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Home › Conditions › Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia

Part of Leukemia

19 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Acute Lymphoblastic Leukemia

103 trials tracked for Acute Lymphoblastic Leukemia: 22 in phase 3 or 4 and 16 with published results. The most-cited published study has 339 citations.

103Trials tracked
22Phase 3 & 4
0Recruiting
16With published results
Phase distribution
Phase 4 4 Phase 3 18 Phase 2 62 Phase 1 10 Other / NA 9
  1. Phase 3 Phase 3 Trial of Blinatumomab vs Standard Chemotherapy in Pediatric Subjects With HIgh-Risk (HR) First Relapse B-precursor Acute Lymphoblastic Leukemia (ALL) Completed · 339 cited
  2. Phase 3 Dasatinib and Combination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Lymphoblastic Leukemia Completed · 256 cited
  3. Phase 3 Combination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Lymphoblastic Leukemia Completed · 236 cited
  4. Phase 3 Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant Completed · 113 cited
  5. Phase 3 Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma Completed · 102 cited
  6. Phase 3 Total Therapy Study XVI for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia Completed · 90 cited
Show 44 more trials
  1. Phase 3 A Study Of Inotuzumab Ozogamicin Versus Investigator's Choice Of Chemotherapy In Patients With Relapsed Or Refractory Acute Lymphoblastic Leukemia Completed · 78 cited
  2. Phase 3 A Study of the Safety and Effectiveness of Apixaban in Preventing Blood Clots in Children With Leukemia Who Have a Central Venous Catheter and Are Treated With Asparaginase Completed · 53 cited
  3. Phase 3 Phase III B in Acute Lymphoblastic Leukemia Completed · 51 cited
  4. Phase 3 Calaspargase Pegol or Pegaspargase and Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed High-Risk Acute Lymphoblastic Leukemia Completed · 48 cited
  5. Phase 3 Combination Chemotherapy With or Without Lestaurtinib in Treating Younger Patients With Newly Diagnosed Acute Lymphoblastic Leukemia Completed · 37 cited
  6. Phase 3 An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL) Completed · 34 cited
  7. Phase 4 Vaccinating Children After Chemotherapy Completed · 25 cited
  8. Phase 4 A Study Of Two Inotuzumab Ozogamicin Doses in Relapsed/ Refractory Acute Lymphoblastic Leukemia Transplant Eligible Patients Completed · 17 cited
  9. Phase 3 Efficacy and Safety of the BiTE Antibody Blinatumomab in Chinese Adult Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (ALL) Completed · 11 cited
  10. Phase 3 Tac, Mini-MTX, MMF Versus Tac, MTX for GVHD Prevention Completed · 11 cited
  11. Phase 4 A Study of Inotuzumab Ozogamicin in Chinese Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia Completed
  12. Phase 4 Learning Impairments Among Survivors of Childhood Cancer Completed
  13. Phase 3 Therapy for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia Completed
  14. Phase 3 Dexamethasone Compared With Prednisone During Induction Therapy and Methotrexate With or Without Leucovorin During Maintenance Therapy in Treating Patients With Newly Diagnosed High-Risk Acute Lymphoblastic Leukemia Completed
  15. Phase 3 Methotrexate or Pentostatin for Graft-versus-host Disease Prophylaxis in Risk-adapted Allogeneic Bone Marrow Transplantation for Hematologic Malignancies Completed
  16. Phase 3 A Severity-Adapted Clinical Trial of Diminished Bone Mineral Density in Acute Lymphoblastic Leukemia Survivors Completed
