Acute Lymphoblastic Leukemia, PediatricPhase 3NCT03020030
Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents
A Phase 3 study testing Pegaspargase, Erwinia asparaginase and Cyclophosphamide in people with Acute Lymphoblastic Leukemia, Pediatric, run by Dana-Farber Cancer Institute at 9 sites in 2 countries.
Trial summary
Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children. The cancer comes from a cell in the blood called a lymphocyte. Normal lymphocytes are produced in the bone marrow (along with other blood cells) and help fight infections. In ALL, the cancerous lymphocytes are called lymphoblasts. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Acute Lymphoblastic Leukemia, Pediatric
- What is being tested?
- Pegaspargase, Erwinia asparaginase and Cyclophosphamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 560 participants
- Where
- 9 sitesin 2 countries
- Study dates
- 3 Mar 2017to Nov 2034
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Confirmed diagnosis of acute lymphoblastic leukemia. Diagnosis should be made by bone marrow aspirate or biopsy demonstrating ≥ 25% involvement by lymphoblasts, with flow cytometry or immunohistochemistry confirming B-precursor or T-ALL phenotype.
- -- For patients with circulating blasts in the peripheral blood, flow cytometry confirmation of B-ALL or T-ALL phenotype is sufficient for registration onto the study. Bone marrow aspirate and/or biopsy should be performed as soon as feasible, preferably prior to the initiation of any therapy.
- Prior Therapy: No prior therapy is allowed except for the following:
- Corticosteroids: Short courses of corticosteroid (defined as ≤ 7 days of corticosteroids within the 4-weeks preceding registration) are allowed prior to registration.
- --- Participants who have been on corticosteroids chronically (defined as more than 7 days of corticosteroids within the 4-weeks preceding registration or more than 28 days of corticosteroids over the preceding 6 months) are not eligible.
- IT cytarabine: A single dose of intrathecal cytarabine (at the time of the diagnostic lumbar puncture) is allowed prior to registration. If patient has received IT cytarabine prior to registration, Day 1 IT cytarabine should not be administered.
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- Emergent Radiation Therapy: Emergent radiation to the mediastinum or other life-threatening masses is allowed prior to registration.
- Age: 365 days to < 22 years
- Direct bilirubin < 1.4 mg/dL (23.9 micromoles/L).
- Ability of parent or guardian to understand and the willingness to sign a written informed consent document.
Who cannot join
- Mature B-cell (Burkitt's) ALL (defined by the presence of surface immunoglobulin and/or the t(8;14)(q24;q32), t(8;22), or t(2;8) translocation and/or c-myc-gene rearrangement).
- World Health Organization diagnostic criteria of mixed phenotype acute leukemia (MPAL) or leukemia of ambiguous lineage
- Any chemotherapy or radiotherapy for previous malignancy are not eligible.
- Treatment in past with any anti-neoplastic agent, including methotrexate, 6-mercaptopurine, 6-thioguanine, vincristine cyclophosphamide, cytarabine (except for IT cytarabine), or any anthracycline, for any reason (eg, rheumatologic or autoimmune condition).
- Currently receiving any investigational agents.
- Known HIV-positivity
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- Uncontrolled intercurrent illness including, but not limited to ongoing infection with vital sign instability (hypotension, respiratory insufficiency), life-threatening acute tumor lysis syndrome (eg, with renal failure), symptomatic congestive heart failure, cardiac arrhythmia, intracranial or other uncontrolled bleeding, or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant women are excluded from this study because many of the agents used on this protocol have potential for teratogenic and/or abortifacient effects. Because there is an unknown but potential risk of adverse events in nursing infants secondary to treatment of the mother with these chemotherapy agents, breastfeeding should be discontinued if the mother is enrolled.
- History of a previous malignancy. Exception: Individuals with a previous malignancy treated with surgery only (no chemotherapy or radiotherapy) more than 5 years prior to registration may be enrolled.
Trial locations and contacts
9 sites in 2 countries.
9 sites in 2 countries
United States7 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Roswell Park Cancer Institute
Buffalo, New York, 14263
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903
Columbia University Medical Center, Morgan Stanley Children's Hospital of New York-Presbyterian
New York, New York, 10032
Hasbro Children's Hospital / Rhode Island Hospital
Providence, Rhode Island, 02903
Montefiore Medical Center
The Bronx, New York, 10467
Canada2 sites
Hospital Sainte Justine, University of Montreal
Montreal, Quebec
Centre Hospitalier U. de Quebec
Québec, Quebec
About this study
Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children. The cancer comes from a cell in the blood called a lymphocyte. Normal lymphocytes are produced in the bone marrow (along with other blood cells) and help fight infections. In ALL, the cancerous lymphocytes are called lymphoblasts. They do not help fight infection and crowd out the normal blood cells in the bone marrow so that the body cannot make enough normal blood cells. ALL is always fatal if it is not treated. With current treatments, most children and adolescents with this disease will be cured.
