NCT05327894

Brief Summary

This study is a treatment protocol with blinatumomab for infants under 1 year old who are diagnosed with acute lymphoblastic leukemia with a specific unfavorable genetic alteration. The purpose of the study is to improve the outcome of this disease in infants.

Trial Health

88
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
160

participants targeted

Target at P25-P50 for phase_3

Timeline
53mo left

Started Dec 2022

Longer than P75 for phase_3

Geographic Reach
23 countries

111 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress44%
Dec 2022Sep 2030

First Submitted

Initial submission to the registry

April 7, 2022

Completed
7 days until next milestone

First Posted

Study publicly available on registry

April 14, 2022

Completed
8 months until next milestone

Study Start

First participant enrolled

December 15, 2022

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2027

Expected
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2030

Last Updated

April 13, 2026

Status Verified

April 1, 2026

Enrollment Period

4.7 years

First QC Date

April 7, 2022

Last Update Submit

April 8, 2026

Conditions

Keywords

infant under one yearKMT2A-wildtype

Outcome Measures

Primary Outcomes (1)

  • Event free survival (EFS).

    The primary endpoint is EFS, defined as the time from diagnosis to resistance to induction, relapse, death from any cause or second malignancy (whichever occurs first), or time to last follow-up (censored) for patients without events.

    5 years

Secondary Outcomes (9)

  • Overall survival

    8 years

  • Endpoints by risk group

    8 years

  • Outcome for the entire study cohort and according to risk group

    8 years

  • Minimal Residual Disease

    8 years

  • CD19 (cluster of differentiation antigen 19) negative relapse

    8 years

  • +4 more secondary outcomes

Study Arms (2)

Medium Risk (MR)

OTHER

Subject is defined as MR if \> 6months of age at diagnosis, OR \< 6 months of age with White Blood cell Count (WBC) \< 300 at diagnosis and good prednisone response. Subject gets 1st cycle of blinatumomab. If MRD is \>0.01%, after 1st cycle of blinatumomab, subject will be allocated to HR treatment from that phase, and will be eligible for HSCT. If MRD is undetectable or \< 0.01% after the 1st cycle of blinatumomab (TP2) patient will be eligible for replacement of MARMA by 2nd cycle of blinatumomab after receipt of lymphoid style consolidation (Protocol IB) or of myeloid style consolidation (ADE/MAE).

Drug: Blinatumomab

High risk (HR)

OTHER

Subject is defined as HR if \< 6 months of age with WBC \> 300 at diagnosis OR poor prednisone response. Also MR patients with end of induction MRD ≥ 1%, or MRD \> 0.01% after the 1st cycle of blinatumomab, will be allocated to HR treatment. Subject gets 1 cycle of blinatumomab. Thereafter patient is eligible for hematopoietic stem cell transplantation (HSCT) with or without experimental therapy in an investigational window.

Drug: Blinatumomab

Interventions

1st cycle: 15 μg/m2/day as a 4 week continuous IV infusion for patients with a M1 marrow. For patients with a M2/M3 marrow a step-dosing strategy is required with a dose of 5 μg/m2/day in week 1 followed by 15 μg/m2/day in weeks 2, 3, and 4.

Also known as: Cycle 1
High risk (HR)Medium Risk (MR)

Eligibility Criteria

Age1 Day - 1 Year
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Patients with newly diagnosed B- precursor ALL or B-cell MPAL (single lineage) according to the WHO classification of tumours of haematopoietic and lymphoid tissues (revised 4th edition 2017), with KMT2A-rearrangement.
  • ≤ 365 days of age at the time of diagnosis of ALL.
  • Written informed consent of the parent(s) or other legally authorized guardian of the patient according to local law and regulations.