  17. Phase 2 T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts Completed
  18. Phase 2 Clinical Study With Blinatumomab in Pediatric and Adolescent Patients With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia Completed
  19. Phase 2 Safety and Efficacy of BL-8040 for the Mobilization of Donor Hematopoietic Stem Cells and Allogeneic Transplantation in Patients With Advanced Hematological Malignancies Completed
  20. Phase 2 Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies Completed
  21. Phase 2 Combination Chemotherapy and Dasatinib in Treating Participants With Philadelphia Positive or BCR-ABL Positive Acute Lymphoblastic Leukemia. Completed
  22. Phase 2 Clofarabine-cyclophosphamide as Salvage Therapy for Refractory and Relapsed Acute Lymphoblastic Leukemia (ALL) Adults Completed
  23. Phase 2 Tocilizumab for the Prevention of Graft Versus Host Disease After Cord Blood Transplantation Completed
  24. Phase 2 Treatment of Adult Ph+ LAL With BMS-354825 Completed
  25. Phase 2 Study Evaluating AMD3100 for Transplantation of Sibling Donor Stem Cells in Patients With Hematological Malignancies Completed
  26. Phase 2 Safety and Efficacy of Donor T-lymphocytes Depleted ex Vivo of Host Alloreactive T-cells (ATIR) in Patients With a Hematologic Malignancy Who Received a Hematopoietic Stem Cell Transplantation From a Haploidentical Donor Completed
  27. Phase 2 Pilot of Abatacept-based Immunosuppression for Prevention of Acute GvHD During Unrelated Donor HCT Completed
  28. Phase 2 ALL Adult Consortium Trial: Adult ALL Trial Completed
  29. Phase 2 Treatment of Older Adults With Acute Lymphoblastic Leukemia Completed
  30. Phase 2 Vyxeos(CPX-351) in Adults w R/R Acute Lymphoblastic Leukemia Completed
  31. Phase 2 Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation Completed
  32. Phase 2 Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients With Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transplant Completed
  33. Phase 2 Therapy for Pediatric Relapsed or Refractory Acute Lymphoblastic Leukemia Completed
  34. Phase 2 Methods to Enhance the Safety and Effectiveness of Stem Cell Transplants Completed
  35. Phase 2 A Roll-over Study to Provide Continued Treatment With Lyophilized Pegaspargase (S95014) in Pediatric Patients With Acute Lymphoblastic Leukemia (ALL) Completed
  36. Phase 2 Sitagliptin Umbilical Cord Blood Transplant Study Completed
  37. Phase 2 Study of Augmented Hyper-CVAD in Acute Lymphoblastic Leukemia Salvage Completed
  38. Phase 2 Administration of GRASPA (Suspension of Erythrocytes Encapsulating L-asparaginase) in Elderly Patients With First Line Acute Lymphoblastic Leukemia Completed
  39. Phase 2 Combination Chemotherapy and Ofatumumab in Treating Patients With Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma Completed
  40. Phase 2 Ibrutinib in Preventing Acute Leukemia in Patients After Reduced-Intensity Conditioning and Stem Cell Transplant Completed
  41. Phase 2 Nebulized Amphotericin B Lipid Complex in Invasive Pulmonary Aspergillosis in Paediatric Patients With Acute Leukaemia Completed
  42. Phase 2 A Study in Adults With Untreated Acute Lymphoblastic Leukemia Completed
  43. Phase 2 Clofarabine and Cyclophosphamide Combination in Acute Lymphoblastic Leukemia Patients Completed
  44. Phase 2 CMC-544 in Relapsed Refractory Acute Lymphoblastic Leukemia (ALL) Completed

