Trial · NCT02303821

Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia

NCT02303821, Phase 1, Completed, Amgen; results first posted 2025-06-04

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia. Trials, record NCT02303821. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02303821

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia
NCT number: NCT02303821
Status: Completed
Phases: Phase 1
Enrollment: 141 participants, actual
Lead sponsor: Amgen
Conditions: Acute Lymphoblastic Leukemia (ALL)
Interventions: Carfilzomib, Dexamethasone, Mitoxantrone, PEG-asparaginase, Vincristine, Intrathecal (IT) Methotrexate, Intrathecal Triple Therapy (Intrathecal Cytarabine, Hydrocortisone, and Methotrexate), 6-Mercaptopurine, Cyclophosphamide, Cytarabine, Daunorubicin
First posted: 2014-12-01
Last updated: 2025-06-04
Other registries' IDs: eudract:2014-001633-84
Results first posted: 2025-06-04

Source ClinicalTrials.gov

Registered outcomes22

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Phase 1b: Number of Participants Who Experienced Treatment-emergent Adverse Events (TEAEs)	From first dose up to 30 days after the last dose of study treatment; maximum duration of treatment was 8 weeks	An adverse event (AE) is any untoward medical occurrence in a clinical study participant irrespective of a causal relationship with the study treatment. TEAEs are AEs that occurred after the start of study treatment and up to the end of the study or 30 days of the last study treatment, whichever is earlier. Any clinically significant changes in vital signs, electrocardiograms, and clinical laboratory tests that occurred after study treatment administration were recorded as TEAEs. Treatment-related AEs (TRAEs) were TEAEs considered related to at least one study drug by the investigator, including those with unknown relationship. A serious AE (SAE) was defined as any untoward medical occurrence that resulted in death, was immediately life threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, or another medically important serious event.
Primary	Phase 1b: Number of Participants Who Experienced Dose Limiting Toxicities (DLTs)	Up to approximately 35 days	A DLT was defined as any of the following toxicities assessed by the investigator as possibly, probably, or definitely attributable to carfilzomib, with protocol defined exclusions: Any Grade 4 nonhematologic toxicity, ≥ Grade 4 neutropenia or ≥ Grade 3 thrombocytopenia.
Primary	Phase 2: Percentage of Participants With Complete Remission (CR) After Induction Therapy	Up to Day 50 (28-day cycle of induction therapy + recovery window from Day 29 to Day 45 [Day 36 to Day 50 for infants])	CR was defined as: 1. Attainment of M1 bone marrow status (less than 5% blasts in a bone marrow aspirate and at least 200 cells counted) with no evidence of circulating blasts or extramedullary disease. 2. Recovery of peripheral counts: * Absolute neutrophil count (ANC) greater than or equal to 1000/µL * Platelet count greater than or equal to 100000/µL. * Assessed between days 29 and 45 Data was adjusted as inverse probability of treatment weight (IPTW) for the average treatment effect of the treated (IPTW-ATTW).
Secondary	Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Lead-in Cycle Day 1 (7-day cycle) and Induction Cycle Day 8 (28-day cycle): pre-dose, 15 minutes (m) after the start of infusion, immediately (within 2m) before the end of infusion (EOI), EOI, 10m, 30m, 1 hour (h), 2 h, and 4h post-dose	The PK parameter estimates for carfilzomib were estimated from plasma concentration-time profiles using standard noncompartmental approaches over the complete sampling interval.
