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A Protocol for the Safety and Tolerance of Intravenous 4-Demethylcholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) in Patients With Advanced Cancer

Phase 1
18 Years
Open (Enrolling)
Advanced Cancer With/Without Brain Involvement.

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Trial Information

A Protocol for the Safety and Tolerance of Intravenous 4-Demethylcholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) in Patients With Advanced Cancer

4-Demethylcholesterylpenclomedine(DM-CHOC-PEN)is a polychlorinated pyridine cholesteryl
carbonate that has demonstrated complete remissions when administrated to mice bearing
intracranially implanted human cancer xenografts (glioblastomas and breast cancers) and has
acceptable preclinical toxicity profiles - mice, rats and dogs.

The drug is being administered intravenously once every 21 days as an infusion. The starting
dose was 39 mg/M2 (based on 1/10 LD10 observed in mice and rats).

A modified accelerated dosing protocol of Simon, Freidlin, et al is in process to escalate
dosage which will reduce the number of patients required and minimize ineffective doses. The
protocol will result in one patient cohorts at 40% dosage escalations. When the 1st instance
of a dose limiting toxicity (DLT) [grade - 3 or 4 toxicity] is observed, or the 2nd instance
of a 1st course grade 2 toxicity of any type is observed, the cohort for that current
dose level will be expanded to 3-6 patients and escalation will proceed at 33% increments.

During the entirety of the study, intra-patient escalation will be allowed. This will occur
if the patient experiences are < grade 2 toxicity at the existing dose, if no > grade 2
toxicity was identified at the existing dose and no DLTs were noted at the higher dose level
(if any patients had been escalated to that dose).

Continuation of therapy - Patients who experience stabilization, partial or complete
remission while being treated will continue to receive the drug as per the last dose prior
to identifying positive results at an every 3 weeks interval.

Discontinuation of therapy - progressive disease and/or toxicities.

Inclusion Criteria:

- All patients must have histological evidence of a solid malignant tumor
(hematological malignancies are excluded) with convincing clinical, radiographic or
isotopic evidence of cancer, for which no effective proven treatment exists. CNS
associated tumors are preferred, but not required. Patients must sign an informed
consent that complies with the investigator/DEKK-TEC policies and approved by a Human
Investigation Review Committee.

- All patients must have a projected survival time of at least 12 weeks and a Karnofsky
performance score: >60% (or Zubrod score of >2).

- All patients must be off previous chemo- and/or radiotherapy for at least three (3)
weeks prior to entrance into the study and have recovered from any toxic effects
induced by such treatment(s). Patients who have received a nitrosourea type drug
must have had no treatment within the last six weeks.

- Measurable lesions are not required for admittance to the study - but are desirable.

- Age initiated after limitation - 18 years or older. A separate pediatric study is
proposed to evaluate tolerance to the drug in children.

- Gender is not a criterion.

Exclusion Criteria:

- Hematology WBC <4,000 mm3 Platelets <100,000 mm3

- Liver Function If bilirubin, AST, and/or ALT are >ULN

- Renal Function Creatinine >1.5 mg%

- Cardiovascular Acute myocardial infarction Congestive heart failure - (NYHA criteria
for uncontrolled) Clinically significant cardiac arrhythmias - uncontrolled

- Concomitant chemotherapy or radiotherapy is not permitted.

- Pregnant or lactating females are excluded. Women of childbearing age, and their
sexual partners, must use an effective contraception program. Males who are having
sexual relations with women capable of child bearing must use birth control while on
the study and for 3-months after the last dose of the study drug.

- Allergies to eggs, lecithin or soy products.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Document maximum tolerated dose (MTD) & SLT

Outcome Time Frame:

one year

Safety Issue:


Principal Investigator

Marcus L Ware, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Tulane University Medical School


United States: Food and Drug Administration

Study ID:




Start Date:

September 2010

Completion Date:

July 2013

Related Keywords:

  • Advanced Cancer With/Without Brain Involvement.
  • Glioblastoma multiforme
  • Advanced cancers
  • Brain Neoplasms, Malignant, Primary
  • Brain Tumor, Recurrent
  • Advanced cancer
  • Neoplasms



Tulane University Medical SchoolNew Orleans, Louisiana  70112