Carcinoma, HepatocellularPhase 1OPTIMNCT03253289
Meclizine for Hepatocellular Carcinoma
A Phase 1 study testing Meclizine Oral Tablet in people with Carcinoma, Hepatocellular, run by Tannaz Armaghnay at 5 sites in United States.
Trial summary
Meclizine hydrochloride is an antihistamine widely used for treatment of vertigo and motion sickness. In HCC it has been used for anti-emetic effects, but it is used here as a CAR (constitutive androstane receptor) inverse agonist. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Carcinoma, Hepatocellular
- What is being tested?
- Meclizine Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 5 sitesUnited States
- Study dates
- 13 Oct 2017to 28 Mar 2023
- Phase
- Phase 1
- 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
- Patients must have imaging: CT or MRI abdomen with and without contrast confirmed or highly suspicious for Hepatocellular carcinoma. Patients must have a liver biopsy confirmed for Hepatocellular carcinoma.
- Patients must have measurable disease, defined as tumor mass which is >10 mm with spiral CT scan or MRI. Baseline imaging scan must be within 8 weeks of registration.
- Patients must have no prior history of treatment for HCC (treatment naïve) on the lesion that is being targeted for biopsy. New HCC lesions can arise in the liver of patients despite local therapy of other areas of the liver due to consistent underlying risk factors such as cirrhosis, and chronic hepatitis B or C infection. Patients with prior local liver directed therapy such as TACE, Ablation, Y-90 or hepatectomy surgery for HCC are eligible if they have developed a new untreated lesion in the liver which can be targeted for a biopsy for this study. There is no required time frame or washout period from when a lesion has been locally treated to the time when a new lesion is found and targeted for biopsy for this trial. Patients who have had prior systemic therapy of any kind are not eligible.
- Patients must be greater than 18 years of age.
- ECOG Performance status less than/equal to 2 (Karnofsky greater than 60%).
- Patients must have normal organ and marrow function as defined below, within 21 days of registration: Leukocytes greater than 3,000/mcL; ANC greater than 1,500/mcL; Platelets greater than 50,000/mcL; Hemoglobin greater than/equal to 8 g/dL; ALT(SGPT) less than/equal to 5X IULN and AST (SGOT) less than/equal to 5X IULN; Creatinine less than/equal to 2X IULN or Creatinine clearance greater than 60 mL/min for patients with creatinine levels greater than IULN; Child Pugh Class A (5-6 points) or B (7 points); INR less than/equal to 2.3; Albumin greater than 2.8 g/dL; Total bilirubin less than/equal to 3X IULN.
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- Patients must be candidate for surgical resection, ablation, TACE, Y90 or systemic therapy.
- Patients should have life expectancy greater than/equal to 10 weeks.
- Willingness to Use Contraception: The effects of Meclizine on the developing human fetus at the recommended therapeutic dose are unknown. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study treatment with meclizine. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. If a male participant impregnates his partner he should inform his treating physician immediately.
- Patients must be informed of the investigational nature of this study, and must sign and give written informed consent in accordance with institutional and federal guidelines.
Who cannot join
- Patients may not be receiving any other concurrent anti-cancer therapy.
- Patients may not be receiving any other concurrent investigational agents.
- Patients taking medications with a narrow therapeutic index including warfarin, digoxin, phenobarbital, carbamazepine, and cyclosporine are not excluded but should be monitored carefully.
- Patients must not be taking Rifampin or St John's Wort.
- Patient must not have a history of allergic reactions like anaphylaxis attributed to compounds of similar chemical or biologic composition to Meclizine such as antihistamine drugs.
- Patient must not be a candidate for liver transplant.
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- Child Pugh Class B (8,9) and Class C are excluded
- Antiviral therapy for HCV and HBV is allowed, but patient should not be on interferon.
- HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with meclizine.
