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Leukemia and Lymphoma Clinical Trials

For questions about new and recurrent leukemia and lymphoma clinical trials, please email the research nurse coordinator, Shari Thompson, or call 202-476-2802.

Title Description Key Eligibility Criteria Sponsor Investigator Disease Type
PEPN22P1: A Pharmacokinetic Study of Vincristine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
NCT05359237
A correlative study of vincristine pharmacokinetics to evaluate a new infant dosing method and determine whether it results in uniform drug exposure (AUC) across the age groups.
  • Age: ≤ 12 years
  • Newly diagnosed and relapsed cancer diagnosis that is being treated with vincristine at the 1.5 mg/m2 dose level
Children's Oncology Group AeRang Kim, M.D. Any
PEPN2113: A Phase 1 and pharmacokinetic study of Uproleselan (GMI-1271, IND #139758, NSC #801708) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane and is in second or greater relapse or that is refractory to relapse therapy
NCT05146739
A Phase 1 study of uproleselan (GMI-1271) in combination with fludarabine and cytarabine (FLA) for pediatric patients with relapsed and refractory acute myeloid leukemia, myelodysplastic syndrome and mixed phenotype acute leukemia that expresses the E-selectin ligand on the cell membrane.
  • Patients with second or greater relapse or refractory AML, MDS, or MPAL that expresses E-selectin ligand on the cell membrane
  • Patients with or without Down syndrome
  • Age: < 18 years
Children's Oncology GroupAeRang Kim, M.D.Acute Myeloid Leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia

TACL T2020-006: A Phase I Study of Tagraxofusp With or Without Chemotherapy in Pediatric Patients with Relapsed or Refractory CD123 Expressing Hematologic Malignancies
NCT05476770

A Phase 1 study to assess safety and tolerability of tagraxofusp monotherapy and determine the recommended dose for combination with chemotherapy in pediatric and young adult patients with relapsed/refractory CD123+ hematologic malignancies, and to assess safety and tolerability of tagraxofusp when given in combination with chemotherapy in pediatric and young adult patients with relapsed/refractory CD123+ hematologic malignancies.
  • Relapsed and/or refractory hematologic malignancy
  • Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry
  • Patients with Down syndrome are eligible
  • Age ≥ 1 year and ≤21 years
Therapeutic Advances in Childhood Leukemia ConsortiumReuven Schore, M.D.Hematologic malignancy including, but not limited to, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated leukemia, blastic plasmacytoid dendritic cell neoplasm, Hodgkin lymphoma, and non-Hodgkin lymphoma

