Phase 3 Trial of Blinatumomab vs Standard Chemotherapy in Pediatric Subjects With HIgh-Risk (HR) First Relapse B-precursor Acute Lymphoblastic Leukemia (ALL)

NCT ID: NCT02393859

Last Updated: 2024-05-29

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

111 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-11-10

Study Completion Date

2022-11-21

Brief Summary

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B-precursor ALL is an aggressive malignant disease. Therapy is usually stratified according to risk characteristics to ensure that appropriate treatment is administered to patients with high-risk of relapse. In general, pediatric treatment regimens are more intense than those employed in adults and include courses of combination chemotherapy. Standard of care chemotherapy is associated with considerable toxicity. There is a lack of novel treatment options for subjects who relapse or are refractory to treatment. Therefore, innovative therapeutic approaches are urgently needed. Blinatumomab is a bispecific single-chain antibody construct designed to link B cells and T cells resulting in T cell activation and a cytotoxic T cell response against CD19 expressing cells. This study will evaluate the event-free survival (EFS) after treatment with blinatumomab when compared to standard of care (SOC) chemotherapy. The effect of blinatumomab on overall survival and reduction of minimal residual disease compared to SOC chemotherapy will also be investigated.

Detailed Description

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Patients will be randomized in a 1:1 ratio to receive either one cycle of blinatumomab or one block of standard high-risk consolidation chemotherapy. Blinatumomab is administered as a continuous intravenous infusion (CIVI). One cycle of blinatumomab treatment includes 4 weeks of CIVI of blinatumomab. After completing consolidation therapy, the patients should undergo alloHSCT depending on their bone marrow status. The patients will be followed up until the last subject on study is 36 months following alloHSCT or has died, whichever is first.

Conditions

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Leukemia, Acute Lymphoblastic

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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High Risk Consolidation 3 (HC3) Chemotherapy

One week of treatment with HC3 followed by 3 weeks of no treatment. The standard intensive consolidation chemotherapy course HC3 includes dexamethasone (10 mg/m\^2/day intravenous \[IV\] on Days 1-6), vincrisitne (1.5 mg/m\^2/day IV on Days 1 and 6), daunorubicin (30 mg/m\^2 IV over 24 hours on Day 5), methotrexate (1 g/m\^2 IV over 36 hours on Day 1), ifosfamide (800 mg/m\^2 IV for 1 hour on Days 2-4), and pegylated \[PEG\]-asparaginase (1000 U/m\^2 IV for 2 hours or intramuscularly \[IM\] on Day 6) or, if allergic, erwinia-asparaginase (20,000 units/m\^2 IV or IM every 48 hours for a total of 6 doses).

Group Type ACTIVE_COMPARATOR

Dexamethasone

Intervention Type DRUG

10 mg/m\^2/day intravenous (IV) on Days 1-6

Vincrisitne

Intervention Type DRUG

1.5 mg/m\^2/day IV on Days 1 and 6

Daunorubicin

Intervention Type DRUG

30 mg/m\^2 IV over 24 hours on Day 5

Methotrexate

Intervention Type DRUG

1 g/m\^2 IV over 36 hours on Day 1

Ifosfamide

Intervention Type DRUG

800 mg/m\^2 IV for 1 hour on Days 2-4

PEG-asparaginase

Intervention Type DRUG

1000 U/m\^2 IV for 2 hours or intramuscularly (IM) on Day 6

Erwinia-asparaginase

Intervention Type DRUG

In case of allergic reaction to PEG-asparaginase, participants could change to erwinia-asparaginase, 20,000 units/m2 every 48 hours for a total of 6 doses

Blinatumomab

15 μg/m\^2/day as a continuous intravenous infusion (CIVI) for 4 weeks

Group Type EXPERIMENTAL

Blinatumomab

Intervention Type DRUG

15 μg/m\^2/day as a continuous intravenous infusion (CIVI) for 4 weeks

Interventions

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Blinatumomab

15 μg/m\^2/day as a continuous intravenous infusion (CIVI) for 4 weeks

Intervention Type DRUG

Dexamethasone

10 mg/m\^2/day intravenous (IV) on Days 1-6

Intervention Type DRUG

Vincrisitne

1.5 mg/m\^2/day IV on Days 1 and 6

Intervention Type DRUG

Daunorubicin

30 mg/m\^2 IV over 24 hours on Day 5

Intervention Type DRUG

Methotrexate

1 g/m\^2 IV over 36 hours on Day 1

Intervention Type DRUG

Ifosfamide

800 mg/m\^2 IV for 1 hour on Days 2-4

Intervention Type DRUG

PEG-asparaginase

1000 U/m\^2 IV for 2 hours or intramuscularly (IM) on Day 6

Intervention Type DRUG

Erwinia-asparaginase

In case of allergic reaction to PEG-asparaginase, participants could change to erwinia-asparaginase, 20,000 units/m2 every 48 hours for a total of 6 doses

