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CompletedNCT03384654Updated May 25, 2025Results posted

A Study to Evaluate the Efficacy and Safety of Daratumumab in Pediatric and Young Adult Participants Greater Than or Equal to (>=)1 and Less Than or Equal to (<=) 30 Years of Age With Relapsed/Refractory Precursor B-cell or T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

A Phase 2 interventional study of Daratumumab and Vincristine in Precursor Cell Lymphoblastic Leukemia-Lymphoma, sponsored by Janssen Research & Development, LLC. Completed at 53 sites in 10 countries. Open to participants aged 1 Year to 30 Years. Per ClinicalTrials.gov, last updated 2025-05-25.

Sponsored by Janssen Research & Development, LLC · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
47
Allocation
Non-randomized
Ages
1 Year to 30 Years
Sex
All
01

Study summary

The purpose of this study is to evaluate the efficacy of daratumumab in addition to standard chemotherapy in pediatric participants with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL)/lymphoblastic lymphoma (LL) and T-cell ALL/LL as measured by the complete response (CR) rate.

Read the detailed description

Screening for eligible participants will be performed within 21 days before administration of the study drug. Participants with B-cell ALL/LL will receive treatment until disease progression, unacceptable toxicity or achievement of CR followed by hematopoietic stem cell transplant (HSCT). Participants with T cell ALL/LL will receive treatment for up to 2 cycles. If disease progression is confirmed, then the participant will discontinue study treatment, complete the End of Treatment Visit, and enter the Posttreatment Period. For those participants who discontinue study drug prior to disease progression, disease evaluations will continue to be performed every 8 weeks until subsequent anticancer therapy is initiated.

02

Conditions studied

03

Who can participate

Ages eligible
1 Year to 30 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Documented acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LL) as defined by the criteria below:

    1. B-cell cohort: Stage 1; ALL in second or greater relapse or refractory to 2 prior induction regimens with greater than or equal to (>=) 5 percent (%) blasts in the bone marrow and aged 1 to less than (\<) 18 years. Stage 2; ALL in second or greater relapse or refractory to 2 prior induction regimens with (>=) 5% blasts in the bone marrow and aged 1 to 30 years. LL in second or greater relapse or refractory to 2 prior induction regimens and biopsy proven and with evidence of measurable disease by radiologic criteria and aged 1 to 30 years.
    2. T-cell cohort: Stage 1; ALL in first relapse or refractory to 1 prior induction/consolidation regimen with (>=) 5% blasts in the bone marrow and aged 1 to \<18 years. Stage 2; ALL in first relapse or refractory to 1 prior induction/consolidation regimen with (>=) 5% blasts in the bone marrow and aged 1 to 30 years. LL in first relapse or refractory to 1 prior induction/consolidation regimen biopsy proven and with evidence of measurable disease by radiologic criteria and aged 1 to 30 years
  • Performance status greater than or equal to (>=) 70 by Lansky scale (for participants less than [\<] 16 years of age) or Karnofsky scale (for participants [>=] 16 years of age)
  • Adequate hematology laboratory values at Cycle 1 Day 1 pre-dosing defined as follows:

    1. Hemoglobin (>=) 7.5 gram per deciliter (g/dL) ([>=] 5 millimole per liter [mmol/L]; prior red blood cell [RBC] transfusion is permitted)
    2. Platelet count (>=) 10*10\^9 per liter (L) (prior platelet transfusion is permitted)
  • Adequate renal function defined as normal serum creatinine for the participant's age or creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 milliliter per minute per 1.73 square meter (mL/min/1.73 m\^2) prior to enrollment
  • Adequate liver function prior to enrollment defined as:

    1. Alanine aminotransferase level less than or equal to (\<=) 2.5* the upper limit of normal (ULN),
    2. Aspartate aminotransferase level (\<=) 2.5* ULN, and
    3. Total bilirubin (\<=) 2* ULN or direct bilirubin level (\<=) 2.0* ULN

Exclusion criteria

Exclusion Criteria:

  • Received an allogeneic hematopoietic transplant within 3 months of screening
  • Active acute graft-versus-host disease of any grade or chronic graft-versus-host disease of Grade 2 or higher
  • Received immunosuppression post hematopoietic transplant within 1 month of study entry
  • Philadelphia chromosome positive (Ph+) B-cell ALL eligible for tyrosine kinase inhibitor therapy
  • Has either of the following:

    1. Evidence of dyspnea at rest or oxygen saturation (\<=) 94 percent (%).
    2. Known moderate or severe persistent asthma within the past 2 years, or uncontrolled asthma of any classification
  • Received an investigational drug, was vaccinated with live attenuated vaccines, or used an invasive investigational medical device within 4 weeks before the planned first dose of study drug, or is currently being treated in an investigational study
  • Known to be seropositive for human immunodeficiency virus (HIV)
  • Any one of the following:

    1. Seropositive for hepatitis B (defined by a positive test for hepatitis B surface antigen [HBsAg]). Participants with resolved infection (ie, participants who are HBsAg negative but positive for antibodies to hepatitis B core antigen [anti-HBc] and/or antibodies to hepatitis B surface antigen [anti-HBs]) must be screened using real-time polymerase chain reaction (PCR) measurement of hepatitis B virus (HBV) deoxyribonucleic acid (DNA) levels. Those who are PCR positive will be excluded
    2. Known to be seropositive for hepatitis C (except in the setting of a sustained virologic response [SVR], defined as aviremia at least 12 weeks after completion of antiviral therapy)
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
47 participants (actual)

Study arms

  • Experimental
    Cohort 1: B-Cell Acute Lymphoblastic Leukemia (ALL)/LL

    Cohort 1 will include participants with B cell ALL/LL in second or greater relapse or refractory to at least 2 prior induction regimens. Participant will receive daratumumab in combination with vincristine and prednisone.

    Drug: Daratumumab · Drug: Vincristine · Drug: Prednisone

  • Experimental
    Cohort 2: T-Cell ALL/LL

    Cohort 2 will include participants with T-cell ALL/LL in first relapse or refractory to at least 1 prior induction/consolidation regimen. Participant will receive daratumumab in combination with vincristine, prednisone, doxorubicin and peg-asparaginase in Cycle 1 and daratumumab in combination with cyclophosphamide, cytarabine, 6- mercaptopurine and methotrexate in Cycle 2.

    Drug: Daratumumab · Drug: Vincristine · Drug: Prednisone · Drug: Doxorubicin · Biological: Peg-asparaginase · Drug: Cyclophosphamide · Drug: Cytarabine · Drug: 6-mercaptopurine · Drug: Methotrexate

Interventions

  • DrugDaratumumab

    Participant will receive daratumumab 16 milligram per kilogram (mg/kg) in cohort 1 and cohort 2.

  • DrugVincristine

    Participant will receive vincristine 1.5 milligram per meter square (mg/m\^2) in cohort 1 and cohort 2.

  • DrugPrednisone

    Participant will receive prednisone 40 mg/m\^2 in cohort 1 and cohort 2.

  • DrugDoxorubicin

    Participant will receive doxorubicin 60 mg/m\^2 in cohort 2.

  • BiologicalPeg-asparaginase

    Participant will receive peg-asparaginase 2500 units per meter square (U/m\^2) in cohort 2.

  • DrugCyclophosphamide

    Participant will receive cyclophosphamide 1 gram per meter square (g/m\^2) once in cohort 2.

  • DrugCytarabine

    Participant will receive cytarabine 75 mg/m\^2 in cohort 2.

  • Drug6-mercaptopurine

    Participant will receive 6-mercaptopurine 60 mg/m\^2 orally daily in cohort 2.

  • DrugMethotrexate

    Participant will receive methotrexate 5 g/m\^2 intravenously (IV) in cohort 2.

05

What researchers measure

Primary outcomes

  1. Cohort 1: Percentage of Participants With Complete Response (CR) for B-cell Acute Lymphoblastic Leukemia (ALL)

    Complete response based on the modified National Comprehensive Cancer Network (NCCN) criteria was defined as: less than 5 percent (%) blasts in the bone marrow; no evidence of circulating blasts or extramedullary disease; full recovery of peripheral blood counts: platelets greater than (\>)100\*10\^9 cells/liter (L) and absolute neutrophil count (ANC) \>1.0\*10\^9 cells/L. This outcome measure was planned to be analyzed for specified arm only.