The standard treatment for ALL involves about 2 years of chemotherapy. The drugs that are used, and the doses of the drugs, are similar but not identical for all children and adolescents with ALL. Some children and adolescents receive stronger treatment, especially during the first several months. A number of factors are used to decide how strong the treatment should be to give the best chance for cure. These factors are called "risk factors". This trial is studying the use of a new, updated set of risk factors to decide how strong the treatment will be. The study also will test a new way of dosing a chemotherapy drug called pegaspargase (which is part of the standard treatment for ALL) based on checking levels of the drug in the blood and adjusting the dose based on the levels.
Read the detailed description
There are a standard set of risk factors which are used to decide how strong treatment should be for a child with ALL. These risk factors include the child's age when the leukemia is diagnosed, how high the white blood cell count (WBC) is in the blood, whether or not leukemia cells are seen in the spinal fluid (referred to as Central Nervous System or CNS status), and whether or not the leukemia has certain abnormalities in their chromosomes (genetic material in the cell). Another risk factor is the amount of leukemia in the marrow that can be measured by a special laboratory test called "MRD" (Minimal Residual Disease) after the first month of treatment.
Over the last several years, new factors have been identified which help predict how well a child's leukemia may respond to treatment. These new risk factors include additional abnormalities in the genes of the leukemia cell, as well the amount of leukemia (MRD level) at second time point (about 2-3 months after starting treatment).
In this trial, the investigators will use the new risk factors along with old risk factors to decide how strong the treatment will be. The goal is to better identify those participants who might benefit from stronger treatment in order to improve their chance for cure. The investigators also hope to better identify participants who have a high chance of being cured with standard treatment in order to reduce their chance of side effects while maintaining the chance of cure.
This trial also aims to study the dosing of a drug called pegaspargase. Pegaspargase is a chemotherapy drug that is an important part of ALL treatment but it is also can cause many side effects. With the standard dose of pegaspargase, levels of the drug in the blood are higher than may be necessary to effectively treat leukemia.
On this research study, the investigators will be comparing the standard dose of pegaspargase with a new way of dosing the drug based on levels of the drug that we can measure in the blood. With the new way of doing, treatment will begin with a lower dose. If the levels are high, the dose will be decreased one more time; however, if at any time the levels are too low, dosing will be switched back up to the standard dose. The goal of this research study is to learn whether this new way of dosing (starting at a lower dose and changing the dose based on drug levels in the blood) will decrease side effects but still be as effective as the standard dosing of the drug.
- Conditions
- Acute Lymphoblastic Leukemia, Pediatric
- Being tested
- Pegaspargase, Erwinia asparaginase, Cyclophosphamide, CYTARABINE, DASATINIB
- Keywords
- acute lymphoblastic leukemia
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegaspargaseDrug
Arm A: Standard/Fixed Dose Pegaspargase (2500 IU/m2 every 2 weeks) Arm B: Reduced Dose (PK-adjusted) Pegaspargase (Starting Dose: 2000 IU/m2) Arm X: Directly Assigned Standard Dose (2500 IU/m2): For all VHR and patients who decline randomization
Erwinia asparaginaseDrug
Also called ERWINAZE®, ERWINIA CHRYSANTHEMI, ERWINASE®
Only for patients with Pegaspargase allergy or silent inactivation.
CyclophosphamideDrug
Also called CYTOXAN
Standard of Care
CYTARABINEDrug
Also called CYTOSINE ARABINOSIDE, ARA-C, CYTOSAR®
Standard of Care
DASATINIBDrug
Standard of Care
DEXAMETHASONEDrug
Also called DECADRON®, HEXADROL®,, DEXONE®,, DEXAMETH®
Standard of Care
DexrazoxaneDrug
Also called Zinecard
Standard of Care
DoxorubicinDrug
Also called ADRIAMYCIN®
Standard of Care
ETOPOSIDEDrug
Also called VePesid
Standard of Care
HYDROCORTISONEDrug
Standard of Care
Standard of Care
MERCAPTOPURINEDrug
Also called 6-MP
Standard of Care
METHOTREXATEDrug
Standard of Care
NELARABINEDrug
Standard of Care
VincristineDrug
Also called Oncovin
Standard of Care
| Study group | Role | What they receive |
|---|---|---|
| Initial Low Risk (Initial LR) | Other | Meets all the following criteria: B-ALL, Age 1-<15 years, WBC < 50,000/microliter, CNS-1 or CNS-2, no BCR-ABL1, no iAMP21, and no VHR characteristics.
