Clinical Trials
A Phase 1/2A, Open Label Dose Escalation and Expansion Study of Orally Administered CA-4948 as a Monotherapy in Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome
Lead Researcher: Jane Larae Liesveld MD
This is a multicenter, open-label, Phase 1/2a dose escalation and expansion study of orally administered emavusertib (CA-4948) monotherapy in adult patients with Acute Myelogenous Leukemia (AML) or high risk Myelodysplastic Syndrome (MDS).
Patients enrolling in the Phase 1 portion of the study must meet one of the following criteria prior to consenting to the study:
R/R AML with FLT3 mutations who have been previously treated with a FLT3 inhibitor
R/R AML with spliceosome mutations of SF3B1 or U2AF1
R/R hrMDS with spliceosome mutations of SF3B1 or U2AF1
Number of pretreatments: 1 or 2
The Phase 2a Dose Expansion will be in 3 Cohorts of patients:
R/R AML with FLT3 mutations who have been previously treated with a FLT3 inhibitor;
R/R AML with spliceosome mutations of SF3B1 or U2AF1; and
R/R hrMDS (IPSS-R score > 3.5) with spliceosome mutations of SF3B1 or U2AF1.
All patients above have had ≤ 2 lines of prior systemic anticancer treatment. In previous versions of this protocol there was a Phase 1b portion of the study, in which patients with AML or hrMDS received CA-4948 in combination with venetoclax. This part of the study is no longer open for enrollment.
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A Single-Arm, Open-Label Pharmacokinetic, Safety, and Efficacy Study of ASTX727 in Combination With Venetoclax in Adult Patients With Acute Myeloid Leukemia
Lead Researcher: Jane Larae Liesveld MD
The Phase 1 portion of this study is a single-arm, open-label, multicenter, non-randomized interventional study to evaluate the pharmacokinetic (PK) interaction, safety, and efficacy of ASTX727 when given in combination with venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy. The primary purpose of the study is to rule out drug-drug interactions between ASTX727 and venetoclax combination therapy by evaluating area under the curve (AUC) and maximum plasma concentration (Cmax) exposure. The Phase 2 portion of the study is to assess the efficacy of ASTX727 and venetoclax when given in combination and to evaluate potential PK interactions. Phase 2 will follow the same overall study design as Phase 1 and has two parts, Part A and Part B.
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A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) for transplantation in pediatric and adult patients with hematologic malignancies and other indications (NMDP 10-CBA)
Lead Researcher: Jane Larae Liesveld MD
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.
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A Program for Monitoring Minimal Residual Disease Following Treatment of Patients With Acute Myeloid Leukemia or High Grade Myelodysplastic Syndrome
Lead Researcher: Jane Larae Liesveld MD
This study is being performed to develop assays to determine the impact of the therapy patients receive for treatment of AML or MDS and to determine if these tests can identify those patients who are at a greater risk for having their disease relapse.
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Publications
Journal Articles
11/12/2023
Cliburn JA, Uy JC, Swift S, Liesveld JL, Iqbal MA, Jajosky AN, Becker MW. "Diagnosing familial platelet disorder with predisposition to myeloid malignancy: Lessons learned from a germline whole-gene deletion of RUNX1." International journal of laboratory hematology.. 2023 Nov 12; Epub 2023 Nov 12.
10/23/2023
Hill BT, Ma S, Zent CS, Baran AM, Wallace DS, Advani AS, Winter AM, Winter JN, Gordon LI, Karmali R, Liesveld JL, Mulford DA, Rowland C, Bui A, Sportelli P, Miskin HP, Weiss MS, Friedberg JW, Barr PM. "Response-Adapted, Time-Limited Venetoclax, Umbralisib and Ublituximab for Relapsed/Refractory Chronic Lymphocytic Leukemia." Blood advances.. 2023 Oct 23; Epub 2023 Oct 23.
8/12/2023
Stein EM, de Botton S, Cluzeau T, Pigneux A, Liesveld JL, Cook RJ, Rousselot P, Rizzieri DA, Braun T, Roboz GJ, Lebon D, Heiblig M, Baker K, Volkert A, Paul S, Rajagopal N, Roth DA, Kelly M, Peterlin P. "Use of tamibarotene, a potent and selective RAR? agonist, in combination with azacitidine in patients with relapsed and refractory AML with gene overexpression." Leukemia & lymphoma.. 2023 Aug 12; :1-10. Epub 2023 Aug 12.
Books & Chapters
2017
Chapter Title: Stem Cell Mobilization
Book Title: In: Encyclopedia of Cancer
Author List: Liesveld, JL
Edited By: Schwab, M
Published By: Springer-Verlag 2017
2016
Chapter Title: Acute Myelogenous Leukemia
Book Title: In: Williams Hematology
Author List: Liesveld, JL and Lichtman, MA
Edited By: Kaushansky K, Lichtman MA, Prchal JT, Levi MM, Press OW, Burns LJ, Caligiuri MA
Published By: McGraw Hill 2016 in New York, NY
2016
Chapter Title: Chronic Myelogenous Leukemia and Related Disorders
Book Title: In: Williams Hematology
Author List: Liesveld, JL and Lichtman, MA
Edited By: Kaushansky K, Lichtman MA, Prchal JT, Levi MM, Press OW, Burns LJ, Caligiuri MA,
Published By: McGraw Hill 2016 in New York, NY
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