- Patient must not have uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, myocardial infarction or cerebrovascular accident within 6 months prior to registration, cardiac arrhythmia, glaucoma, asthma or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant women are excluded from this study because meclizine is a Class B agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with meclizine, breastfeeding should be discontinued if the mother is treated with meclizine.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Baylor College of Medicine -McNair Campus
Houston, Texas, 77030
Baylor College of Medicine
Houston, Texas, 77030
Baylor St. Luke's Medical Center
Houston, Texas, 77030
Ben Taub General Hospital
Houston, Texas, 77030
Harris Health System- Smith Clinic
Houston, Texas, 77030
About this study
Official title: A Window of Opportunity Trial with Meclizine in Hepatocellular Carcinoma
Meclizine hydrochloride is an antihistamine widely used for treatment of vertigo and motion sickness. In HCC it has been used for anti-emetic effects, but it is used here as a CAR (constitutive androstane receptor) inverse agonist.
The hypothesis of this study is that Meclizine, CAR inverse agonist, will have beneficial therapeutic effect in patients with hepatocellular carcinoma who are candidates for surgical resection, ablation, TACE, Y90 or systemic therapy by blocking tumorigenesis and inducing apoptosis. The effects of Meclizine will be analyzed by measuring messenger RNA level of CAR target genes CYP2B6, c-Myc and FoxM1, the downstream effectors of CAR, by real time quantitative PCR.
Read the detailed description
The constitutive androstane receptor (CAR, NR1I3) is a nuclear receptor that plays a central role in hepatic detoxification of potentially toxic compounds, or xenobiotics. Chronic CAR activation by specific agonists induces tumors in wild type mice, and strongly promotes hepatocarcinogenesis in combination with initiating mutagens. Both effects are absent in CAR null mice. These tumorigenic effects are associated with an acute induction of hepatocyte proliferation in mice. Preliminary results using partially humanized mice demonstrate a very similar proliferative effect of CAR activation in human hepatocytes.
The transcriptional activity of CAR can be reversed by specific inverse agonists. These ligands are analogous to steroid receptor antagonists, converting the transcriptional activation of the agonist bound receptor into transcriptional repression. These compounds are termed inverse agonists because they do not depend on the presence of agonist ligands to exert their repressive effects. Mouse and human CAR proteins are more divergent than other nuclear receptors, and respond to quite different profiles of agonists and inverse agonists. Preliminary results demonstrate that the specific mouse CAR inverse agonist androstanol blocks proliferation and induces apoptosis in mouse liver tumors. This raises the possibility that targeting CAR may represent a new modality of treatment for hepatocellular cancer (HCC) analogous to estrogen and androgen receptor antagonists in breast and prostate cancers. Meclizine, a widely used antihistamine medication for vertigo and motion sickness, is an inverse agonist ligand of human CAR. Investigators hypothesize that reversing CAR function with meclizine will have a beneficial therapeutic effect in patients with HCC by blocking proliferation and inducing apoptosis.
Investigators therefore propose a novel window of opportunity trial in which biopsy proven HCC patients will receive oral meclizine daily for 28 (up to 35) days while awaiting surgical resection, radiofrequency ablation, transarterial chemoembolization, Y90 or systemic therapy. The primary test of treatment outcome will be the predicted decrease in expression of downstream CAR target genes (CYP2B6, MYC and FOXM1) in pre and post treatment tumor specimens. Investigators will also measure the change in tumor proliferation and apoptosis by measuring Ki-67 proliferation index and TUNEL assays (terminal deoxynucleotidyl transferase dUTP Nick-End Labeling assay), serum levels of AFP and GDF 15, and overall tumor response by imaging. HCC is the most rapidly increasing cause of cancer mortality in the United States and medical treatment options are limited. Successful completion of this study may identify a new approach to treatment of HCC.
- Conditions
- Carcinoma, Hepatocellular
- Being tested
- Meclizine Oral Tablet
- Keywords
- Hepatocellular carcinoma, HCC, Liver cancer
- 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.
Meclizine Oral TabletDrug
All subjects will receive 50 mg of meclizine taken orally, twice a day (daily dose 100 mg) for 28 (up to 35) days.
| Study group | Role | What they receive |
|---|---|---|
| Meclizine 100 mg | Experimental | Meclizine 50 mg will be taken by the patient orally twice daily for a total of 28 days(up to 35 days). Receives: Drug: Meclizine Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in mRNA levels
Quantitative real time PCR(qPCR) can give change in expression level compared to control Delta CT value. Downstream target genes of CAR (CYP2b6,c-Myc, and FoxM) will be measured by qPCR in the pre and post treatment Hepatocellular cancer tissue specimens. The qPCR machine measures the intensity of fluorescence emitted by the probe at each cycle. The Ct measure is a determined PCR cycle and represents the basic result of a qPCR experience. The Ct is the value where the PCR curve crosses the threshold.
