Trial · NCT00400946
05-001: Treatment of Acute Lymphoblastic Leukemia in Children
NCT00400946, Phase 3, Completed, Dana-Farber Cancer Institute; results first posted 2017-06-14
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ConductScience Library. 05-001: Treatment of Acute Lymphoblastic Leukemia in Children. Trials, record NCT00400946. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00400946
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 05-001: Treatment of Acute Lymphoblastic Leukemia in Children
- NCT number
- NCT00400946
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 800 participants, actual
- Lead sponsor
- Dana-Farber Cancer Institute
- Conditions
- Drug/Agent Toxicity by Tissue/Organ, Leukemia
- Interventions
asparaginase, cyclophosphamide, cytarabine, dexamethasone, dexrazoxane hydrochloride, doxorubicin hydrochloride, etoposide, leucovorin calcium, mercaptopurine, methotrexate, methylprednisolone, pegaspargase, prednisolone, therapeutic hydrocortisone, vincristine sulfate, radiation therapy
- First posted
- 2006-11-17
- Last updated
- 2026-07-30
- Results first posted
- 2017-06-14
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Asparaginase-Related Toxicity Rate | 30-week post-induction asparaginase treatment period | Asparaginase-related toxicity rate is defined as the percentage of patients who experience allergy (all grades), symptomatic pancreatitis (grade 2 or worse), thrombotic or bleeding complications requiring intervention (grade 2 or worse) with treatment attribution of possibly, probably or definite based on CTCAEv3. |
| Secondary | 5-Year Disease-Free Survival | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | Disease-free survival (DFS) in a landmark analysis is defined as the duration of time from asparaginase randomization (which occurred after patients achieved complete remission and were assigned to a final risk group) to documented relapse, death during remission or second malignant neoplasm. DFS is estimated based on the Kaplan-Meier method and 5-year DFS is the probability of patients remaining alive, relapse-free and without occurrence of second malignant neoplasm 5 years from asparaginase randomization. Disease relapse is defined as \>25% lymphoblasts identified morphologically in bone marrow aspirate/biopsy, or identification of lymphoblasts in marrow (any percentage) identified to be leukemic by flow cytometry, cytogenetics, FISH, immunohistochemistry, or other tests. Appearance of leukemic cells at any extramedullary site (a single, unequivocal lymphoblast in the CSF may qualify as CNS leukemia) also qualifies if confirmed by the PI. |
| Secondary | Post-Induction Nadir Serum Asparaginase Activity Level | Samples for nadir serum asparaginase activity analyses were obtained before doses administered at weeks 5, 11, 17, 23 and 29 of post-induction asparaginase treatment. | Nadir serum asparaginase activity (NSAA) levels were estimated based on established methods. |
| Secondary | Post-Induction Therapeutic Nadir Serum Asparaginase Activity Rate | Samples for nadir serum asparaginase activity analyses were obtained before doses administered at weeks 5, 11, 17, 23 and 29 of post-induction asparaginase treatment. | Nadir serum asparaginase activity (NSAA) levels were estimated based on established methods. Post-Induction therapeutic NSAA rate is defined as the percentage of patients achieving a NSAA level above 0.1 IU/mL ever during post-induction therapy. |
| Secondary | Induction Infection Toxicity Rate | Assessed daily during remission induction days 4-32. | Infection toxicity rate is defined as the percentage of patients who experience bacterial or fungal infection of grade 3 or higher with treatment attribution of possibly, probably or definite based on CTCAEv3 during remission induction phase of combination chemotherapy. |
| Secondary | Induction Serum Asparaginase Activity Level | Samples for serum asparaginase activity analyses were obtained days 4, 11, 18 and 25 post one-dose of IV-PEG on day 7 of the induction phase. | Serum asparaginase activity (NSAA) levels were estimated based on established methods. |