    Time frame: Up to 2 cycles, that is, up to 56 days (each cycle of 28-days)

  2. Cohort 2: Percentage of Participants With Complete Response (CR) for T-cell ALL

    Complete response based on the modified NCCN criteria was defined as: less than 5% blasts in the bone marrow; no evidence of circulating blasts or extramedullary disease; full recovery of peripheral blood counts: platelets \>100\*10\^9 cells/L and ANC \>1.0\*10\^9 cells/L. This outcome measure was planned to be analyzed for specified arms only.

    Time frame: End of Cycle 1 (that is, up to 28 days)

Secondary outcomes

  1. Overall Response Rate (ORR)

    For ALL participants, ORR was defined as percentage of participants who achieved CR or CR with only partial hematological recovery(CRi) as per NCCN criteria. CR for ALL: less than 5% blasts in bone marrow; no evidence of circulating blasts or extramedullary disease; full recovery of peripheral blood counts: platelets \>100\*10\^9 cells/L and ANC \>1.0\*10\^9 cells/L. CRi for ALL: less than 5% blasts in bone marrow; no evidence of circulating blasts or extramedullary disease; partial recovery of peripheral blood counts not meeting criteria for CR. For LL participants, ORR was defined as percentage of participants who had CR or PR during or after treatment administration but prior to start of subsequent anti-cancer therapy or allogeneic hematopoietic stem cell transplant(HSCT). CR for LL: disappearance of all evidence of disease from all sites. PR for LL: decrease of \>=50% in sum of products of diameter of lesions of up to 6 of largest dominant nodes or nodal masses with no new lesions.

    Time frame: Up to 4 years 4 months

  2. Event-free Survival (EFS)

    EFS was defined as the time (in months) from the date of first treatment to the first documented treatment failure (that is \[ie\], disease progression) or date of relapse from CR or death due to any cause, whichever occurred first. Per NCCN criteria, relapse from CR is defined as: reappearance of leukemia blasts in the peripheral blood or \>5% blasts in the bone marrow; reappearance of extramedullary disease or new extramedullary disease. Progressive disease: increase of at least 25% in the absolute number of circulating peripheral or bone marrow blasts, or development of new extramedullary disease. Kaplan-Meier method was used for the analysis.

    Time frame: Up to 4 years 4 months

  3. Relapse-free Survival (RFS)

    RFS was defined as the time (in months) from CR to relapse from CR, or disease progression or death due to any cause, whichever occurred first. For ALL, as per NCCN criteria, relapse from CR is defined as: reappearance of leukemia blasts in the peripheral blood or \>5% blasts in the bone marrow, or reappearance of extramedullary disease or new extramedullary disease. Progressive disease: increase of at least 25% in absolute number of circulating peripheral or bone marrow blasts, or development of new extramedullary disease. Kaplan-Meier method was used for analysis.

    Time frame: Up to 4 years 4 months

  4. Overall Survival (OS)

    OS was defined as the time (in months) from the date of first study drug administration to the date of death due to any cause. Kaplan-Meier method was used for the analysis. Participants who died after consent withdrawal were considered as having an OS event.

    Time frame: Up to 4 years 4 months

  5. Minimal Residual Disease (MRD) Negative Rate

    MRD negative rate was defined as the percentage of participants who were considered MRD negative after MRD testing by bone marrow aspirate at any timepoint after first study treatment administration and before disease progression or starting subsequent anti-cancer therapy or allogeneic hematopoietic stem cell transplant (HSCT). MRD negative was defined as less than (\<) 0.01% abnormal population counts to nucleated mononuclear cells when measured by flow cytometry.

    Time frame: Up to 4 years 4 months

  6. Percentage of Participants Who Received an Allogeneic Hematopoietic Stem Cell Transplant (HSCT)

    Percentage of participants who received an allogeneic HSCT after treatment with daratumumab were reported.