You may not qualify if:

  • KMT2A-wildtype patients.
  • Multilineage MPAL
  • T-ALL.
  • Age \> 365 days at the time of diagnosis.
  • Down syndrome.
  • Relapsed ALL.
  • Treatment with systemic corticosteroids (equivalent prednisone \>10 mg/m2/day) for more than one week and/or any chemotherapeutic agent in the 4-week interval prior to diagnosis. Patients who received corticosteroids by aerosol are eligible for the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (115)

Hospital de Pediatría S.A.M.I.C. "Juan P. Garrahan"

Buenos Aires, Argentina

SUSPENDED

Australian and New Zealand Children's Haematology/Oncology Group

Clayton, Victoria, Australia

RECRUITING

North Adelaide- Womens and Childrens Hospital

Adelaide, Australia

ACTIVE NOT RECRUITING

Monash Children's Hosptial

Clayton, Australia

ACTIVE NOT RECRUITING

New Lambton Heights- John Hunter Children's Hospital

New Lambton Heights, Australia

ACTIVE NOT RECRUITING

Royal Children's Hospital (Children's Cancer Centre)

Parkville, Australia

ACTIVE NOT RECRUITING

Perth Children's Hospital

Perth, Australia

RECRUITING

Queensland Children's Hospital

South Brisbane, Australia

ACTIVE NOT RECRUITING

Sydney Childrens Hospital

Sydney, Australia

ACTIVE NOT RECRUITING

The Childrens Hospital at Westmead

Westmead, Australia

ACTIVE NOT RECRUITING

Medical University Of Graz

Graz, Austria

ACTIVE NOT RECRUITING

Medical University Of Innsbruck

Innsbruck, Austria

RECRUITING

Gemeinnutzige Salzburger Landes kliniken Betriebsgesellschaft mbH

Salzburg, Austria

RECRUITING

St. Anna Children's Hospital

Vienna, Austria

RECRUITING

Antwerp University Hospital

Antwerp, Belgium

ACTIVE NOT RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, Belgium

ACTIVE NOT RECRUITING

Hôpital Universitaire des Enfants Reine Fabiola

Brussels, Belgium

ACTIVE NOT RECRUITING

Universitair Ziekenhuis Gent

Ghent, Belgium

ACTIVE NOT RECRUITING

UZ Leuven

Leuven, Belgium

ACTIVE NOT RECRUITING

Centre Hospitalier Regional De La Citadelle

Liège, Belgium

ACTIVE NOT RECRUITING

CHC MontLegia

Liège, Belgium

ACTIVE NOT RECRUITING

University Hospital Brno

Brno, Czechia

ACTIVE NOT RECRUITING

University Hospital Olomouc

Olomouc, Czechia

ACTIVE NOT RECRUITING

Hospital Motol V Uvalu 841

Prague, Czechia

RECRUITING

AUH Skejby

Aarhus, Denmark

RECRUITING

Copenhagen-Rigshospitalet

Copenhagen, Denmark

RECRUITING

Odense University Hospital

Odense, Denmark

ACTIVE NOT RECRUITING

New Children's Hospital

Helsinki, Finland

ACTIVE NOT RECRUITING

Kuopio University Hospital

Kuopio, Finland

RECRUITING

Oulu University Hospital

Oulu, Finland

ACTIVE NOT RECRUITING

Tampere University Hospital

Tampere, Finland

RECRUITING

CHU Amiens

Amiens, France

RECRUITING

CHU Besancon

Besançon, France

ACTIVE NOT RECRUITING

CHU de Bordeaux

Bordeaux, France

RECRUITING

HCE

Grenoble, France

ACTIVE NOT RECRUITING

CHRU de Lille

Lille, France

ACTIVE NOT RECRUITING

Institute of Hematology and Pediatric Oncology

Lyon, France

ACTIVE NOT RECRUITING

CHU Timone

Marseille, France

ACTIVE NOT RECRUITING

CHI Montpellier

Montpellier, France

ACTIVE NOT RECRUITING

CHU Nancy

Nancy, France

ACTIVE NOT RECRUITING

CHU Nantes

Nantes, France

ACTIVE NOT RECRUITING

CHU de Nice

