Recurrent Hepatocellular CarcinomaPhase 2NCT04805788
Stereotactic Body Proton Radiotherapy for the Treatment of Liver Cancer
A Phase 2 study testing Proton Stereotactic Body Radiation Therapy and Quality-of-Life Assessment in people with Recurrent Hepatocellular Carcinoma and Stage I Hepatocellular Carcinoma AJCC v8, run by Mayo Clinic at 2 sites in United States.
Trial summary
This phase II trial studies the effect of stereotactic body proton radiotherapy in treating patients with liver cancer. Proton radiotherapy is a type of treatment that uses high-energy beams to treat tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Recurrent Hepatocellular Carcinoma, Stage I Hepatocellular Carcinoma AJCC v8, Stage II Hepatocellular Carcinoma AJCC v8
- What is being tested?
- Proton Stereotactic Body Radiation Therapy and Quality-of-Life Assessment
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesUnited States
- Study dates
- 24 Aug 2021to 28 Jan 2025
- Phase
- Phase 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
- Age >= 18 years
- Radiologically presumed or histologically proven, newly diagnosed or recurrent, hepatocellular carcinoma
- Clinical stage T0-T4N0M0 (American Joint Committee on Cancer [AJCC] 8th edition).
- One to three discrete Liver Reporting and Data System-5 (LIRADS-5) lesions that can be encompassed within a single radiation treatment plan
- Patients treated with external beam radiation as a bridge to transplant are allowed
- Minimum single lesion size >= 1 cm, maximum cumulative diameter =< 15 cm
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- Vascular involvement (including portal vein, inferior vena cava [IVC] and/or hepatic vein) is allowed
- Target lesion must be amenable to a SBRT regimen utilizing proton beam therapy (i.e. SBPT)
- Prior local liver treatment including surgery, percutaneous ablation, transarterial bland or chemoembolization (TACE), or Y-90 radioembolization is allowed if completed at least 6 weeks prior to treatment start date
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 to 2
- Life expectance >= 3 months based on medical comorbidities, tumor extent and other clinical factors as determined by treating physician
- Child Pugh score of A or B7
- Absolute neutrophil count (ANC) >= 1000 cells/mm^3 (within 45 days of study entry)
- Platelets (Plt) >= 30,000 cells/mm^3 (the use of transfusion or other intervention to achieve the minimum platelet level is allowed) (within 45 days of study entry)
- Hemoglobin (Hgb) >= 8.0 g/dL (the use of transfusion or other intervention to achieve the minimum hemoglobin level is allowed) (within 45 days of study entry)
- Total bilirubin < 2 mg/dL (within 45 days of study entry)
- Able to and provides Institutional Review Board (IRB) approved study specific written informed consent
- Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)
- Enrollment on a second independent protocol is allowed if the second protocol intervention will not significantly affect results of the current protocol as determined by the study investigators
Who cannot join
- Medical contraindication to receipt of radiotherapy
- Severe active co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
- Uncontrolled concurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements or providing informed consent
- Active systemic lupus or scleroderma
- Women of childbearing potential who are sexually active and not willing/able to use medically acceptable forms of contraception
- Prior receipt of external beam radiation to the current active disease site or if additional radiotherapy to the current site would be unsafe as determined by the treating radiation oncologist
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- More than 3 LIRADS-5 lesions or disease extent such that organ at risk constraints cannot be met
- Tumor extension into common or main branch biliary duct or adjacent organs including stomach, small or large bowel
- Extrahepatic metastases or lymph node involvement
- History of other malignancies (except for in situ cancers treated only by local excision and basal cell and squamous cell carcinomas of the skin) within 1 year prior to study entry
- Patient is unable to undergo intravenous contrast enhanced liver imaging (either computed tomography [CT] or magnetic resonance imaging [MRI]) based on clinical imaging protocols established at the treating institution
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
Mayo Clinic in Arizona
Scottsdale, Arizona, 85259
About this study
Official title: A Phase II Single Arm Trial of Stereotactic Body Proton Radiotherapy (SBPT) for Hepatocellular Carcinoma
This phase II trial studies the effect of stereotactic body proton radiotherapy in treating patients with liver cancer. Proton radiotherapy is a type of treatment that uses high-energy beams to treat tumors.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine the 3-month rate of patients experiencing an increase in Child-Pugh (CP) score by 2 or more points of 5-fraction stereotactic body proton radiotherapy (SBPT) in the treatment of hepatocellular carcinoma (HCC).
SECONDARY OBJECTIVES:
- To determine the 3-month complication rate as defined by grade 3 or higher acute adverse event.
