24 Participants Needed

OTX-2002 for Liver Cancer

(MYCHELANGELO I Trial)

Recruiting at 15 trial locations
CM
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Overseen ByHead of Clinical Development Operations
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Omega Therapeutics
Must be taking: Antivirals
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications for this trial?

The trial protocol does not specify if you need to stop your current medications. However, if you have chronic hepatitis B, you must continue antiviral therapy for at least 12 weeks before starting the study drug.

What data supports the idea that OTX-2002 for Liver Cancer is an effective treatment?

The available research does not provide specific data on the effectiveness of OTX-2002 for Liver Cancer. Instead, it focuses on liver transplantation and other treatments for liver cancer, such as hepatic resection and systemic therapies. These studies show improved survival rates for liver transplantation over the years and compare it to other treatments, but they do not mention OTX-2002. Therefore, there is no direct evidence from the provided information to support the effectiveness of OTX-2002 for Liver Cancer.12345

What safety data is available for OTX-2002 in liver cancer treatment?

The provided research does not contain specific safety data for OTX-2002 or OTX 2002 in liver cancer treatment. The articles focus on general topics such as radiation oncology toxicity, immune-related adverse events in PD-1 blockade immunotherapy, adverse event reporting in cancer trials, liver injury in pediatric oncology, and real-time toxicity monitoring in clinical trials. None of these directly address safety data for OTX-2002.678910

Is the drug OTX-2002 a promising treatment for liver cancer?

The information provided does not directly mention OTX-2002 or its effects on liver cancer, so we cannot determine if it is a promising treatment based on the given research articles.211121314

Research Team

YM

Yan Moore, MD

Principal Investigator

Omega Therapeutics

Eligibility Criteria

Adults over 18 with advanced liver cancer (hepatocellular carcinoma) or solid tumors linked to the MYC gene, who've tried standard treatments without success. They must have a good performance status and controlled hepatitis B if present. Those with severe liver complications, brain metastases, recent bleeding from esophageal varices, or specific types of mixed liver cancers cannot join.

Inclusion Criteria

My liver cancer is at an intermediate or advanced stage and cannot be treated with local therapies.
I am 18 years old or older.
My condition worsened or didn't improve after at least 1 treatment, and no standard treatment options are left.
See 3 more

Exclusion Criteria

My cancer has spread to the main blood vessel of my liver.
I have brain metastases or carcinomatous meningitis that hasn't been treated.
I have not had hepatic encephalopathy in the last 3 months.
See 5 more

Treatment Details

Interventions

  • OTX-2002
Trial OverviewOTX-2002 is being tested alone and alongside standard treatments for its safety and effectiveness against certain cancers. The trial has two parts: first testing OTX-2002 by itself (monotherapy), then in combination with other cancer drugs (combination therapy).
Participant Groups
4Treatment groups
Experimental Treatment
Group I: OTX-2002 + Tyrosine Kinase Inhibitor TwoExperimental Treatment2 Interventions
OTX-2002 + Tyrosine Kinase Inhibitor Two : (Cycle length = 4 weeks) OTX-2002 will be administered as an IV infusion over 80-120 minutes every 2 weeks. Tyrosine Kinase Inhibitor Two will be standard per the respective fixed local approved dose
Group II: OTX-2002 + Tyrosine Kinase Inhibitor OneExperimental Treatment2 Interventions
OTX-2002 + Tyrosine Kinase Inhibitor One: (Cycle length = 4 weeks) OTX-2002 will be administered as an IV infusion over 80-120 minutes every 2 weeks. Tyrosine Kinase Inhibitor One will be standard per the respective fixed local approved dose
Group III: OTX-2002 + Checkpoint InhibitorExperimental Treatment2 Interventions
OTX-2002 + Immune Checkpoint Blockade: (Cycle length = 6 weeks) OTX-2002 will be administered as an IV infusion over 80-120 minutes every 2 weeks. Checkpoint Inhibitor will be standard per the respective fixed local approved dose
Group IV: OTX-2002Experimental Treatment1 Intervention
Monotherapy: OTX-2002 (Cycle length = 4 weeks) OTX-2002 will be administered as an IV infusion over 80-120 minutes every 2 weeks

OTX-2002 is already approved in United States for the following indications:

🇺🇸
Approved in United States as OTX-2002 for:
  • Hepatocellular carcinoma (Orphan Drug Designation)

Find a Clinic Near You

Who Is Running the Clinical Trial?

