Acute Lymphoblastic Leukemia (ALL)Phase 2/3FORUM2NCT07297914
Framework for Optimizing, Refining, and Unifying Management of HSCT in Pediatric ALL
A Phase 2/3 study testing Total Body Irradiation 8 Gy, Ruxolitinib and Blinatumomab in people with Acute Lymphoblastic Leukemia (ALL) and Stem Cell Transplant, run by Bambino Gesù Hospital and Research Institute at 9 sites in 9 countries.
Trial summary
Current therapeutic strategies for high-risk or relapsed ALL patients often involve intensive treatments, including allogeneic hematopoietic stem cell transplantation (HSCT). HSCT remains a cornerstone of therapy, offering curative potential; however, it is associated with considerable risks, including non-relapse… Read more about this study
- Who is it for?
- Ages 3 months to 25 years
- Conditions the study is for
- Acute Lymphoblastic Leukemia (ALL), Stem Cell Transplant, Graft -Versus-host-disease
- What is being tested?
- Total Body Irradiation 8 Gy, Ruxolitinib and Blinatumomab
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1,000 participants
- Where
- 9 sitesin 9 countries
- Study dates
- 7 Jan 2026to 1 Dec 2032
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male and female patients with allogenic transplant indication for ALL, as determined by national frontline protocols
- Age ≥3 months to ≤25 years at the time of HSCT.
- Patients must be in complete remission (with <5% blasts and absence of leukemia cells in extramedullary sites) prior to undergoing HSCT.
- Selected donor must be either a matched donor (matched donor category includes 9/10 identical siblings and 10/10 or 9/10 HLA-matched unrelated donors) or a mismatched family donor (≤8/10 HLA match). Either bone marrow or peripheral blood stem cell grafts are permitted. Cord blood is permitted, as well, provided that the unit is at least 6/8 HLA matched and with a cryopreserved cellularity of at least 3x107 nucleated cells/Kg recipient body weight.
- Female patients of childbearing potential must have a negative pregnancy test at screening, and all patients must agree to adhere to effective contraception during the study period.
- Written study informed consent and/or assent from the patient and/or the parent, or guardian
Who cannot join
- Patients < 3 months and > 25 years of age at the time of HSCT.
- Patients not in complete morphological remission at the time of enrollment.
- Patients with an initial diagnosis of Non-Hodgkin Lymphoma (NHL).
- Patients with ALL as a secondary malignancy.
- Patients with a history of previous autologous or allogeneic HSCT (prior allogeneic transplantation is permitted for subjects receiving post-transplant interventions, such as those enrolled in the R2 and P1 substudies, provided that this is their first allogeneic HSCT).
- Female patients who are pregnant or breast feeding.
Show all 28Show fewer
- Fertile male or female patients of childbearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of contraception.
- Active clinically uncontrolled bacterial, fungal, parasitic, or viral infection. Infections are considered controlled if appropriate therapy has been instituted and, at the time of screening, no physical or radiographic signs of infection progression are present.
- Active HBV or HCV infection that requires treatment, or at risk for HBV reactivation (e.g. positive HBsAg). Subjects with negative HbsAg and positive total HB core antibody may be included if HBV DNA is undetectable at the time of screening. Subjects who are positive for HCV antibody are eligible only if polymerase chain reaction test is negative for HCV RNA. Subjects whose immune status is unknown or uncertain must have results confirming immune status before enrollment. Prior serology results are acceptable for determining eligibility.
- Known human immunodeficiency virus infection (HIV).
- Significant respiratory disease including patients who are on mechanical ventilation or who have resting O2 saturation <90% by pulse-oximetry on room-air.
