A Phase I Trial of High-Dose Ascorbate in Glioblastoma Multiforme

NCT ID: NCT01752491

Last Updated: 2024-12-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-04-01

Study Completion Date

2019-11-15

Brief Summary

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This is a phase 1 (first in man) study testing the safety of adding high dose ascorbate (vitamin C) to standard radiation and chemotherapy for initial treatment of glioblastoma multiforme (GBM).

Detailed Description

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This phase 1 study will test the safety of adding high dose ascorbate (vitamin C) to standard chemoradiation and, after the radiation is completed, during 6 cycles of temozolomide.

Standard treatment for glioblastoma multiforme (GBM) involves surgery followed by radiation combined with temozolomide (a chemotherapy). After radiation, patients receive cycles of temozolomide (adjuvant chemotherapy)

Participants will:

* receive high doses of intravenous (IV) ascorbate three times a week during chemoradiation
* receive high doses of intravenous (IV) ascorbate twice a week during adjuvant chemotherapy (after radiation)

This is a phase 1 study will evaluate the side effects of adding this drug to the standard therapy. The dose given to a participant will be determined by how well other participants have tolerated the drug.

Conditions

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Glioblastoma GBM Glioblastoma Multiforme

Keywords

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Ascorbate Ascorbic acid Vitamin C Radiation Temozolomide

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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15g Ascorbate

During radiation therapy:

* Radiation: 61.2 Gray (1.8 Gray / fraction / day), 5 days/week, for approximately 8 weeks.
* Temozolomide: 75 mg/m2, taken orally, once daily, every day, until radiation is completed.
* Ascorbate: 15 g administered by IV three times a week until 1 month after radiation is completed (approximately 12 weeks).

After radiation therapy:

* Temozolomide: Starting 1 month after radiation. 150 mg/m2 and then 200 mg/m2 daily. Starting 28 days after the completion of radiation therapy. Taken for 5 days followed by 23 days of rest for 6 cycles.
* Ascorbate: escalating weekly doses of ascorbate (up to 125 grams) to target a serum level of 350 mg/dL (20 mM). Ascorbate is administered twice weekly, each week, for up to 6 months.

Group Type EXPERIMENTAL

Ascorbate

Intervention Type DRUG

Intravenous infusion of high-dose ascorbate

Temozolomide

Intervention Type DRUG

Oral chemotherapeutic

Radiation therapy

Intervention Type RADIATION

External beam radiation therapy

25g Ascorbate

If the 15g arm is tolerated, the study opens the 25g arm.

During radiation therapy:

* Radiation: 61.2 Gray (1.8 Gy/fraction/day), 5 days/wk, for about 8 weeks.
* Temozolomide: 75 mg/m2, taken orally, once every day, until radiation is completed.
* Ascorbate: 25 g administered by IV three times/wk until 1 month after radiation is completed (about 12 weeks).

After radiation therapy:

* Temozolomide: Starting 1 month after radiation. 150 mg/m2 and then 200 mg/m2 daily. Starting 28 days after the completion of radiation therapy. Taken for 5 days followed by 23 days of rest for 6 cycles.
* Ascorbate: escalating weekly doses of ascorbate (up to 125 grams) to target a serum level of 350 mg/dL (20 mM). Ascorbate is administered twice weekly, each week, for up to 6 months.

Group Type EXPERIMENTAL

Ascorbate

Intervention Type DRUG

Intravenous infusion of high-dose ascorbate

Temozolomide

Intervention Type DRUG

Oral chemotherapeutic

Radiation therapy

Intervention Type RADIATION

External beam radiation therapy

50g arm

If the 25g arm is tolerated, the study opens the 50g arm.

During radiation therapy:

* Radiation: 61.2 Gray (1.8 Gy/fraction/day), 5 days/wk, for about 8 weeks.
* Temozolomide: 75 mg/m2, taken orally, once every day, until radiation is completed.
* Ascorbate: 50 g administered by IV three times a week until 1 month after radiation is completed (about 12 weeks).

After radiation therapy:

* Temozolomide: Starting 1 month after radiation. 150 mg/m2 and then 200 mg/m2 daily. Starting 28 days after the completion of radiation therapy. Taken for 5 days followed by 23 days of rest for 6 cycles.
* Ascorbate: escalating weekly doses of ascorbate (up to 125 grams) to target a serum level of 350 mg/dL (20 mM). Ascorbate is administered twice weekly, each week, for up to 6 months.