Intervention Type DRUG

Other Intervention Names

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Blincyto, AMG103

Eligibility Criteria

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Inclusion Criteria

* Subjects with Philadelphia chromosome negative (Ph-) high-risk (HR) first relapse B-precursor acute lymphoblastic leukemia (ALL; as defined by International Berlin-Frankfurt-Muenster study group/International study for treatment of childhood relapsed ALL \[I-BFM SG/IntReALL\] criteria)
* Subjects with bone marrow blast percentage \< 5% (M1) or bone marrow blast percentage \< 25% and ≥5% (M2) marrow at the time of randomization,
* Age \> 28 days and \< 18 years at the time of informed consent/assent
* Subject's legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated
* Availability of the following material from relapse diagnosis for central analysis of minimal residual disease (MRD) by polymerase chain reaction (PCR): clone-specific primers and reference deoxyribonucleic acid (DNA), as well as primer sequences and analyzed sequences of clonal rearrangements (cases with isolated extramedullary relapse or cases with technical and/or logistic hurdles to obtain and process bone marrow material are exempt from providing this material. In these cases, central MRD analysis only by Flow is permitted).

Exclusion Criteria

* Clinically relevant central nervous system (CNS) pathology requiring treatment (eg, unstable epilepsy). Evidence of current CNS (CNS 2, CNS 3) involvement by ALL. Subjects with CNS relapse at the time of relapse are eligible if CNS is successfully treated prior to enrollment
* Peripheral neutrophils \< 500/μL prior to start of treatment
* Peripheral platelets \< 50,000/μL prior to start of treatment
* Currently receiving treatment in another investigational device or drug study or less than 4 weeks since ending treatment on another investigational device or drug study(s), procedures required by IntReALL high-risk (HR) guidelines are allowed
* Symptoms and/or clinical signs and/or radiological and/or sonographic signs that indicate an acute or uncontrolled chronic infection, any other concurrent disease or medical condition that could be exacerbated by the treatment or would seriously complicate compliance with the protocol
* Abnormal renal or hepatic function prior to start of treatment (day 1) as defined below
* Abnormal serum creatinine based on age/gender
* Total bilirubin \> 3.0 mg/dL prior to start of treatment (unless related to Gilbert's or Meulengracht disease)
* Documented infection with human immunodeficiency virus (HIV)
* Known hypersensitivity to immunoglobulins or any of the products or components to be administered during dosing (excluding asparaginase)
* Post-menarchal female subject who is pregnant or breastfeeding, or is planning to become pregnant or breastfeed while receiving protocol-specified therapy and for at least 12 months after the last dose of chemotherapy
* Post-menarchal female subject who is not willing to practice true sexual abstinence or use a highly effective form of contraception while receiving protocol-specified therapy and for at least 12 months after the last dose of chemotherapy
* Sexually mature male subject who is not willing to practice true sexual abstinence or use a condom with spermicide while receiving protocol-specified therapy and for at least 6 months after last dose of chemotherapy. In countries where spermicide is not available, a condom without spermicide is acceptable
* Sexually mature male subject who is not willing to abstain from sperm donation while receiving protocol-specified therapy and for at least 6 months after last dose of chemotherapy
* Subject likely to not be available to complete all protocol-required study visits or procedures, including follow-up visits, and/or to comply with all required study procedures to the best of the subject's and investigator's knowledge
* History or evidence of any other clinically significant disorder, condition or disease (with the exception of those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion
* Placed into an institution due to juridical or regulatory ruling.
Minimum Eligible Age

0 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amgen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MD

Role: STUDY_DIRECTOR

Amgen

Locations

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Research Site

Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina

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Randwick, New South Wales, Australia

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Westmead, New South Wales, Australia

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South Brisbane, Queensland, Australia

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Parkville, Victoria, Australia

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Graz, , Austria

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Innsbruck, , Austria

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Vienna, , Austria

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Brussels, , Belgium

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Brussels, , Belgium

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Ghent, , Belgium

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Leuven, , Belgium

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Liège, , Belgium

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Curitba, Paraná, Brazil

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Porto Alegre, Rio Grande do Sul, Brazil

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São Paulo, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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Prague, , Czechia

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København Ø, , Denmark

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Bordeaux, , France

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Lille, , France

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Lyon, , France

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Marseille, , France

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Montpellier, , France

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Nantes, , France

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Paris, , France

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Paris, , France

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Strasbourg, , France

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Vandœuvre-lès-Nancy, , France

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Berlin, , Germany

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Düsseldorf, , Germany

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Erlangen, , Germany

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Essen, , Germany

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Frankfurt am Main, , Germany