Treated with Induction IA (vincristine, dexamethasone, pegaspargase), Induction IB (cyclophosphamide, cytarabine, mercaptopurine), Consolidation IA (vincristine, high-dose methotrexate + leucovorin, mercaptopurine). IT chemotherapy in all phases. Final risk group assigned by end of Consolidation IA. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase; Drug: Cyclophosphamide; Drug: CYTARABINE; Drug: DEXAMETHASONE; Drug: HYDROCORTISONE; Drug: LEUCOVORIN CALCIUM; Drug: MERCAPTOPURINE; Drug: METHOTREXATE; Drug: Vincristine |
| Initial High Risk (Initial HR) | Other | Meets at least one of the following criteria: Age >=15 years, WBC >=50,000/microliter, CNS-3, T-ALL, iAMP21, BCR-ABL1
And: No VHR characteristics
Treated with Induction IA (vincristine, dexamethasone, pegaspargase, doxorubicin + dexrazoxane), Induction IB (cyclophosphamide, cytarabine, mercaptopurine), Consolidation IA (vincristine, high-dose methotrexate + leucovorin, mercaptopurine). IT chemotherapy in all phases. Final risk group assigned by end of Consolidation IA. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase; Drug: Cyclophosphamide; Drug: CYTARABINE; Drug: DEXAMETHASONE; Drug: Dexrazoxane; Drug: Doxorubicin; Drug: HYDROCORTISONE; Drug: LEUCOVORIN CALCIUM; Drug: MERCAPTOPURINE; Drug: METHOTREXATE; Drug: Vincristine |
| Initial Very High Risk (Initial VHR) | Other | Any of the following are present: IKZF1 deletion, MLL (KMT2A) rearrangement, low hypodiploidy, t(17;19)
Treated with Induction IA (vincristine, dexamethasone, pegaspargase, doxorubicin + dexrazoxane), Induction IB (cyclophosphamide, cytarabine, mercaptopurine), Consolidation IA (vincristine, high-dose methotrexate + leucovorin, mercaptopurine). IT chemotherapy in all phases. Final risk group assigned by end of Consolidation IA. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase; Drug: Cyclophosphamide; Drug: CYTARABINE; Drug: DASATINIB; Drug: DEXAMETHASONE; Drug: Dexrazoxane; Drug: Doxorubicin; Drug: ETOPOSIDE; Drug: HYDROCORTISONE; Drug: LEUCOVORIN CALCIUM; Drug: MERCAPTOPURINE; Drug: METHOTREXATE; Drug: NELARABINE; Drug: Vincristine |
| Final Low Risk (Final LR) | Other | Initial Low Risk and Low MRD (<0.0001) at first time point (Day 32)
Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows:
CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by randomization or direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, methotrexate, pegaspargase [by randomization or direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy).
All treatment completed 24 months from date of complete remission. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase; Drug: CYTARABINE; Drug: DEXAMETHASONE; Drug: HYDROCORTISONE; Drug: MERCAPTOPURINE; Drug: METHOTREXATE; Drug: Vincristine |
| Final Intermediate Risk (Final IR) | Other | Initial High Risk and Low MRD (<0.0001) at first time point (Day 32)
Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows:
CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by randomization or direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, doxorubicin + dexrazoxane, pegaspargase [by randomization or direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy).
All treatment completed 24 months from date of complete remission. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase; Drug: CYTARABINE; Drug: DEXAMETHASONE; Drug: Dexrazoxane; Drug: Doxorubicin; Drug: HYDROCORTISONE; Drug: MERCAPTOPURINE; Drug: METHOTREXATE; Drug: Vincristine |
| Final High Risk (Final HR) | Other | Initial Low Risk or Initial High Risk with High MRD (>=0.0001) at first time point (Day 32) but low MRD (<0.001) at second time point (week 10-12)
Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows:
CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by randomization or direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, doxorubicin + dexrazoxane, pegaspargase [by randomization or direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy).
All treatment completed 24 months from date of complete remission. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase; Drug: CYTARABINE; Drug: DEXAMETHASONE; Drug: Dexrazoxane; Drug: Doxorubicin; Drug: HYDROCORTISONE; Drug: MERCAPTOPURINE; Drug: METHOTREXATE; Drug: Vincristine |
| Final Very High Risk (Final VHR) | Other | Initial VHR or any patient with high MRD (>=0.001) at second time point (week 10-12)
Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows:
Consolidation IB/B-ALL (High-dose methotrexate + leucovorin, cyclophosphamide, etoposide, IT chemotherapy); Consolidation IB/T-ALL (nelararbine, cyclophosphamide, etoposide); Consolidation IC (High-dose cytarabine, etoposide, dexamethasone, pegaspargase [by direct assignment], IT chemotherapy); CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, doxorubicin + dexrazoxane, pegaspargase [by direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy).
