Trial Outcomes & Findings for Study of 4-Demethyl-4-cholesteryloxycarbonylpenclome (DM-CHOC-PEN) in Patients With Brain Tumors (NCT NCT02038218)

NCT ID: NCT02038218

Last Updated: 2020-09-16

Results Overview

Tumor Diameter from patient scans

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 29 participants
• Primary outcome timeframe: Until remission or off treatment due to progression
• Results posted on: 2020-09-16

Participant Flow

Study completed - Tulane Medical Center (2017-19), Detroit Research Center (Lansing, Detroit and Owasso) 2017-19, Mt Sinai Medical Center, NYNY, 2014-18 and Ochsner Medical Centers (New Orleans, 2017-19.

Patients with cancer involving the CNS were treated with intravenous DM-CHOC-PEN. The drug is metabolized by the liver. Abnormal neurological and hepatic values excluded the patients from treatments. If the patients were on steroids, this also excluded treatments. Steroids initiate the break down of DM-CHOC-PEN.

Participant milestones

Measure	DM-CHOC-PEN - Arm 1 Cohort 1: Patients without liver involvement or history of liver disease, will be treated at a dose of 98.7 mg/m2. Patients in both Arms 1 \& 2 will discontinue dosing with DM-CHOC-PEN when there are any unacceptable toxicities, progression of cancer or patient compliance. 4-Demethyl-4-cholestryloxycarbonylpenclomedine: This will be an open-label, uncontrolled two-arm, multi-center study in patients with CNS involvement from melanoma, breast, lung cancers or primary malignancies of the CNS. Patients can be previously treated with radiation and systemic therapies and are eligible if they also have other sites of cancer involvement. Two Cohorts of patients will be treated every 21 days with a single infusion of	DM-CHOC-PEN - Arm 2 Cohort 2: Patients with liver involvement or history of disease will be treated at a dose of 85.8 mg/m2; 5 patients were in this group
Overall Study STARTED	22	7
Overall Study COMPLETED	22	7
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of 4-Demethyl-4-cholesteryloxycarbonylpenclome (DM-CHOC-PEN) in Patients With Brain Tumors

Baseline characteristics by cohort

Measure	ARM 1 n=22 Participants Arm-1: Patients with without liver involvement or history of liver disease, will be treated at a dose of 98.7 mg/m2. Patients will be treated every once every 21 days with a single infusion of DM-CHOC-PEN as an out-patient. 4-Demethyl-4-cholestryloxycarbonylpenclomedine: This will be an open-label, uncontrolled two-arm, multi-center study in patients with CNS involvement from melanoma, breast, lung cancers or primary malignancies of the CNS.	ARM 2 n=7 Participants Arm-2: Patients with liver involvement or history of liver disease, will be treated at a dose of 85.8mg/m2. Patients will be treated every once every 21 days with a single infusion of DM-CHOC-PEN as an out-patient. 4-Demethyl-4-cholestryloxycarbonylpenclomedine: This will be an open-label, uncontrolled two-arm, multi-center study in patients with CNS involvement from melanoma, breast, lung cancers or primary malignancies of the CNS.	Total n=29 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	22 Participants n=5 Participants	6 Participants n=7 Participants	28 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Age, Continuous	51 years STANDARD_DEVIATION 8 • n=5 Participants	61 years STANDARD_DEVIATION 10 • n=7 Participants	52 years STANDARD_DEVIATION 8 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	2 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants	5 Participants n=7 Participants	19 Participants n=5 Participants
Region of Enrollment United States	22 Participants n=5 Participants	7 Participants n=7 Participants	29 Participants n=5 Participants
Average Initial Tumor Diameter	2.8 cm STANDARD_DEVIATION 1.3 • n=5 Participants	2.6 cm STANDARD_DEVIATION 0.8 • n=7 Participants	2.8 cm STANDARD_DEVIATION 1.2 • n=5 Participants

PRIMARY outcome

Timeframe: Until remission or off treatment due to progression

Population: Mean Tumor size after Treatment

Tumor Diameter from patient scans

Outcome measures

Measure	DM-CHOC-PEN - Arm 1 n=22 Participants Cohort 1: Patients without liver involvement or history of liver disease, will be treated at a dose of 98.7 mg/m2. Patients will discontinue dosing with DM-CHOC-PEN when there are any unacceptable toxicities, progression of cancer or patient compliance. 4-Demethyl-4-cholestryloxycarbonylpenclomedine: This will be an open-label, uncontrolled two-arm, multi-center study in patients with CNS involvement from melanoma, breast, lung cancers or primary malignancies of the CNS. Patients can be previously treated with radiation and systemic therapies and are eligible if they also have other sites of cancer involvement.	DM-CHOC-PEN - Arm 2 n=7 Participants Cohort 2: Patients with liver involvement or history of disease will be treated at a dose of 85.8 mg/m2. Patients will discontinue dosing with DM-CHOC-PEN when there are any unacceptable toxicities, progression of cancer or patient compliance. 4-Demethyl-4-cholestryloxycarbonylpenclomedine: This will be an open-label, uncontrolled multi-center study in patients with CNS involvement from melanoma, breast, lung cancers or primary malignancies of the CNS. Patients can be previously treated with radiation and systemic therapies and are eligible if they also have other sites of cancer involvement.
Tumor Diameter	2.7 cm Standard Deviation 1.1	4.4 cm Standard Deviation 0.8

PRIMARY outcome

Timeframe: Until death

Overall survival in months

Outcome measures

Measure	DM-CHOC-PEN - Arm 1 n=22 Participants Cohort 1: Patients without liver involvement or history of liver disease, will be treated at a dose of 98.7 mg/m2. Patients will discontinue dosing with DM-CHOC-PEN when there are any unacceptable toxicities, progression of cancer or patient compliance. 4-Demethyl-4-cholestryloxycarbonylpenclomedine: This will be an open-label, uncontrolled two-arm, multi-center study in patients with CNS involvement from melanoma, breast, lung cancers or primary malignancies of the CNS. Patients can be previously treated with radiation and systemic therapies and are eligible if they also have other sites of cancer involvement.	DM-CHOC-PEN - Arm 2 n=7 Participants Cohort 2: Patients with liver involvement or history of disease will be treated at a dose of 85.8 mg/m2. Patients will discontinue dosing with DM-CHOC-PEN when there are any unacceptable toxicities, progression of cancer or patient compliance. 4-Demethyl-4-cholestryloxycarbonylpenclomedine: This will be an open-label, uncontrolled multi-center study in patients with CNS involvement from melanoma, breast, lung cancers or primary malignancies of the CNS. Patients can be previously treated with radiation and systemic therapies and are eligible if they also have other sites of cancer involvement.
Overall Survival	11 Months Standard Deviation 8	8 Months Standard Deviation 3

Adverse Events

ARM 1

Serious events: 0 serious events

Other events: 3 other events

Deaths: 19 deaths

ARM 2

Serious events: 0 serious events

Other events: 1 other events

Deaths: 7 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	ARM 1 n=22 participants at risk Patients without liver involvement	ARM 2 n=7 participants at risk Patients with liver involvement
Nervous system disorders Headache	4.5% 1/22 • Number of events 1 • Data was collected over 2.5 years	0.00% 0/7 • Data was collected over 2.5 years
Nervous system disorders Seizures	9.1% 2/22 • Data was collected over 2.5 years	14.3% 1/7 • Data was collected over 2.5 years

Additional Information

Dr. Lee Roy Morgan

DEKK-TEC, Inc.

Phone: 504-583-6135

Email: lrm1579@aol.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place