    Time frame: Up to 4 years 4 months

  7. Maximum Observed Serum Concentration (Cmax) of Daratumumab

    Cmax was defined as maximum observed serum concentration of daratumumab.

    Time frame: Cohort 1: B-cell ALL: End of infusion (EOI) on Day 1 of Cycle 2; Cohort 2: T-cell ALL (1-17 years): EOI on Day 22 of Cycle 2; Cohort 2: T-cell ALL (18-30 years): EOI on Day 1 of Cycle 2; Cohort 2: T-cell LL (1-30 years): EOI on Day 22 of Cycle 2

  8. Minimum Observed Serum Concentration (Cmin) of Daratumumab

    Cmin was defined as minimum observed serum concentration of daratumumab.

    Time frame: Cohort 1: B-cell ALL: Predose on Day 1 of Cycle 2; Cohort 2: T-cell ALL (1-17 years): Predose on Day 22 of Cycle 2; Cohort 2: T-cell ALL (18-30 years): Predose on Day 1 of Cycle 2; Cohort 2: T-cell LL (1-30 years): Predose on Day 22 of Cycle 2

  9. Number of Participants With Anti-daratumumab Antibodies

    Number of participants with anti-daratumumab antibodies was reported.

    Time frame: Up to 4 years 4 months

  10. Concentration of Daratumumab in Cerebrospinal Fluid (CSF)

    Concentration of daratumumab in CSF was reported. '0' in the number analyzed field signifies that none of the participants were available for the analysis at the specified time points.

    Time frame: Pre-dose on Cohort 1: Cycles 1 and 2: Day 1; Cohort 2: Cycle 1 Day 1 and Day 15 and Cycle 2 Day 2 and Day 15

06

Results

Posted Oct 13, 2023

Participant flow

Participant flow — Overall Study
MilestoneCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Started724510
Completed724410
Not completed0010
Withdrew: Withdrawal by subject0010

Outcome measures

PrimaryCohort 1: Percentage of Participants With Complete Response (CR) for B-cell Acute Lymphoblastic Leukemia (ALL)

Complete response based on the modified National Comprehensive Cancer Network (NCCN) criteria was defined as: less than 5 percent (%) blasts in the bone marrow; no evidence of circulating blasts or extramedullary disease; full recovery of peripheral blood counts: platelets greater than (\>)100\*10\^9 cells/liter (L) and absolute neutrophil count (ANC) \>1.0\*10\^9 cells/L. This outcome measure was planned to be analyzed for specified arm only.

Time frame:
Up to 2 cycles, that is, up to 56 days (each cycle of 28-days)
Reported as:
Number · Percentage of participants
Cohort 1: Percentage of Participants With Complete Response (CR) for B-cell Acute Lymphoblastic Leukemia (ALL)
Percentage of participantsCohort 1: B-cell ALL (1-17 Years)
Cohort 1: Percentage of Participants With Complete Response (CR) for B-cell Acute Lymphoblastic Leukemia (ALL)0 (NA to NA)
PrimaryCohort 2: Percentage of Participants With Complete Response (CR) for T-cell ALL

Complete response based on the modified NCCN criteria was defined as: less than 5% blasts in the bone marrow; no evidence of circulating blasts or extramedullary disease; full recovery of peripheral blood counts: platelets \>100\*10\^9 cells/L and ANC \>1.0\*10\^9 cells/L. This outcome measure was planned to be analyzed for specified arms only.

Time frame:
End of Cycle 1 (that is, up to 28 days)
Reported as:
Number · Percentage of participants
Cohort 2: Percentage of Participants With Complete Response (CR) for T-cell ALL
Percentage of participantsCohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Cohort 2: Percentage of Participants With Complete Response (CR) for T-cell ALL41.7 (24.6 to 60.3)60.0 (18.9 to 92.4)30.0 (8.7 to 60.7)
SecondaryOverall Response Rate (ORR)