Dasatinib administered daily during all phases to pts with ABL1-class fusions. All treatment completed 24 months from date of complete remission. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase; Drug: Cyclophosphamide; Drug: CYTARABINE; Drug: DASATINIB; Drug: DEXAMETHASONE; Drug: Dexrazoxane; Drug: Doxorubicin; Drug: ETOPOSIDE; Drug: HYDROCORTISONE; Drug: LEUCOVORIN CALCIUM; Drug: MERCAPTOPURINE; Drug: METHOTREXATE; Drug: NELARABINE; Drug: Vincristine |
| Fixed Dose Pegaspargase | Active comparator | Final LR, IR, HR patients who consent to randomization and are assigned to receive 15 doses of pegaspargase every 2-weeks at standard fixed-dose (2500 IU/m2/dose). Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase |
| Reduced Dose (PK-Adjusted) Pegaspargase | Experimental | Final LR, IR, HR patients who consent to randomization and are assigned to receive 15 doses of pegaspargase every 2-weeks beginning at a reduced dose (2000 IU/m2/dose); subsequent doses adjusted based on nadir serum asparaginase activity (NSAA) levels, with goal of maintaining NSAA between 0.4 and 1.0 IU/mL. Closed to Enrollment. Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase |
| Direct Assignment | Other | All VHR patients, and any Final LR, IR, HR patients who decline randomization: Assigned to receive standard dosing of pegaspargase (15 doses of pegaspargase every 2-weeks at standard fixed-dose; 2500 IU/m2/dose). Receives: Drug: Pegaspargase; Drug: Erwinia asparaginase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Remission Rate
Measured: After 1 month of treatment (Induction IA) for all participants, assessed at the end of first month of treatment in all participants through study completion (expected to take 4-5 years to accrue)
Event-Free Survival
Measured: From registration to the time of induction failure, relapse, death, or second malignancy, whichever came first, assessed up to 60 months.
Secondary outcomes (4)
Overall Survival
Measured: From registration to the time of death from any cause, assessed up to 60 months.
Disease Free Survival
Measured: From randomization or direct assignment (for participants who achieved a complete remission and were assigned a final risk group) to the time of relapse, death, or second malignancy, whichever came first, assessed up to 60 months.
Nadir Serum Asparaginase Activity (NSAA)
Proportion of patients receiving pegaspargase with NSAA >= 1.0 IU/mL
Measured: During post-induction therapy with 30-weeks of pegaspargase (15 doses), collected during the first 6 months of therapy for all participants through study completion (expected to take 4-5 years to accrue)
Non-allergic Asparaginase Toxicity
Frequency of non-allergic asparaginase-related toxicities, an average of 5 years.
Measured: During post-induction therapy with 30-weeks of pegaspargase (15 doses). collected during the first 6 months of therapy for all participants through study completion (expected to take 4-5 years to accrue)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2017 (estimated)
- Study start3 Mar 2017
- Primary completion30 Nov 2026 (estimated)
- Study completionNov 2034 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Acute Lymphoblastic Leukemia, PediatricStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 560 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Servier
- Responsible party
- Principal investigator, Melissa Burns, Dana-Farber Cancer Institute
- Investigators
- Melissa Burns, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03020030
- Sponsor's protocol ID
- 16-001
- Status last verified
- Jul 2026
- First submitted
- 6 Jan 2017
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Willekens J, Ramjan S, Sands SA, Park Y, K C N, Burns MA, Welch JJG, Kahn J, Kelly KM, Tran TH, Michon B, Gennarini L, Place A, Silverman LB, Cole PD. CSF metabolomic signature during therapy for childhood acute lymphoblastic leukemia predicts subsequent working memory impairment. Mol Med. 2025 Dec 30;32(1):12. doi: 10.1186/s10020-025-01414-z.
PubMed 41469545Park Y, K C N, Willekens J, Patel C, Savage BA, Lin H, Paneque A, Daly R, Thrope A, Burns MA, Welch JJG, Kahn JM, Kelly KM, Tran TH, Michon B, Gennarini L, Silverman LB, Sands SA, Cole PD. Treatment-Related Changes in Cerebrospinal Fluid Markers of Oxidative Stress and Neurodegeneration during Therapy for Childhood Acute Lymphoblastic Leukemia. Cancer Epidemiol Biomarkers Prev. 2025 Nov 3;34(11):2015-2024. doi: 10.1158/1055-9965.EPI-25-1058.
PubMed 40905823Ramjan S, Blair Thies M, Li Y, Thrope A, Silverman L, Welch JJG, Kahn J, Kelly KM, Tran TH, Michon B, Gennarini L, Bona K, Park Y, Cole PD, Sands SA. Baseline Neurocognitive Functioning Among Children Treated for Acute Lymphoblastic Leukemia (ALL): Dana Farber Cancer Institute ALL Consortium Study 16-001. Pediatr Blood Cancer. 2025 Aug;72(8):e31767. doi: 10.1002/pbc.31767. Epub 2025 May 13.
