Metastatic Hepatocellular CarcinomaPhase 1/2NCT01754987
A Study of Vitamin C in the Treatment of Liver Cancer to Determine if it is Safe and Effective
A Phase 1/2 study testing Ascorbic Acid + Sorafenib and Sorafenib in people with Metastatic Hepatocellular Carcinoma and Advanced Liver Cancer, run by Thomas Jefferson University at 1 site in United States.
Trial summary
This protocol is a phase I/II, study of ascorbic acid (AA) infusions combined with treatment with sorafenib versus treatment with sorafenib alone in subjects with metastatic hepatocellular carcinoma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Metastatic Hepatocellular Carcinoma, Advanced Liver Cancer
- What is being tested?
- Ascorbic Acid + Sorafenib and Sorafenib
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2012to Jan 2016
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Metastatic hepatocellular carcinoma
- G6PD (glucose-6-phosphate dehydrogenase) status > lower limit of normal
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
- Laboratory at baseline evaluation for inclusion in the study: creatinine ≤1.5X upper limit (if the creatinine is elevated, but ≤1.5X the ULN, a 24 hour creatinine clearance will be obtained); transaminase (AST/ALT) ≤2.0X upper limit of normal; bilirubin levels ≥ 2 mg/dL; ANC ≥1,500/mm3; Hemoglobin > 8g/dL; platelet ≥ 100,000/mm3
- Women of childbearing potential will confirm a negative pregnancy test and must practice effective contraception during the study.
- Willing and able to provide informed consent and participate in the study procedures.
Who cannot join
- Patients with evidence of a significant current psychiatric disorder that would prevent completion of the study as determined by the PI will not be allowed to participate.
- Co-morbid medical condition that would affect survival or tolerance as determined by the PI. This includes patients who have not fully recovered from toxicities associated with prior therapy. It also includes subjects who, as determined by the PI, are at risk of experiencing fluid overload (i.e., congestive heart failure).
- Patients who currently abuse alcohol or drugs.
- Patients with known glomerular filtration rate of <60ml/min or with nephrotic range proteinuria.
- Pregnant or lactating women
- Enrollment in active clinical trial/ experimental therapy or IND study within the prior 30 days.
- Contraindication for CT or PET/CT as per the PI.
Trial locations and contacts
1 site in 1 country.
United States1 site
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
About this study
Official title: Phase I/II Study of Intravenous Ascorbic Acid in Treatment of Metastatic Hepatocellular Carcinoma
This protocol is a phase I/II, study of ascorbic acid (AA) infusions combined with treatment with sorafenib versus treatment with sorafenib alone in subjects with metastatic hepatocellular carcinoma. The phase I aspect will assess the safety and efficacy of the concurrent treatments and the phase II aspect will utilize CT (computer-tomography) scans to assess overall tumor response rate and evaluate disease progression
Read the detailed description
Intravenous Ascorbic Acid (Vitamin C) is a widely used alternative cancer treatment. This trial will study an intravenous Vitamin C treatment for persons with liver cancer that has spread, who are also receiving Sorafenib (a standard cancer drug), to see whether the combination of Vitamin C and Sorafenib is safe and well tolerated. Phase I will involve 6 persons who will receive the Vitamin C for 8 weeks to more specifically assess the safety of getting Vitamin C in combination with Sorafenib. Phase II will be randomized to receive either Vitamin C plus Sorafenib or Sorafenib alone for 16 weeks. If Vitamin C has a beneficial effect on tumour cells, patients may experience a regression of tumor or tumor markers. Additional benefits include scans at no charge to the patient.
