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Clinical Trials/NCT03455504
NCT03455504
Active, not recruiting
Phase 2

Study Investigating Safety, Tolerability and Effectiveness of Venetoclax Add in Combination With Fludarabine, Cyratabine and Idarubicine in Induction of New Onset Non-M3 Acute Myelid Leukemia

Gruppo Italiano Malattie EMatologiche dell'Adulto22 sites in 1 country124 target enrollmentStarted: October 26, 2018Last updated:
InterventionsVenetoclax

Overview

Phase
Phase 2
Status
Active, not recruiting
Enrollment
124
Locations
22
Primary Endpoint
Number of patients achieving complete response (CR) after course 1 or course 2 if course 2 is administered (CR + CRi + CRp)

Overview

Brief Summary

This study will test the effectiveness and safety of Venetoclax in combination with standard induction regimen for patients with acute leukemia and poor prognosis.

Detailed Description

Intensive treatment of Acute Leukemia, multidrug based chemotherapy induction, achieves complete remission (CR) rates ranging from 50% to 80%. Nonetheless, the majority (60- 70%) of responding patients will eventually relapse.

Patients with non-low risk leukemia according most diffuse risk scores have a poor prognosis and a short survival. For these patient traditional chemotherapy seems to be ineffective, even with BMT consolidation, probably due to activation of survival pathways in Leukemia cells, so that novel therapeutic interventions should be attempted.

This study will test the effectiveness and safety of Venetoclax, a selective BCL2 inhibitor, in combination with standard induction regimen.

Inhibiting anti-apoptotic pathway of BCL2, the investigators expect to improve success rate of standard chemotherapies.

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
18 Years to 65 Years (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Patients with documented/confirmed new onset non-m3 acute myeloid leukemia, according to WHO 2017 will be eligible for this study.
  • Hematological illness has to be graded intermediate or high risk according ELN criteria
  • Patients ≥ 18 years old and ≤ 65 years old
  • ECOG performance status ≤
  • Patients with a life expectancy \>12 weeks
  • Patients may have AML which has arisen from prior therapies or other antecedent disorder
  • Adequate hepatic function
  • Adequate pancreatic function
  • Adequate renal function assessed by: Serum creatinine within reference laboratory ranges or creatinine clearance (by Cockcroft Gault formula, see Appendix 2) ≥ 50 mL/min for patients in whom, in the Investigator's judgment, serum creatinine level may not adequately reflect renal function.
  • All non-hematological adverse events must have resolved to NCI-CTCAE Grade ≤ 2 prior to starting therapy.

Exclusion Criteria

  • Patients with low risk AML according ELN criteria
  • Patients with current clinical evidence of CNS leukemia.
  • Patients receiving any other investigational or commercial agents or therapies administered with the intention to treat their malignancy with the exception of Hydroxyurea (HU) or 6-Mercaptopurine (6MP) in patients who need to continue this agent to maintain WBC count ≤10,000/mm
  • HU and 6MP must be discontinued at the time of initiation of study medications.
  • Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study including but not limited at:
  • unstable angina
  • Patients who are on anti-microbial agents with therapeutic intent

Arms & Interventions

Cohort 1

Experimental

FLAI + V400 mg

Intervention: Venetoclax (Drug)

Cohort 2

Experimental

FLAI + V600 mg

Intervention: Venetoclax (Drug)

Outcomes

Primary Outcomes

Number of patients achieving complete response (CR) after course 1 or course 2 if course 2 is administered (CR + CRi + CRp)

Time Frame: At 24 months from study entry

Efficacy in new onset, non ELN Low risk AML (safety run-in, part1, and part2). Effectiveness of Venetoclax added in combination with chemotherapy in obtaining Complete remission in new onset, non ELN Low risk AML (safety run-in, part1, and part2).

Secondary Outcomes

No secondary outcomes reported

Investigators

Sponsor Class
Other
Responsible Party
Sponsor

Study Sites (22)

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