For ALL participants, ORR was defined as percentage of participants who achieved CR or CR with only partial hematological recovery(CRi) as per NCCN criteria. CR for ALL: less than 5% blasts in bone marrow; no evidence of circulating blasts or extramedullary disease; full recovery of peripheral blood counts: platelets \>100\*10\^9 cells/L and ANC \>1.0\*10\^9 cells/L. CRi for ALL: less than 5% blasts in bone marrow; no evidence of circulating blasts or extramedullary disease; partial recovery of peripheral blood counts not meeting criteria for CR. For LL participants, ORR was defined as percentage of participants who had CR or PR during or after treatment administration but prior to start of subsequent anti-cancer therapy or allogeneic hematopoietic stem cell transplant(HSCT). CR for LL: disappearance of all evidence of disease from all sites. PR for LL: decrease of \>=50% in sum of products of diameter of lesions of up to 6 of largest dominant nodes or nodal masses with no new lesions.

Time frame:
Up to 4 years 4 months
Reported as:
Number · Percentage of participants
Overall Response Rate (ORR)
Percentage of participantsCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Overall Response Rate (ORR)14.3 (0.7 to 52.1)83.3 (65.8 to 94.1)80.0 (34.3 to 99.0)50.0 (22.2 to 77.8)
SecondaryEvent-free Survival (EFS)

EFS was defined as the time (in months) from the date of first treatment to the first documented treatment failure (that is \[ie\], disease progression) or date of relapse from CR or death due to any cause, whichever occurred first. Per NCCN criteria, relapse from CR is defined as: reappearance of leukemia blasts in the peripheral blood or \>5% blasts in the bone marrow; reappearance of extramedullary disease or new extramedullary disease. Progressive disease: increase of at least 25% in the absolute number of circulating peripheral or bone marrow blasts, or development of new extramedullary disease. Kaplan-Meier method was used for the analysis.

Time frame:
Up to 4 years 4 months
Reported as:
Median · Months
Event-free Survival (EFS)
MonthsCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Event-free Survival (EFS)1.1 (0.9 to 2.1)8.9 (5.3 to NA)10.3 (2.6 to NA)2.9 (1.3 to 4.9)
SecondaryRelapse-free Survival (RFS)

RFS was defined as the time (in months) from CR to relapse from CR, or disease progression or death due to any cause, whichever occurred first. For ALL, as per NCCN criteria, relapse from CR is defined as: reappearance of leukemia blasts in the peripheral blood or \>5% blasts in the bone marrow, or reappearance of extramedullary disease or new extramedullary disease. Progressive disease: increase of at least 25% in absolute number of circulating peripheral or bone marrow blasts, or development of new extramedullary disease. Kaplan-Meier method was used for analysis.

Time frame:
Up to 4 years 4 months
Reported as:
Median · Months
Relapse-free Survival (RFS)
MonthsCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Relapse-free Survival (RFS)—19.4 (5.3 to NA)9.4 (3.6 to NA)NA (1.8 to NA)
SecondaryOverall Survival (OS)

OS was defined as the time (in months) from the date of first study drug administration to the date of death due to any cause. Kaplan-Meier method was used for the analysis. Participants who died after consent withdrawal were considered as having an OS event.

Time frame:
Up to 4 years 4 months
Reported as:
Median · Months
Overall Survival (OS)
MonthsCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Overall Survival (OS)3.2 (1.0 to 3.6)10.9 (6.7 to NA)12.0 (4.5 to NA)4.2 (1.7 to 5.6)
SecondaryMinimal Residual Disease (MRD) Negative Rate

MRD negative rate was defined as the percentage of participants who were considered MRD negative after MRD testing by bone marrow aspirate at any timepoint after first study treatment administration and before disease progression or starting subsequent anti-cancer therapy or allogeneic hematopoietic stem cell transplant (HSCT). MRD negative was defined as less than (\<) 0.01% abnormal population counts to nucleated mononuclear cells when measured by flow cytometry.

Time frame:
Up to 4 years 4 months
Reported as:
Number · Percentage of participants
Minimal Residual Disease (MRD) Negative Rate
Percentage of participantsCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Minimal Residual Disease (MRD) Negative Rate0 (0 to 0)45.8 (28.2 to 64.2)20.0 (1.0 to 65.7)50.0 (22.2 to 77.8)
SecondaryPercentage of Participants Who Received an Allogeneic Hematopoietic Stem Cell Transplant (HSCT)

Percentage of participants who received an allogeneic HSCT after treatment with daratumumab were reported.