PubMed 40361276Tran TH, Langlois S, Meloche C, Caron M, Saint-Onge P, Rouette A, Bataille AR, Jimenez-Cortes C, Sontag T, Bittencourt H, Laverdiere C, Lavallee VP, Leclerc JM, Cole PD, Gennarini LM, Kahn JM, Kelly KM, Michon B, Santiago R, Stevenson KE, Welch JJG, Schroeder KM, Koch V, Cellot S, Silverman LB, Sinnett D. Whole-transcriptome analysis in acute lymphoblastic leukemia: a report from the DFCI ALL Consortium Protocol 16-001. Blood Adv. 2022 Feb 22;6(4):1329-1341. doi: 10.1182/bloodadvances.2021005634.
PubMed 34933343
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents
- ClinicalTrials.gov ID
- NCT03020030
- Acronym / sponsor ID
- 16-001
- Conditions
- Acute Lymphoblastic Leukemia, Pediatric
- Keywords
- acute lymphoblastic leukemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 560 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Servier
- Study start
- 3 Mar 2017 (actual)
- Primary completion
- 30 Nov 2026 (estimated)
- Study completion
- Nov 2034 (estimated)
- First posted
- 13 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children. The cancer comes from a cell in the blood called a lymphocyte. Normal lymphocytes are produced in the bone marrow (along with other blood cells) and help fight infections. In ALL, the cancerous lymphocytes are called lymphoblasts. They do not help fight infection and crowd out the normal blood cells in the bone marrow so that the body cannot make enough normal blood cells. ALL is always fatal if it is not treated. With current treatments, most children and adolescents with this disease will be cured.
The standard treatment for ALL involves about 2 years of chemotherapy. The drugs that are used, and the doses of the drugs, are similar but not identical for all children and adolescents with ALL. Some children and adolescents receive stronger treatment, especially during the first several months. A number of factors are used to decide how strong the treatment should be to give the best chance for cure. These factors are called "risk factors". This trial is studying the use of a new, updated set of risk factors to decide how strong the treatment will be. The study also will test a new way of dosing a chemotherapy drug called pegaspargase (which is part of the standard treatment for ALL) based on checking levels of the drug in the blood and adjusting the dose based on the levels.
Detailed description
There are a standard set of risk factors which are used to decide how strong treatment should be for a child with ALL. These risk factors include the child's age when the leukemia is diagnosed, how high the white blood cell count (WBC) is in the blood, whether or not leukemia cells are seen in the spinal fluid (referred to as Central Nervous System or CNS status), and whether or not the leukemia has certain abnormalities in their chromosomes (genetic material in the cell). Another risk factor is the amount of leukemia in the marrow that can be measured by a special laboratory test called "MRD" (Minimal Residual Disease) after the first month of treatment.
Over the last several years, new factors have been identified which help predict how well a child's leukemia may respond to treatment. These new risk factors include additional abnormalities in the genes of the leukemia cell, as well the amount of leukemia (MRD level) at second time point (about 2-3 months after starting treatment).
In this trial, the investigators will use the new risk factors along with old risk factors to decide how strong the treatment will be. The goal is to better identify those participants who might benefit from stronger treatment in order to improve their chance for cure. The investigators also hope to better identify participants who have a high chance of being cured with standard treatment in order to reduce their chance of side effects while maintaining the chance of cure.
This trial also aims to study the dosing of a drug called pegaspargase. Pegaspargase is a chemotherapy drug that is an important part of ALL treatment but it is also can cause many side effects. With the standard dose of pegaspargase, levels of the drug in the blood are higher than may be necessary to effectively treat leukemia.
On this research study, the investigators will be comparing the standard dose of pegaspargase with a new way of dosing the drug based on levels of the drug that we can measure in the blood. With the new way of doing, treatment will begin with a lower dose. If the levels are high, the dose will be decreased one more time; however, if at any time the levels are too low, dosing will be switched back up to the standard dose. The goal of this research study is to learn whether this new way of dosing (starting at a lower dose and changing the dose based on drug levels in the blood) will decrease side effects but still be as effective as the standard dosing of the drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 10
| Arm | Type | Receives |
|---|---|---|
| Initial Low Risk (Initial LR) | Other | Drug: Pegaspargase, Drug: Erwinia asparaginase, Drug: Cyclophosphamide, Drug: CYTARABINE, Drug: DEXAMETHASONE, Drug: HYDROCORTISONE, Drug: LEUCOVORIN CALCIUM, Drug: MERCAPTOPURINE, Drug: METHOTREXATE, Drug: VincristineMeets all the following criteria: B-ALL, Age 1-<15 years, WBC < 50,000/microliter, CNS-1 or CNS-2, no BCR-ABL1, no iAMP21, and no VHR characteristics. Treated with Induction IA (vincristine, dexamethasone, pegaspargase), Induction IB (cyclophosphamide, cytarabine, mercaptopurine), Consolidation IA (vincristine, high-dose methotrexate + leucovorin, mercaptopurine). IT chemotherapy in all phases. Final risk group assigned by end of Consolidation IA. |