Measured: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Secondary outcomes (6)
Change in Ki-67 proliferation index
The proliferative index is a measure of the number of cells in a tumor that are dividing (proliferating).
Measured: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
change in apoptosis by TUNEL assay
apoptosis will be measured by TUNEL (terminal deoxynucleotidyl transferase dUTP Nick-End Labeling assay)
Measured: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Tumor response
Assess tumor response by RECIST criteria
Measured: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Change in Serum AFP
This will be measured in peripheral blood samples
Measured: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Change in growth differentiation factor (GDF-15)
This will be measured in peripheral blood samples
Measured: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
A panel of CAR downstream target genes
expression of a panel of CAR downstream target genes
Measured: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2017
- Study start13 Oct 2017
- Primary completion28 Mar 2023
- Study completion28 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Carcinoma, HepatocellularStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tannaz Armaghnay
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Tannaz Armaghnay, Baylor College of Medicine
- Investigators
- Tannaz Armaghany, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03253289
- Sponsor's protocol ID
- H-40370
- Acronym
- OPTIM
- Status last verified
- Sep 2024
- First submitted
- 19 May 2017
- Last updated
- 2 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: IPD will not be shared, as indicated above.
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Window of Opportunity Trial with Meclizine in Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT03253289
- Acronym / sponsor ID
- OPTIM / H-40370
- Conditions
- Carcinoma, Hepatocellular
- Keywords
- Hepatocellular carcinoma, HCC, Liver cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Tannaz ArmaghnayUniversity, hospital or other
- Study start
- 13 Oct 2017 (actual)
- Primary completion
- 28 Mar 2023 (actual)
- Study completion
- 28 Mar 2023 (actual)
- First posted
- 17 Aug 2017 (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.
Meclizine hydrochloride is an antihistamine widely used for treatment of vertigo and motion sickness. In HCC it has been used for anti-emetic effects, but it is used here as a CAR (constitutive androstane receptor) inverse agonist.
The hypothesis of this study is that Meclizine, CAR inverse agonist, will have beneficial therapeutic effect in patients with hepatocellular carcinoma who are candidates for surgical resection, ablation, TACE, Y90 or systemic therapy by blocking tumorigenesis and inducing apoptosis. The effects of Meclizine will be analyzed by measuring messenger RNA level of CAR target genes CYP2B6, c-Myc and FoxM1, the downstream effectors of CAR, by real time quantitative PCR.
Detailed description
The constitutive androstane receptor (CAR, NR1I3) is a nuclear receptor that plays a central role in hepatic detoxification of potentially toxic compounds, or xenobiotics. Chronic CAR activation by specific agonists induces tumors in wild type mice, and strongly promotes hepatocarcinogenesis in combination with initiating mutagens. Both effects are absent in CAR null mice. These tumorigenic effects are associated with an acute induction of hepatocyte proliferation in mice. Preliminary results using partially humanized mice demonstrate a very similar proliferative effect of CAR activation in human hepatocytes.
The transcriptional activity of CAR can be reversed by specific inverse agonists. These ligands are analogous to steroid receptor antagonists, converting the transcriptional activation of the agonist bound receptor into transcriptional repression. These compounds are termed inverse agonists because they do not depend on the presence of agonist ligands to exert their repressive effects. Mouse and human CAR proteins are more divergent than other nuclear receptors, and respond to quite different profiles of agonists and inverse agonists. Preliminary results demonstrate that the specific mouse CAR inverse agonist androstanol blocks proliferation and induces apoptosis in mouse liver tumors. This raises the possibility that targeting CAR may represent a new modality of treatment for hepatocellular cancer (HCC) analogous to estrogen and androgen receptor antagonists in breast and prostate cancers. Meclizine, a widely used antihistamine medication for vertigo and motion sickness, is an inverse agonist ligand of human CAR. Investigators hypothesize that reversing CAR function with meclizine will have a beneficial therapeutic effect in patients with HCC by blocking proliferation and inducing apoptosis.