Time frame:
Up to 4 years 4 months
Reported as:
Number · Percentage of participants
Percentage of Participants Who Received an Allogeneic Hematopoietic Stem Cell Transplant (HSCT)
Percentage of participantsCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Percentage of Participants Who Received an Allogeneic Hematopoietic Stem Cell Transplant (HSCT)14.3 (0.7 to 52.1)75.0 (56.5 to 88.5)60.0 (18.9 to 92.4)30.0 (8.7 to 60.7)
SecondaryMaximum Observed Serum Concentration (Cmax) of Daratumumab

Cmax was defined as maximum observed serum concentration of daratumumab.

Time frame:
Cohort 1: B-cell ALL: End of infusion (EOI) on Day 1 of Cycle 2; Cohort 2: T-cell ALL (1-17 years): EOI on Day 22 of Cycle 2; Cohort 2: T-cell ALL (18-30 years): EOI on Day 1 of Cycle 2; Cohort 2: T-cell LL (1-30 years): EOI on Day 22 of Cycle 2
Reported as:
Mean · Micrograms per milliliter (mcg/mL)
Maximum Observed Serum Concentration (Cmax) of Daratumumab
Micrograms per milliliter (mcg/mL)Cohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Maximum Observed Serum Concentration (Cmax) of Daratumumab494 ± 184763 ± 185501 ± 347758 ± 157
SecondaryMinimum Observed Serum Concentration (Cmin) of Daratumumab

Cmin was defined as minimum observed serum concentration of daratumumab.

Time frame:
Cohort 1: B-cell ALL: Predose on Day 1 of Cycle 2; Cohort 2: T-cell ALL (1-17 years): Predose on Day 22 of Cycle 2; Cohort 2: T-cell ALL (18-30 years): Predose on Day 1 of Cycle 2; Cohort 2: T-cell LL (1-30 years): Predose on Day 22 of Cycle 2
Reported as:
Mean · mcg/mL
Minimum Observed Serum Concentration (Cmin) of Daratumumab
mcg/mLCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Minimum Observed Serum Concentration (Cmin) of Daratumumab172 ± 115369 ± 105172 ± 177365 ± 204
SecondaryNumber of Participants With Anti-daratumumab Antibodies

Number of participants with anti-daratumumab antibodies was reported.

Time frame:
Up to 4 years 4 months
Reported as:
Count of participants · Participants
Number of Participants With Anti-daratumumab Antibodies
ParticipantsCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Number of Participants With Anti-daratumumab Antibodies0000
SecondaryConcentration of Daratumumab in Cerebrospinal Fluid (CSF)

Concentration of daratumumab in CSF was reported. '0' in the number analyzed field signifies that none of the participants were available for the analysis at the specified time points.

Time frame:
Pre-dose on Cohort 1: Cycles 1 and 2: Day 1; Cohort 2: Cycle 1 Day 1 and Day 15 and Cycle 2 Day 2 and Day 15
Reported as:
Mean · mcg/mL
Concentration of Daratumumab in Cerebrospinal Fluid (CSF)
mcg/mLCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Cycle 1 Day 1 predoseNA ± NANA ± NANA ± NANA ± NA
Cycle 1 Day 15 predose—0.907 ± 1.960.319 ± 0.2030.456 ± 0.280
Cycle 2 Day 1 predose0.573 ± 0.545———
Cycle 2 Day 2 predose—0.915 ± 0.9160.296 ± 0.1911.23 ± 0.728
Cycle 2 Day 15 predose—0.934 ± 0.5490.1631.06 ± 0.282