Nice, France

ACTIVE NOT RECRUITING

Hôpital Robert Debré, APHP

Paris, France

ACTIVE NOT RECRUITING

TRS

Paris, France

RECRUITING

CHU Reims

Reims, France

RECRUITING

CHU Rennes

Rennes, France

RECRUITING

CHU Charles Nicolle

Rouen, France

ACTIVE NOT RECRUITING

CHRU Strasbourg Hautepierre

Strasbourg, France

ACTIVE NOT RECRUITING

Universitätsklinikum Augsburg

Augsburg, Germany

ACTIVE NOT RECRUITING

Charite Universitaetsmedizin Berlin KöR

Berlin, Germany

RECRUITING

Universitaetsklinikum Bonn AöR

Bonn, Germany

ACTIVE NOT RECRUITING

Klinikum Dortmund gGmbH

Dortmund, Germany

RECRUITING

Universitaetsklinikum Erlangen AöR

Erlangen, Germany

ACTIVE NOT RECRUITING

Justus-Liebig-Universitaet Giessen

Giessen, Germany

ACTIVE NOT RECRUITING

Universitaetsklinikum Halle (Saale) AöR

Halle, Germany

ACTIVE NOT RECRUITING

University Medical Center Hamburg-Eppendorf

Hamburg, Germany

RECRUITING

Universtitätsklinikum Eppendorf

Hamburg, Germany

ACTIVE NOT RECRUITING

Universitätsklinikum Heidelberg AöR

Heidelberg, Germany

ACTIVE NOT RECRUITING

Universitaetsklinikum Schleswig-Holstein AöR

Kiel, Germany

ACTIVE NOT RECRUITING

HELIOS Klinikum Krefeld GmbH

Krefeld, Germany

ACTIVE NOT RECRUITING

Johannes Gutenberg University Mainz

Mainz, Germany

ACTIVE NOT RECRUITING

Universitaetsklinikum Muenster AöR

Münster, Germany

ACTIVE NOT RECRUITING

Klinikum Der Landeshauptstadt Stuttgart gKAöR

Stuttgart, Germany

RECRUITING

Universitaetsklinikum Tuebingen AöR

Tübingen, Germany

ACTIVE NOT RECRUITING

Universitätsklinikum Ulm

Ulm, Germany

RECRUITING

Aghia Sophia' Children's Hospital

Athens, Greece

ACTIVE NOT RECRUITING

HeSPHO

Athens, Greece

ACTIVE NOT RECRUITING

University General Hospital Of Heraklion

Heraklion, Greece

ACTIVE NOT RECRUITING

Mitera

Marousi, Greece

ACTIVE NOT RECRUITING

University Semmelweis

Budapest, Hungary

ACTIVE NOT RECRUITING

University of Pécs

Pécs, Hungary

RECRUITING

National Children's Cancer Service

Dublin, Ireland

RECRUITING

Schneider Childrens Medical Center

Petah Tikva, Israel

RECRUITING

L'Azienda Ospedaliera Di Rilievo Nazionale Santobono-Pausilipon

Naples, Italy

RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù

Roma, Italy

RECRUITING

Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo

Trieste, Italy

RECRUITING

University Of Verona Medical School

Verona, Italy

RECRUITING

Chiba University Hospital

Chiba, Japan

ACTIVE NOT RECRUITING

Ehime University Hospital

Ehime, Japan

ACTIVE NOT RECRUITING

Hiroshima University Hospital

Hiroshima, Japan

ACTIVE NOT RECRUITING

Hokkaido University Hospital

Hokkaido, Japan

ACTIVE NOT RECRUITING

Hyogo Prefectural Kobe Childrens Hospital

Hyōgo, Japan

ACTIVE NOT RECRUITING

Kagoshima University Hospital

Kagoshima, Japan

ACTIVE NOT RECRUITING

Kanagawa Childrens Medical Center

Kanagawa, Japan

ACTIVE NOT RECRUITING

Kyoto Prefectural University of Medicine

Kyoto, Japan

ACTIVE NOT RECRUITING

Kyoto University Hospital

Kyoto, Japan

ACTIVE NOT RECRUITING

Mie University Hospital

Mie, Japan

ACTIVE NOT RECRUITING

Nagoya University Graduate School of Medicine

Nagoya, Japan

ACTIVE NOT RECRUITING

Osaka City General Hospital

Osaka, Japan

ACTIVE NOT RECRUITING