II. To assess late toxicity with 2 year follow-up. III. To estimate the 6-month local control, liver disease control, progression free survival and overall survival.
IV. To determine objective imaging response rates at 6 months by Modified (m) Response Evaluation Criteria in Solid Tumors (RECIST) and RECIST criteria.
- To determine the value of volumetric imaging analysis compared to mRECIST and RECIST in the setting of HCC treated with stereotactic body radiotherapy (SBRT).
CORRELATIVE AND EXPLORATORY OBJECTIVES:
- To evaluate patient-reported outcomes. II. To evaluate clinical features, treatment technique, and dose-volume parameters associated with better local control and fewer adverse events.
III. To determine the value of additional imaging analyses and techniques for assessing tumor response after treatment with SBRT IV. To blood bank patient specimens for future analysis.
OUTLINE:
Patients undergo 1 SBPT fraction over 20-30 minutes per day for a total of 5 fractions.
After completion of study treatment, patients are followed up at 2 weeks, 3 , 6, 12 and 24 months, then annually for 3 years.
- Conditions
- Recurrent Hepatocellular Carcinoma, Stage I Hepatocellular Carcinoma AJCC v8, Stage II Hepatocellular Carcinoma AJCC v8, Stage III Hepatocellular Carcinoma AJCC v8
- Being tested
- Proton Stereotactic Body Radiation Therapy, Quality-of-Life Assessment
- Medical subject headings
- Carcinoma, Hepatocellular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Proton Stereotactic Body Radiation TherapyRadiation
Also called Proton SBRT
Undergo SBPT
Quality-of-Life AssessmentOther
Also called Quality of Life Assessment
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (SBPT) | Experimental | Patients undergo 1 SBPT fraction over 20-30 minutes per day for a total of 5 fractions. Receives: Radiation: Proton Stereotactic Body Radiation Therapy; Other: Quality-of-Life Assessment |
What is being measured
The primary outcome is the main question the study is designed to answer.
3-month rate of patients experiencing an increase in Child-Pugh score by 2 or more points of 5-fraction stereotactic body proton radiotherapy in the treatment of hepatocellular carcinoma (HCC)
The rate will be estimated and a 95% exact binomial confidence interval reported, and then tested utilizing a one-sample test for proportions versus the historical control of 15%.
Measured: 3 months
Secondary outcomes (8)
3-month complication rate
Defined by grade 3 or higher acute adverse event. Will be calculated and presented with 95% confidence intervals (CI), while continuous outcomes will be presented with mean, median, 95% CI, and interquartile range (IQR).
Measured: 3 months
Incidence of late toxicity
Defined by grade 3 or higher acute adverse event.
Measured: Up to 2 years
6-month local control
Will be reported via Kaplan-Meier estimates.
Measured: 6 months
Liver disease control
Will be reported via Kaplan-Meier estimates.
Measured: Up to 2 years
Progression free survival
Will be reported via Kaplan-Meier estimates.
Measured: Up to 2 years
Overall survival
Will be reported via Kaplan-Meier estimates.
Measured: Up to 2 years
Imaging response rates
Will be determined by Modified (m) Response Evaluation Criteria in Solid Tumors (RECIST) and RECIST criteria. Will be calculated and presented with 95% CI, while continuous outcomes will be presented with mean, median, 95% CI, and IQR.
Measured: 6 months
Value of volumetric imaging analysis
Will be compared to mRECIST and RECIST in the setting of HCC treated with stereotactic body radiotherapy (SBRT).
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2021
- Study start24 Aug 2021
- Primary completion15 Feb 2024
- Study completion28 Jan 2025
Why it stopped: slow accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent Hepatocellular CarcinomaStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jonathan B. Ashman, M.D., Ph.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04805788
- Sponsor's protocol ID
- MC200804
- Other IDs
- NCI-2021-01357, MC200804, 20-005638
- Status last verified
- Dec 2025
- First submitted
- 24 Feb 2021
- Last updated
- 31 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Single Arm Trial of Stereotactic Body Proton Radiotherapy (SBPT) for Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT04805788
- Acronym / sponsor ID
- MC200804
- Conditions
- Recurrent Hepatocellular Carcinoma, Stage I Hepatocellular Carcinoma AJCC v8, Stage II Hepatocellular Carcinoma AJCC v8, Stage III Hepatocellular Carcinoma AJCC v8
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: slow accrual
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 24 Aug 2021 (actual)
- Primary completion
- 15 Feb 2024 (actual)
- Study completion
- 28 Jan 2025 (actual)
- First posted
- 18 Mar 2021 (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 phase II trial studies the effect of stereotactic body proton radiotherapy in treating patients with liver cancer. Proton radiotherapy is a type of treatment that uses high-energy beams to treat tumors.