Showing the 50 most-cited and recently-updated of 103 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Acute Lymphoblastic Leukemia: what the trials found

Several targeted and chimeric antigen receptor (CAR) therapies have demonstrated efficacy in patients with Acute Lymphoblastic Leukemia. Blinatumomab showed significant improvements in event-free survival (EFS) 7 and high rates of hematological response, including CR/CRh* or CRi, during the first two treatment cycles 20. Inotuzumab ozogamicin demonstrated a significantly higher percentage of patients achieving hematologic remission compared to standard treatments 13, with 81.8% to 47.7% reaching CR or CRi depending on the cohort 5. Additionally, Calaspargase Pegol-mknl was associated with high rates of complete remission at the end of induction (92.4% to 97.6%) and favorable event-free survival 16.

Asparaginase remains a cornerstone of treatment; studies show that its use, particularly in combination with other agents like Dasatinib, significantly impacts minimal residual disease (MRD) status at the end of induction 8. High-dose versus conventional dose PEG-asparaginase showed comparable rates of continuous complete remission 12. Additionally, while some trials investigated the impact of Lestaurtinib on MLL-R infants treated with combination chemotherapy and Asparaginase, no significant difference in event-free survival was observed between treatment arms 17.

Clinical assessments of supportive care and complications indicate that Cyclophosphamide is a standard component of therapy [9, 21]. Research into the impact of various agents on complications shows that tacrolimus may reduce the incidence of severe mucositis and acute GVHD compared to other regimens 19, while certain interventions like inotuzumab ozogamicin were associated with low rates of significant laboratory test shifts 4.

Research regarding the physiological impacts of the disease has identified significant differences in brain white matter volume between patients with Acute Lymphoblastic Leukemia and sibling controls, as well as between those with ALL and those with other types of brain tumors 6.

Recent results — preliminary, needs further review

  • Siltuximab was evaluated in a Phase 2 trial (N=24) for the treatment of this condition 1.
  • Cyclosporine was evaluated in a Phase 2 study where the median time to onset of Grade II-IV aGVHD by Day 100 was 34 days 2.

For the clinician treating this condition

  • Blinatumomab and Inotuzumab ozogamicin are established options for achieving high rates of hematologic response and improved event-free survival.
  • Asparaginase remains a core component of treatment, with significant implications for minimal residual disease (MRD) status.
  • Calaspargase Pegol-mknl is associated with high induction remission rates and favorable event-free survival.

AI synthesis of 13 cited trials, updated Jun 26, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
6-MP mini-tablets show bioequivalence to reference tablets in fasted children with ALL
Oncology RCT
6-MP mini-tablets show bioequivalence to reference tablets in fasted children with ALL A Tiny New Pill Could Transform Cancer Care for Children
Two open-label, randomized, crossover trials in children with acute lymphoblastic leukemia compared a novel 6-MP mini-tablet to a reference …
A tiny new pill for childhood leukemia works just as well as the standard version, but eating food before taking it dramatically reduces its…
Apr 10, 2026
Red blood cell parameters predict sepsis in children with ALL and chemotherapy-induced neutropenia
Pediatrics Cohort
Red blood cell parameters predict sepsis in children with ALL and chemotherapy-induced neutropenia A Simple Blood Test Could Warn of Sepsis in Children on Chemotherapy
This retrospective cohort study evaluated red blood cell parameters in children with acute lymphoblastic leukemia who developed chemotherapy…
Doctors may have found a faster, simpler way to spot a deadly infection in children undergoing cancer treatment.
Frontiers Apr 10, 2026
Meta-analysis compares CAR T-cell constructs in 1540 relapsed/refractory B-ALL patients
Hematology Meta-analysis
Meta-analysis compares CAR T-cell constructs in 1540 relapsed/refractory B-ALL patients Which CAR T-cell therapy works best for relapsed leukemia?
A systematic review and meta-analysis of 40 clinical trials involving 1540 R/R B-ALL patients found pooled complete remission rates of 83.4%…
For relapsed leukemia, CAR T-cell therapies using 4-1BB components targeting CD19 achieved the highest remission rates with fewer side effec…
Apr 8, 2026
In children with Ph+ ALL, TKI-based therapy reduced HSCT use while maintaining comparable outcomes versus Ph+ALL04
Hematology Phase II
In children with Ph+ ALL, TKI-based therapy reduced HSCT use while maintaining comparable outcomes versus Ph+ALL04 Reduced transplant use showed similar outcomes in children with Philadelphia chromosome-positive leukemia
This single-arm Phase 2 trial evaluated chemotherapy with tyrosine kinase inhibitors (TKIs) guided by minimal residual disease in 41 eligibl…
Children with a specific leukemia type achieved similar survival rates without stem cell transplants, offering a less intensive treatment op…
Apr 8, 2026
Systematic review shows increased fat mass and decreased fat-free mass in children with acute lymphoblastic leukemia during treatment
Hematology Meta-analysis
Systematic review shows increased fat mass and decreased fat-free mass in children with acute lymphoblastic leukemia during treatment ALL Treatment Linked to Changes in Body Fat and Muscle in Young Patients
This systematic review and meta-analysis of 13 studies examined body composition changes in children and young people aged 0-21 years with a…
ALL treatment causes early fat gain and muscle loss in young patients that often persists long after therapy ends.
Apr 6, 2026
AI tool predicts acute leukemia subtypes with AUROC 0.94 for AML, 0.98 for promyelocytic, 0.84 for ALL in international cohort
Oncology
AI tool predicts acute leukemia subtypes with AUROC 0.94 for AML, 0.98 for promyelocytic, 0.84 for ALL in international cohort Can a simple blood test help diagnose leukemia? An AI tool shows promise but still needs work
An AI tool was tested on a retrospective, international cohort of 6206 leukemia patients from 20 centers to predict acute leukemia subtypes …
An AI tool can spot leukemia from standard blood tests but must refuse to guess for most patients until doctors refine it to help more peopl…
Mar 30, 2026
Phase I/II trial tests IMRT with chemotherapy before transplant for relapsed/refractory ALL/AML
Hematology Phase II
Phase I/II trial tests IMRT with chemotherapy before transplant for relapsed/refractory ALL/AML Can a new radiation technique make stem cell transplants safer for leukemia?
A phase I/II trial record describes a study of intensity-modulated radiation therapy with etoposide and cyclophosphamide followed by allogen…
Doctors are testing a precise radiation technique to protect healthy organs while preparing patients for life-saving stem cell transplants.
CT.gov Mar 30, 2026