Secondary	Phase 1b: Area Under the Curve (AUC) From Time 0 to the Last Quantifiable Timepoint (AUClast) of Carfilzomib	Lead-in Cycle Day 1 (7-day cycle) and Induction Cycle Day 8 (28-day cycle): pre-dose, 15m after the start of infusion, immediately (within 2m) before the EOI, EOI, 10m, 30m, 1h, 2 h, and 4h post-dose	The PK parameter estimates for carfilzomib were estimated from plasma concentration-time profiles using standard noncompartmental approaches over the complete sampling interval.
Secondary	Phase 1b: AUC From Time 0 to Infinity (AUCinf) of Carfilzomib	Lead-in Cycle Day 1 (7-day cycle) and Induction Cycle Day 8 (28-day cycle): pre-dose, 15m after the start of infusion, immediately (within 2m) before the EOI, EOI, 10m, 30m, 1h, 2 h, and 4h post-dose	The PK parameter estimates for carfilzomib were estimated from plasma concentration-time profiles using standard noncompartmental approaches over the complete sampling interval.
Secondary	Phase 1b: Percentage of Participants Who Achieved a Combined CR and CR Without Platelet Recovery (CRp) at the End of Induction Cycle	Up to approximately Day 29 of Induction Cycle (28-days cycle, assessment on Day 29)	CR was defined as the attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease and with recovery of peripheral counts (ANC \> 750 mcl and platelet count \> 75,000 mcl). CRp was defined as the attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease and with recovery of ANCs (ANC \> 750 mcl), but with insufficient recovery of platelets (\< 75,000 mcl).
Secondary	Phase 1b: Percentage of Participants Achieving Minimal Residual Disease (MRD) Status of <10-³ and <10-⁴ Lymphoblasts at the End of Induction Cycle	Up to approximately Day 29 of Induction Cycle (28-days cycle, assessment on Day29)	MRD was defined as the quantification of residual lymphoblast in the blood. Assessed by next generation sequencing (NGS).
Secondary	Phase 2: Number of Participants Who Experienced TEAEs	From first dose up to 30 days after the last dose of study treatment; maximum duration of treatment was 8 weeks	An AE is any untoward medical occurrence in a clinical study participant irrespective of a causal relationship with the study treatment. TEAEs are AEs that occurred after the start of study treatment and up to the end of the study or 30 days of the last study treatment, whichever is earlier. Any clinically significant changes in vital signs, electrocardiograms, and clinical laboratory tests that occurred after study treatment administration were recorded as TEAEs. TRAEs were TEAEs considered related to at least one study drug by the investigator, including those with unknown relationship. An SAE was defined as any untoward medical occurrence that resulted in death, was immediately life threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, or another medically important serious event.
Secondary	Phase 2: Percentage of Participants With CR With Incomplete Hematologic Recovery (CRi) After Induction Therapy or Better Remission Status	Up to Day 50 (28-day cycle of induction therapy + recovery window from Day 29 to Day 45 [up to Day 50 for infants])	CR was defined as: a. Attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease. b. Recovery of peripheral counts: - ANC ≥ 1000/µL - Platelet count ≥ 100 000/µL. CRi was defined as: 1. Attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease 2. ANC and platelet counts not fulfilling criteria for CR with partial hematologic recovery (CRh), CR without platelet recovery (CRp), or CR. CRp was defined as: a. Attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease b. Recovery of peripheral counts: i. ANC ≥ 1000/µL ii. Platelet count \< 100 000/µL. CRh was defined as: 1. Attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease 2. Recovery of peripheral counts: i. ANC ≥ 500/µL but \< 1000/µL ii. Platelet count ≥ 50 000/µL but \< 100 000/µL. Data was IPTW adjusted.

