
Condition:
Hepatocellular Carcinoma
Solid Tumor
Hepatocellular Carcinoma Non-resectable
Hepatocellular Carcinoma Recurrent
Hepatocellular Cancer
Liver Cancer
Liver, Cancer Of, Non-Resectable
Conditions: Official terms:
Carcinoma
Carcinoma, Hepatocellular
Liver Neoplasms
Immune Checkpoint Inhibitors
Tyrosine Kinase Inhibitors
Conditions: Keywords:
MYC
C-MYC
MYC Amplification
MYC Overexpression
MRNA
Epigenetics
Study type:
Interventional
Study phase:
Phase 1/Phase 2
Overall status:
Recruiting
Study design:
Allocation:
Non-Randomized
Intervention model:
Parallel Assignment
Intervention model description:
Part 1 OTX-2002 monotherapy dose escalation and expansion in participants with
hepatocellular carcinoma (HCC) and other solid tumors
During the monotherapy dose escalation phase, participants will be enrolled in sequential
cohorts of increasing doses of OTX-2002.
Part 2 of the study is a safety run-in and expansion study of OTX-2002 participants with
HCC will be administered OTX-2002 in combination with tyrosine kinase inhibitor One (Part
2A), Tyrosine Kinase Inhibitor Two (Part 2B), or Checkpoint Inhibitor (Part 2C).
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
OTX-2002
Description:
OTX-2002 is a lipid nanoparticle (LNP) that contains a biscistronic mRNA that codes for 2
independent epigenomic controllers.
Arm group label:
OTX-2002
Arm group label:
OTX-2002 + Checkpoint Inhibitor
Arm group label:
OTX-2002 + Tyrosine Kinase Inhibitor One
Arm group label:
OTX-2002 + Tyrosine Kinase Inhibitor Two
Intervention type:
Drug
Intervention name:
Tyrosine kinase inhibitor One
Description:
Tyrosine Kinase Inhibitor
Arm group label:
OTX-2002 + Tyrosine Kinase Inhibitor One
Intervention type:
Drug
Intervention name:
Tyrosine kinase inhibitor Two
Description:
tyrosine kinase inhibitor
Arm group label:
OTX-2002 + Tyrosine Kinase Inhibitor Two
Intervention type:
Drug
Intervention name:
Checkpoint Inhibitor, Immune
Description:
monoclonal antibody that binds to PD-1 or PD-L1
Arm group label:
OTX-2002 + Checkpoint Inhibitor
Summary:
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.
Detailed description:
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).
In Part 1, during dose escalation, participants with HCC and other solid tumors that
progressed on, relapsed after, are refractory to, or are intolerant of standard of care
for which no treatment options are available will be administered an intravenous infusion
of OTX-2002 as a single agent. The escalation will be conducted using a 3+3 design, with
the primary endpoint of dose limiting toxicity (DLT), maximum tolerated dose (MTD), and
incidence of treatment emergent adverse events (TEAEs). In Part 1 expansion, 15-25
participants with BCLC stage B or C, Child-Pugh Class A HCC who have received at least 1
prior line of systemic anticancer treatment, and without available subsequent standard of
care, will receive OTX-2002 at the recommended dose for expansion (RDE) for monotherapy.
The primary endpoint of Part 1 expansion will be overall response rate (ORR) and duration
of response (DoR).
In Part 2, during safety run-in, participants with BCLC stage B or C, Child-Pugh Class A
HCC who have received at least 1 prior line of systemic anticancer treatment, and without
available subsequent standard of care, will receive OTX-2002 at the selected dose in
combination with standard of care therapies at the local approved dose. The primary
endpoint of Part 2 safety run-in will be DLT, MTD, and incidence of TEAE. Once the
combination therapies have been determined to be tolerable in the safety run-in, 15-25
HCC participants will be enrolled in Part 2 expansion for each of the combination
therapies. The primary endpoint of Part 2 expansion will be ORR and DoR.
Criteria for eligibility:
Criteria:
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.