- Presence of severely impaired renal function (confirmed within 72 hours prior to study treatment start) defined by:
- Glomerular Filtration Rate (GFR) < 30 mL/min/1.73 m2 using estimated creatinine clearance calculated by updated bedside Schwartz equation or Cockcroft Gault equation OR
- Renal dialysis requirement
- Clinically significant or uncontrolled cardiac disease including any of the following:
- Uncontrolled hypertension
- New York Heart Association Class III or IV congestive heart failure
- Clinically significant cardiac arrhythmias
- Severe hepatic insufficiency, defined by any of the following:
- Child-Pugh Class C liver disease
- AST (aspartate aminotransferase) or ALT (alanine aminotransferase) levels > 5 times the upper limit of normal (ULN), unless attributable to GvHD
- Total bilirubin > 3.0 mg/dL, unless attributable to GvHD
- INR (International Normalized Ratio) ≥ 1.7
- Clinical evidence of hepatic encephalopathy or ascites
- Presence of severe concomitant constitutional disease that precludes treatment as per protocol, based on the investigator's judgment. Examples include but are not limited to: Down syndrome with severe comorbidities, significant cardiac malformations, and metabolic disorders affecting treatment feasibility.
- Underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere participation in the study, pose a significant risk to the patient or interfere with interpretation of study data.
- Karnofsky or Lansky performance score <50%, indicating significant functional impairment.
- Patients who are unwilling or unable to comply with study procedures, including follow-up requirements and treatment schedules.
Trial locations and contacts
9 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 9 countries
Austria1 site, 1 recruiting
- Not yet recruiting
St'Anna Children Hospital
Vienna
Denmark1 site, 1 recruiting
- Not yet recruiting
Rigshopsitalet, University Hospital
Copenhagen
Finland1 site, 1 recruiting
- Not yet recruiting
HUS-Yhtymae (HUS Helsinki University Hospital)
Helsinki
France1 site, 1 recruiting
- Not yet recruiting
Robert- Debré Academic Hospital
Paris
Italy1 site, 1 recruiting
- Not yet recruiting
IRCCS Ospedale Pediatrico Bambino Gesù
Roma, RM, 00165
Poland1 site, 1 recruiting
- Not yet recruiting
University of Medical Sciences
Poznan
About this study
Current therapeutic strategies for high-risk or relapsed ALL patients often involve intensive treatments, including allogeneic hematopoietic stem cell transplantation (HSCT). HSCT remains a cornerstone of therapy, offering curative potential; however, it is associated with considerable risks, including non-relapse mortality (NRM), significant morbidity, and long-term complications that continue to be major concerns.
In response to these challenges, the FORUM consortium has made substantial progress in improving outcomes for children with ALL undergoing HSCT. The consortium focuses on reducing life-threatening and lifelong complications, ultimately aiming to enhance quality of life for these high-risk patients. Building on the robust evidence generated by FORUM1, the FORUM2 study has been designed to further optimize the role of HSCT in ALL across all age groups and donor settings within a harmonized and internationally coordinated framework.
The FORUM2 study introduces a master protocol structure that encompasses multiple hypothesis-driven substudies, each addressing a specific determinant of HSCT outcomes. This design enables simultaneous or sequential evaluation of novel strategies while ensuring uniform governance, endpoint definitions, and data-quality standards. The overarching objective is to refine the role of HSCT in ALL by reducing treatment-related toxicity while preserving the essential graft-versus-leukemia effect.
Read the detailed description
The key focus areas and objectives include:
- Optimization of conditioning regimens
- Advancements in GvHD prevention and treatment
- Integration of novel immunotherapies
- Improvement of long-term survivorship
- Harmonization of supportive care and post-transplant monitoring
- Expansion of donor availability The FORUM2 study comprises two randomized comparisons (R1 and R2 substudies), one stratified cohort (S1 substudy), and one pilot cohort (P1 substudy). The protocol is structured as a master protocol, with the R1 substudy serving as the central component because it represents the continuation of the FORUM1 study (which compared TBI-based and chemotherapy-based conditioning) and is expected to enroll the majority of patients.