| Secondary | Induction Therapeutic Nadir Serum Asparaginase Activity Rate | Samples for serum asparaginase activity analyses were obtained days 4, 11, 18 and 25 post one-dose of IV-PEG on day 7 of the induction phase. | Nadir serum asparaginase activity (NSAA) levels were estimated based on established methods. Induction therapeutic NSAA rate is defined as the percentage of patients achieving a NSAA level above 0.1 IU/mL at a given timepoint. |
| Secondary | 5-Year Disease-Free Survival by MRD Day 32 Status | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | Disease-free survival (DFS) in a landmark analysis is defined as the duration of time from asparaginase randomization (which occurred after patients achieved complete remission and were assigned to a final risk group) to documented relapse, death during remission or second malignant neoplasm. DFS is estimated based on the Kaplan-Meier method and 5-year DFS is the probability of patients remaining alive, relapse-free and without occurrence of second malignant neoplasm 5 years from asparaginase randomization. Disease relapse is defined as \>25% lymphoblasts identified morphologically in bone marrow aspirate/biopsy, or identification of lymphoblasts in marrow (any percentage) identified to be leukemic by flow cytometry, cytogenetics, FISH, immunohistochemistry, or other tests. Appearance of leukemic cells at any extramedullary site (a single, unequivocal lymphoblast in the CSF may qualify as CNS leukemia) also qualifies if confirmed by the PI. |
| Secondary | 5-Year Disease-Free Survival by Bone Marrow Day 18 Status | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | Disease-free survival (DFS) in a landmark analysis is defined as the duration of time from asparaginase randomization (which occurred after patients achieved complete remission and were assigned to a final risk group) to documented relapse, death during remission or second malignant neoplasm. DFS is estimated based on the Kaplan-Meier method and 5-year DFS is the probability of patients remaining alive, relapse-free and without occurrence of second malignant neoplasm 5 years from asparaginase randomization. Disease relapse is defined as \>25% lymphoblasts identified morphologically in bone marrow aspirate/biopsy, or identification of lymphoblasts in marrow (any percentage) identified to be leukemic by flow cytometry, cytogenetics, FISH, immunohistochemistry, or other tests. Appearance of leukemic cells at any extramedullary site (a single, unequivocal lymphoblast in the CSF may qualify as CNS leukemia) also qualifies if confirmed by the PI. |
| Secondary | 5-year Disease-Free Survival by CNS Directed Treatment Group | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | Disease-free survival (DFS) in a landmark analysis is defined as the duration of time from asparaginase randomization (which occurred after patients achieved complete remission and were assigned to a final risk group) to documented relapse, death during remission or second malignant neoplasm. DFS is estimated based on the Kaplan-Meier method and 5-year DFS is the probability of patients remaining alive, relapse-free and without occurrence of second malignant neoplasm 5 years from asparaginase randomization. Disease relapse is defined as \>25% lymphoblasts identified morphologically in bone marrow aspirate/biopsy, or identification of lymphoblasts in marrow (any percentage) identified to be leukemic by flow cytometry, cytogenetics, FISH, immunohistochemistry, or other tests. Appearance of leukemic cells at any extramedullary site (a single, unequivocal lymphoblast in the CSF may qualify as CNS leukemia) also qualifies if confirmed by the PI. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Asparaginase-Related Toxicity Rate | 30-week post-induction asparaginase treatment period | The analysis dataset is comprised of all randomized patients. | percentage of participants | NUMBER |
| SECONDARY | 5-Year Disease-Free Survival | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | The analysis dataset is comprised of all randomized patients. | probability | NUMBER |
