DM-CHOC-PEN for Brain Tumors in AYA Subjects

NCT03668847 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: DTI-026
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) is a polychlorinated pyridyl cholesterol carbonate that is lipophilic, electrically neural, crosses the blood brain barrier (BBB), ability to localize in intracranial tumor tissue, lacks neurotoxicity and not transported out of the brain via Pgp (p-glycoprotein). DM-CHOC-PEN has completed a Phase I Adolescent and Young Adult (AYA) trial in humans, some of which possessed primary and secondary tumors involving the brain. Complete remissions in both primary (astrocytoma, GBM) and metastatic lung cancers were reported. This Phase II trial is closed for adolescent and young adults (AYA) subjects with advanced cancer - brain involvement is required.

Conditions

Advanced Cancer; Brain Metastases

Outcome Measures

Primary Outcomes (1)

• Brain Tumor Responses to Therapy

  MRI of the brain

  From date of entry into the study until the date of first documented progression or date of of death from any cause, which ever came first, assessed up to 100 months.

Secondary Outcomes (1)

• Maximum Plasma Concentration [Cmax], Area Under the Curve [AUC]

  From initiation of treatment until end of therapy or death from any cause or which ever comes first, assessed up to 8-months.

Study Arms (1)

DM-CHOC-PEN

EXPERIMENTAL

4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) - 75 or 98.7 mg/m2 emulsion will be administered IV once every 21-days until relapse

Drug: DM-CHOC-PEN

Interventions

DM-CHOC-PEN DRUG

DM-CHOC-PEN administration by IV infusion

DM-CHOC-PEN

Eligibility Criteria

• Age: 15 Years - 39 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects must have histological proof of a malignancy, which has been treated with standard treatments, which may include radiation, and measurable lesions are not required but must have evidence that the disease is advanced.
• Subjects must have life expectancy of at least 12 weeks and a Karnofsky performance score: \> 60 % (or a Zubrod performance status of \< 2).
• The age limit - a limit of 39 years of age. Gender is not a criterion.
• All subjects 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); no nitrosourea type drug or ipilumimab treatments are permitted within the last six (6) weeks prior to enrollment. No major surgery within 14 days of enrollment. Subjects may continue to receive anti- estrogen/steroid therapy that has been initiated at least eight weeks prior to enrollment in the study.
• Subjects should have adequate bone marrow function defined as a peripheral WBC \>3,000/mm3 with an ANC \>1500/mm3 and a platelet count \>100,000/mm3.
• Subjects should have hepatic function (alkaline phosphatase, AST and ALT) \< ULN and renal functions with serum creatinine - \<1.5 x UNL. If a patient has liver metastasis and/or a history of liver disease - they will receive a lower dose of the drug per treatment protocol.
• Subjects should not be allergic to eggs or soy beans.
• Subjects must be medically, psychologically and neurologically stable and have triplicate baseline ECG's with a mean QTc interval \<500 ms and \>300 ms and neither a history of congenital prolonged or short QT syndrome. Subjects with a history of cardiac disease must be stable.
• Subjects and/or legal guardian must understand the nature of the study and be willing to sign an informed consent that complies with the investigator/DEKK-TEC policies and approved by the Human Investigation Review Committee.

You may not qualify if:

• Subjects with concurrent severe and/or uncontrolled medical co-morbidities - including active infections, unstable uncontrolled diabetes, cardiovascular and pulmonary, renal, psychiatric or social conditions that could compromise the safety or compliance of treatment are not eligible.
• Concomitant chemotherapy or radiotherapy was not permitted.
• Pregnant or lactating females were 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 the barrier birth control while on the study and for 3-months after the last dose of the study drug.
• Subjects taking CYP3A4 inducers or inhibitors were not eligible since it is not known whether the study drug is metabolized through this pathway. The following CYP3A4 inhibitors/inducers are not permitted during the trial - phenobarbital, fluconazole, erythromycin, verapamil; the latter 3-drugs are moderate CYP3A4 inhibitors.
• Subjects taking the following medications may experience QT/QTc interval prolongation and are not eligible for the trial - most anti-arrhythmia drugs (incl. amiodarone), erythromycin, quinolone antibiotics, ketoconazole, Zithromax, and phenothiazine and were denied enrollment in the study. The possible interactions of these drugs and DM-CHOC-PEN have not been established.
• Coagulopathies - patients requiring full dose anticoagulation with warfarin were excluded.

Sponsors & Collaborators

• DEKK-TEC, Inc.: lead

Study Sites (1)

Tulane University Medical Center

New Orleans, Louisiana, 70112, United States

Location

MeSH Terms

Conditions

Brain Neoplasms

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Ali Baghian, MD

  Tulane University Medical Center

  PRINCIPAL INVESTIGATOR

• Tallat Mahmmod, MD

  Detroit Clinical Research Center

  PRINCIPAL INVESTIGATOR

• Marcus L Ware, MD

  Ochsner Health System

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 5, 2018
• First Posted: September 13, 2018
• Study Start: January 1, 2019
• Primary Completion: August 31, 2021
• Study Completion: September 30, 2021
• Last Updated: November 8, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF, CSR
• Time Frame: The data summary is being prepared as a manuscript for distribution. December 31, 2022
• Access Criteria: The requesting individuals must be established investigators.

Locations

US (1)