Advanced CancerPhase 1NCT03371004
DM-CHOC-PEN Plus Radiation for Brain Tumors
A Phase 1 study testing 4-Demethyl-4-cholesteryloxycarbonylpenclomedine in people with Advanced Cancer, run by DEKK-TEC, Inc. at 3 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Advanced Cancer
- What is being tested?
- 4-Demethyl-4-cholesteryloxycarbonylpenclomedine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 3 sitesUnited States
- Study dates
- 5 Feb 2016to 1 Oct 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- 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 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 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 are 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 will be denied enrollment in the study. The possible interactions of these drugs and DM-CHOC-PEN have not been established. Subjects receiving these drugs will only be eligible if they discontinue the drugs and have an acceptable ECG.
- Coagulopathies - patients requiring full dose anticoagulation with warfarin are excluded. However, patients stable and on other anticoagulants can be incl
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Detroit Clinical Research Centers
Detroit, Michigan, 48336
Tulane University Medical Center
New Orleans, Louisiana, 70112
Tulane University Medical School
New Orleans, Louisiana, 70112
About this study
Official title: A Phase I Trial to Evaluate Safety and Tolerance of Intravenous 4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) in Adult Subjects With Cancer Involving the CNS
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) (1). DM-CHOC-PEN has completed Phase I/II trials in humans with primary and secondary tumors involving the brain with success. Complete remissions in both primary astrocytoma and metastatic lung and leukemia malignancies.
This trial is open for adult subjects with advanced cancer - brain involvement is required.
Read the detailed description
The primary goal of this Phase I oncology clinical trial will be to evaluate the safety and use of 4-demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) plus radiation, as anticancer therapy for adults with advanced cancer involving the central nervous system (CNS) involvement.
DM-CHOC-PEN is a polychlorinated pyridine cholesteryloxycarbonate that crosses the blood brain barrier (BBB), accumulates in CNS tumor tissue in humans and has produced objective responses, with acceptable/reversible hepatic toxicities (in patients with prior liver disease) and no evidence of hematological, renal, neuro-toxicities with improved quality of life and overall survival in adult Phase I/II clinical trials - IND - 68,876.
The drug recently received Orphan Drug designation for NSCLC involving the brain.
The FDA supports the proposed Phase I clinical trial designed to identify safety, toxicities and an acceptable MTD of the drug in combination with radiation in adult cancers subjects with CNS involvement.
Almost 700,000 people in the US are living with tumors involving the CNS or spinal nervous system (SNS) tumors. Trends in CNS tumors have sharply increased since 1989 for individuals with a history of cancer, who appeared to have 'beaten the odds', only to have a reoccurrence from cancer involving the CNS after years of remission; the most common types of cancer in AYA individuals are - from lung, breast, melanoma, and sarcoma malignancies. This group of individuals deserves special attention.
A critical component in designing an agent that will cross the protective blood brain barrier (BBB) is that the agent must be readily transported intracerebrally, does not produce local irritation/neurotoxicity and is not recycled back into the general circulation. After IV administration DM-CHOC-PEN readily penetrates the BBB, is not a substrate for the transporter protein P-glycoprotein (P-gp) and has shown anticancer activity in CNS tumors. The effective transport of DM-CHOC-PEN into CNS tumors in adults without neurotoxic behavioral alterations and associated events supports the drug's use in combination with radiation in the treatment of CNS tumors. The observed responses noted in adults with metastatic cancers involving the CNS and cerebellum treated with DM-CHOC-PEN is encouraging. Thus, the drug's unique properties and lack of toxicities noted in the adult studies merits the Phase I trial proposed here in combination with radiation.
The specific objectives of this Phase I study will be to:
- Conduct a Phase I clinical trial with DM-CHOC-PEN plus radiation in adults with advanced cancers involving the central nervous system to document toxicities, define an acceptable maximum tolerated dose (MTD), and identify anticancer activity for the binary treatment DM-CHOC-PEN plus radiation. All data will be communicated through an e-RAP program. This will be accomplished through IND - 68.876.
- Studying the pharmacokinetic/dynamic profiles of DM-CHOC-PEN and metabolites in adults after being treated with DM-CHOC-PEN and radiation.
- Analyze data and prepare a Phase II clinical trial or a Designation Orphan Drug application for FDA review.