PEPN2111: A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
NCT04870944

This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with relapsed, or refractory solid tumors, including central nervous system (CNS) tumors or lymphoma.
  • Parts A and B1: ≥ 12 months and ≤ 21 years old
  • Part B2 (relapsed/refractory osteosarcoma): ≥ 12 months and ≤ 30 years old
Children's Oncology GroupAeRang Kim, M.D.Solid tumors, lymphoma
PEPN2011: A Phase 1/2 Study of Tegavivint (IND#156033, NSC#826393) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors
NCT04851119
 A Phase 1/2 study to Investigate the safety, dosing and efficacy of Tegavivint in children and young adults with relapsed or refractory solid tumors including desmoid tumors and lymphoma.
  • Part A: ≥ 12 months and ≤ 21 years old 
  • Relapsed or refractory solid tumors, including patients with non-Hodgkin lymphoma and desmoid tumors
  • Part B: ≥ 12 months and ≤ 30 years
  • Recurrent or refractory Ewing sarcoma, desmoid tumors, osteosarcoma, liver tumors (HCC and hepatoblastoma), Wilms tumor, and tumors with Wnt pathway aberrations.
Children's Oncology GroupAeRang Kim, M.D.Solid tumors, including non-Hodgkin lymphoma and desmoid tumors
PEPN2112: A Phase 1/ 2 Study of BAY 1895344 (elimusertib, IND#152153, NSC#810486) in Pediatric Patients with Relapsed
or Refractory Solid Tumors
NCT05071209
A Phase 1/2 study to investigate the safety, dosing and efficacy of BAY 1895344 (elimusertib) in children and young adults with relapsed or refractory solid tumors. 
  • Part A1: Patients with specified recurrent or refractory solid tumors or lymphomas without bone marrow involvement  ≥ 12 months and < 18 years of age
  • Part B1a: Patients with any Ewing Sarcoma or any EWS-fusion positive solid tumor ≥ 18 years of age
  • Part B2a: Patients with alveolar rhabdomyosarcoma (ARMS) with the PAX3-FOXO1 fusion ≥ 18 years of age
  • Part B3a: Patients with any (non-CNS primary) solid tumor including lymphoma with inactivation of any of the DNA Damage Repair (DDR) genes ≥ 18 years of age
Children's Oncology GroupAeRang Kim, M.D., Ph.D.Ewing sarcoma, alveolar rhabdomyosarcoma with PAX-FOXO1 fusion, non-CNS primary solid tumor including lymphoma with inactivation of DDR genes
AALL1721: A Phase 2 trial of Tisagenlecleucel in First-Line High-Risk (HR) Pediatric and Young Adult Patients with B-cell Acute Lymphoblastic Leukemia (B-ALL) who are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) therapy.
NCT03876769
A phase 2 trial to evaluate the efficacy of tisagenlecleucel in first-line high-risk (HR) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (B-ALL) who are minimal residual disease (MRD) positive at the end of consolidation (EOC) therapy.
  • Relapsed/refractory CD19 expressing B-Cell acute lymphoblastic leukemia
  • MRD positive at EOC therapy
  • Ages 1 years to 25 years