Group Type EXPERIMENTAL

Ascorbate

Intervention Type DRUG

Intravenous infusion of high-dose ascorbate

Temozolomide

Intervention Type DRUG

Oral chemotherapeutic

Radiation therapy

Intervention Type RADIATION

External beam radiation therapy

62.5g

If the 50g arm is tolerated, the study opens the 62.5g arm.

During radiation therapy:

* Radiation: 61.2 Gray (1.8 Gy/fraction/day), 5 days/wk, for about 8 weeks.
* Temozolomide: 75 mg/m2, taken orally, once every day, until radiation is completed.
* Ascorbate: 62.5 g administered by IV three times a week until 1 month after radiation is completed (about 12 weeks).

After radiation therapy:

* Temozolomide: Starting 1 month after radiation. 150 mg/m2 and then 200 mg/m2 daily. Starting 28 days after the completion of radiation therapy. Taken for 5 days followed by 23 days of rest for 6 cycles.
* Ascorbate: escalating weekly doses of ascorbate (up to 125 grams) to target a serum level of 350 mg/dL (20 mM). Ascorbate is administered twice weekly, each week, for up to 6 months.

Group Type EXPERIMENTAL

Ascorbate

Intervention Type DRUG

Intravenous infusion of high-dose ascorbate

Temozolomide

Intervention Type DRUG

Oral chemotherapeutic

Radiation therapy

Intervention Type RADIATION

External beam radiation therapy

75g Ascorbate

If the 62.5g arm is tolerated, the study opens the 75g arm.

During radiation therapy:

* Radiation: 61.2 Gray (1.8 Gy/fraction/day), 5 days/wk, for about 8 weeks.
* Temozolomide: 75 mg/m2, taken orally, once every day, until radiation is completed.
* Ascorbate: 75 g administered by IV three times a week until 1 month after radiation is completed (about 12 weeks).

After radiation therapy:

* Temozolomide: Starting 1 month after radiation. 150 mg/m2 and then 200 mg/m2 daily. Starting 28 days after the completion of radiation therapy. Taken for 5 days followed by 23 days of rest for 6 cycles.
* Ascorbate: escalating weekly doses of ascorbate (up to 125 grams) to target a serum level of 350 mg/dL (20 mM). Ascorbate is administered twice weekly, each week, for up to 6 months.

Group Type EXPERIMENTAL

Ascorbate

Intervention Type DRUG

Intravenous infusion of high-dose ascorbate

Temozolomide

Intervention Type DRUG

Oral chemotherapeutic

Radiation therapy

Intervention Type RADIATION

External beam radiation therapy

87.5g Ascorbate

If the 75g arm is tolerated, the study opens the 87.5g arm.

During radiation therapy:

* Radiation: 61.2 Gray (1.8 Gy/fraction/day), 5 days/wk, for about 8 weeks.
* Temozolomide: 75 mg/m2, taken orally, once every day, until radiation is completed.
* Ascorbate: 87.5 g administered by IV three times a week until 1 month after radiation is completed (about 12 weeks).

After radiation therapy:

* Temozolomide: Starting 1 month after radiation. 150 mg/m2 and then 200 mg/m2 daily. Starting 28 days after the completion of radiation therapy. Taken for 5 days followed by 23 days of rest for 6 cycles.
* Ascorbate: escalating weekly doses of ascorbate (up to 125 grams) to target a serum level of 350 mg/dL (20 mM). Ascorbate is administered twice weekly, each week, for up to 6 months.

Group Type EXPERIMENTAL

Ascorbate

Intervention Type DRUG

Intravenous infusion of high-dose ascorbate

Temozolomide

Intervention Type DRUG

Oral chemotherapeutic

Radiation therapy

Intervention Type RADIATION

External beam radiation therapy

Interventions

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Ascorbate

Intravenous infusion of high-dose ascorbate

Intervention Type DRUG

Temozolomide

Oral chemotherapeutic

Intervention Type DRUG

Radiation therapy

External beam radiation therapy

Intervention Type RADIATION

Other Intervention Names

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Ascorbic Acid Vitamin C Temodar External beam radiation therapy

Eligibility Criteria

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Inclusion Criteria

* Patients must have newly diagnosed (i.e., within 5 weeks), histologically or cytologically confirmed glioblastoma multiforme.
* Diagnosis must be made by surgical biopsy or excision.
* Therapy must begin ≤ 5 weeks after surgery.
* Age ≥ 18 years
* ECOG performance status 0-2 (Karnofsky \> 50%).
* A complete blood count and differential must be obtained within 21 days prior to the first dose of radiation, with adequate bone marrow functions as defined below:

* Absolute neutrophil count (ANC) ≥ 1500 cells per mm3
* Platelets ≥ 100,000 per mm3
* Hemoglobin ≥ 8 g/dL
* Serum blood chemistries within 21 days before the first day of radiation, as defined below:

* Creatinine ≤ 2.0 mg
* Total bilirubin ≤ 1.5 mg/dL
* ALT (Alanine Aminotransferase)≤ 3 times the institutional upper limit of normal
* AST (Aspartate Aminotransferase) ≤ 3 times the institutional upper limit of normal
* Tolerate one text dose (15g) of ascorbate
* Not pregnant
* Ability to understand and willingness to sign a written informed consent document

Exclusion Criteria

* Recurrent high grade glioma
* G6PD (glucose-6-phosphate dehydrogenase) deficiency
* Patients actively receiving insulin unless approved by the study medical monitor, study sponsor, and the study principal investigator.
* History of allergic reactions attributed to compounds of similar chemical or biologic composition to temozolomide.
* Significant co-morbid central nervous system disease, including but not limited to, multiple sclerosis.
* Patients who are on the following drugs and cannot have a drug substitution: flecainide, methadone, amphetamines, quinidine, and chlorpropamide. High dose ascorbic acid may affect urine acidification and, as a result, may affect clearance rates of these drugs.
* Prior invasive malignancies (except non-melanomatous skin cancers and carcinoma in situ of the cervix or bladder) unless disease free for ≥ 5 years.
* Patients who have received prior chemotherapy (including Gliadel wafers) for the current glioma.
* Prior radiation therapy to the head or neck, which would result in overlap of radiation therapy fields.
* Patients may not be receiving any other investigational agents.
* Uncontrolled intercurrent 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.
* Pregnant women are excluded from this study because ionizing radiation is a known teratogen, and temozolomide is a Class D agent with the potential for teratogenic or abortifacient effects.
* Known HIV-positive individuals. High-dose ascorbate acid is a known CYP450 3A4 (an enzyme pathway) inducer, which results in lower serum levels of antiretroviral drugs
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Joseph J. Cullen, MD, FACS

OTHER

Sponsor Role lead

Responsible Party

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John M. Buatti

Professor and Chair, Department of Radiation Oncology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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John M. Buatti, MD

Role: PRINCIPAL_INVESTIGATOR

Department of Radiation Oncology, The University of Iowa

Joseph J Cullen, MD

Role: STUDY_DIRECTOR

Professor of Surgery, The University of Iowa

Locations

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Holden Comprehensive Cancer Center at the University of Iowa

Iowa City, Iowa, United States

Site Status

Countries

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United States

References

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Du J, Cullen JJ, Buettner GR. Ascorbic acid: chemistry, biology and the treatment of cancer. Biochim Biophys Acta. 2012 Dec;1826(2):443-57. doi: 10.1016/j.bbcan.2012.06.003. Epub 2012 Jun 20.

Reference Type BACKGROUND
PMID: 22728050 (View on PubMed)

Du J, Martin SM, Levine M, Wagner BA, Buettner GR, Wang SH, Taghiyev AF, Du C, Knudson CM, Cullen JJ. Mechanisms of ascorbate-induced cytotoxicity in pancreatic cancer. Clin Cancer Res. 2010 Jan 15;16(2):509-20. doi: 10.1158/1078-0432.CCR-09-1713. Epub 2010 Jan 12.

Reference Type BACKGROUND
PMID: 20068072 (View on PubMed)

Schoenfeld JD, Sibenaller ZA, Mapuskar KA, Wagner BA, Cramer-Morales KL, Furqan M, Sandhu S, Carlisle TL, Smith MC, Abu Hejleh T, Berg DJ, Zhang J, Keech J, Parekh KR, Bhatia S, Monga V, Bodeker KL, Ahmann L, Vollstedt S, Brown H, Shanahan Kauffman EP, Schall ME, Hohl RJ, Clamon GH, Greenlee JD, Howard MA, Schultz MK, Smith BJ, Riley DP, Domann FE, Cullen JJ, Buettner GR, Buatti JM, Spitz DR, Allen BG. O2⋅- and H2O2-Mediated Disruption of Fe Metabolism Causes the Differential Susceptibility of NSCLC and GBM Cancer Cells to Pharmacological Ascorbate. Cancer Cell. 2017 Apr 10;31(4):487-500.e8. doi: 10.1016/j.ccell.2017.02.018. Epub 2017 Mar 30.

Reference Type RESULT
PMID: 28366679 (View on PubMed)

Other Identifiers

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P30CA086862

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01CA140206

Identifier Type: NIH

Identifier Source: secondary_id

View Link

201211713

Identifier Type: -

Identifier Source: org_study_id