Research across Leukemia

Related studies from across the Leukemia family.

MRG mutations correlate with worse overall survival and lower remission in NPM1-mutated AML
Hematology Meta-analysis
MRG mutations correlate with worse overall survival and lower remission in NPM1-mutated AML Specific gene mutations linked to worse outcomes in certain leukemia
This meta-analysis of 4,363 patients found that MRG mutations (such as ASXL1, BCOR, and EZH2) are associated with inferior overall survival …
New data shows that specific gene mutations can predict poorer survival and lower remission rates for patients with a specific type of leuke…
Sep 1, 2026
Treosulfan-fludarabine improves 24-month event-free survival to 65% versus 53% in AML patients
Hematology RCT
Treosulfan-fludarabine improves 24-month event-free survival to 65% versus 53% in AML patients Treosulfan Shows Better Outcomes for Patients with Acute Myeloid Leukemia
This Phase 3 randomized trial subgroup analysis of 352 patients with AML undergoing alloHCT found treosulfan-fludarabine superior to reduced…
A new analysis of a Phase 3 trial suggests treosulfan may offer better survival rates and fewer complications for patients with acute myeloi…
Sep 1, 2026
Dasatinib 50 mg maintains efficacy while reducing pleural effusion and thrombocytopenia in CP-CML
Hematology Meta-analysis
Dasatinib 50 mg maintains efficacy while reducing pleural effusion and thrombocytopenia in CP-CML Lower Dasatinib Dose May Improve Tolerability for Leukemia Patients
This meta-analysis of 7 nonoverlapping reports compared dasatinib 50 mg and 100 mg in adults with chronic-phase chronic myeloid leukemia. Th…
A lower daily dose of dasatinib may offer similar effectiveness with fewer side effects for adults with chronic-phase chronic myeloid leukem…
Sep 1, 2026
6-month IS value predicts MR4.5 attainment and TKI discontinuation eligibility in CML patients
Hematology Phase III
6-month IS value predicts MR4.5 attainment and TKI discontinuation eligibility in CML patients Trial Shows Early Markers Predict Success in Chronic Myeloid Leukemia
This observational study of 431 patients with chronic myeloid leukemia (CML) on second-generation TKIs found that the 6-month IS value is a …
New data from a Phase 3 trial shows that early markers can help predict which patients with chronic myeloid leukemia may reach specific trea…
Aug 25, 2026
Pirtobrutinib Combined with Venetoclax and Rituximab Improves Progression Free Survival
Oncology RCT
Pirtobrutinib Combined with Venetoclax and Rituximab Improves Progression Free Survival Trial shows pirtobrutinib improves survival for chronic lymphocytic leukemia
Adding pirtobrutinib to a venetoclax-rituximab regimen significantly extends progression-free survival in patients with chronic lymphocytic …
A new combination treatment involving pirtobrutinib may help patients with chronic lymphocytic leukemia live longer without their cancer pro…
Aug 9, 2026
Deep learning for leukemia diagnosis faces fragmented research and limited multimodal integration in current literature
Hematology Sys. Review
Deep learning for leukemia diagnosis faces fragmented research and limited multimodal integration in current literature AI for leukemia diagnosis still fragmented, review finds
This scoping review evaluates deep learning techniques for leukemia detection across morphological, immunophenotyping, and molecular modalit…
A new review shows AI tools for leukemia diagnosis are promising but fragmented, with few using multiple data types.
Frontiers Aug 8, 2026
Leukapheresis not clearly linked to lower 30-day mortality in AML with hyperleukocytosis
Hematology Meta-analysis
Leukapheresis not clearly linked to lower 30-day mortality in AML with hyperleukocytosis Leukapheresis Shows No Clear Early Death Benefit in AML