| Initial High Risk (Initial HR) | Other | Drug: Pegaspargase, Drug: Erwinia asparaginase, Drug: Cyclophosphamide, Drug: CYTARABINE, Drug: DEXAMETHASONE, Drug: Dexrazoxane, Drug: Doxorubicin, Drug: HYDROCORTISONE, Drug: LEUCOVORIN CALCIUM, Drug: MERCAPTOPURINE, Drug: METHOTREXATE, Drug: VincristineMeets at least one of the following criteria: Age >=15 years, WBC >=50,000/microliter, CNS-3, T-ALL, iAMP21, BCR-ABL1 And: No VHR characteristics Treated with Induction IA (vincristine, dexamethasone, pegaspargase, doxorubicin + dexrazoxane), Induction IB (cyclophosphamide, cytarabine, mercaptopurine), Consolidation IA (vincristine, high-dose methotrexate + leucovorin, mercaptopurine). IT chemotherapy in all phases. Final risk group assigned by end of Consolidation IA. |
| Initial Very High Risk (Initial VHR) | Other | Drug: Pegaspargase, Drug: Erwinia asparaginase, Drug: Cyclophosphamide, Drug: CYTARABINE, Drug: DASATINIB, Drug: DEXAMETHASONE, Drug: Dexrazoxane, Drug: Doxorubicin, Drug: ETOPOSIDE, Drug: HYDROCORTISONE, Drug: LEUCOVORIN CALCIUM, Drug: MERCAPTOPURINE, Drug: METHOTREXATE, Drug: NELARABINE, Drug: VincristineAny of the following are present: IKZF1 deletion, MLL (KMT2A) rearrangement, low hypodiploidy, t(17;19) Treated with Induction IA (vincristine, dexamethasone, pegaspargase, doxorubicin + dexrazoxane), Induction IB (cyclophosphamide, cytarabine, mercaptopurine), Consolidation IA (vincristine, high-dose methotrexate + leucovorin, mercaptopurine). IT chemotherapy in all phases. Final risk group assigned by end of Consolidation IA. |
| Final Low Risk (Final LR) | Other | Drug: Pegaspargase, Drug: Erwinia asparaginase, Drug: CYTARABINE, Drug: DEXAMETHASONE, Drug: HYDROCORTISONE, Drug: MERCAPTOPURINE, Drug: METHOTREXATE, Drug: VincristineInitial Low Risk and Low MRD (<0.0001) at first time point (Day 32) Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows: CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by randomization or direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, methotrexate, pegaspargase [by randomization or direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy). All treatment completed 24 months from date of complete remission. |
| Final Intermediate Risk (Final IR) | Other | Drug: Pegaspargase, Drug: Erwinia asparaginase, Drug: CYTARABINE, Drug: DEXAMETHASONE, Drug: Dexrazoxane, Drug: Doxorubicin, Drug: HYDROCORTISONE, Drug: MERCAPTOPURINE, Drug: METHOTREXATE, Drug: VincristineInitial High Risk and Low MRD (<0.0001) at first time point (Day 32) Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows: CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by randomization or direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, doxorubicin + dexrazoxane, pegaspargase [by randomization or direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy). All treatment completed 24 months from date of complete remission. |
| Final High Risk (Final HR) | Other | Drug: Pegaspargase, Drug: Erwinia asparaginase, Drug: CYTARABINE, Drug: DEXAMETHASONE, Drug: Dexrazoxane, Drug: Doxorubicin, Drug: HYDROCORTISONE, Drug: MERCAPTOPURINE, Drug: METHOTREXATE, Drug: VincristineInitial Low Risk or Initial High Risk with High MRD (>=0.0001) at first time point (Day 32) but low MRD (<0.001) at second time point (week 10-12) Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows: CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by randomization or direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, doxorubicin + dexrazoxane, pegaspargase [by randomization or direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy). All treatment completed 24 months from date of complete remission. |
| Final Very High Risk (Final VHR) | Other | Drug: Pegaspargase, Drug: Erwinia asparaginase, Drug: Cyclophosphamide, Drug: CYTARABINE, Drug: DASATINIB, Drug: DEXAMETHASONE, Drug: Dexrazoxane, Drug: Doxorubicin, Drug: ETOPOSIDE, Drug: HYDROCORTISONE, Drug: LEUCOVORIN CALCIUM, Drug: MERCAPTOPURINE, Drug: METHOTREXATE, Drug: NELARABINE, Drug: VincristineInitial VHR or any patient with high MRD (>=0.001) at second time point (week 10-12) Final Risk Group assigned at end of Consolidation IA. Subsequent therapy as follows: Consolidation IB/B-ALL (High-dose methotrexate + leucovorin, cyclophosphamide, etoposide, IT chemotherapy); Consolidation IB/T-ALL (nelararbine, cyclophosphamide, etoposide); Consolidation IC (High-dose cytarabine, etoposide, dexamethasone, pegaspargase [by direct assignment], IT chemotherapy); CNS phase (vincristine, dexamethasone, mercaptopurine, pegaspargase [by direct assignment], IT chemotherapy); Consolidation II (vincristine, dexamethasone, mercaptopurine, doxorubicin + dexrazoxane, pegaspargase [by direct assignment], IT chemotherapy); Continuation (vincristine, dexamethasone, mercaptopurine, methotrexate, IT chemotherapy). Dasatinib administered daily during all phases to pts with ABL1-class fusions. All treatment completed 24 months from date of complete remission. |
