Ignite Creation Date:
2024-05-06 @ 12:51 PM
Last Modification Date:
2024-10-26 @ 1:05 PM
Study NCT ID:
NCT03866109
Status:
RECRUITING
Last Update Posted:
2023-01-20
First Post:
2019-03-05
Brief Title:
A Study Evaluating Temferon in Patients With Glioblastoma Unmethylated MGMT
Sponsor:
Genenta Science
Organization:
Genenta Science
Study Overview
Official Title:
A Phase IIIa Dose Escalation Study Evaluating the Safety and Efficacy of Autologous CD34-Enriched HSPCs Genetically Modified With Human Interferon-α2 in Patients With Glioblastoma Multiforme and Unmethylated MGMT Gene Promoter
Status:
RECRUITING
Status Verified Date:
2022-01
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:
TEM-GBM
Brief Summary:
This is a non-randomized open label phase IIIa dose-escalation study involving a single injection of Temferon an investigational advanced therapy consisting of autologous CD34-enriched hematopoietic stem and progenitor cells exposed to transduction with a lentiviral vector driving myeloid specific interferon-alpha2 expression which will be administered to up to 27 patients affected by GBM who have an unmethylated MGMT promoter Part A will evaluate the safety and tolerability of 5 escalating doses of Temferon and 3 different conditioning regimens in up to 27 patients following first line treatment
Detailed Description:
This is a non-randomized open label multicenter phase IIIa therapeutic-exploratory dose escalation prospective study involving a single injection of Temferon an investigational ATMP consisting of autologous CD34-enriched HSPCs exposed to transduction with a 3rd generation lentiviral vector driving myeloid-specific IFN-alpha2 expression which will be administered to up to 27 patients affected by GBM who have an unmethylated MGMT promoter The study will recruit and follow-up patients at a specialist neurosurgical and neuro-oncology units in Italy Administration of Temferon and hematological follow up will take place at specialist hematology and bone marrow transplantation units
Potentially eligible patients will be identified immediately after surgical resection of GBM once the MGMT promoter methylator status is known Once written informed consent is obtained and screening procedures have been completed harvesting of HSPCs will occur A standard of care regimen lasting approximately 6 weeks will then take place During this time Temferon manufacturing will occur Following completion of radiotherapy patients will be admitted for receipt of a conditioning regimen consisting of BCNU and thiotepa Cohorts 1-6 busulfan and thiotepa Cohort 5 or busulfan Cohorts 7 and 8 This will be followed by administration of Temferon In-patient monitoring will occur until hematological recovery occurs Thereafter regular follow-up of patients will occur up to 2 years 720 days with the majority of assessments and procedures At the 720 day visit patients will be invited to participate in a long term follow-up study which will last for an additional 6 years
In Part A of the study 8 cohorts of 3 patients will receive 5 escalating doses of Temferon On completion of dose escalation in Part A a conditioning regimen and single dose of Temferon will be selected to be studied in up to a further 6 patients
In the event that GBM disease progression occurs patients will be managed with second line therapies including second surgery TMZ BCNU fotemustine or any other approved therapy for GBM
Potentially eligible patients will be identified immediately after surgical resection of GBM once the MGMT promoter methylator status is known Once written informed consent is obtained and screening procedures have been completed harvesting of HSPCs will occur A standard of care regimen lasting approximately 6 weeks will then take place During this time Temferon manufacturing will occur Following completion of radiotherapy patients will be admitted for receipt of a conditioning regimen consisting of BCNU and thiotepa Cohorts 1-6 busulfan and thiotepa Cohort 5 or busulfan Cohorts 7 and 8 This will be followed by administration of Temferon In-patient monitoring will occur until hematological recovery occurs Thereafter regular follow-up of patients will occur up to 2 years 720 days with the majority of assessments and procedures At the 720 day visit patients will be invited to participate in a long term follow-up study which will last for an additional 6 years
In Part A of the study 8 cohorts of 3 patients will receive 5 escalating doses of Temferon On completion of dose escalation in Part A a conditioning regimen and single dose of Temferon will be selected to be studied in up to a further 6 patients
In the event that GBM disease progression occurs patients will be managed with second line therapies including second surgery TMZ BCNU fotemustine or any other approved therapy for GBM
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2018-001404-11 | EUDRACT_NUMBER | None | None |