Clinical trial NCT01754987: A Study of Vitamin C in the Treatment of Liver Cancer to Determine if it is Safe and Effective
Official title: Phase I/II Study of Intravenous Ascorbic Acid in Treatment of Metastatic Hepatocellular Carcinoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Metastatic Hepatocellular Carcinoma
Advanced Liver Cancer
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
Lead sponsor: Thomas Jefferson University
Sponsor class: OTHER
First posted: 2012-12-21
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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. 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Number of Participants That Experience Serious Adverse Events.
timeFrame: 16 weeks +/- 2 weeks
Secondary outcomes:
measure: Overall Tumor Response Rate
timeFrame: 16 weeks +/- 2 weeks
measure: Mean Value Collected Using the Functional Assessment of Cancer Therapy-General (FACT-G) Quality Assessment Instrument
timeFrame: 16 weeks +/- 2 weeks
measure: Number of Participants That Are Alive After 15 Weeks of Treatment.
timeFrame: 15 weeks+
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
Study officials:
affiliation: Thomas Jefferson University
name: Daniel A Monti, MD, MBA
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.