Omega Therapeutics

Lead Sponsor

Trials
1
Recruited
20+

Findings from Research

In a study of 181 patients with hepatocellular carcinoma (HCC) treated between 1980 and 1989, those who underwent orthotopic liver transplantation (TX) had significantly better survival rates compared to those who had subtotal hepatic resection (HX) when HCC was associated with liver cirrhosis.
Both treatment methods had high tumor recurrence rates, particularly in advanced stages, indicating a need for additional nonsurgical anti-cancer therapies to improve long-term survival outcomes.
Hepatic resection versus transplantation for hepatocellular carcinoma.Iwatsuki, S., Starzl, TE., Sheahan, DG., et al.[2022]
Liver transplantation (LTx) can lead to prolonged cancer-free survival in patients with cirrhosis and hepatocellular carcinoma (HCC), with an 82% survival rate at 22.5 months post-transplant for those meeting selection criteria.
Among the 28 patients with cirrhosis and HCC studied, 87% survived for 1 year and 76% for 3 years post-LTx, showing comparable survival rates to those undergoing LTx for other malignancies and benign conditions, indicating the efficacy of LTx in this patient group.
An active liver transplant programme for hepatocellular carcinoma in cirrhotic patients: is it justified?Chui, AK., Rao, AR., McCaughan, GW., et al.[2022]
Objective response (OR) measured by modified RECIST (mRECIST) is an independent predictor of overall survival (OS) in patients with advanced hepatocellular carcinoma (HCC), based on a systematic review of 34 randomized clinical trials involving 14,056 patients.
While OR-mRECIST shows a better correlation with OS than OR-RECIST, the surrogacy is considered modest, suggesting it can be used as an endpoint in early phase trials but not as a primary endpoint in larger phase III studies.
Objective Response Predicts Survival in Advanced Hepatocellular Carcinoma Treated with Systemic Therapies.Kudo, M., Montal, R., Finn, RS., et al.[2022]

References

Hepatic resection versus transplantation for hepatocellular carcinoma. [2022]
An active liver transplant programme for hepatocellular carcinoma in cirrhotic patients: is it justified? [2022]
Objective Response Predicts Survival in Advanced Hepatocellular Carcinoma Treated with Systemic Therapies. [2022]
Present status and future prospects in liver transplantation. [2009]
Improved results of transplantation for hepatocellular carcinoma: a report from the International Registry of Hepatic Tumors in Liver Transplantation. [2010]
Electronic Medical Record-Based Radiation Oncology Toxicity Recording Instrument Aids Benchmarking and Quality Improvement in the Clinic. [2019]
Immune-related adverse events predict responses to PD-1 blockade immunotherapy in hepatocellular carcinoma. [2021]
Early detection of toxicity and adjustment of ongoing clinical trials: the history and performance of the North Central Cancer Treatment Group's real-time toxicity monitoring program. [2016]
Use and misuse of common terminology criteria for adverse events in cancer clinical trials. [2018]
10.United Statespubmed.ncbi.nlm.nih.gov
Incidence and Sequelae of Liver Injury Among Children Treated for Solid Tumors: Analysis of a Large Single-Center Prospective Cohort. [2022]
Impact of CDX2 expression status on the survival of patients after curative resection for colorectal cancer liver metastasis. [2022]
Orthotopic liver transplantation in hepatocellular carcinoma: comparison of results in incidental and known hepatocellular carcinoma. [2009]
Liver transplantation for hepatocellular carcinoma: prognostic factors associated long-term survival. [2019]
14.Korea (South)pubmed.ncbi.nlm.nih.gov
OTX1 Contributes to Hepatocellular Carcinoma Progression by Regulation of ERK/MAPK Pathway. [2018]