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
City of Hope
Address:
City:
Duarte
Zip:
91010
Country:
United States
Status:
Recruiting
Contact:
Last name:
Daneng Li
Phone:
626-471-9200
Email:
danli@coh.org
Facility:
Name:
University of Florida Health Cancer Center
Address:
City:
Gainesville
Zip:
32610
Country:
United States
Status:
Recruiting
Contact:
Last name:
Taryn King
Phone:
352-733-0299
Email:
King.taryn@ufl.edu
Facility:
Name:
University of Chicago
Address:
City:
Chicago
Zip:
60637
Country:
United States
Status:
Recruiting
Contact:
Last name:
Aurelie Desgardin
Phone:
773-834-7188
Email:
adesgard@bsd.uchicago.edu
Contact backup:
Last name:
Hang Chang
Phone:
773-702-3482
Email:
hchang@bsd.uchicago.edu
Facility:
Name:
Ochsner Clinic Foundation
Address:
City:
New Orleans
Zip:
70121
Country:
United States
Status:
Recruiting
Contact:
Last name:
Sharon Jerdonek
Phone:
15048423929
Email:
sharon.jerdonek@ochsner.org
Facility:
Name:
Stephenson Cancer Center at Oklahoma University
Address:
City:
Oklahoma City
Zip:
73104
Country:
United States
Status:
Recruiting
Contact:
Last name:
Susanna V Ulahannan
Phone:
405-217-3020
Phone ext:
48955
Email:
susanna-ulahannan@ouhsc.edu
Facility:
Name:
Fred Hutch / University of Washington
Address:
City:
Seattle
Zip:
98109
Country:
United States
Status:
Recruiting
Contact:
Last name:
GI Nurse Navigator
Phone:
206-606-4800
Email:
ginursenavslu@seattlecca.org
Facility:
Name:
Prince of Wales Hospital
Address:
City:
Hong Kong
Country:
Hong Kong
Status:
Recruiting
Contact:
Last name:
Stephen Lam Cham
Phone:
85-2-3505-1042
Email:
chanlam_stephen@cuhk.edu.hk
Facility:
Name:
Queen Mary Hospital
Address:
City:
Hong Kong
Country:
Hong Kong
Status:
Recruiting
Contact:
Last name:
Thomas Chung Cheung Yau
Phone:
852-2255-1661
Email:
tyaucc@hku.hk
Facility:
Name:
Asan Medical Center
Address:
City:
Seoul
Country:
Korea, Republic of
Status:
Recruiting
Contact:
Last name:
Min-hee Ryu
Phone:
82-2-3010-5935
Email:
miniryu@amc.seoul.kr
Facility:
Name:
Seoul National University Hospital
Address:
City:
Seoul
Country:
Korea, Republic of
Status:
Recruiting
Contact:
Last name:
Tae-Yong Kim
Phone:
82-2-2072-4748
Email:
ktyongmd@gmail.com
Facility:
Name:
Severance Hospital, Yonsei University Health System
Address:
City:
Seoul
Country:
Korea, Republic of
Status:
Recruiting
Contact:
Last name:
Choong-Kun Lee
Phone:
82-2-2228-8122
Email:
cklee512@yuhs.ac
Facility:
Name:
National Cancer Center Singapore
Address:
City:
Singapore
Country:
Singapore
Status:
Recruiting
Contact:
Last name:
David Tai Wai-Meng
Phone:
65-6436-8000
Email:
clinical.trials@nccs.com.sg
Facility:
Name:
National University Hospital
Address:
City:
Singapore
Country:
Singapore
Status:
Recruiting
Contact:
Last name:
Yong Wei Peng
Phone:
65-6773-7888
Email:
ncis@nuhs.edu.sg
Facility:
Name:
National Cheng Kung University Hospital
Address:
City:
Tainan
Country:
Taiwan
Status:
Recruiting
Contact:
Last name:
Chia-Jui Yen
Phone:
886-912-377366
Email:
yencj@mail.ncku.edu.tw
Facility:
Name:
National Taiwan University Hospital
Address:
City:
Taipei
Country:
Taiwan
Status:
Recruiting
Contact:
Last name:
Chia-Chi Lin
Phone:
886-919-171-263
Email:
cclin1@ntu.edu.tw
Start date:
August 19, 2022
Completion date:
December 2028
Lead sponsor:
Agency:
Omega Therapeutics
Agency class:
Industry
Source:
Omega Therapeutics
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05497453