1 to 10 of 22Source ClinicalTrials.gov

See all 22 registered outcomes

Outcomes22

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
SECONDARY	Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Lead-in Cycle Day 1 (7-day cycle) and Induction Cycle Day 8 (28-day cycle): pre-dose, 15 minutes (m) after the start of infusion, immediately (within 2m) before the end of infusion (EOI), EOI, 10m, 30m, 1 hour (h), 2 h, and 4h post-dose	Phase 1b PK Analysis Set: All participants who received the study treatment regimen during the lead-in cycle and induction cycles, and had available data. Phase 1b Dose Escalation 2 arms did not have a 1-week lead-in cycle.	ng/mL	MEAN
SECONDARY	Phase 1b: Area Under the Curve (AUC) From Time 0 to the Last Quantifiable Timepoint (AUClast) of Carfilzomib	Lead-in Cycle Day 1 (7-day cycle) and Induction Cycle Day 8 (28-day cycle): pre-dose, 15m after the start of infusion, immediately (within 2m) before the EOI, EOI, 10m, 30m, 1h, 2 h, and 4h post-dose	Phase 1b PK Analysis Set: All participants who received the study treatment regimen during the lead-in cycle and induction cycles, and had available data. Phase 1b Dose Escalation 2 arms did not have a 1-week lead-in cycle.	hr*ng/mL	MEAN
SECONDARY	Phase 1b: AUC From Time 0 to Infinity (AUCinf) of Carfilzomib	Lead-in Cycle Day 1 (7-day cycle) and Induction Cycle Day 8 (28-day cycle): pre-dose, 15m after the start of infusion, immediately (within 2m) before the EOI, EOI, 10m, 30m, 1h, 2 h, and 4h post-dose	Phase 1b PK Analysis Set: All participants who received the study treatment regimen during the lead-in cycle and induction cycles, and had available data. Phase 1b Dose Escalation 2 arms did not have a 1-week lead-in cycle.	hr*ng/ mL	MEAN
SECONDARY	Phase 1b: Percentage of Participants Who Achieved a Combined CR and CR Without Platelet Recovery (CRp) at the End of Induction Cycle	Up to approximately Day 29 of Induction Cycle (28-days cycle, assessment on Day 29)	Phase 1b Induction SAS: All participants who received any amount of the study treatment regimen during the induction period and had available data.	Percentage of participants	NUMBER
SECONDARY	Phase 1b: Percentage of Participants Achieving Minimal Residual Disease (MRD) Status of <10-³ and <10-⁴ Lymphoblasts at the End of Induction Cycle	Up to approximately Day 29 of Induction Cycle (28-days cycle, assessment on Day29)	Phase 1b Induction SAS: All participants who received any amount of the study treatment regimen during the induction period and had available data.	Percentage of Participants	NUMBER
SECONDARY	Phase 2: Number of Participants Who Experienced TEAEs	From first dose up to 30 days after the last dose of study treatment; maximum duration of treatment was 8 weeks	Phase 2 Safety Analysis Set: All participants enrolled to study 20140106 who received at least 1 dose of carfilzomib.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Phase 2: Percentage of Participants With CR With Incomplete Hematologic Recovery (CRi) After Induction Therapy or Better Remission Status	Up to Day 50 (28-day cycle of induction therapy + recovery window from Day 29 to Day 45 [up to Day 50 for infants])	Phase 2: Participants enrolled in 20140106, in the PAS, who received at least 1 dose of carfilzomib.	Percentage of Participants	NUMBER
SECONDARY	Phase 2: Event Free Survival (EFS)	Up to approximately 2 years	Phase 2: Participants enrolled in 20140106, in the PAS, who received at least 1 dose of carfilzomib.	Months	MEDIAN
SECONDARY	Phase 2: Overall Survival (OS)	Up to approximately 2 years	Phase 2: Participants enrolled in 20140106, in the PAS, who received at least 1 dose of carfilzomib.	Months	MEDIAN
SECONDARY	Phase 2: Duration of Remission (DOR)	Up to approximately 2 years	Phase 2: Participants enrolled in 20140106, in the PAS, who received at least 1 dose of carfilzomib. Only participants who achieved CRi or a better remission status were included in the analysis. Data was IPTW adjusted. Medians were estimated using the KM method.	Months	MEDIAN