This meta-analysis of 1406 adults with AML and hyperleukocytosis found that leukapheresis was not clearly associated with lower 30-day morta…
A review of 1406 AML patients found leukapheresis did not clearly lower 30-day death risk. Evidence is weak.
Aug 3, 2026
AML infiltration of bile duct identified as primary cause of acute pancreatitis in case report
Rheumatology Sys. Review
AML infiltration of bile duct identified as primary cause of acute pancreatitis in case report Leukemia Cells Found as Cause of Severe Pancreatitis
This case report and literature review describes a 34-year-old man with acute pancreatitis and type 2 diabetes, where acute myeloid leukemia…
A rare case shows how a specific type of leukemia can infiltrate the pancreas and cause severe inflammation in patients with diabetes.
Frontiers Aug 3, 2026
TKI plus chemotherapy achieves deep molecular remission in Ph+ leukemia case with diagnostic framework
Allergy & Immunology Meta-analysis
TKI plus chemotherapy achieves deep molecular remission in Ph+ leukemia case with diagnostic framework One patient's leukemia remission offers clues for rare diagnosis
This systematic review with a case report describes a patient with Philadelphia chromosome-positive leukemia who achieved complete remission…
A patient with a rare leukemia achieved deep remission for 35 months. The case highlights how to tell apart two similar-looking blood cancer…
Frontiers Jul 29, 2026
Imatinib achieves complete cytogenetic response in a pediatric patient with suspected chronic myeloid leukemia
Pediatrics Sys. Review
Imatinib achieves complete cytogenetic response in a pediatric patient with suspected chronic myeloid leukemia Imatinib works for CML in child with high platelets
This case report describes a pediatric patient with isolated thrombocytosis who achieved a complete cytogenetic response to imatinib after f…
A child with high platelets and no response to standard therapy was found to have CML and responded to imatinib.
Frontiers Jul 21, 2026
Venetoclax plus intensive chemo yields 63.79% ORR in relapsed/refractory AML meta-analysis
Hematology Meta-analysis
Venetoclax plus intensive chemo yields 63.79% ORR in relapsed/refractory AML meta-analysis Venetoclax combo shows promise for hard-to-treat leukemia
A meta-analysis of 235 patients with relapsed/refractory AML found that venetoclax combined with intensive chemotherapy achieved a pooled ov…
For patients with relapsed AML, venetoclax plus intensive chemo led to a 64% response rate in a new analysis.
Jul 1, 2026
GSTO1 inhibition induces apoptosis in AML cells while LMRS model stratifies patient risk
Allergy & Immunology Guideline
GSTO1 inhibition induces apoptosis in AML cells while LMRS model stratifies patient risk New lipid metabolism markers help predict outcomes for leukemia patients
This guideline synthesizes in vitro and observational data regarding lipid metabolism in acute myeloid leukemia (AML). The findings identify…
Researchers identified a new way to group and predict survival for patients with acute myeloid leukemia using lipid metabolism patterns.
Frontiers Jun 30, 2026

Questions about Acute Lymphoblastic Leukemia

Do factors like age or where a child lives change survival chances for Acute Lymphoblastic Leukemia?

Yes, age and geographic location affect survival in pediatric ALL; younger children and those in high-income countries generally have better outcomes.

Full answer →
Does taking fluoroquinolone medicine help reduce high fevers in children with Acute Lymphoblastic Leukemia?

Fluoroquinolone prophylaxis reduces febrile neutropenia (fever with low white blood cells) in children with ALL, but it is not a treatment for high fevers; it is a preventive measure given during chemotherapy.

Full answer →