- Being tested
- Ascorbic Acid + Sorafenib, Sorafenib
- Keywords
- Vitamins, Malignant Tumor, Tumour, Integrative Medicine, Complementary Medicine, Alternative Medicine Antioxidants, Molecular Mechanisms of Pharmacological Action, Pharmacologic Actions, Protective Agents, Physiological Effects of Drugs, Micronutrients, Growth Substances, Antineoplastic Agents, Phytogenic, Antineoplastic Agents, Therapeutic Uses, Radiation-Sensitizing Agents, Liver Cancer
- Medical subject headings
- Carcinoma, Hepatocellular, Neoplasms, Liver Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ascorbic Acid + SorafenibDrug
Also called Vitamin C, Ascorbate
SorafenibDrug
Also called Nexavar
| Study group | Role | What they receive |
|---|---|---|
| Ascorbic Acid + Sorafenib | Experimental | Drug: Vitamin C Other Names: Ascorbic Acid, Ascorbate
Dosage:
Vitamin C : 100 grams (infusion) Phase I: 3x a week for 8 weeks Phase II: 3x a week for 16 weeks Sorafenib: taken daily (oral) Receives: Drug: Ascorbic Acid + Sorafenib |
| Sorafenib alone | Other | Sorafenib: taken daily (oral) Receives: Drug: Sorafenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants That Experience Serious Adverse Events.
The primary aim is to assess whether or not (IV) Ascorbic Acid (AA) with sorafenib therapy is relatively safe and well-tolerated according to Common Terminology Criteria for Adverse Events (CTCAE)v4.0
Measured: 16 weeks +/- 2 weeks
Secondary outcomes (3)
Overall Tumor Response Rate
To utilize CT or PET/CT scans to assess overall tumor response rate (complete) in subjects with advanced metastatic hepatocellular cancer treated with the combination of ascorbic acid and sorafenib versus sorafenib alone.
Measured: 16 weeks +/- 2 weeks
Mean Value Collected Using the Functional Assessment of Cancer Therapy-General (FACT-G) Quality Assessment Instrument
To evaluate quality of life using Functional Assessment of Cancer Therapy-General (FACT-G) quality assessment instrument. The FACT-G questionnaire will be used to assess quality-of-life longitudinally. Quality-of-life scores obtained from the FACT-G will be summarized at multiple time points. Five-point scale from 0 (not at all) to 4 (very much)
Measured: 16 weeks +/- 2 weeks
Number of Participants That Are Alive After 15 Weeks of Treatment.
To evaluate duration of tumor response and progression-free survival
Measured: 15 weeks+
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2012 (estimated)
- Study startSep 2012
- Primary completionJan 2016
- Study completionJan 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Metastatic Hepatocellular CarcinomaStudies of the same condition, open and under way.
- More trials run by Thomas Jefferson UniversityThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Thomas Jefferson University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Daniel A Monti, MD, MBA, Principal investigator, Thomas Jefferson University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01754987
- Sponsor's protocol ID
- 12D.424
- Other IDs
- JT 2306
- Status last verified
- May 2025
- First submitted
- 18 Dec 2012
- Last updated
- 4 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Monti DA, Mitchell E, Bazzan AJ, Littman S, Zabrecky G, Yeo CJ, Pillai MV, Newberg AB, Deshmukh S, Levine M. Phase I evaluation of intravenous ascorbic acid in combination with gemcitabine and erlotinib in patients with metastatic pancreatic cancer. PLoS One. 2012;7(1):e29794. doi: 10.1371/journal.pone.0029794. Epub 2012 Jan 17.
