Study of 4-Demethylcholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) in Patients With Advanced Cancer (DM-CHOC-PEN)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01048008
Recruitment Status : Completed
First Posted : January 13, 2010
Last Update Posted : January 10, 2014
National Cancer Institute (NCI)
Information provided by (Responsible Party):

Brief Summary:

DM-CHOC-PEN is a polychlorinated pyridine cholesteryl carbonate that has demonstrated antineoplastic activities in human xenograft intracerebrally implanted tumor mouse models, acceptable preclinical toxicities in mouse, rat and dog models; and no behavioral cognitive impairment/neurotoxicities were noted in mouse and rat models.

The drug is ready for human use as an soy bean oil/lecithin/glycerin water emulsion, the latter which has been documented - chemically and biologically to be stable and safe. Patients are currently being enrolled and treated with the protocol.

Patients with advanced cancer, with or without central nervous system involvement will be eligible for enrollment, providing the required blood and other eligibility requirements are met.

Condition or disease Intervention/treatment Phase
Advanced Cancer With/Without Brain Involvement. Drug: DM-CHOC-PEN Phase 1

Detailed Description:

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Protocol for the Safety and Tolerance of Intravenous 4-Demethylcholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) in Patients With Advanced Cancer
Study Start Date : September 2010
Actual Primary Completion Date : July 2013
Actual Study Completion Date : November 2013

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: 4-DM-CHOC-PEN

DM-CHOC-PEN is an intervention and will be dosed @ 39 mg/M2,escalated in 1-patient cohorts at 40% dosage.

  • At the 1st DLT - expand to 3-6 patient cohorts/dose with escalations at 33% increments.
  • The MTD will be where 2 DLTs are noted and the study is discontinued.

DM-CHOC-PEN will be dosed @ 39 mg/M2, escalated in 1-patient cohorts at 40% dosage.

At the 1st DLT - expand to 3-6 patient cohorts/dose with escalations at 33% increments.

The MTD will be where 2 DLTs are noted and the study is discontinued.

Other Name: 4-Demethylcholesteryloxycarbonylpenclomedine

Primary Outcome Measures :
  1. Document maximum tolerated dose (MTD) & SLT [ Time Frame: one year ]

Secondary Outcome Measures :
  1. Document a toxicity profile for the drug [ Time Frame: one year ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01048008

United States, Louisiana
Tulane University Medical School
New Orleans, Louisiana, United States, 70112
Sponsors and Collaborators
National Cancer Institute (NCI)
Principal Investigator: Marcus L Ware, MD Tulane University Medical School

Responsible Party: DEKK-TEC, Inc. Identifier: NCT01048008     History of Changes
Other Study ID Numbers: DTI-021
5R43CA132257-02 ( U.S. NIH Grant/Contract )
First Posted: January 13, 2010    Key Record Dates
Last Update Posted: January 10, 2014
Last Verified: January 2014

Keywords provided by DEKK-TEC, Inc.:
Glioblastoma multiforme
Advanced cancers
Brain Neoplasms, Malignant, Primary
Brain Tumor, Recurrent
Advanced cancer

Additional relevant MeSH terms: