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|Molecular Profile||Indication/Tumor Type||Response Type||Relevant Treatment Approaches||Therapy Name||Approval Status||Evidence Type||Efficacy Evidence||References|
|IDH1 R132X||acute myeloid leukemia||sensitive||IDH1 Inhibitor||IDH305||Phase I||Actionable||In a Phase I trial, IDH305 treatment resulted in objective response in 33% (7/21) of acute myeloid leukemia patients harboring IDH1 R132 mutations, including complete remission in 3 (14%) and partial remission in 4 (19%) patients (Blood 2016 128 (22):1073).||detail...|
|IDH1 R132X||acute myeloid leukemia||predicted - sensitive||CG-806||Preclinical - Patient cell culture||Actionable||In a preclinical study, patient-derived acute myeloid leukemia cells harboring IDH1 R132X mutations demonstrated increased sensitivity to CG-806 compared to wild-type cells in culture (Proceedings of the American Association for Cancer Research, Vol 60, Mar 2019, Abstract #1323).||detail...|
|IDH1 R132X||acute myeloid leukemia||no benefit||IDH1 Inhibitor||BAY1436032||Phase I||Actionable||In a Phase I trial, treatment with BAY1436032 in acute myeloid leukemia patients harboring an IDH1 R132X mutation demonstrated safety and resulted in an overall response rate of 15% (4/27), including one complete remission, one partial remission, and morphologic leukemia-free state in two patients, stable disease in 67% (18/27), and a median overall survival of 6.6 months, however, due to low response rates for all doses, further clinical development was not supported (PMID: 32733012; NCT03127735).||32733012|
|IDH1 R132X||low grade glioma||predicted - sensitive||IDH1 Inhibitor||Ivosidenib||Phase I||Actionable||In a Phase I trial, low grade glioma patients with an IDH1 mutation (n=66; R132H=57, R132C/G/S=1 each, R132X=5) treated with Tibsovo (ivosidenib) demonstrated an overall response rate of 2.9% (1/35, 1 partial response) and stable disease in 85.7% (30/35) of patients with a non-enhancing glioma versus no responses and stable disease in 45.2% (14/31) of patients with an enhancing glioma, and led to a median progression-free survival of 13.6 months and 1.4 months, respectively (PMID: 32530764; NCT02073994).||32530764|
|IDH1 R132X||acute myeloid leukemia||predicted - sensitive||IDH1 Inhibitor||LY3410738||Phase I||Actionable||In a Phase I trial, LY3410738 demonstrated safety and inhibited D-2-HG in patients with relapsed or refractory IDH-mutant acute myeloid leukemia, resulting in a composite complete remission (CRc) rate of 38% (12/32, 5 CR, 2 CRh, 5 CRi/CRp) in IDH inhibitor-naive patients harbor IDH1 R132 mutations (Cancer Res (2023) 83 (8_Supplement): CT026; NCT04603001).||detail...|
|IDH1 R132X||brain glioma||predicted - sensitive||IDH1 Inhibitor||Olutasidenib||Phase Ib/II||Actionable||In a Phase Ib/II trial, Rezlidhia (olutasidenib) treatment was well tolerated and did not meet the primary endpoint, with an objective response rate of 8% (2/25, both partial responses) but led to a disease control rate of 48% (12/25) in patients with glioma harboring IDH1 R132H (n=22), R132L (n=2), R132C (n=1), or R132G (n=1) (PMID: 35639513; NCT04380012).||35639513|