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Freiburg im Breisgau, , Germany

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Giessen, , Germany

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Hamburg, , Germany

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Hanover, , Germany

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Jena, , Germany

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Kiel, , Germany

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München, , Germany

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Münster, , Germany

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Tübingen, , Germany

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Ulm, , Germany

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Würzburg, , Germany

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Goudi, , Greece

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Haifa, , Israel

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Jerusalem, , Israel

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Petah Tikva, , Israel

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Tel Aviv, , Israel

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Tel Litwinsky, , Israel

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Bologna, , Italy

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Genova, , Italy

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Monza (MB), , Italy

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Napoli, , Italy

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Padua, , Italy

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Pavia, , Italy

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Roma, , Italy

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Roma, , Italy

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Torino, , Italy

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Guadalajara, Jalisco, Mexico

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Mexico City, Mexico City, Mexico

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Monterrey, Nuevo León, Mexico

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Rotterdam, , Netherlands

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Utrecht, , Netherlands

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Oslo, , Norway

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Bydgoszcz, , Poland

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Krakow, , Poland

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Lublin, , Poland

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Wroclaw, , Poland

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Zabrze, , Poland

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Lisbon, , Portugal

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Porto, , Portugal

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Bucharest, , Romania

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Cluj-Napoca, , Romania

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Moscow, , Russia

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Saint Petersburg, , Russia

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Málaga, Andalusia, Spain

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Seville, Andalusia, Spain

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Santander, Cantabria, Spain

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Barcelona, Catalonia, Spain

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Barcelona, Catalonia, Spain

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Santiago de Compostela, Galicia, Spain

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Boadilla del Monte, Madrid, Spain

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El Palmar, Murcia, Spain

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Valencia, Valencia, Spain

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Madrid, , Spain

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Madrid, , Spain

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Stockholm, , Sweden

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Basel, , Switzerland

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Zurich, , Switzerland

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Adana, , Turkey (Türkiye)

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Antalya, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Kayseri, , Turkey (Türkiye)

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Birmingham, , United Kingdom

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Bristol, , United Kingdom

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Glasgow, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Newcastle upon Tyne, , United Kingdom

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Sheffield, , United Kingdom

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Sutton, , United Kingdom

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Countries

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Argentina Australia Austria Belgium Brazil Czechia Denmark France Germany Greece Israel Italy Mexico Netherlands Norway Poland Portugal Romania Russia Spain Sweden Switzerland Turkey (Türkiye) United Kingdom

References

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Locatelli F, Eckert C, Hrusak O, Buldini B, Sartor M, Zugmaier G, Zeng Y, Pilankar D, Morris J, von Stackelberg A. Blinatumomab overcomes poor prognostic impact of measurable residual disease in pediatric high-risk first relapse B-cell precursor acute lymphoblastic leukemia. Pediatr Blood Cancer. 2022 Aug;69(8):e29715. doi: 10.1002/pbc.29715. Epub 2022 Apr 28.

Reference Type BACKGROUND
PMID: 35482538 (View on PubMed)

Blinatumomabe em pacientes pediátricos com leucemia linfoblástica aguda B em primeira recidiva de alto risco: um estudo de custo-efetividade. van Beurden-Tan CHY, Ribeiro RA, Seber A, de Martino Lee ML, Marçola M, Schuetz R, Loggetto SR, Maiolino A. Jornal Brasileiro de Economia da Saúde 14(1): 41-50. 10.21115/JBES.v14.n1.p41-50

Reference Type BACKGROUND

Locatelli F, Zugmaier G, Rizzari C, Morris JD, Gruhn B, Klingebiel T, Parasole R, Linderkamp C, Flotho C, Petit A, Micalizzi C, Mergen N, Mohammad A, Kormany WN, Eckert C, Moricke A, Sartor M, Hrusak O, Peters C, Saha V, Vinti L, von Stackelberg A. Effect of Blinatumomab vs Chemotherapy on Event-Free Survival Among Children With High-risk First-Relapse B-Cell Acute Lymphoblastic Leukemia: A Randomized Clinical Trial. JAMA. 2021 Mar 2;325(9):843-854. doi: 10.1001/jama.2021.0987.

Reference Type BACKGROUND
PMID: 33651091 (View on PubMed)

Caillon M, Brethon B, van Beurden-Tan C, Supiot R, Le Mezo A, Chauny JV, Majer I, Petit A. Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France. Pharmacoecon Open. 2023 Jul;7(4):639-653. doi: 10.1007/s41669-023-00411-4. Epub 2023 Apr 18.

Reference Type BACKGROUND
PMID: 37071263 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

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2014-002476-92

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

20120215

Identifier Type: -

Identifier Source: org_study_id

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