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Disease

Acute leukemia

Late-stage therapeutic development
Also known as acute leukaemia (disease), acute leukemia (disease), leukemia, acute, X-linked, stem cell leukaemia+3 more

acute leukaemia (disease), acute leukemia (disease), leukemia, acute, X-linked, stem cell leukaemia, stem cell leukaemia (disease), stem cell leukemia, stem cell leukemia (disease).

21
Clinical trials
3
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 4 clinical trials expected to report results, the earliest in Q3 2027.
Upcoming Milestones
Clinical Milestones6View
Results posted 1
Results expected 4
Other updates 1
Activity timeline6

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Melphalan hydrochlorideapproved

Approval — High-dose of Phelinun used alone or in combination with other cytotoxic medicinal product… (2020)

Posaconazoleapproved

Approval — Posaconazole Accord is indicated for use in the treatment of the following fungal infecti… (2019)

Clinical trials

14 sponsors · 2 new · 0 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
21
All trials
6
Active
8
Late-stage
6
Completed
Late-stage studies

A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy

Phase 3 · Recruiting · Children's Oncology Group

Interfant-21 International Collaborative Treatment Protocol for Infants Under One Year With KMT2A-rearranged Acute Lymphoblastic Leukemia or Mixed Phenotype Acute Leukemia.

Phase 3 · Recruiting · Princess Maxima Center for Pediatric Oncology

A Randomized Phase III Trial of Patients With Advanced Hematologic Malignancies Undergoing Allogeneic Hematopoietic Cell Transplantation With Either Orca-T, a T-cell-Depleted Graft With Additional Infusion of Conventional T Cells and Regulatory T Cells, or Standard-of-Care Allogeneic Graft

Phase 3 · Active, not recruiting · Orca Biosystems, Inc.

A Randomized, Multicenter, Phase III Trial of Tacrolimus/Methotrexate Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation (BMT CTN 1703; Progress III); Companion Study: Microbiome and Immune Reconstitution in Cellular Therapies and Hematopoietic Stem Cell Transplantation (BMT CTN 1801; Mi-Immune)

Phase 3 · Completed · Medical College of Wisconsin

A Randomized, Multi-Center, Phase III Trial of Calcineurin Inhibitor-Free Interventions for Prevention of Graft-versus-Host Disease (BMT CTN 1301; Progress II)

Phase 3 · Completed · National Heart, Lung, and Blood Institute (NHLBI)

A Randomized Trial of Levofloxacin to Prevent Bacteremia in Children Being Treated for Acute Leukemia (AL) or Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Phase 3 · Completed · Children's Oncology Group
Recruiting

A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy

Phase 3 · Recruiting · Children's Oncology Group

Interfant-21 International Collaborative Treatment Protocol for Infants Under One Year With KMT2A-rearranged Acute Lymphoblastic Leukemia or Mixed Phenotype Acute Leukemia.

Phase 3 · Recruiting · Princess Maxima Center for Pediatric Oncology

SJALL23T: Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)

Phase 2 · Recruiting · St. Jude Children's Research Hospital

A Multicenter Phase II Study of Subcutaneous Blinatumomab for Treatment of Adult Patients With CD19-Positive Mixed Phenotype Acute Leukemia (MPAL)

Phase 2 · Recruiting · West Virginia University

Phase 2 Trial of Menin Inhibitor BN104 as Post Hematopoietic Stem Cell Transplantation Maintenance in Patients With Acute Leukemia

Phase 2 · Recruiting · The First Affiliated Hospital of Soochow University
Recently completed

A Multicenter Phase II Study of Blinatumomab for Treatment of Adult Patients With Morphologic Relapsed/Refractory or Measurable Residual Disease (MRD) CD19-Positive Mixed Phenotype Acute Leukemia (MPAL)

Phase 2 · Completed · University of Maryland, Baltimore

A Phase Ⅱ, Open Label, Single Arm, Single-Center Study to Evaluate the Efficacy and Safety of Azacitidine，Venetoclax，and Flumatinib in Newly Diagnosed Ph-positive Acute Leukemia and CML-AP/BP Patients

Phase 2 · Completed · The First Affiliated Hospital of Soochow University

A Randomized, Multicenter, Phase III Trial of Tacrolimus/Methotrexate Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation (BMT CTN 1703; Progress III); Companion Study: Microbiome and Immune Reconstitution in Cellular Therapies and Hematopoietic Stem Cell Transplantation (BMT CTN 1801; Mi-Immune)