Osaka University Graduate School of Medicine 2-2

Osaka, Japan

ACTIVE NOT RECRUITING

Saitama Prefectural Childrens Medical Center

Saitama, Japan

ACTIVE NOT RECRUITING

Shizuoka Childrens Hospital

Shizuoka, Japan

ACTIVE NOT RECRUITING

National Center for Child Health and Development

Tokyo, Japan

ACTIVE NOT RECRUITING

The University of Tokyo Hospital

Tokyo, Japan

ACTIVE NOT RECRUITING

Tokyo Metropolitan Childre&#39;s Medical Center

Tokyo, Japan

ACTIVE NOT RECRUITING

Tohoku University Hospital

Tōhoku, Japan

ACTIVE NOT RECRUITING

Vilnius University Hospital Santaros Klinikos

Vilnius, Lithuania

ACTIVE NOT RECRUITING

Princess Máxima Center for pediatric oncology

Utrecht, Utrecht, 3584 CS, Netherlands

RECRUITING

Christchurch Children's Hospital

Christchurch, New Zealand

ACTIVE NOT RECRUITING

Haukeland University Hospital

Bergen, Norway

ACTIVE NOT RECRUITING

St. Olavs Hospital

Trondheim, Norway

RECRUITING

Instituto Portugues de Oncologica Lisboa

Lisbon, Portugal

ACTIVE NOT RECRUITING

Jeddah-King Abdulaziz Medical City

Jeddah, Saudi Arabia

ACTIVE NOT RECRUITING

King Abdulaziz Medical City, King Abdullah International Medical Research Center

Riyadh, Saudi Arabia

ACTIVE NOT RECRUITING

National Institute of Children's Diseases

Bratislava, Slovakia

ACTIVE NOT RECRUITING

Vall D'hebron Institut De Recerca

Barcelona, Spain

RECRUITING

Hospital Infantil Universitario Nino Jesus

Madrid, Spain

RECRUITING

University Hospital Virgen Del Rocio S.L.

Seville, Spain

ACTIVE NOT RECRUITING

Hospital Universitario Y Politecnico La Fe

Valencia, Spain

ACTIVE NOT RECRUITING

Childrens Cancer Center Queen Silvia Children´s Hospital, Sahlgrenska University Hospital

Gothenburg, Sweden

RECRUITING

Linkoping University Hospital

Linköping, Sweden

RECRUITING

Skane University Hospital

Lund, Sweden

ACTIVE NOT RECRUITING

Karolinska University Hospital

Stockholm, Sweden

ACTIVE NOT RECRUITING

Umea University Hospital

Umeå, Sweden

RECRUITING

Children's UH

Uppsala, Sweden

RECRUITING

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Biphenotypic, Acute

Interventions

blinatumomab

Condition Hierarchy (Ancestors)

Leukemia, LymphoidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Study Officials

  • Janine Stutterheim, Dr

    Princess Maxima Center for Pediatric Oncology in The Netherlands

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Definition: Provide details about the Interventional Study Model. Limit: 1000 characters.
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 7, 2022

First Posted

April 14, 2022

Study Start

December 15, 2022

Primary Completion (Estimated)

September 1, 2027

Study Completion (Estimated)

September 1, 2030

Last Updated

April 13, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

all individual participant data that underlie results in a publication

Shared Documents
CSR
Time Frame
The Clinical Study Report (CSR) will be made available within 6 months upon study end.
Access Criteria
A summary of the study results will be made public via www.clinical trials.gov as well as to Ethical committees/ Health Authorities and all participating patients by providing them through their treating physicians a patient letter with a summary of the results.

Locations