Clinical trial · Interventional
05-001: Treatment of Acute Lymphoblastic Leukemia in Children
Treatment of Acute Lymphoblastic Leukemia in Children
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
RATIONALE: L-asparaginase is an important component of treatment for childhood acute lymphoblastic leukemia, but is also associated with notable side-effects, including hypersensitivity, pancreatitis, and thrombosis. We have previously reported that patients with acute lymphoblastic leukemia in whom asparaginase treatment was discontinued because of intolerable side-effects had survival outcomes that were inferior to those who received all or nearly all of their intended doses. Two bacterial sources of asparaginase exist: Escherichia coli (E coli) and Erwinia chrysanthemia (Erwinia). Generally, the E coli-derived enzyme has been used as front-line therapy and the Erwinia-derived preparation has been reserved for patients who develop hypersensitivity reactions. Pegylated E coli asparaginase (PEG-asparaginase) has a longer half-life and is potentially less immunogenic than native E coli L-asparaginase, and has been used as the initial asparaginase preparation in some pediatric acute lymphoblastic leukemia treatment regimens. PURPOSE: Although the pharmacokinetics of each of these asparaginase preparations: intravenous PEG-asparaginase (IV-PEG) and intramuscular native E coli L-asparaginase (IM-EC) have been well characterized, their relative efficacy and toxicity have not been studied extensively.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Drug/Agent Toxicity by Tissue/Organ | — | UNRESOLVED | — |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (16)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| asparaginase | Drug | Asparaginase | ALIAS |
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| cytarabine | Drug | Cytarabine | ALIAS |
| dexamethasone | Drug | Dexamethasone | ALIAS |
| dexrazoxane hydrochloride | Drug | — | UNRESOLVED |
| doxorubicin hydrochloride | Drug | Doxorubicin | ALIAS |
| etoposide | Drug | Etoposide | ALIAS |
| leucovorin calcium | Drug | Leucovorin | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- ACTIVE_COMPARATOR
- label
- Intramuscular native E coli L-asparaginase (IM-EC)
- description
- Patients in this arm were randomized to intramuscular native E coli L-asparaginase 25 000 IU/m2 weekly for 30 doses. Protocol therapy was comprised of 5 phases: Induction, Consolidation I, CNS, Consolidation II, Continuation and varied dependent on risk classification. Patients who achieved complete remission after induction were eligible for post-induction asparaginase randomization. Further details are provided in the study description section.
- interventionNames
- Drug: asparaginase
- Drug: cyclophosphamide
- Drug: cytarabine
- Drug: dexamethasone
- Drug: dexrazoxane hydrochloride
- Drug: doxorubicin hydrochloride
- Drug: etoposide
- Drug: leucovorin calcium
- Drug: mercaptopurine
- Drug: methotrexate
- Drug: methylprednisolone
- Drug: prednisolone
- Drug: therapeutic hydrocortisone
- Drug: vincristine sulfate
- Radiation: radiation therapy
- type
- EXPERIMENTAL
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Year
- Maximum age
- 18 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Diagnosis of acute lymphoblastic leukemia (ALL)
* No known mature B-cell ALL, defined by the presence of any of the following:
* Surface immunoglobulin
* L3 morphology
* t(8;14)(q24;q32)
* t(8;22)
* t(2;8)
* T-cell surface markers and t(8;14)(q24;q11) allowed
* No secondary ALL
PATIENT CHARACTERISTICS:
* No known HIV positivity
* Not pregnant or nursing
* Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
* No prior therapy except steroids of ≤ 1 week in duration and/or emergent radiation therapy to the mediastinum
* Patients treated with steroids within the past 7 days will not receive steroid prophase during study treatmentReferences
Publications (4)
- RESULTSilverman LB, Supko JG, Stevenson KE, Woodward C, Vrooman LM, Neuberg DS, Asselin BL, Athale UH, Clavell L, Cole PD, Kelly KM, Laverdiere C, Michon B, Schorin M, Schwartz CL, O'Brien JE, Cohen HJ, Sallan SE. Intravenous PEG-asparaginase during remission induction in children and adolescents with newly diagnosed acute lymphoblastic leukemia. Blood. 2010 Feb 18;115(7):1351-3. doi: 10.1182/blood-2009-09-245951. Epub 2009 Dec 10. PMID 20007809
- RESULTPlace AE, Stevenson KE, Vrooman LM, Harris MH, Hunt SK, O'Brien JE, Supko JG, Asselin BL, Athale UH, Clavell LA, Cole PD, Kelly KM, Laverdiere C, Leclerc JM, Michon B, Schorin MA, Welch JJ, Lipshultz SE, Kutok JL, Blonquist TM, Neuberg DS, Sallan SE, Silverman LB. Intravenous pegylated asparaginase versus intramuscular native Escherichia coli L-asparaginase in newly diagnosed childhood acute lymphoblastic leukaemia (DFCI 05-001): a randomised, open-label phase 3 trial. Lancet Oncol. 2015 Dec;16(16):1677-90. doi: 10.1016/S1470-2045(15)00363-0. Epub 2015 Nov 6. PMID 26549586
- DERIVEDVrooman LM, Blonquist TM, Harris MH, Stevenson KE, Place AE, Hunt SK, O'Brien JE, Asselin BL, Athale UH, Clavell LA, Cole PD, Kelly KM, Laverdiere C, Leclerc JM, Michon B, Schorin MA, Sulis ML, Welch JJG, Neuberg DS, Sallan SE, Silverman LB. Refining risk classification in childhood B acute lymphoblastic leukemia: results of DFCI ALL Consortium Protocol 05-001. Blood Adv. 2018 Jun 26;2(12):1449-1458. doi: 10.1182/bloodadvances.2018016584. PMID 29941458
- DERIVEDKahn JM, Cole PD, Blonquist TM, Stevenson K, Jin Z, Barrera S, Davila R, Roberts E, Neuberg DS, Athale UH, Clavell LA, Laverdiere C, Leclerc JM, Michon B, Schorin MA, Welch JJG, Sallan SE, Silverman LB, Kelly KM. An investigation of toxicities and survival in Hispanic children and adolescents with ALL: Results from the Dana-Farber Cancer Institute ALL Consortium protocol 05-001. Pediatr Blood Cancer. 2018 Mar;65(3):10.1002/pbc.26871. doi: 10.1002/pbc.26871. Epub 2017 Nov 1. PMID 29090520