| SECONDARY | Post-Induction Nadir Serum Asparaginase Activity Level | Samples for nadir serum asparaginase activity analyses were obtained before doses administered at weeks 5, 11, 17, 23 and 29 of post-induction asparaginase treatment. | The analysis dataset is comprised of all randomized patients who consented to research studies with an evaluable sample for analysis of serum asparaginase activity at the respective post-induction assessment timepoints. | IU/mL | MEAN |
| SECONDARY | Post-Induction Therapeutic Nadir Serum Asparaginase Activity Rate | Samples for nadir serum asparaginase activity analyses were obtained before doses administered at weeks 5, 11, 17, 23 and 29 of post-induction asparaginase treatment. | The analysis dataset is comprised of all randomized patients who consented to research studies with a one post-induction evaluable sample for analysis of serum asparaginase activity. | percentage of participants | NUMBER |
| SECONDARY | Induction Infection Toxicity Rate | Assessed daily during remission induction days 4-32. | The analysis dataset is comprised of eligible and treated patients. This excludes the 6 enrolled but ineligible patients. Rates in this overall study cohort will be compared against historical controls (in particular patients treated with a more intensive induction regimen). | percentage of participants | NUMBER |
| SECONDARY | Induction Serum Asparaginase Activity Level | Samples for serum asparaginase activity analyses were obtained days 4, 11, 18 and 25 post one-dose of IV-PEG on day 7 of the induction phase. | The analysis dataset is comprised of all randomized patients who consented to research studies with an evaluable sample for analysis of serum asparaginase activity at the respective induction assessment timepoints. | IU/mL | MEDIAN |
| SECONDARY | Induction Therapeutic Nadir Serum Asparaginase Activity Rate | Samples for serum asparaginase activity analyses were obtained days 4, 11, 18 and 25 post one-dose of IV-PEG on day 7 of the induction phase. | The analysis dataset is comprised of all randomized patients who consented to research studies with an evaluable sample for analysis of serum asparaginase activity at the respective induction assessment timepoints. | percentage of participants | NUMBER |
| SECONDARY | 5-Year Disease-Free Survival by MRD Day 32 Status | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | The analysis dataset is comprised of B cell ALL patients who achieved an induction complete remission with an evaluable sample at day 32 for analysis of MRD. | probability | NUMBER |
| SECONDARY | 5-Year Disease-Free Survival by Bone Marrow Day 18 Status | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | The analysis dataset is comprised of patients who achieved an induction complete remission with an evaluable sample at day 18 (optional submission) for analysis of marrow morphology. | probability | NUMBER |
| SECONDARY | 5-year Disease-Free Survival by CNS Directed Treatment Group | Disease evaluations occurred continuously on treatment. Suggested long-term follow-up was monthly for 6m, bi-monthly for 6m, every 4 months for 1y, semi-annually for 1y, then annually. Median follow-up in this study cohort is 6 yrs, up to 10y. | The analysis dataset is comprised of patients who achieved an induction complete remission with an evaluable sample at diagnosis for analysis of CNS-directed therapy . | probability | NUMBER |
Source ClinicalTrials.gov
Results by group35
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Asparaginase-Related Toxicity Rate | Intramuscular Native E Coli L-asparaginase (IM-EC) | 26 | 95% Confidence Interval 20 to 32 | percentage of participants | |
| Asparaginase-Related Toxicity Rate | Intravenous PEG-asparaginase (IV-PEG) | 28 | 95% Confidence Interval 22 to 34 | percentage of participants | |
| 5-Year Disease-Free Survival | Intramuscular Native E Coli L-asparaginase (IM-EC) | .89 | 95% Confidence Interval .85 to .93 | probability | |
| 5-Year Disease-Free Survival | Intravenous PEG-asparaginase (IV-PEG) | .90 | 95% Confidence Interval .86 to .94 | probability | |
| Post-Induction Nadir Serum Asparaginase Activity Level | Intramuscular Native E Coli L-asparaginase (IM-EC) | Week 5 NSAA Level | 0.129 | Standard Deviation 0.108 | IU/mL |
| Post-Induction Nadir Serum Asparaginase Activity Level | Intravenous PEG-asparaginase (IV-PEG) | Week 5 NSAA Level | 0.726 | Standard Deviation 0.322 | IU/mL |
