Discontinuation of Hypomethylating Agent and Venetoclax in Patients With AML MRD
NCT Number: NCT06511882
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Acute Myeloid Leukemia
Interventions: DRUG: Azacitidine|DRUG: Decitabine|DRUG: Venetoclax
Sponsor: H. Lee Moffitt Cancer Center and Research Institute
Collaborators: AbbVie
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 37
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-06-28
Completion Date: 2028-06
Locations: Moffitt Cancer Center, Tampa, Florida, 33612, United States

Summary:
The purpose of this research study is to see if people whose Acute myeloid leukemia (AML) is being successfully treated with azacitidine or decitabine in combination with venetoclax can discontinue this chemotherapy for some period of time after a year of treatment without increasing the likelihood that their AML will return.

Primary Outcome Measures: Rates of Complete Response (CR)/Complete Response with incomplete hematologic recovery (CRi), Rates of CR/CRi at 18 months from the time of initial CR/CRi in patients who discontinue frontline HMA (azacitidine or decitabine)/VEN (venetoclax) after achieving Measurable Residual Disease (MRD) negativity by MFC within 12 months of starting therapy.

The Null hypothesis (p0) will be tested against the alternative hypothesis (p1).

Null hypothesis: p0 ≤ 50% will remain in CR/CRi at 18 months from the time of initial CR/CRi Alternative hypothesis: p1 ≥ 70% will remain in CR/CRi at 18 months from the time of initial CR/CRi, Up to 18 Months
Secondary Outcome Measures: Overall Survival (OS), Overall survival will be assessed in MRD-negative patients who discontinue HMA (azacitidine or decitabine)/VEN (venetoclx).

Overall Survival (OS) is defined as the length of time from the start of treatment until death due to any cause., Up to 36 Months|Rates to Re-Treatment, Rates to re-treatment is defined as Measurable Residual Disease (MRD)-negative CR/CRi and CR/CRi and rates to re-treatment following molecular or morphologic relapse duration discontinuation., Up to 36 Months|Treatment Free Molecular Remission (TFMR), Defined as time from Measurable Residual Disease (MRD) negativity to first MRD positivity, Up to 36 Months|European Organization for Research and Treatment of Cancer (EORTC-QLQ-C30)/Quality of Life (QoL), EORTC-QLQ-C30/QoL consists of a 30-item subject self-report questionnaire composed of five functional scales (physical, role, emotional, social, and cognitive), three symptom scales (fatigue, nausea and vomiting, and pain), a global health status/quality of life scale, and six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties).

Scale scores range from 0-100., Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)|Patient Reported Outcome Measurement Information System (PROMIS), PROMIS scale provides response options 1-5 to indicate patient-reported health status measures for physical, mental, and social well-being.

It measures concepts such as pain, fatigue, physical function, depression, anxiety, and social function., Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)|EuroQoL-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analog Scale (VAS), EQ-5D-5L VAS is an instrument for measuring the general health status of an individual. It consists of five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.

Scale scores range from 0-100., Day 1 (start of enrollment), start of Discontinuation Phase, and monthly until EOT (Up to 36 Months)