| Fixed Dose Pegaspargase | Active comparator | Drug: Pegaspargase, Drug: Erwinia asparaginaseFinal LR, IR, HR patients who consent to randomization and are assigned to receive 15 doses of pegaspargase every 2-weeks at standard fixed-dose (2500 IU/m2/dose). |
| Reduced Dose (PK-Adjusted) Pegaspargase | Experimental | Drug: Pegaspargase, Drug: Erwinia asparaginaseFinal LR, IR, HR patients who consent to randomization and are assigned to receive 15 doses of pegaspargase every 2-weeks beginning at a reduced dose (2000 IU/m2/dose); subsequent doses adjusted based on nadir serum asparaginase activity (NSAA) levels, with goal of maintaining NSAA between 0.4 and 1.0 IU/mL. Closed to Enrollment. |
| Direct Assignment | Other | Drug: Pegaspargase, Drug: Erwinia asparaginaseAll VHR patients, and any Final LR, IR, HR patients who decline randomization: Assigned to receive standard dosing of pegaspargase (15 doses of pegaspargase every 2-weeks at standard fixed-dose; 2500 IU/m2/dose). |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Confirmed diagnosis of acute lymphoblastic leukemia. Diagnosis should be made by bone marrow aspirate or biopsy demonstrating ≥ 25% involvement by lymphoblasts, with flow cytometry or immunohistochemistry confirming B-precursor or T-ALL phenotype.
- -- For patients with circulating blasts in the peripheral blood, flow cytometry confirmation of B-ALL or T-ALL phenotype is sufficient for registration onto the study. Bone marrow aspirate and/or biopsy should be performed as soon as feasible, preferably prior to the initiation of any therapy.
- Prior Therapy: No prior therapy is allowed except for the following:
- --- Participants who have been on corticosteroids chronically (defined as more than 7 days of corticosteroids within the 4-weeks preceding registration or more than 28 days of corticosteroids over the preceding 6 months) are not eligible.
- Age: 365 days to < 22 years
Key exclusion criteria (9 listed)
- Mature B-cell (Burkitt's) ALL (defined by the presence of surface immunoglobulin and/or the t(8;14)(q24;q32), t(8;22), or t(2;8) translocation and/or c-myc-gene rearrangement).
- World Health Organization diagnostic criteria of mixed phenotype acute leukemia (MPAL) or leukemia of ambiguous lineage
- Any chemotherapy or radiotherapy for previous malignancy are not eligible.
- Treatment in past with any anti-neoplastic agent, including methotrexate, 6-mercaptopurine, 6-thioguanine, vincristine cyclophosphamide, cytarabine (except for IT cytarabine), or any anthracycline, for any reason (eg, rheumatologic or autoimmune condition).
- Currently receiving any investigational agents.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PegaspargaseArm A: Standard/Fixed Dose Pegaspargase (2500 IU/m2 every 2 weeks) Arm B: Reduced Dose (PK-adjusted) Pegaspargase (Starting Dose: 2000 IU/m2) Arm X: Directly Assigned Standard Dose (2500 IU/m2): For all VHR and patients who decline randomization | Drug | Direct Assignment, Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Fixed Dose Pegaspargase, Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR), Reduced Dose (PK-Adjusted) Pegaspargase |
| Erwinia asparaginaseAlso: ERWINAZE®, ERWINIA CHRYSANTHEMI, ERWINASE®Only for patients with Pegaspargase allergy or silent inactivation. | Drug | Direct Assignment, Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Fixed Dose Pegaspargase, Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR), Reduced Dose (PK-Adjusted) Pegaspargase |
| CyclophosphamideAlso: CYTOXANStandard of Care | Drug | Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
| CYTARABINEAlso: CYTOSINE ARABINOSIDE, ARA-C, CYTOSAR®Standard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
| DASATINIBStandard of Care | Drug | Final Very High Risk (Final VHR), Initial Very High Risk (Initial VHR) |
| DEXAMETHASONEAlso: DECADRON®, HEXADROL®,, DEXONE®,, DEXAMETH®Standard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
| DexrazoxaneAlso: ZinecardStandard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Very High Risk (Initial VHR) |
| DoxorubicinAlso: ADRIAMYCIN®Standard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Very High Risk (Initial VHR) |
| ETOPOSIDEAlso: VePesidStandard of Care | Drug | Final Very High Risk (Final VHR), Initial Very High Risk (Initial VHR) |
| HYDROCORTISONEStandard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
| LEUCOVORIN CALCIUMStandard of Care | Drug | Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
| MERCAPTOPURINEAlso: 6-MPStandard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
| METHOTREXATEStandard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
| NELARABINEStandard of Care | Drug | Final Very High Risk (Final VHR), Initial Very High Risk (Initial VHR) |
| VincristineAlso: OncovinStandard of Care | Drug | Final High Risk (Final HR), Final Intermediate Risk (Final IR), Final Low Risk (Final LR), Final Very High Risk (Final VHR), Initial High Risk (Initial HR), Initial Low Risk (Initial LR), Initial Very High Risk (Initial VHR) |
Primary outcome
Complete Remission Rate
Time frame: After 1 month of treatment (Induction IA) for all participants, assessed at the end of first month of treatment in all participants through study completion (expected to take 4-5 years to accrue)
Event-Free Survival
Time frame: From registration to the time of induction failure, relapse, death, or second malignancy, whichever came first, assessed up to 60 months.