Detailed description
PRIMARY OBJECTIVE:
- To determine the 3-month rate of patients experiencing an increase in Child-Pugh (CP) score by 2 or more points of 5-fraction stereotactic body proton radiotherapy (SBPT) in the treatment of hepatocellular carcinoma (HCC).
SECONDARY OBJECTIVES:
- To determine the 3-month complication rate as defined by grade 3 or higher acute adverse event.
II. To assess late toxicity with 2 year follow-up. III. To estimate the 6-month local control, liver disease control, progression free survival and overall survival.
IV. To determine objective imaging response rates at 6 months by Modified (m) Response Evaluation Criteria in Solid Tumors (RECIST) and RECIST criteria.
- To determine the value of volumetric imaging analysis compared to mRECIST and RECIST in the setting of HCC treated with stereotactic body radiotherapy (SBRT).
CORRELATIVE AND EXPLORATORY OBJECTIVES:
- To evaluate patient-reported outcomes. II. To evaluate clinical features, treatment technique, and dose-volume parameters associated with better local control and fewer adverse events.
III. To determine the value of additional imaging analyses and techniques for assessing tumor response after treatment with SBRT IV. To blood bank patient specimens for future analysis.
OUTLINE:
Patients undergo 1 SBPT fraction over 20-30 minutes per day for a total of 5 fractions.
After completion of study treatment, patients are followed up at 2 weeks, 3 , 6, 12 and 24 months, then annually for 3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (SBPT) | Experimental | Radiation: Proton Stereotactic Body Radiation Therapy, Other: Quality-of-Life AssessmentPatients undergo 1 SBPT fraction over 20-30 minutes per day for a total of 5 fractions. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Age >= 18 years
- Radiologically presumed or histologically proven, newly diagnosed or recurrent, hepatocellular carcinoma
- Clinical stage T0-T4N0M0 (American Joint Committee on Cancer [AJCC] 8th edition).
- Target lesion must be amenable to a SBRT regimen utilizing proton beam therapy (i.e. SBPT)
- Prior local liver treatment including surgery, percutaneous ablation, transarterial bland or chemoembolization (TACE), or Y-90 radioembolization is allowed if completed at least 6 weeks prior to treatment start date
Key exclusion criteria (11 listed)
- Medical contraindication to receipt of radiotherapy
- Severe active co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
- Uncontrolled concurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements or providing informed consent
- Active systemic lupus or scleroderma
- Women of childbearing potential who are sexually active and not willing/able to use medically acceptable forms of contraception
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 |
|---|---|---|
| Proton Stereotactic Body Radiation TherapyAlso: Proton SBRTUndergo SBPT | Radiation | Treatment (SBPT) |
| Quality-of-Life AssessmentAlso: Quality of Life AssessmentAncillary studies | Other | Treatment (SBPT) |
Primary outcome
3-month rate of patients experiencing an increase in Child-Pugh score by 2 or more points of 5-fraction stereotactic body proton radiotherapy in the treatment of hepatocellular carcinoma (HCC)
Time frame: 3 months
The rate will be estimated and a 95% exact binomial confidence interval reported, and then tested utilizing a one-sample test for proportions versus the historical control of 15%.
8 secondary outcomes
3-month complication rate
Time frame: 3 months
Incidence of late toxicity
Time frame: Up to 2 years
6-month local control
Time frame: 6 months
Liver disease control
Time frame: Up to 2 years
Progression free survival
Time frame: Up to 2 years
Overall survival
Time frame: Up to 2 years
Imaging response rates
Time frame: 6 months
Value of volumetric imaging analysis
Time frame: 6 months
Results and safety
The study was stopped early (reason given: slow accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2025.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
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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.
Jonathan B. Ashman, M.D., Ph.D. principal investigator
Mayo Clinic
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Proton Stereotactic Body Radiation Therapy and Quality-of-Life Assessment in people with Recurrent Hepatocellular Carcinoma and Stage I Hepatocellular Carcinoma AJCC v8, run by Mayo Clinic at 2 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 19 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: stopped early, as of the sponsor's last update on 31 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 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: slow accrual). 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?
3-month rate of patients experiencing an increase in Child-Pugh score by 2 or more points of 5-fraction stereotactic body proton radiotherapy in the treatment of hepatocellular carcinoma (HCC) (time frame: 3 months). 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 31 Dec 2025.