An open-label, single-arm, pilot study of Venetoclax in combination with 5 days Azacitidine in treatment-nai¨ve subjects with acute myelogenous leukemia who are =18 years of age and not eligible for standard induction therapy (VENAZA-5S PILOT TRIAL)
- Conditions
- Acute myeloid leukemiaMedDRA version: 21.0Level: LLTClassification code: 10000886Term: Acute myeloid leukemia Class: 10029104Therapeutic area: Diseases [C] - Neoplasms [C04]
- Registration Number
- CTIS2022-501537-23-00
- Lead Sponsor
- niversity of Leipzig
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 45
Confirmed diagnosis of AML by WHO criteria 2016 AND ineligible for treatment with a standard cytarabine and anthracycline induction regimen due to age or comorbidities, Age = 18 years, Projected life expectancy of at least 12 weeks, Adequate renal and liver function
Prior treatment for AML or MDS with one of the following: HMA, Chemotherapeutic agent, CAR-T cell therapy, Experimental therapies; (NOTE: Prior use of hydroxyurea is allowed), History of other malignancies with the exception of adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast; Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; previous malignancy confined and surgically resected (or treated with other modalities) with curative intent, History of myeloproliferative neoplasm (MPN), Diagnosis of acute promyelocytic leukemia (APL), Presence of favorable-risk karyotype abnormalities, i.e. t(15;17), t(8;21), inv(16) or t(16;16), Known active CNS involvement with AML, Known to be positive for HIV, Hepatitis B or C, Cardiovascular disability status of NYHA > 2, Chronic respiratory disease that requires continuous oxygen, or significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, or any other medical condition that in the opinion of the investigator would adversely affect his/her participating in this study, Evidence of other clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial or fungal)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of the trial is to generate first data on the efficacy of the combination of Venetoclax and 5-day courses of Azacitidine in the studied patient population in a descriptive manner.;Secondary Objective: To describe the depth and duration of response with the combination regimen, To describe survival with the combination regimen, To describe the safety and toxicity profile of the combination regimen, To describe potential treatment interruptions and discontinuations, To describe potential hospitalizations, To describe Quality of life (QoL, EORTC QLQ-C30) with the combination regimen, To describe MRD levels on study treatment;Primary end point(s): The primary outcome measure is the response rate defined as the rate of CR or CRi after up to 6 cycles of therapy (best response).
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Rate of CR or CRi by the Initiation of Cycle 2;Secondary end point(s):Rate of CR with partial hematologic recovery (CRh) after up to 6 cycles of therapy;Secondary end point(s):Time from initiation of treatment (C1D1) until achievement of CR or CRi;Secondary end point(s):Objective response rate (CR, CRh, CRi, MLFS);Secondary end point(s):Event free survival (EFS);Secondary end point(s):Overall survival (OS);Secondary end point(s):Descriptive assessment of MRD levels on study treatment;Secondary end point(s):Time to treatment discontinuation;Secondary end point(s):Rate of patients with at least one treatment interruption;Secondary end point(s):Delay of subsequent cycles, dose reductions or shortening/interruption of study drug administration;Secondary end point(s):Duration of patient hospitalization;Secondary end point(s):Quality of life (QoL) measured through EORTC QLQ-C30