This trial is currently open and accepting patients.
The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.
Enrollment: 260 patients (estimated)View More
November 15, 2022
As of June 1, 2022, 51 pts with AML were treated (16 in cohort 1; 17 in cohort 2; 14 in cohort 3; 4 in cohort 4). Median age was 77 yrs (TN, n=28) and 64 yrs (R/R, n=23) (Table 1). At baseline, 23 pts (45.1%) had adverse-risk cytogenetics, 37 (72.5%) had grade ≥3 neutropenia, and 27 (52.9%) had grade ≥3 thrombocytopenia.
At a median follow-up of 2.8 mo (range 0.1-12.3) and median duration of treatment of 1.9 mo (range 0.0-12.3), 2 of 41 evaluable pts experienced DLTs (grade 4 neutropenia and grade 4 thrombocytopenia at 80 mg) (Table 2), which did not meet the safety stopping protocol criteria. Laboratory tumor lysis syndrome (TLS; Howard criteria) was observed in 1 pt (160 mg x 10 days) with a known history of chronic kidney disease; TLS resolved within 4 days.
Twenty-one (41%) pts discontinued study drugs: 7 (13.7%) due to AEs, 5 (9.8%) due to disease progression, 4 (7.8%) proceeded to transplant, 3 (5.9%) withdrew consent, 1 (2.0%) per investigator decision, and 1 (2.0%) started new anti-cancer treatment.
The most common TEAEs were neutropenia (58.8%), thrombocytopenia (45.1%), anemia (43.1%), febrile neutropenia (33.3%), nausea (39.2%), and constipation (37.3%). Neutropenia was the most common grade ≥3 TEAE observed in 30 (58.8%) pts; most cases did not require dose modification. Grade ≥3 infections occurred in 23 (45.1%) pts, mostly in cycle 1. The general incidence of febrile neutropenia and infections decreased with subsequent cycles. Four (7.8%) pts died due to unrelated TEAEs: bronchopulmonary aspergillosis, pneumonia, pulmonary sepsis, and possible intracranial hemorrhage after a fall. TEAEs leading to dose reduction (5.9%) or dose interruption (11.8%) were infrequent.
In TN AML, complete remission (CR)/CR with partial hematologic recovery (CRh) was achieved in 16 (59.3%) and CR in 13 (48.1%) evaluable pts (Table 1). Of the 13 pts in CR, 7 (53.8 %) reached CR by end of cycle 1. In cohort 2 with the longest follow-up, CR was achieved in 8 (72.7%) pts with TN AML. Notably, 7 of these pts are continuing study treatment (median of 5 cycles, range 1-12) without progression. In R/R AML, CR/CRh was seen in 50% of evaluable pts, with a CR rate of 25%. Nine (50.0%; 6 TN, 3 R/R) of 18 evaluable pts with CR/CRh achieved MRD negativity (<0.1% per ELN 2018).
In pts with AML treated with 40, 80, and 160 mg, PK increased in a dose-dependent manner at steady state with no drug-drug interaction observed with aza.
View all clinical trial locations sorted by state.
If you are interested in keeping an eye on this trial, you can add it to your list of favorite trials. We'll send you alerts when this trial is updated.
You can explore trial locations from around the US and connect directly with a trial coordinator.Find Nearby Locations
You can print an overview of this trial to take in to your next appointment. Your doctor can help you understand if this trial may be right for you.
Still need help? Send us a message