Adverse events

Collected over From baseline (Day 1) up to 4 years 4 months. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Cohort 1: B-cell ALL (1-17 Years)7/7 (100%)3/7 (42.9%)7/7 (100%)
Cohort 2: T-Cell ALL (1-17 Years)14/24 (58.3%)16/24 (66.7%)24/24 (100%)
Cohort 2: T-Cell ALL (18-30 Years)3/5 (60%)4/5 (80%)5/5 (100%)
Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)8/10 (80%)7/10 (70%)10/10 (100%)
Most frequent serious events
Showing 10 of 54
Most frequent serious events
EventCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
Febrile NeutropeniaBlood and lymphatic system disorders3/76/241/52/10
PyrexiaGeneral disorders0/77/241/51/10
ThrombocytopeniaBlood and lymphatic system disorders0/70/241/50/10
StomatitisGastrointestinal disorders0/71/241/50/10
PainGeneral disorders0/70/241/50/10
CellulitisInfections and infestations0/71/241/50/10
Septic ShockInfections and infestations0/72/241/50/10
Weight DecreasedInvestigations0/70/241/50/10
Decreased AppetiteMetabolism and nutrition disorders0/70/241/50/10
DeliriumPsychiatric disorders0/70/241/50/10
Most frequent other events
Showing 10 of 193
Most frequent other events
EventCohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)
AnaemiaBlood and lymphatic system disorders4/716/242/510/10
ThrombocytopeniaBlood and lymphatic system disorders2/718/243/59/10
ConstipationGastrointestinal disorders2/76/244/52/10
HyperbilirubinaemiaHepatobiliary disorders0/77/244/54/10
Blood Alkaline Phosphatase IncreasedInvestigations0/70/244/50/10
HyponatraemiaMetabolism and nutrition disorders0/76/244/52/10
StomatitisGastrointestinal disorders0/710/242/57/10
PyrexiaGeneral disorders4/713/243/57/10
NeutropeniaBlood and lymphatic system disorders2/715/242/56/10
Abdominal PainGastrointestinal disorders2/712/243/54/10

Baseline characteristics

Age, Continuous
Age, Continuous(years)Cohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)Total
Mean8.1 ± 5.019.8 ± 4.1722.2 ± 2.7713.5 ± 5.6811.7 ± 6.03
Sex: Female, Male
Sex: Female, Male(Participants)Cohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)Total
Female4100115
Male3145931
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Cohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)Total
Hispanic or Latino14218
Not Hispanic or Latino4163730
Unknown or Not Reported24028
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Cohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)Total
American Indian or Alaska Native00101
Asian01102
Native Hawaiian or Other Pacific Islander00000
Black or African American00101
White5182833
More than one race00000
Unknown or Not Reported25029
Region of Enrollment
Region of Enrollment(Participants)Cohort 1: B-cell ALL (1-17 Years)Cohort 2: T-Cell ALL (1-17 Years)Cohort 2: T-Cell ALL (18-30 Years)Cohort 2: T-Cell Lymphoblastic Leukemia (LL) (1-30 Years)Total
BELGIUM01001
FRANCE03025
GERMANY20002
ISRAEL02002
ITALY24006
NETHERLANDS02002
SPAIN251412
UNITED KINGDOM03115
UNITED STATES143311
07

