Ignite Creation Date:
2024-05-06 @ 12:03 PM
Last Modification Date:
2024-10-26 @ 12:53 PM
Study NCT ID:
NCT03668847
Status:
COMPLETED
Last Update Posted:
2022-11-08
First Post:
2018-09-05
Brief Title:
DM-CHOC-PEN for Brain Tumors in AYA Subjects
Sponsor:
DEKK-TEC Inc
Organization:
DEKK-TEC Inc
Study Overview
Official Title:
A Phase II Safety and Tolerance of 4-Demethyl-4-cholesteryloxycarbonylpenclomedine DM-CHOC-PEN in Adolescent and Young Adults AYA With Malignancies Involving the CNS
Status:
COMPLETED
Status Verified Date:
2022-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
This Phase II trial is closed for adolescent and young adults AYA subjects with advanced cancer - brain involvement is required
Detailed Description:
The primary goal of this Phase II AYA oncology clinical trial was to evaluate the safety and efficacy of 4-demethyl-4-cholesteryloxycarbonylpenclomedine DM-CHOC-PEN as anticancer therapy in AYA individuals with advanced cancer involving the central or spinal nervous system CNS SNS
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 acceptablereversible 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 adolescent young adult and adult Phase III clinical trials - IND - 68876
The FDA has supported the Phase II clinical trial designed to identify safety and efficacy in AYA cancers subjects and the trial has been completed with acceptable toxicity and MTDs identified
Almost 700000 people in the US are living with tumors involving the CNS or spinal nervous system SNS tumors Nearly 15 of these tumors involve the adolescentyoung adult AYA population aged 15-39 years of age It is predicted that 10617 AYA individuals will be diagnosed with brain or CNS tumors resulting in 434 deaths this year in the US Trends in CNS tumors have sharply increased since 1989 for AYA individuals with a history of cancer who appeared to have beaten the odds only to have a re-occurrence from cancer involving the CNS after years of remission the most common types of cancer in AYA individuals are - melanoma leukemia and sarcomas This group of individuals deserves special attention
For males and female individuals 20 years of age primary brain and secondary cancers of the CNS and spinal nervous system SNS are the most common causes of death from cancer and in the 20-39 year age group the first cause of cancer-related deaths in males and the fifth cause of cancer-related deaths in females The incidence and histology of cancer types does vary according to subject age
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 irritationneurotoxicity 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 neurotic behavioral alterations and associated events supports the drugs use in children with CNS tumors at an age in which brain development and maturation is still very active with cognitive lability The observed responses noted in adults with metastatic cancers involving the CNS and cerebellum treated with DM-CHOC-PEN may also occur in medulloblastoma in AYA Thus the drugs unique properties and lack of toxicities noted in the adult studies merits the Phase I trial proposed here in children
The specific objectives of this Phase I study were to
1 Conduct a Phase II clinical trial with DM-CHOC-PEN in AYA individuals that have advanced cancers with central or spinal nervous systems involvements and monitor safety and document anticancer activity for the drug All data was between investigators communicated through an e-RAP program This was accomplished through IND - 68876
2 Verify the pharmacokineticdynamic profiles of DM-CHOC-PEN and metabolites in AYA subjects with advanced cancers involving the central nervous system
3 Analyze data and prepare an Orphan Drug Designated package for FDA submission for AYA subjects with CNS involvement from cancer for review
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 acceptablereversible 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 adolescent young adult and adult Phase III clinical trials - IND - 68876
The FDA has supported the Phase II clinical trial designed to identify safety and efficacy in AYA cancers subjects and the trial has been completed with acceptable toxicity and MTDs identified
Almost 700000 people in the US are living with tumors involving the CNS or spinal nervous system SNS tumors Nearly 15 of these tumors involve the adolescentyoung adult AYA population aged 15-39 years of age It is predicted that 10617 AYA individuals will be diagnosed with brain or CNS tumors resulting in 434 deaths this year in the US Trends in CNS tumors have sharply increased since 1989 for AYA individuals with a history of cancer who appeared to have beaten the odds only to have a re-occurrence from cancer involving the CNS after years of remission the most common types of cancer in AYA individuals are - melanoma leukemia and sarcomas This group of individuals deserves special attention
For males and female individuals 20 years of age primary brain and secondary cancers of the CNS and spinal nervous system SNS are the most common causes of death from cancer and in the 20-39 year age group the first cause of cancer-related deaths in males and the fifth cause of cancer-related deaths in females The incidence and histology of cancer types does vary according to subject age
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 irritationneurotoxicity 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 neurotic behavioral alterations and associated events supports the drugs use in children with CNS tumors at an age in which brain development and maturation is still very active with cognitive lability The observed responses noted in adults with metastatic cancers involving the CNS and cerebellum treated with DM-CHOC-PEN may also occur in medulloblastoma in AYA Thus the drugs unique properties and lack of toxicities noted in the adult studies merits the Phase I trial proposed here in children
The specific objectives of this Phase I study were to
1 Conduct a Phase II clinical trial with DM-CHOC-PEN in AYA individuals that have advanced cancers with central or spinal nervous systems involvements and monitor safety and document anticancer activity for the drug All data was between investigators communicated through an e-RAP program This was accomplished through IND - 68876
2 Verify the pharmacokineticdynamic profiles of DM-CHOC-PEN and metabolites in AYA subjects with advanced cancers involving the central nervous system
3 Analyze data and prepare an Orphan Drug Designated package for FDA submission for AYA subjects with CNS involvement from cancer for review
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None