Hepatocellular CarcinomaPhase 1/2MYCHELANGELO INCT05497453
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
A Phase 1/2 study testing OTX-2002, Tyrosine kinase inhibitor One and Tyrosine kinase inhibitor Two in people with Hepatocellular Carcinoma and Solid Tumor, run by Omega Therapeutics at 15 sites in 5 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatocellular Carcinoma, Solid Tumor, Hepatocellular Carcinoma Non-resectable
- What is being tested?
- OTX-2002, Tyrosine kinase inhibitor One and Tyrosine kinase inhibitor Two
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 15 sitesin 5 countries
- Study dates
- 19 Aug 2022to 21 Nov 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
15 sites in 5 countries.
15 sites in 5 countries
United States6 sites
University of Chicago
Chicago, Illinois, 60637
City of Hope
Duarte, California, 91010
University of Florida Health Cancer Center
Gainesville, Florida, 32610
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
Stephenson Cancer Center at Oklahoma University
Oklahoma City, Oklahoma, 73104
Fred Hutch / University of Washington
Seattle, Washington, 98109
South Korea3 sites
Asan Medical Center
Seoul
Seoul National University Hospital
Seoul
Severance Hospital, Yonsei University Health System
Seoul
Hong Kong2 sites
Prince of Wales Hospital
Hong Kong
Queen Mary Hospital
Hong Kong
Singapore2 sites
National Cancer Center Singapore
Singapore
National University Hospital
Singapore
Taiwan2 sites
National Cheng Kung University Hospital
Tainan
National Taiwan University Hospital
Taipei
About this study
Official title: 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 and Other Solid Tumor Types Known for Association with the MYC Oncogene
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.
Read the 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.
- Conditions
- Hepatocellular Carcinoma, Solid Tumor, Hepatocellular Carcinoma Non-resectable, Hepatocellular Carcinoma Recurrent, Hepatocellular Cancer, Liver Cancer
- Being tested
- OTX-2002, Tyrosine kinase inhibitor One, Tyrosine kinase inhibitor Two, Checkpoint Inhibitor, Immune
- Keywords
- MYC, C-MYC, MYC Amplification, MYC Overexpression, MRNA, Epigenetics
- Medical subject headings
- Carcinoma, Hepatocellular, Liver Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OTX-2002Drug
OTX-2002 is a lipid nanoparticle (LNP) that contains a biscistronic mRNA that codes for 2 independent epigenomic controllers.
Tyrosine kinase inhibitor OneDrug
Tyrosine Kinase Inhibitor
Tyrosine kinase inhibitor TwoDrug
tyrosine kinase inhibitor
Checkpoint Inhibitor, ImmuneDrug
monoclonal antibody that binds to PD-1 or PD-L1
| Study group | Role | What they receive |
|---|---|---|
| OTX-2002 | Experimental | Monotherapy: OTX-2002 (Cycle length = 4 weeks) OTX-2002 will be administered as an IV infusion over 80-120 minutes every 2 weeks Receives: Drug: OTX-2002 |
| OTX-2002 + Tyrosine Kinase Inhibitor One | Experimental | 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 Receives: Drug: OTX-2002; Drug: Tyrosine kinase inhibitor One |
| OTX-2002 + Tyrosine Kinase Inhibitor Two | Experimental | 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 Receives: Drug: OTX-2002; Drug: Tyrosine kinase inhibitor Two |
| OTX-2002 + Checkpoint Inhibitor | Experimental | 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 Receives: Drug: OTX-2002; Drug: Checkpoint Inhibitor, Immune |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine Dose limiting toxicities (DLT)and maximum tolerated dose ( MTD) (Part 1 escalation and Part 2 safety run-in)
The frequency of DLTs will be tabulated by dose for participants in the dose escalation phase, and information about all DLTs will be listed by dose.
Measured: 28 days/4 weeks from the first dose of OTX-2002
Incidence of TEAEs including all AEs,Grade 3-5 AEs, drug-related AEs, and SAEs (Part 1 escalation and Part 2 safety run-in)
The incidence of TEAEs, SAEs, and TEAEs leading to study drug discontinuation will be summarized for all patients.
Measured: 30 days after the last dose of study drug
Overall response rate (ORR)(for Part 1 and Part 2 expansion)
ORR (proportion of participants who achieve CR or PR), DCR (proportion of participants who achieve CR, PR, or SD)per mRECIST (for HCC) and RECIST 1.1(for solid tumors), during Parts 1, 2A, 2B,and 2C
•ORR (proportion of participants who achieve CR or PR), DCR (proportion of participants who achieve CR, PR, or SD)per irRECIST during Part 2C
Measured: through treatment completion, up to two years
Duration of Response (DOR) (for Part 1 and Part 2 expansion)
Duration of Complete Response and Partial Response
Measured: through treatment completion, up to two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2022
- Study start19 Aug 2022
- Primary completion21 Nov 2024
- Study completion21 Nov 2024
Why it stopped: Sponsor decision to terminate after monotherapy dose escalation.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatocellular CarcinomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- 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).
