Ignite Creation Date:
2025-12-24 @ 4:16 PM
Ignite Modification Date:
2025-12-29 @ 12:19 AM
Study NCT ID:
NCT05379166
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-12-22
First Post:
2022-03-15
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Venetoclax and Azacitidine for Treatment of Therapy Related or Secondary Myelodysplastic Syndrome
Sponsor:
Uma Borate
Organization:
Study Overview
Official Title:
Phase II Study of Clinical Efficacy of Venetoclax in Combination With Azacitidine in Patients With Therapy Related Myelodysplastic Syndrome (t-MDS)
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-10
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:
This phase II trial studies the effect of venetoclax and azacitidine in treating patients with therapy related or secondary myelodysplastic syndrome. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Chemotherapy drugs, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving venetoclax in combination with azacitidine may work better in treating patients with therapy related or secondary myelodysplastic syndrome.
Detailed Description:
PRIMARY OBJECTIVE:
I. Determine the proportion of participants that achieve a complete remission following treatment with azacitidine and venetoclax.
SECONDARY OBJECTIVES:
I. Assess safety of azacitidine and venetoclax combination therapy. II. Determine the overall response rate (ORR). III. Determine the complete cytogenetic response rate (CCyR). IV. Determine the duration of response (DOR). V. Estimate event-free survival (EFS). VI. Estimate overall survival (OS). VII. Determine combined hematologic improvement rate (HIR). VIII. Determine red blood cell transfusion independence rate. IX. Determine platelet transfusion independence rate. X. Determine proportion of participants whose disease transforms to acute myeloid leukemia (AML).
XI. Determine proportion of participants that proceed to allogeneic hematopoietic stem cell transplantation (alloHSCT).
XII. Compare response criteria between the 2006 International Working Group (IWG) and 2023 IWG criteria.
EXPLORATORY OBJECTIVES:
I. Determine baseline frequencies of cytogenetic aberrations and their relationships to response to study therapy.
II. Estimate progression-free survival (PFS). III. Obtain quality of life (QoL) information from patient-reported responses to Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 7a and European Organisation for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) - Core 30 (C30) questionnaires.
OUTLINE:
Patients receive venetoclax orally (PO) once daily (QD) on days 1-14 and azacitidine intravenously (IV) over 10-40 minutes on days 1-7 or days 1-5 of week 1 and days 1 and 2 of week 2. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days and every 6 months for up to 24 months.
I. Determine the proportion of participants that achieve a complete remission following treatment with azacitidine and venetoclax.
SECONDARY OBJECTIVES:
I. Assess safety of azacitidine and venetoclax combination therapy. II. Determine the overall response rate (ORR). III. Determine the complete cytogenetic response rate (CCyR). IV. Determine the duration of response (DOR). V. Estimate event-free survival (EFS). VI. Estimate overall survival (OS). VII. Determine combined hematologic improvement rate (HIR). VIII. Determine red blood cell transfusion independence rate. IX. Determine platelet transfusion independence rate. X. Determine proportion of participants whose disease transforms to acute myeloid leukemia (AML).
XI. Determine proportion of participants that proceed to allogeneic hematopoietic stem cell transplantation (alloHSCT).
XII. Compare response criteria between the 2006 International Working Group (IWG) and 2023 IWG criteria.
EXPLORATORY OBJECTIVES:
I. Determine baseline frequencies of cytogenetic aberrations and their relationships to response to study therapy.
II. Estimate progression-free survival (PFS). III. Obtain quality of life (QoL) information from patient-reported responses to Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 7a and European Organisation for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) - Core 30 (C30) questionnaires.
OUTLINE:
Patients receive venetoclax orally (PO) once daily (QD) on days 1-14 and azacitidine intravenously (IV) over 10-40 minutes on days 1-7 or days 1-5 of week 1 and days 1 and 2 of week 2. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days and every 6 months for up to 24 months.
Study Oversight
Has Oversight DMC:
True
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?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| NCI-2021-08484 | REGISTRY | CTRP (Clinical Trial Reporting Program) | View |