Investigators therefore propose a novel window of opportunity trial in which biopsy proven HCC patients will receive oral meclizine daily for 28 (up to 35) days while awaiting surgical resection, radiofrequency ablation, transarterial chemoembolization, Y90 or systemic therapy. The primary test of treatment outcome will be the predicted decrease in expression of downstream CAR target genes (CYP2B6, MYC and FOXM1) in pre and post treatment tumor specimens. Investigators will also measure the change in tumor proliferation and apoptosis by measuring Ki-67 proliferation index and TUNEL assays (terminal deoxynucleotidyl transferase dUTP Nick-End Labeling assay), serum levels of AFP and GDF 15, and overall tumor response by imaging. HCC is the most rapidly increasing cause of cancer mortality in the United States and medical treatment options are limited. Successful completion of this study may identify a new approach to treatment of HCC.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Meclizine 100 mg | Experimental | Drug: Meclizine Oral TabletMeclizine 50 mg will be taken by the patient orally twice daily for a total of 28 days(up to 35 days). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients must have imaging: CT or MRI abdomen with and without contrast confirmed or highly suspicious for Hepatocellular carcinoma. Patients must have a liver biopsy confirmed for Hepatocellular carcinoma.
- Patients must have measurable disease, defined as tumor mass which is >10 mm with spiral CT scan or MRI. Baseline imaging scan must be within 8 weeks of registration.
- Patients must have no prior history of treatment for HCC (treatment naïve) on the lesion that is being targeted for biopsy. New HCC lesions can arise in the liver of patients despite local therapy of other areas of the liver due to consistent underlying risk factors such as cirrhosis, and chronic hepatitis B or C infection. Patients with prior local liver directed therapy such as TACE, Ablation, Y-90 or hepatectomy surgery for HCC are eligible if they have developed a new untreated lesion in the liver which can be targeted for a biopsy for this study. There is no required time frame or washout period from when a lesion has been locally treated to the time when a new lesion is found and targeted for biopsy for this trial. Patients who have had prior systemic therapy of any kind are not eligible.
- Patients must be greater than 18 years of age.
- ECOG Performance status less than/equal to 2 (Karnofsky greater than 60%).
Key exclusion criteria (11 listed)
- Patients may not be receiving any other concurrent anti-cancer therapy.
- Patients may not be receiving any other concurrent investigational agents.
- Patients taking medications with a narrow therapeutic index including warfarin, digoxin, phenobarbital, carbamazepine, and cyclosporine are not excluded but should be monitored carefully.
- Patients must not be taking Rifampin or St John's Wort.
- Patient must not have a history of allergic reactions like anaphylaxis attributed to compounds of similar chemical or biologic composition to Meclizine such as antihistamine drugs.
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 |
|---|---|---|
| Meclizine Oral TabletAll subjects will receive 50 mg of meclizine taken orally, twice a day (daily dose 100 mg) for 28 (up to 35) days. | Drug | Meclizine 100 mg |
Primary outcome
Change in mRNA levels
Time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Quantitative real time PCR(qPCR) can give change in expression level compared to control Delta CT value. Downstream target genes of CAR (CYP2b6,c-Myc, and FoxM) will be measured by qPCR in the pre and post treatment Hepatocellular cancer tissue specimens. The qPCR machine measures the intensity of fluorescence emitted by the probe at each cycle. The Ct measure is a determined PCR cycle and represents the basic result of a qPCR experience. The Ct is the value where the PCR curve crosses the threshold.
6 secondary outcomes
Change in Ki-67 proliferation index
Time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
change in apoptosis by TUNEL assay
Time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Tumor response
Time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Change in Serum AFP
Time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Change in growth differentiation factor (GDF-15)
Time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
A panel of CAR downstream target genes
Time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2024.
- Individual participant dataSharing planned: No. IPD will not be shared, as indicated above.
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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.
Tannaz Armaghany, MD principal investigator
Baylor College of Medicine
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Meclizine Oral Tablet in people with Carcinoma, Hepatocellular, run by Tannaz Armaghnay at 5 sites in United States.
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 10 inclusion and 11 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: finished, as of the sponsor's last update on 2 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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 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.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change in mRNA levels (time frame: day1 and last day of treatment(last day would be one time point between day 28 and day 35 of treatment)). 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 2 Oct 2024.