Phase 3 · Completed · Medical College of Wisconsin

A Randomized, Multi-Center, Phase III Trial of Calcineurin Inhibitor-Free Interventions for Prevention of Graft-versus-Host Disease (BMT CTN 1301; Progress II)

Phase 3 · Completed · National Heart, Lung, and Blood Institute (NHLBI)

A Randomized Trial of Levofloxacin to Prevent Bacteremia in Children Being Treated for Acute Leukemia (AL) or Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Phase 3 · Completed · Children's Oncology Group

Clofarabine, Cyclophosfamide, And Etoposide For The Treatment Of Relapsed Or Resistant Acute Leukemia In Pediatric Patients

Phase 2/3 · Completed · Istituto Giannina Gaslini

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2020emaApproval — Melphalan hydrochloride· High-dose of Phelinun used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: multiple myeloma, malignant lymphoma (Hodgkin, non-Hodgkin lymphoma), acute lymphoblastic and myeloblastic leukemia, childhood neuroblastoma, ovarian cancer, mammary adenocarcinoma. Phelinun in combination with other cytotoxic medicinal products is indicated as reduced intensity conditioning (RIC) treatment prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in malignant haematological diseases in adults. Phelinun in combination with other cytotoxic medicinal products is indicated as conditioning regimen prior to allogeneic haematopoietic stem cell transplantation in haematological diseases in the paediatric population as: Myeloablative conditioning (MAC) treatment in case of malignant haematological diseases RIC treatment in case of non-malignant haematological diseases. source ↗
2019emaApproval — Posaconazole· Posaconazole Accord is indicated for use in the treatment of the following fungal infections in adults: Invasive aspergillosis; Fusariosis in patients with disease that is refractory to amphotericin B or in patients who are intolerant of amphotericin B; Chromoblastomycosis and mycetoma in patients with disease that is refractory to itraconazole or in patients who are intolerant of itraconazole; Coccidioidomycosis in patients with disease that is refractory to amphotericin B, itraconazole or fluconazole or in patients who are intolerant of these medicinal products. Refractoriness is defined as progression of infection or failure to improve after a minimum of 7 days of prior therapeutic doses of effective antifungal therapy. Posaconazole Accord is also indicated for prophylaxis of invasive fungal infections in the following patients:  Patients receiving remission-induction chemotherapy for acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) expected to result in prolonged neutropenia and who are at high risk of developing invasive fungal infections; Hematopoietic stem cell transplant (HSCT) recipients who are undergoing high-dose immunosuppressive therapy for graft versus host disease and who are at high risk of developing invasive fungal infections. source ↗
2019emaApproval — Posaconazole· Posaconazole AHCL oral suspension is indicated for use in the treatment of the following fungal infections in adults: Invasive aspergillosis in patients with disease that is refractory to amphotericin B or itraconazole or in patients who are intolerant of these medicinal products; Fusariosis in patients with disease that is refractory to amphotericin B or in patients who are intolerant of amphotericin B; Chromoblastomycosis and mycetoma in patients with disease that is refractory to itraconazole or in patients who are intolerant of itraconazole; Coccidioidomycosis in patients with disease that is refractory to amphotericin B, itraconazole or fluconazole or in patients who are intolerant of these medicinal products. Oropharyngeal candidiasis: as first-line therapy in patients who have severe disease or are immunocompromised, in whom response to topical therapy is expected to be poor. Refractoriness is defined as progression of infection or failure to improve after a minimum of 7 days of prior therapeutic doses of effective antifungal therapy. Posaconazole AHCL oral suspension is also indicated for prophylaxis of invasive fungal infections in the following patients: Patients receiving remission-induction chemotherapy for acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) expected to result in prolonged neutropenia and who are at high risk of developing invasive fungal infections; Hematopoietic stem cell transplant (HSCT) recipients who are undergoing high-dose immunosuppressive therapy for graft versus host disease and who are at high risk of developing invasive fungal infections. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

3 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

CD380.26AGAP30.02UBE2D40.01

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1via CD380.26

Reference

Authoritative identity, definition & identifiers.

Synonyms

acute leukaemia (disease), acute leukemia (disease), leukemia, acute, X-linked, stem cell leukaemia, stem cell leukaemia (disease), stem cell leukemia, stem cell leukemia (disease)

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.