PubMed 22272248
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Intravenous Ascorbic Acid in Treatment of Metastatic Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT01754987
- Acronym / sponsor ID
- 12D.424
- Keywords
- Vitamins, Malignant Tumor, Tumour, Integrative Medicine, Complementary Medicine, Alternative Medicine Antioxidants, Molecular Mechanisms of Pharmacological Action, Pharmacologic Actions, Protective Agents
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Thomas Jefferson UniversityUniversity, hospital or other
- Study start
- Sep 2012
- Primary completion
- Jan 2016 (actual)
- Study completion
- Jan 2016 (actual)
- First posted
- 21 Dec 2012 (estimated)
- Results
- Posted 23 Aug 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This protocol is a phase I/II, study of ascorbic acid (AA) infusions combined with treatment with sorafenib versus treatment with sorafenib alone in subjects with metastatic hepatocellular carcinoma. The phase I aspect will assess the safety and efficacy of the concurrent treatments and the phase II aspect will utilize CT (computer-tomography) scans to assess overall tumor response rate and evaluate disease progression
Detailed description
Intravenous Ascorbic Acid (Vitamin C) is a widely used alternative cancer treatment. This trial will study an intravenous Vitamin C treatment for persons with liver cancer that has spread, who are also receiving Sorafenib (a standard cancer drug), to see whether the combination of Vitamin C and Sorafenib is safe and well tolerated. Phase I will involve 6 persons who will receive the Vitamin C for 8 weeks to more specifically assess the safety of getting Vitamin C in combination with Sorafenib. Phase II will be randomized to receive either Vitamin C plus Sorafenib or Sorafenib alone for 16 weeks. If Vitamin C has a beneficial effect on tumour cells, patients may experience a regression of tumor or tumor markers. Additional benefits include scans at no charge to the patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ascorbic Acid + Sorafenib | Experimental | Drug: Ascorbic Acid + SorafenibDrug: Vitamin C Other Names: Ascorbic Acid, Ascorbate Dosage: Vitamin C : 100 grams (infusion) Phase I: 3x a week for 8 weeks Phase II: 3x a week for 16 weeks Sorafenib: taken daily (oral) |
| Sorafenib alone | Other | Drug: SorafenibSorafenib: taken daily (oral) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age > 18 years
- Metastatic hepatocellular carcinoma
- G6PD (glucose-6-phosphate dehydrogenase) status > lower limit of normal
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
- Laboratory at baseline evaluation for inclusion in the study: creatinine ≤1.5X upper limit (if the creatinine is elevated, but ≤1.5X the ULN, a 24 hour creatinine clearance will be obtained); transaminase (AST/ALT) ≤2.0X upper limit of normal; bilirubin levels ≥ 2 mg/dL; ANC ≥1,500/mm3; Hemoglobin > 8g/dL; platelet ≥ 100,000/mm3
Key exclusion criteria (7 listed)
- Patients with evidence of a significant current psychiatric disorder that would prevent completion of the study as determined by the PI will not be allowed to participate.
- Co-morbid medical condition that would affect survival or tolerance as determined by the PI. This includes patients who have not fully recovered from toxicities associated with prior therapy. It also includes subjects who, as determined by the PI, are at risk of experiencing fluid overload (i.e., congestive heart failure).
- Patients who currently abuse alcohol or drugs.
- Patients with known glomerular filtration rate of <60ml/min or with nephrotic range proteinuria.
- Pregnant or lactating women
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 |
|---|---|---|
| Ascorbic Acid + SorafenibAlso: Vitamin C, Ascorbate | Drug | Ascorbic Acid + Sorafenib |
| SorafenibAlso: Nexavar | Drug | Sorafenib alone |
Primary outcome
Number of Participants That Experience Serious Adverse Events.
Time frame: 16 weeks +/- 2 weeks
The primary aim is to assess whether or not (IV) Ascorbic Acid (AA) with sorafenib therapy is relatively safe and well-tolerated according to Common Terminology Criteria for Adverse Events (CTCAE)v4.0
3 secondary outcomes
Overall Tumor Response Rate
Time frame: 16 weeks +/- 2 weeks
Mean Value Collected Using the Functional Assessment of Cancer Therapy-General (FACT-G) Quality Assessment Instrument
Time frame: 16 weeks +/- 2 weeks
Number of Participants That Are Alive After 15 Weeks of Treatment.
Time frame: 15 weeks+
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 22272248Monti DA, Mitchell E, Bazzan AJ, Littman S, Zabrecky G, Yeo CJ, Pillai MV, Newberg AB, Deshmukh S, Levine M. Phase I evaluation of intravenous ascorbic acid in combination with gemcitabine and erlotinib in patients with metastatic pancreatic cancer. PLoS One. 2012;7(1):e29794. doi: 10.1371/journal.pone.0029794. Epub 2012 Jan 17.
- Individual participant dataSharing planned: No
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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.
Daniel A Monti, MD, MBA principal investigator
Thomas Jefferson University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Ascorbic Acid + Sorafenib and Sorafenib in people with Metastatic Hepatocellular Carcinoma and Advanced Liver Cancer, run by Thomas Jefferson University at 1 site 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 7 inclusion and 7 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 4 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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?
Yes. Results are posted in the registry (first posted 23 Aug 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants That Experience Serious Adverse Events. (time frame: 16 weeks +/- 2 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01754987, which also lists 1 linked publication.
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 4 May 2025.