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Omega Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yan Moore, MD, Study director, Omega Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05497453
- Sponsor's protocol ID
- OTX-2002-101
- Acronym
- MYCHELANGELO I
- Status last verified
- Sep 2024
- First submitted
- 8 Aug 2022
- Last updated
- 3 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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 and Other Solid Tumor Types Known for Association with the MYC Oncogene
- ClinicalTrials.gov ID
- NCT05497453
- Acronym / sponsor ID
- MYCHELANGELO I / OTX-2002-101
- Conditions
- Hepatocellular Carcinoma, Solid Tumor, Hepatocellular Carcinoma Non-resectable, Hepatocellular Carcinoma Recurrent, Hepatocellular Cancer, Liver Cancer
- Keywords
- MYC, C-MYC, MYC Amplification, MYC Overexpression, MRNA, Epigenetics
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision to terminate after monotherapy dose escalation.
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Omega TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 19 Aug 2022 (actual)
- Primary completion
- 21 Nov 2024 (actual)
- Study completion
- 21 Nov 2024 (actual)
- First posted
- 11 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
- 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).
| Arm | Type | Receives |
|---|---|---|
| OTX-2002 | Experimental | Drug: OTX-2002Monotherapy: OTX-2002 (Cycle length = 4 weeks) OTX-2002 will be administered as an IV infusion over 80-120 minutes every 2 weeks |
| OTX-2002 + Tyrosine Kinase Inhibitor One | Experimental | Drug: OTX-2002, Drug: Tyrosine kinase inhibitor OneOTX-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 |
| OTX-2002 + Tyrosine Kinase Inhibitor Two | Experimental | Drug: OTX-2002, Drug: Tyrosine kinase inhibitor TwoOTX-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 |
| OTX-2002 + Checkpoint Inhibitor | Experimental | Drug: OTX-2002, Drug: Checkpoint Inhibitor, ImmuneOTX-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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OTX-2002OTX-2002 is a lipid nanoparticle (LNP) that contains a biscistronic mRNA that codes for 2 independent epigenomic controllers. | Drug | OTX-2002, OTX-2002 + Checkpoint Inhibitor, OTX-2002 + Tyrosine Kinase Inhibitor One, OTX-2002 + Tyrosine Kinase Inhibitor Two |
| Tyrosine kinase inhibitor OneTyrosine Kinase Inhibitor | Drug | OTX-2002 + Tyrosine Kinase Inhibitor One |
| Tyrosine kinase inhibitor Twotyrosine kinase inhibitor | Drug | OTX-2002 + Tyrosine Kinase Inhibitor Two |
| Checkpoint Inhibitor, Immunemonoclonal antibody that binds to PD-1 or PD-L1 | Drug | OTX-2002 + Checkpoint Inhibitor |
Primary outcome
Determine Dose limiting toxicities (DLT)and maximum tolerated dose ( MTD) (Part 1 escalation and Part 2 safety run-in)
Time frame: 28 days/4 weeks from the first dose of OTX-2002
The frequency of DLTs will be tabulated by dose for participants in the dose escalation phase, and information about all DLTs will be listed by dose.
Incidence of TEAEs including all AEs,Grade 3-5 AEs, drug-related AEs, and SAEs (Part 1 escalation and Part 2 safety run-in)
Time frame: 30 days after the last dose of study drug
The incidence of TEAEs, SAEs, and TEAEs leading to study drug discontinuation will be summarized for all patients.
Overall response rate (ORR)(for Part 1 and Part 2 expansion)
Time frame: through treatment completion, up to two years
ORR (proportion of participants who achieve CR or PR), DCR (proportion of participants who achieve CR, PR, or SD)per mRECIST (for HCC) and RECIST 1.1(for solid tumors), during Parts 1, 2A, 2B,and 2C •ORR (proportion of participants who achieve CR or PR), DCR (proportion of participants who achieve CR, PR, or SD)per irRECIST during Part 2C
Duration of Response (DOR) (for Part 1 and Part 2 expansion)
Time frame: through treatment completion, up to two years
Duration of Complete Response and Partial Response
Results and safety
The study was stopped early (reason given: Sponsor decision to terminate after monotherapy dose escalation.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2025.
- Individual participant dataSharing planned: Undecided
Related trials for Hepatocellular Carcinoma
Not yet recruitingPhase 1
Study of ACC-1898 in Adult Participants With Advanced Solid Tumors
Hepatocellular Carcinoma, Renal Cell Carcinoma
RecruitingObservational study
Observational Study Protocol: LIVER-R
Hepatobiliary Cancers
Enrolling by invitationPhase 3
Long-Term Extension Study for Participants Previously Enrolled in an Exelixis-Sponsored Study
Cancer, Renal Cell Carcinoma (RCC)
Active, not recruiting
BioPearl™ Microspheres Loaded With Doxorubicin for the Treatment of Unresectable Hepatocellular Carcinoma (HCC)
Hepatocellular Carcinoma Non-resectable
Active, not recruitingPhase 1
Nivolumab With or Without Relatlimab for Patients With Potentially Resectable Hepatocellular Carcinoma
Hepatocellular Carcinoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yan Moore, MD study director
Omega Therapeutics
15 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing OTX-2002, Tyrosine kinase inhibitor One and Tyrosine kinase inhibitor Two in people with Hepatocellular Carcinoma and Solid Tumor, run by Omega Therapeutics at 15 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, South Korea, Hong Kong, Singapore, Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision to terminate after monotherapy dose escalation.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine Dose limiting toxicities (DLT)and maximum tolerated dose ( MTD) (Part 1 escalation and Part 2 safety run-in) (time frame: 28 days/4 weeks from the first dose of OTX-2002). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2025.