Patients ineligible for the R1 substudy-due to age (<2 years), donor type, physician discretion, or personal preference-will still be included in the master protocol and monitored accordingly. Patients transplanted from a mismatched family donor will be stratified within the S1 substudy. Additionally, patients younger than 2 years of age with B-ALL are eligible for the P1 pilot substudy, which will investigate the use of post-HSCT blinatumomab to reduce relapse incidence in this high-risk population.
Primary and secondary endpoints, general assessment timelines, and supportive care guidelines will remain consistent for both R1 substudy participants and patients included in the broader master protocol. Additional assessments, endpoints, and interventions specific to other study groups are outlined in their respective protocol sections (or appendices) and will be conducted exclusively for patients enrolled in those specific cohorts.
- Being tested
- Total Body Irradiation 8 Gy, Ruxolitinib, Blinatumomab, Cyclophosphamide, Total Body Irradiation 12 Gy
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Total Body Irradiation 8 GyRadiation
Total Body Irradiation 8 Gy administered in combination with VP16 as part of the conditioning regimen
RuxolitinibCombination product
Ruxolitinib plus corticosteroids in treatment-naïve acute graft-versus-host disease
BlinatumomabDrug
Up to four cycles of blinatumomab as post-HSCT maintenance therapy
CyclophosphamideDrug
In vivo T-cells depletion/modulation with post-transplant cyclophosphamide
Total Body Irradiation 12 GyRadiation
Total Body Irradiation 12 Gy administered in combination with VP16 as part of the conditioning regimen
CorticosteroidsDrug
Corticosteroids alone in treatment-naïve acute graft-versus-host disease
αβ T-cells depletionOther
Ex vivo graft manipulation based on selective depletion of T-cell receptor αβ (TCR αβ+)/CD19+ lymphocytes from the graft (αβ T-cells depletion)
| Study group | Role | What they receive |
|---|---|---|
| R1 substudy | Experimental | A phase III, randomized, multi-center study comparing TBI 8 Gy versus TBI 12 Gy in children and young adults with ALL for conditioning of allogeneic HSCT Receives: Radiation: Total Body Irradiation 8 Gy; Radiation: Total Body Irradiation 12 Gy |
| R2 substudy | Experimental | A phase III, randomized, open-label multi-center study comparing ruxolitinib plus corticosteroids versus corticosteroids alone in children with ALL and treatment-naïve grade II-IV aGvHD following allogeneic HSCT Receives: Combination Product: Ruxolitinib; Drug: Corticosteroids |
| S1 substudy | Experimental | A prospective stratified cohort study comparing between in vivo PT-Cy and ex vivo αβ T-cell depletion in mismatched donor transplantation for pediatric and young adult ALL. Receives: Drug: Cyclophosphamide; Other: αβ T-cells depletion |
| P1 substudy | Active comparator | A phase II, open-label, multi-center study of blinatumomab following allogeneic HSCT for B-lineage ALL in children younger than 2 years not receiving TBI as part of the conditioning regimen Receives: Drug: Blinatumomab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event Free Survival (EFS)
EFS is defined as the time from randomization (intention-to-treat analysis) or HSCT (per-protocol/as treated) to first failure event defined as follows:
Failure events are:
- Relapse
- Graft failure
- Death from any cause
- Diagnosis of a second malignant neoplasm
Measured: at year 4
Overall response rate (ORR) at day 28
Overall response rate (ORR) at day 28 after randomization, defined as the proportion of patients in each arm demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for earlier progression, mixed response or nonresponse.
Measured: day 28
Event Free Survival (EFS)
EFS is defined as the time from HSCT to first failure event defined as follows:
Failure events are:
- Relapse
- Graft failure
- Death from any cause
- Diagnosis of a second malignant neoplasm
Measured: at year 4
Cumulative incidence of relapse (CIR) at 2 years after HSCT
CIR at 2 years after HSCT in blinatumomab-treated patents and historical controls. CIR is calculated from the time of study enrolment until the date of relapse (defined as either bone marrow aspirate or biopsy with ≥ 5% blasts or as appearance of leukemia cells in an extramedullary site) or last follow-up (death from any cause other than leukemia relapse and secondary malignancies will be considered a competing event)
Measured: 2 years after HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2025
- Study start7 Jan 2026
- Primary completion30 Nov 2032 (estimated)
- Study completion1 Dec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Acute Lymphoblastic Leukemia (ALL)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The FORUM2 study has a flexible design which consists of: * Two randomized comparison groups (R1 SUBSTUDY and R2 SUBSTUDY), * One stratified cohort (S1 SUBSTUDY), * One pilot cohort (P1 SUBSTUDY).