- Conditions
- Advanced Cancer
- Being tested
- 4-Demethyl-4-cholesteryloxycarbonylpenclomedine
- Keywords
- DM-CHOC-PEN, Radiation, Cancer
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4-Demethyl-4-cholesteryloxycarbonylpenclomedineDrug
Also called DM-CHOC-PEN
IV administration as described
| Study group | Role | What they receive |
|---|---|---|
| DM-CHOC-PEN + Radiation | Experimental | 4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) - 39-89.7 MG/M2 iv once and then 3-weeks later radiation - 15-30 Gy will be administered Receives: Drug: 4-Demethyl-4-cholesteryloxycarbonylpenclomedine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Laboratory and imaging studies to verify responses
Measured: 1 year
Secondary outcomes (1)
Imaging studies
MRI Exams of the Brain after Treatments
Measured: 6-weeks after treatments
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2017
- Study start5 Feb 2016
- Primary completion1 Oct 2022
- Study completion1 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- DEKK-TEC, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Steven DiBiase, MD, Principal investigator, Tulane University Medical CenterLee Roy Morgan, MD, PhD, Principal investigator, DEKK-TEC, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03371004
- Sponsor's protocol ID
- DTI-025
- Status last verified
- Nov 2022
- First submitted
- 19 Nov 2017
- Last updated
- 4 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Other trial centers are being evaluated
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial to Evaluate Safety and Tolerance of Intravenous 4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) in Adult Subjects With Cancer Involving the CNS
- ClinicalTrials.gov ID
- NCT03371004
- Acronym / sponsor ID
- DTI-025
- Conditions
- Advanced Cancer
- Keywords
- DM-CHOC-PEN, Radiation, Cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- DEKK-TEC, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 5 Feb 2016 (actual)
- Primary completion
- 1 Oct 2022 (actual)
- Study completion
- 1 Oct 2022 (actual)
- First posted
- 13 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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) (1). DM-CHOC-PEN has completed Phase I/II trials in humans with primary and secondary tumors involving the brain with success. Complete remissions in both primary astrocytoma and metastatic lung and leukemia malignancies.
This trial is open for adult subjects with advanced cancer - brain involvement is required.
Detailed description
The primary goal of this Phase I oncology clinical trial will be to evaluate the safety and use of 4-demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) plus radiation, as anticancer therapy for adults with advanced cancer involving the central nervous system (CNS) involvement.
DM-CHOC-PEN is a polychlorinated pyridine cholesteryloxycarbonate that crosses the blood brain barrier (BBB), accumulates in CNS tumor tissue in humans and has produced objective responses, with acceptable/reversible hepatic toxicities (in patients with prior liver disease) and no evidence of hematological, renal, neuro-toxicities with improved quality of life and overall survival in adult Phase I/II clinical trials - IND - 68,876.
The drug recently received Orphan Drug designation for NSCLC involving the brain.
The FDA supports the proposed Phase I clinical trial designed to identify safety, toxicities and an acceptable MTD of the drug in combination with radiation in adult cancers subjects with CNS involvement.
Almost 700,000 people in the US are living with tumors involving the CNS or spinal nervous system (SNS) tumors. Trends in CNS tumors have sharply increased since 1989 for individuals with a history of cancer, who appeared to have 'beaten the odds', only to have a reoccurrence from cancer involving the CNS after years of remission; the most common types of cancer in AYA individuals are - from lung, breast, melanoma, and sarcoma malignancies. This group of individuals deserves special attention.
A critical component in designing an agent that will cross the protective blood brain barrier (BBB) is that the agent must be readily transported intracerebrally, does not produce local irritation/neurotoxicity and is not recycled back into the general circulation. After IV administration DM-CHOC-PEN readily penetrates the BBB, is not a substrate for the transporter protein P-glycoprotein (P-gp) and has shown anticancer activity in CNS tumors. The effective transport of DM-CHOC-PEN into CNS tumors in adults without neurotoxic behavioral alterations and associated events supports the drug's use in combination with radiation in the treatment of CNS tumors. The observed responses noted in adults with metastatic cancers involving the CNS and cerebellum treated with DM-CHOC-PEN is encouraging. Thus, the drug's unique properties and lack of toxicities noted in the adult studies merits the Phase I trial proposed here in combination with radiation.
The specific objectives of this Phase I study will be to:
- Conduct a Phase I clinical trial with DM-CHOC-PEN plus radiation in adults with advanced cancers involving the central nervous system to document toxicities, define an acceptable maximum tolerated dose (MTD), and identify anticancer activity for the binary treatment DM-CHOC-PEN plus radiation. All data will be communicated through an e-RAP program. This will be accomplished through IND - 68.876.
- Studying the pharmacokinetic/dynamic profiles of DM-CHOC-PEN and metabolites in adults after being treated with DM-CHOC-PEN and radiation.
- Analyze data and prepare a Phase II clinical trial or a Designation Orphan Drug application for FDA review.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| DM-CHOC-PEN + Radiation | Experimental | Drug: 4-Demethyl-4-cholesteryloxycarbonylpenclomedine4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) - 39-89.7 MG/M2 iv once and then 3-weeks later radiation - 15-30 Gy will be administered |
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (6 listed)
- 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 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 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 are 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 will be denied enrollment in the study. The possible interactions of these drugs and DM-CHOC-PEN have not been established. Subjects receiving these drugs will only be eligible if they discontinue the drugs and have an acceptable ECG.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 4-Demethyl-4-cholesteryloxycarbonylpenclomedineAlso: DM-CHOC-PENIV administration as described | Drug | DM-CHOC-PEN + Radiation |
Primary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 1 year
Laboratory and imaging studies to verify responses
1 secondary outcome
Imaging studies
Time frame: 6-weeks after treatments
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2022.
- Individual participant dataSharing planned: Yes. Other trial centers are being evaluated
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven DiBiase, MD principal investigator
Tulane University Medical Center
Lee Roy Morgan, MD, PhD principal investigator
DEKK-TEC, Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 4-Demethyl-4-cholesteryloxycarbonylpenclomedine in people with Advanced Cancer, run by DEKK-TEC, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2022.