Study locations

53 sites
  • University of Alabama at Birmingham
    Birmingham, Alabama 35233-1711, United States
  • Phoenix Children's Hospital
    Phoenix, Arizona 85016, United States
  • UCSF Benioff Children's Hospital Oakland
    Oakland, California 94609, United States
  • Children's Hospital Orange County
    Orange, California 92868, United States
  • Stanford University
    Palo Alto, California 94304, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Connecticut Children's Medical Center
    Hartford, Connecticut 06106, United States
  • Children'S Healthcare Of Atlanta/Emory Univ. Dept. Of Pediatrics
    Atlanta, Georgia 30342, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois 60611, United States
  • Riley Hospital for Children
    Indianapolis, Indiana 46202, United States
  • Johns Hopkins University
    Baltimore, Maryland 21231-1000, United States
  • Dana Farber Cancer Institute
    Boston, Massachusetts 02215-5418, United States
  • C.S. Mott Children's Hospital
    Ann Arbor, Michigan 48109-4257, United States
  • Washington Univeristy School of Medicine/ Pediatrics
    Saint Louis, Missouri 63110, United States
  • Newark Beth Israel Medical Center
    Newark, New Jersey 07112, United States
  • New York University Langone Medical Center
    New York, New York 10016, United States
  • Memorial Sloan Kettering Cancer Center
    New York, New York 10065, United States
  • Stony Brook University Medical Center
    Stony Brook, New York 11733, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43214, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Dell Children's Medical Center of Central Texas/Children's Blood and Cancer Center
    Austin, Texas 78723, United States
  • UT Southwestern Medical Center
    Dallas, Texas 75235, United States
  • Baylor College of Medicine
    Houston, Texas 77030, United States
  • University of Utah Primary Children's Medical Center
    Salt Lake City, Utah 84113, United States
  • Medical College Of Wisconsin
    Milwaukee, Wisconsin 53226-3522, United States
  • Universitair Ziekenhuis Gent - UZ GENT
    Gent, 9000, Belgium
  • CHU de Bordeaux, Hopital des Enfants
    Bordeaux, 33076, France
  • IHOPE - Hospices civils de Lyon
    Lyon, 69008, France
  • Hopital trousseau- APHP
    Paris, 75012, France
  • Hôpital Robert Debré
    Paris, 75019, France
  • Hôpital D'Enfants
    Vandoeuvre les Nancy, 54500, France
  • Charite-Universitätsmedizin Berlin - Berlin
    Berlin, 13353, Germany
  • Medizinische Hochschule Hannover
    Hannover, 30625, Germany
  • Universitatsklinikum Munster
    Münster, 48149, Germany
  • Schneider Children's Medical Center
    Petach Tiquva, 4920235, Israel
  • Istituto Giannina Gaslini
    Genova, 16147, Italy
  • Fondazione MBBM, ASST Monza
    Monza, 20900, Italy
  • Ospedale Pediatrico Bambin Gesù
    Roma, 00165, Italy
  • AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
    Torino, 10126, Italy
  • Princess Maxima Center
    Utrecht, 3584 EA, Netherlands
  • Hosp Univ Vall D Hebron
    Barcelona, 8035, Spain
  • Hosp. Sant Joan de Deu
    Esplugues de Llobregat, 08950, Spain
  • Hosp. Infantil Univ. Nino Jesus
    Madrid, 28009, Spain
  • Hosp. Univ. I Politecni La Fe
    Valencia, 46026, Spain
  • Karolinska University Hospital
    Stockholm, 17176, Sweden
  • Bristol Royal Hospital for Children
    Bristol, BS2 8BJ, United Kingdom
  • Royal Hospital for Sick Children
    Glasgow, G51 4TF, United Kingdom
  • Leeds Children's Hospital
    Leeds, LS1 3EX, United Kingdom
  • University College London Hospitals
    London, NW1 2BU, United Kingdom
  • Great Ormond Street Hospital
    London, WC1N 2JH, United Kingdom
  • Royal Manchester Children's Hospital
    Manchester, M13 9WL, United Kingdom
  • Royal Marsden Hospital
    Surrey, SM2 5PT, United Kingdom
08

References and documents

Publications

  • Ruhayel SD, Valvi S. Daratumumab in T-cell acute lymphoblastic leukaemia: A case report and review of the literature. Pediatr Blood Cancer. 2021 May;68(5):e28829. doi: 10.1002/pbc.28829. Epub 2020 Nov 27. No abstract available. PubMed 33245179 ↗

Study documents

  • Study protocol · Jan 22, 2019
  • Statistical analysis plan · Nov 9, 2022

Documents are hosted by the registry — open the source record to download them.

09

Registry details

Key details

Study ID
NCT03384654
Lead sponsor
Janssen Research & Development, LLC
Responsible party
Sponsor
First posted
Dec 27, 2017
Start date
May 14, 2018
Primary completion
Sep 22, 2022
Completion
Sep 27, 2022
Results posted
Oct 13, 2023
Last update
May 25, 2025

Study contacts

Janssen Research & Development, LLC Clinical Trial
study director · Janssen Research & Development, LLC

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in May 2025. You cannot join it, but the record below documents what was studied.

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