Ignite Creation Date:
2024-05-06 @ 9:02 AM
Last Modification Date:
2024-10-26 @ 12:09 PM
Study NCT ID:
NCT02889445
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2020-08-18
First Post:
2016-08-31
Brief Title:
A Phase I Trial of DM-CHOC-PEN in Adolescent and Young Adult AYA Subjects With Advanced Cancers
Sponsor:
DEKK-TEC Inc
Organization:
DEKK-TEC Inc
Study Overview
Official Title:
A Phase I Trial to Evaluate Safety and Tolerance of Intravenous 4-Demethyl-4-cholesteryloxycarbonylpenclomedine DM-CHOC-PEN in Adolescent and Young Adults AYA Subjects With Cancers
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2020-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
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 1 DM-CHOC-PEN has completed Phase III trials in humans with primary and secondary tumors involving the brain with success Complete remissions in both primary astrocytomas GBM and metastatic lung cancers
This trial is open for adolescent and young adults AYA subjects with advanced cancer - brain involvement is not required
This trial is open for adolescent and young adults AYA subjects with advanced cancer - brain involvement is not required
Detailed Description:
The primary goal of this Phase I pediatric oncology clinical trial will be to evaluate the safety and use of 4-demethyl-4-cholesteryloxycarbonylpenclomedine DM-CHOC-PEN as anticancer therapy for AYA with advanced cancer - 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 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 adult Phase III clinical trials - IND - 68876 1-6
The FDA supports the proposed Phase I clinical trial designed to identify safety toxicities and an acceptable MTD in AYA cancers subjects now that the adult Phase I trial has been completed with acceptable toxicity and MTDs identified 2 3 5 6
Almost 700000 people in the US are living with tumors involving the CNS or spinal nervous system SNS tumors 6 Nearly 15 of these tumors involve the adolescentyoung adult AYA population aged 15-39 years of age 6 It is predicted that 10617 AYA individuals will be diagnosed with brain or CNS tumors resulting in 434 deaths this year in the US 6 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 reoccurrence from cancer involving the CNS after years of remission the most common types of cancer in AYA individuals are - melanoma leukemia and sarcomas 7 This group of individuals deserve special care
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 4 The effective transport of DM-CHOC-PEN into CNS tumors in adults without neurotoxic 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 Table 1 may also occur in medulloblastoma in AYA 7 9 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 will be to
1 Conduct a Phase I clinical trial with DM-CHOC-PEN in AYA that have advanced cancers with - the central nervous system to document toxicities define an acceptable maximum tolerated dose MTD and identify anticancer activity for the drug All data will be communicated through an e-RAP program This will be accomplished through IND - 68876
2 Studying the pharmacokineticdynamic profiles of DM-CHOC-PEN and metabolites in AYAs with advanced cancers involving the central nervous system
3 Analyze data and prepare a Phase II clinical trial in AYA subjects for FDA 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 adult Phase III clinical trials - IND - 68876 1-6
The FDA supports the proposed Phase I clinical trial designed to identify safety toxicities and an acceptable MTD in AYA cancers subjects now that the adult Phase I trial has been completed with acceptable toxicity and MTDs identified 2 3 5 6
Almost 700000 people in the US are living with tumors involving the CNS or spinal nervous system SNS tumors 6 Nearly 15 of these tumors involve the adolescentyoung adult AYA population aged 15-39 years of age 6 It is predicted that 10617 AYA individuals will be diagnosed with brain or CNS tumors resulting in 434 deaths this year in the US 6 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 reoccurrence from cancer involving the CNS after years of remission the most common types of cancer in AYA individuals are - melanoma leukemia and sarcomas 7 This group of individuals deserve special care
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 4 The effective transport of DM-CHOC-PEN into CNS tumors in adults without neurotoxic 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 Table 1 may also occur in medulloblastoma in AYA 7 9 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 will be to
1 Conduct a Phase I clinical trial with DM-CHOC-PEN in AYA that have advanced cancers with - the central nervous system to document toxicities define an acceptable maximum tolerated dose MTD and identify anticancer activity for the drug All data will be communicated through an e-RAP program This will be accomplished through IND - 68876
2 Studying the pharmacokineticdynamic profiles of DM-CHOC-PEN and metabolites in AYAs with advanced cancers involving the central nervous system
3 Analyze data and prepare a Phase II clinical trial in AYA subjects for FDA review
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None