| Post-Induction Nadir Serum Asparaginase Activity Level | Intramuscular Native E Coli L-asparaginase (IM-EC) | Week 11 NSAA Level | 0.143 | Standard Deviation 0.131 | IU/mL |
| Post-Induction Nadir Serum Asparaginase Activity Level | Intravenous PEG-asparaginase (IV-PEG) | Week 11 NSAA Level | 0.773 | Standard Deviation 0.231 | IU/mL |
| Post-Induction Nadir Serum Asparaginase Activity Level | Intramuscular Native E Coli L-asparaginase (IM-EC) | Week 17 NSAA Level | 0.159 | Standard Deviation 0.161 | IU/mL |
| Post-Induction Nadir Serum Asparaginase Activity Level | Intravenous PEG-asparaginase (IV-PEG) | Week 17 NSAA Level | 0.787 | Standard Deviation 0.303 | IU/mL |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 results by groupParticipant flow35
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Intramuscular Native E Coli L-asparaginase (IM-EC) | 231 | |
| Overall Study | STARTED | Intravenous PEG-asparaginase (IV-PEG) | 232 | |
| Overall Study | STARTED | IM-EC [Directly Assigned] | 42 | |
| Overall Study | STARTED | Ineligible for Randomization | 51 | |
| Overall Study | STARTED | Expansion Cohort | 244 | |
| Overall Study | Evaluable for CNS Directed-Treatment | Intramuscular Native E Coli L-asparaginase (IM-EC) | 231 | |
| Overall Study | Evaluable for CNS Directed-Treatment | Intravenous PEG-asparaginase (IV-PEG) | 232 | |
| Overall Study | Evaluable for CNS Directed-Treatment | IM-EC [Directly Assigned] | 42 | |
| Overall Study | Evaluable for CNS Directed-Treatment | Ineligible for Randomization | 21 | |
| Overall Study | Evaluable for CNS Directed-Treatment | Expansion Cohort | 227 |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 participant flowAdverse events903
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Agitation | Psychiatric disorders | Intramuscular Native E Coli L-asparaginase (IM-EC) | 0 | 231 | |
| other | Agitation | Psychiatric disorders | Intravenous PEG-asparaginase (IV-PEG) | 1 | 232 | |
| other | Agitation | Psychiatric disorders | Overall | 1 | 794 | |
| other | Allergy-other | Immune system disorders | Intramuscular Native E Coli L-asparaginase (IM-EC) | 0 | 231 | |
| other | Allergy-other | Immune system disorders | Intravenous PEG-asparaginase (IV-PEG) | 1 | 232 | |
| other | Allergy-other | Immune system disorders | Overall | 1 | 794 | |
| other | Atelectasis | Respiratory, thoracic and mediastinal disorders | Intramuscular Native E Coli L-asparaginase (IM-EC) | 0 | 231 | |
| other | Atelectasis | Respiratory, thoracic and mediastinal disorders | Intravenous PEG-asparaginase (IV-PEG) | 1 | 232 | |
| other | Atelectasis | Respiratory, thoracic and mediastinal disorders | Overall | 1 | 794 | |
| other | Chemoradiation dermatitis | Injury, poisoning and procedural complications | Intramuscular Native E Coli L-asparaginase (IM-EC) | 0 | 231 |
1 to 10 of 903Source ClinicalTrials.gov
See all 903 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Intramuscular Native E Coli L-asparaginase (IM-EC) | 1 | 231 |
| Total, all-cause mortality | Intravenous PEG-asparaginase (IV-PEG) | 1 | 232 |
| Total, all-cause mortality | Overall | 2 | 794 |
| Total, other adverse events | Intramuscular Native E Coli L-asparaginase (IM-EC) | 40 | 231 |
| Total, other adverse events | Intravenous PEG-asparaginase (IV-PEG) | 38 | 232 |
| Total, other adverse events | Overall | 118 | 794 |
| Total, serious adverse events | Intramuscular Native E Coli L-asparaginase (IM-EC) | 132 | 231 |
| Total, serious adverse events | Intravenous PEG-asparaginase (IV-PEG) | 142 | 232 |
| Total, serious adverse events | Overall | 574 | 794 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants2
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|---|---|---|---|---|---|
| P30CA006516 | Dana-Farber/Harvard Cancer Center | EBERT, BENJAMIN LEVINE | DANA-FARBER CANCER INST | NATIONAL CANCER INSTITUTE | 1985 to 2025 | NIH RePORTER |
| P01CA068484 | Therapeutic Index of Acute Lymphoblastic Leukemia | SALLAN, STEPHEN E | DANA-FARBER CANCER INST | NATIONAL CANCER INSTITUTE | 1995 to 2010 | NIH RePORTER |
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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