1 to 10 of 22Source ClinicalTrials.gov

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Results by group136

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 1 (20 mg/m^2)	Dose Escalation 1 - Lead in Cycle Day 1	927	Standard Deviation 259	ng/mL
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	Dose Escalation 1 - Lead in Cycle Day 1	637	Standard Deviation 241	ng/mL
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 1 (20 mg/m^2)	Induction cycle Day 8	771	Standard Deviation 151	ng/mL
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	Induction cycle Day 8	792	Standard Deviation 580	ng/mL
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 2 (20/27 mg/m^2)	Induction cycle Day 8	445	Standard Deviation 260	ng/mL
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 2 (20/36 mg/m^2)	Induction cycle Day 8	1070	Standard Deviation 359	ng/mL
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 2 (20/45 mg/m^2)	Induction cycle Day 8	1630	Standard Deviation 875	ng/mL
Phase 1b: Maximum Concentration (Cmax) of Carfilzomib Alone and in Combination	Phase 1b: Dose Escalation 2 (20/56 mg/m^2)	Induction cycle Day 8	11200	Standard Deviation 16400	ng/mL
Phase 1b: Area Under the Curve (AUC) From Time 0 to the Last Quantifiable Timepoint (AUClast) of Carfilzomib	Phase 1b: Dose Escalation 1 (20 mg/m^2)	Dose Escalation 1 - Lead in Cycle Day 1	387	Standard Deviation 125	hr*ng/mL
Phase 1b: Area Under the Curve (AUC) From Time 0 to the Last Quantifiable Timepoint (AUClast) of Carfilzomib	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	Dose Escalation 1 - Lead in Cycle Day 1	240	Standard Deviation 113	hr*ng/mL

1 to 10 of 136Source ClinicalTrials.gov

See all 136 results by group

Participant flow40

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Phase 1b: Dose Escalation 1 (20 mg/m^2)	5
Overall Study	STARTED	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	6
Overall Study	STARTED	Phase 1b: Dose Escalation 2 (20/27 mg/m^2)	3
Overall Study	STARTED	Phase 1b: Dose Escalation 2 (20/36 mg/m^2)	7
Overall Study	STARTED	Phase 1b: Dose Escalation 2 (20/45 mg/m^2)	4
Overall Study	STARTED	Phase 1b: Dose Escalation 2 (20/56 mg/m^2)	10
Overall Study	STARTED	Phase 2: B-cell (20/56 mg/m^2)	62
Overall Study	STARTED	Phase 2: T-cell (20/56 mg/m^2)	44
Overall Study	Received Carfilzomib During Induction Therapy	Phase 1b: Dose Escalation 1 (20 mg/m^2)	5
Overall Study	Received Carfilzomib During Induction Therapy	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	6

1 to 10 of 40Source ClinicalTrials.gov

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Adverse events2,880

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
other	Anaemia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 1 (20 mg/m^2)	4	5
other	Anaemia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	5	6
other	Anaemia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 2 (20/27 mg/m^2)	2	3
other	Anaemia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 2 (20/36 mg/m^2)	4	7
other	Anaemia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 2 (20/45 mg/m^2	3	4
other	Anaemia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 2 (20/56 mg/m^2)	3	10
other	Anaemia	Blood and lymphatic system disorders	Phase 2: B-cell (20/56 mg/m^2)	37	61
other	Anaemia	Blood and lymphatic system disorders	Phase 2: T-cell (20/56 mg/m^2)	23	44
other	Febrile neutropenia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 1 (20 mg/m^2)	1	5
other	Febrile neutropenia	Blood and lymphatic system disorders	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	1	6

1 to 10 of 2,880Source ClinicalTrials.gov

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Adverse event totals24

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Phase 1b: Dose Escalation 1 (20 mg/m^2)	0	5
Total, all-cause mortality	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	2	6
Total, all-cause mortality	Phase 1b: Dose Escalation 2 (20/27 mg/m^2)	0	3
Total, all-cause mortality	Phase 1b: Dose Escalation 2 (20/36 mg/m^2)	0	7
Total, all-cause mortality	Phase 1b: Dose Escalation 2 (20/45 mg/m^2	0	4
Total, all-cause mortality	Phase 1b: Dose Escalation 2 (20/56 mg/m^2)	3	10
Total, all-cause mortality	Phase 2: B-cell (20/56 mg/m^2)	41	62
Total, all-cause mortality	Phase 2: T-cell (20/56 mg/m^2)	29	44
Total, other adverse events	Phase 1b: Dose Escalation 1 (20 mg/m^2)	5	5
Total, other adverse events	Phase 1b: Dose Escalation 1 (20/27 mg/m^2)	6	6

1 to 10 of 24Source ClinicalTrials.gov

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Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
36215217	DERIVED	PubMed
42234929	DERIVED	PubMed

Grants0

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.

None in this release.

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

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