Enrollment of trial assessing triplet for leukemia halted after ‘unexpected’ outcome
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Enrollment for a randomized phase 2 trial designed to compare a triplet regimen and doublet regimen for certain patients with leukemia has been discontinued due to low likelihood of benefit.
The SELECT-AML-1 trial included patients with newly diagnosed acute myeloid leukemia with retinoic acid receptor alpha gene overexpression.
Researchers assigned patients 1:1 to venetoclax (Venclexta; AbbVie, Genentech) and azacitidine with or without tamibarotene (Syros Pharmaceuticals), a selective retinoic acid receptor alpha agonist.
As part of a prespecified interim analysis, researchers performed a nonbinding futility analysis that included the first 40 randomly assigned patients who had either received 3 months of treatment on study or discontinued therapy.
Results showed a similar rate of complete response or complete response with incomplete hematologic recovery (CR/CRi) in the triplet group vs. the doublet group (65% vs. 70%).
The probability that the study would demonstrate superiority with the triplet regimen at final analysis “was considered low,” according to a Syros press release, prompting the decision to discontinue enrollment.
Researchers observed no new safety signals with the addition of tamibarotene to venetoclax and azacitidine. Patients enrolled in the trial may remain on study at the discretion of investigators.
Complete data from SELECT-AML-1 will be presented at Society of Hematologic Oncology Annual Meeting, scheduled for Sept. 4-7.
“We are disappointed by this unexpected outcome,” David A. Roth, MD, chief medical officer of Syros, said in the release. “In our prior phase 2 clinical trial, the doublet combination of tamibarotene and azacitidine delivered a 61% CR/CRi rate [among patients with newly diagnosed AML] with [retinoic acid receptor alpha] overexpression. This supports our conviction in pursuing a doublet strategy in higher-risk [myelodysplastic syndrome], where we are comparing tamibarotene and azacitidine to azacitidine alone.”
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