Novartis Pharmaceuticals

Reuven Schore, M.D.B-Cell Acute Lymphoblastic Leukemia
ADVL1822: A Phase 1/2, Multi-Center, Dose-Escalating Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Quizartinib Administered in Combination with Re-Induction Chemotherapy, and as a Single- Agent Continuation Therapy, in Pediatric Relapsed/ Refractory AML Subjects Aged 1 Month to <18 Years (And Young Adults Aged Up to 21 Years) With FLT3-ITD Mutations
NCT03793478
This is a Phase 1/2 study of Quizartinib, administered in combination with fludarabine and cytarabine (FLA) (Re-Induction Cycle 1) and FLA (Re-Induction Cycle 2) chemotherapy for re-induction, and as a single agent continuation therapy. It is being investigated as a treatment for relapsed or refractory AML in pediatric subjects aged ≥1 month to <18 years (and young adults aged up to 21 years) old with FLT3-internal tandem duplication (ITD) mutations following failure of front-line intensive chemotherapy.
  • Diagnosis of acute myeloid leukemia with FLT3-ITD mutation
  • 1st recurrence/relapse of acute myeloid leukemia
  • Ages 1 months to ≤ 21 years
Daiichi Sankyo, Inc.AeRang Kim, M.D., Ph.D.Acute Myeloid Leukemia
BIANCA: A Phase II, single arm, multicenter open label trial to determine the safety and efficacy of tisagenlecleucel in pediatric subjects with relapsed or refractory mature B-cell non-Hodgkin lymphoma (NHL)
NCT03610724
A Phase II trial of tisagenlecleucel in pediatric subjects with relapsed or refractory mature B-cell non-Hodgkin lymphoma (BIANCA study).
  • Relapsed/refractory B-cell non-Hodgkin lymphoma
  • Ages ≤ 25 years old
Novartis PharmaceuticalsKeri Toner, M.D.B-cell non-Hodgkin lymphoma
T2017-002, A TACL Phase 1/2 Study of PO Ixazomib in Combination with Chemotherapy for Childhood Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma NCT03817320Phase 1/2 study of Ixazomib administered in conjunction with re-induction chemotherapy.
  • Relapsed/ refractory acute lymphoblastic leukemia or lymphoblastic lymphoma
  • Ages ≤21 years old but will be restricted to <18 years old for the first 9 patients enrolled on each phase
Therapeutic Advances in Childhood Leukemia ConsortiumReuven Schore, M.D.Acute lymphoblastic leukemia, lymphoblastic lymphoma
ADVL1414: A Phase 1 Study of Selinexor (KPT-330, IND #125052), a Selective XPO1 Inhibitor, in Recurrent and Refractory Pediatric Solid Tumors, including CNS Tumors
NCT02323880
This phase 1 trial of selinexor in relapsed or refractory pediatric solid tumors or high-grade gliomas.
  • Part A: Recurrent/refractory solid tumors, including lymphoma and CNS tumors
  • Part B: Recurrent/refractory high grade glioma (WHO Grade III/IV) including disseminated tumors (excluding diffuse intrinsic pontine glioma), not requiring surgical resection
  • Part C: Patients with recurrent/refractory high grade glioma (WHO Grade III/IV) and requiring surgicalresection (excluding diffuse intrinsic pontine glioma and disseminated tumors)
  • Ages ≥ 12 months old to ≤ 21 years old
Children's Oncology GroupJeffrey Dome, M.D., Ph.D.High grade glioma, all solid tumors, all CNS, lymphoma
ADVL18P1: An Open-Label Feasibility Study to Assess the Safety and Pharmacokinetics of Enasidenib in Pediatric Patients with Relapsed/Refractory Acute Myeloid Leukemia R/R-AML with an Isocitrate Dehydrogenase-2 IDH2 Mutation
NCT04203316
This trial studies enasidenib for patients with relapsed or refractory AML with a mutation in a protein called IDH2
  • Acute myeloid leukemia with an IDH2 mutation identified from a peripheral blood or bone marrow sample at the time of diagnosis and/or relapsed/refractory disease.
  • Ages ≥ 24 months to ≤ 18 years old
Children's Oncology GroupAeRang Kim, M.D., Ph.D.Acute myeloid leukemia
AALL1931: An Open Label, Multicenter Study of RC-P in Patients with Acute Lymphoblastic Leukemia ALL/Lymphoblastic Lymphoma LBL Following Hypersensitivity to E. coli-derived Asparaginases
NCT04145531
This is a pivitol phase 2/3 study to determine the efficacy, safety and tolerability of RC-P, a novel asparaginase agent, in patients who are hypersensitive to E. coli derived asparaginases.
  • Pediatric or adult patients diagnosed with B-cell acute lymphoblastic leukemia or acute lymphoblastic leukemia/lymphoma
  • Patients that have had an allergic reaction to long acting E. coli-derived asparaginase or have a silent inactivation
Jazz PharmaceuticalsReuven Schore, M.D.Acute lymphoblastic leukemia, acute lymphoblastic lymphoma
ANHL12P1: Brentuximab Vedotin or Crizotinib and Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage II-IV Anaplastic Large Cell Lymphoma
NCT01979536
This partially randomized phase II trial studies how well Brentuximab Vedotin or Crizotinib, and combination chemotherapy works in treating patients with newly diagnosed stage II-IV anaplastic large cell lymphoma.
  • Newly diagnosed histologically proven anaplastic large cell lymphoma
  • Stage II, III, or IV disease
  • Ages < 22 years
Children's Oncology GroupBirte Wistinghausen, M.D.Anaplastic large cell lymphoma (ALK-positive), Ann Arbor stage II - IV noncutaneous childhood anaplastic large cell lymphoma, CD30-positive neoplastic cells present
ANHL1522: Rituximab and LMP-Specific T-Cells in Treating Pediatric Solid Organ Recipients With EBV-Positive Cluster of Differentiation(CD) 20-Positive Post-Transplant Lymphoproliferative Disorder
NCT02900976
This pilot clinical trial studies how rituximab and latent membrane protein (LMP)-specific T-cells work in treating pediatric solid organ recipients with Epstein-Barr virus-positive, cluster of differentiation (CD)20-positive post-transplant lymphoproliferative disorder.
  • History of solid organ transplantation
  • Newly diagnosed, relapsed/refractory polymorphic or monomorphic post-transplant lymphoproliferative disorder
  • Ages < 30 years
Children's Oncology GroupBirte Wistinghausen, M.D.Epstein-Barr virus-related post-transplant lymphoproliferative disorder, monomorphic post-transplant lymphoproliferative disorder, polymorphic post-transplant lymphoproliferative disorder