Ignite Creation Date:
2024-05-06 @ 3:58 PM
Last Modification Date:
2024-10-26 @ 2:01 PM
Study NCT ID:
NCT04827719
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-04-19
First Post:
2021-03-18
Brief Title:
BST-236 as a Single Agent in Adults With Relapsed or Refractory AML or HR-MDS
Sponsor:
Groupe Francophone des Myelodysplasies
Organization:
Groupe Francophone des Myelodysplasies
Study Overview
Official Title:
Phase 2 Open-Label Single Arm Multi-Center Study to Assess the Efficacy and Safety of BST-236 as a Single Agent in Adults Unfit for Intensive Chemotherapy With Relapsed or Refractory Acute Myeloid Leukemia or HR-MDS
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-02
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:
To assess the efficacy and safety of BST-236 in patients unfit for intensive chemotherapy with AML or HR MDS that failed or relapsed following first line therapy
Detailed Description:
This is a prospective phase 2 open-label multi-center single arm single agent study in unfit adult patients with AML or HR MDS who failed or relapsed following first-line therapy
The study consists of three periods
Screening Up to 28 days Treatment Recurrent 6 days treatment courses with BST-236 Follow-up The period of time from last day of BST-236 treatment to completion of one year 365 days 7 days from the first day of BST-236 treatment
Post-study Follow-up The period of time from last day of BST-236 treatment to completion of one year 365 days 7 days from the first day of BST-236 treatment
Follow-up One-Year follow-up of survival starting at the end of study visit
Treatment period After signing the Informed Consent Form ICF meeting all eligibility criteria and undergoing screening assessments for up to four weeks eligible patients will receive the first induction course with BST-236 45 gm2d administered IV over 1 hour for 6 consecutive days The first day of the induction is defined as Study Day 1 Following the first treatment course
1 AML patients in complete remission CR or CR with incomplete count recovery CRi or CR with partial hematologic recovery CRh and HR MDS patients with CR will receive between 2 to 4 maintenance courses of 6-day treatment with BST-236 every 4-6 weeks The BST-236 dose in these subsequent treatment courses will be 23g m2d or 45 gm2d The reduced dose must be used in case of previous courses toxicity and is allowed in certain cases according to physician discretion following consultation with the study medical monitor The interval between treatment courses may be extended to up to 12 weeks to allow resolution of toxicities per investigator discretion and in consultation with principal investigators
2 Patients with stable disease defined as failure to achieve at least PR but with no evidence of disease progression or patients in partial response PR will receive a second 6-day course with BST-236 at a dose of 45 gm2d Following which
1 AML patients in PR or CR including CR CRh or CRi and HR MDS patients in PR or CR will be treated with maintenance courses as detailed above Patients in stable disease or disease progression may receive alternative treatment outside of the protocol and will be followed for overall survival OS until the completion of 1 year of participation in the study 365 days7 days from the first day of BST-236 treatment Additional 1 year of post study follow-up will be implemented for these patients in order to follow up on their survival status and their current AMLMDS treatments
Patients that have a disease progression while treated in the maintenance courses will not receive any additional BST-236 treatment and will be followed until the completion of 1 year of participation in the study These patients may also receive alternative treatment outside of the protocol in such case they will be followed in regular intervals for progression free survival OS and development of new malignancies
Follow-up period All patients will be followed by periodic in person in-clinic visits until the completion of 1 year of participation in the study 365 days7 days from the first BST-236 treatment day
End of Study EOS visits will be performed on the last day of participation in the study for all patients
Scheduled in-clinic visits to assess safety and efficacy outcomes will be conducted during the study periods as specified in the Schedule of Activities
Unscheduled visits may be conducted at any time for safety reasons or for any other reason
Post study follow-up All patients will be followed for survival for an additional 1 year from the EOS
QT Evaluation with Pharmacokinetics PK sub-study Up to 20 patients participating in the main study in pre-selected sites will be asked to participate in a QT evaluation with PK sub-study to assess the effect of BST-236 on the cardiac electrophysiology In this study up to 14 blood samples for PK analysis will be taken from each patient during the first induction course and a continuous electrocardiogram ECG recording by a Holter monitor will be done in the first 24 hours of BST-236 administration Patients participating in this study will be asked to sign an additional ICF for the collection of the PK blood samples and ECG recording
The study consists of three periods
Screening Up to 28 days Treatment Recurrent 6 days treatment courses with BST-236 Follow-up The period of time from last day of BST-236 treatment to completion of one year 365 days 7 days from the first day of BST-236 treatment
Post-study Follow-up The period of time from last day of BST-236 treatment to completion of one year 365 days 7 days from the first day of BST-236 treatment
Follow-up One-Year follow-up of survival starting at the end of study visit
Treatment period After signing the Informed Consent Form ICF meeting all eligibility criteria and undergoing screening assessments for up to four weeks eligible patients will receive the first induction course with BST-236 45 gm2d administered IV over 1 hour for 6 consecutive days The first day of the induction is defined as Study Day 1 Following the first treatment course
1 AML patients in complete remission CR or CR with incomplete count recovery CRi or CR with partial hematologic recovery CRh and HR MDS patients with CR will receive between 2 to 4 maintenance courses of 6-day treatment with BST-236 every 4-6 weeks The BST-236 dose in these subsequent treatment courses will be 23g m2d or 45 gm2d The reduced dose must be used in case of previous courses toxicity and is allowed in certain cases according to physician discretion following consultation with the study medical monitor The interval between treatment courses may be extended to up to 12 weeks to allow resolution of toxicities per investigator discretion and in consultation with principal investigators
2 Patients with stable disease defined as failure to achieve at least PR but with no evidence of disease progression or patients in partial response PR will receive a second 6-day course with BST-236 at a dose of 45 gm2d Following which
1 AML patients in PR or CR including CR CRh or CRi and HR MDS patients in PR or CR will be treated with maintenance courses as detailed above Patients in stable disease or disease progression may receive alternative treatment outside of the protocol and will be followed for overall survival OS until the completion of 1 year of participation in the study 365 days7 days from the first day of BST-236 treatment Additional 1 year of post study follow-up will be implemented for these patients in order to follow up on their survival status and their current AMLMDS treatments
Patients that have a disease progression while treated in the maintenance courses will not receive any additional BST-236 treatment and will be followed until the completion of 1 year of participation in the study These patients may also receive alternative treatment outside of the protocol in such case they will be followed in regular intervals for progression free survival OS and development of new malignancies
Follow-up period All patients will be followed by periodic in person in-clinic visits until the completion of 1 year of participation in the study 365 days7 days from the first BST-236 treatment day
End of Study EOS visits will be performed on the last day of participation in the study for all patients
Scheduled in-clinic visits to assess safety and efficacy outcomes will be conducted during the study periods as specified in the Schedule of Activities
Unscheduled visits may be conducted at any time for safety reasons or for any other reason
Post study follow-up All patients will be followed for survival for an additional 1 year from the EOS
QT Evaluation with Pharmacokinetics PK sub-study Up to 20 patients participating in the main study in pre-selected sites will be asked to participate in a QT evaluation with PK sub-study to assess the effect of BST-236 on the cardiac electrophysiology In this study up to 14 blood samples for PK analysis will be taken from each patient during the first induction course and a continuous electrocardiogram ECG recording by a Holter monitor will be done in the first 24 hours of BST-236 administration Patients participating in this study will be asked to sign an additional ICF for the collection of the PK blood samples and ECG recording
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