4 secondary outcomes
Overall Survival
Time frame: From registration to the time of death from any cause, assessed up to 60 months.
Disease Free Survival
Time frame: From randomization or direct assignment (for participants who achieved a complete remission and were assigned a final risk group) to the time of relapse, death, or second malignancy, whichever came first, assessed up to 60 months.
Nadir Serum Asparaginase Activity (NSAA)
Time frame: During post-induction therapy with 30-weeks of pegaspargase (15 doses), collected during the first 6 months of therapy for all participants through study completion (expected to take 4-5 years to accrue)
Non-allergic Asparaginase Toxicity
Time frame: During post-induction therapy with 30-weeks of pegaspargase (15 doses). collected during the first 6 months of therapy for all participants through study completion (expected to take 4-5 years to accrue)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2026.
- Derived from this study: PubMed 41469545Willekens J, Ramjan S, Sands SA, Park Y, K C N, Burns MA, Welch JJG, Kahn J, Kelly KM, Tran TH, Michon B, Gennarini L, Place A, Silverman LB, Cole PD. CSF metabolomic signature during therapy for childhood acute lymphoblastic leukemia predicts subsequent working memory impairment. Mol Med. 2025 Dec 30;32(1):12. doi: 10.1186/s10020-025-01414-z.
- Derived from this study: PubMed 40905823Park Y, K C N, Willekens J, Patel C, Savage BA, Lin H, Paneque A, Daly R, Thrope A, Burns MA, Welch JJG, Kahn JM, Kelly KM, Tran TH, Michon B, Gennarini L, Silverman LB, Sands SA, Cole PD. Treatment-Related Changes in Cerebrospinal Fluid Markers of Oxidative Stress and Neurodegeneration during Therapy for Childhood Acute Lymphoblastic Leukemia. Cancer Epidemiol Biomarkers Prev. 2025 Nov 3;34(11):2015-2024. doi: 10.1158/1055-9965.EPI-25-1058.
- Derived from this study: PubMed 40361276Ramjan S, Blair Thies M, Li Y, Thrope A, Silverman L, Welch JJG, Kahn J, Kelly KM, Tran TH, Michon B, Gennarini L, Bona K, Park Y, Cole PD, Sands SA. Baseline Neurocognitive Functioning Among Children Treated for Acute Lymphoblastic Leukemia (ALL): Dana Farber Cancer Institute ALL Consortium Study 16-001. Pediatr Blood Cancer. 2025 Aug;72(8):e31767. doi: 10.1002/pbc.31767. Epub 2025 May 13.
- Derived from this study: PubMed 34933343Tran TH, Langlois S, Meloche C, Caron M, Saint-Onge P, Rouette A, Bataille AR, Jimenez-Cortes C, Sontag T, Bittencourt H, Laverdiere C, Lavallee VP, Leclerc JM, Cole PD, Gennarini LM, Kahn JM, Kelly KM, Michon B, Santiago R, Stevenson KE, Welch JJG, Schroeder KM, Koch V, Cellot S, Silverman LB, Sinnett D. Whole-transcriptome analysis in acute lymphoblastic leukemia: a report from the DFCI ALL Consortium Protocol 16-001. Blood Adv. 2022 Feb 22;6(4):1329-1341. doi: 10.1182/bloodadvances.2021005634.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Melissa Burns, MD principal investigator
Dana-Farber Cancer Institute
9 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pegaspargase, Erwinia asparaginase and Cyclophosphamide in people with Acute Lymphoblastic Leukemia, Pediatric, run by Dana-Farber Cancer Institute at 9 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 16 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Remission Rate (time frame: After 1 month of treatment (Induction IA) for all participants, assessed at the end of first month of treatment in all participants through study completion (expected to take 4-5 years to accrue)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03020030, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2026.