- Masking
- None (open label)
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bambino Gesù Hospital and Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Franco Locatelli, Professor, Principal investigator, IRCSS Ospedale Pediatrico Bambino Gesù
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07297914
- Sponsor's protocol ID
- FORUM2
- Other IDs
- 2025-522052-13-00, 2025
- Acronym
- FORUM2
- Status last verified
- Oct 2026
- First submitted
- 25 Nov 2025
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Oostenbrink LVE, Von Asmuth EGJ, Jol-van der Zijde CM, Jansen-Hoogendijk AM, Vervat C, Bredius RGM, Van Tol MJD, Schilham MW, Sedlacek P, Ifversen M, Balduzzi A, Bader P, Peters C, Moes DJAR, Lankester AC. Anti-T-lymphocyte globulin exposure is associated with acute graft-versus-host disease and relapse in pediatric acute lymphoblastic leukemia patients undergoing hematopoietic stem cell transplantation: a multinational prospective study. Haematologica. 2024 Sep 1;109(9):2854-2863. doi: 10.3324/haematol.2023.284632.
PubMed 38721739Peters C, Dalle JH, Locatelli F, Poetschger U, Sedlacek P, Buechner J, Shaw PJ, Staciuk R, Ifversen M, Pichler H, Vettenranta K, Svec P, Aleinikova O, Stein J, Gungor T, Toporski J, Truong TH, Diaz-de-Heredia C, Bierings M, Ariffin H, Essa M, Burkhardt B, Schultz K, Meisel R, Lankester A, Ansari M, Schrappe M; IBFM Study Group;; von Stackelberg A; IntReALL Study Group; Balduzzi A; I-BFM SCT Study Group; Corbacioglu S; EBMT Paediatric Diseases Working Party; Bader P. Total Body Irradiation or Chemotherapy Conditioning in Childhood ALL: A Multinational, Randomized, Noninferiority Phase III Study. J Clin Oncol. 2021 Feb 1;39(4):295-307. doi: 10.1200/JCO.20.02529. Epub 2020 Dec 17.
PubMed 33332189Bader P, Potschger U, Dalle JH, Moser LM, Balduzzi A, Ansari M, Buechner J, Gungor T, Ifversen M, Krivan G, Pichler H, Renard M, Staciuk R, Sedlacek P, Stein J, Heusel JR, Truong T, Wachowiak J, Yesilipek A, Locatelli F, Peters C. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study. Blood Adv. 2024 Jan 23;8(2):416-428. doi: 10.1182/bloodadvances.2023010591.
PubMed 37738088Kalwak K, Moser LM, Potschger U, Bader P, Kleinschmidt K, Meisel R, Dalle JH, Yesilipek A, Balduzzi A, Krivan G, Goussetis E, Staciuk R, Sedlacek P, Pichler H, Svec P, Gabriel M, Gungor T, Bilic E, Buechner J, Renard M, Vettenranta K, Ifversen M, Diaz-de-Heredia C, Stein J, Toporski J, Bierings M, Peters C, Ansari M, Locatelli F. Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM. Blood Adv. 2025 Feb 25;9(4):741-751. doi: 10.1182/bloodadvances.2024014548.
PubMed 39602342
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Framework for Optimizing, Refining, and Unifying Management of HSCT in Pediatric ALL
- ClinicalTrials.gov ID
- NCT07297914
- Acronym / sponsor ID
- FORUM2 / FORUM2
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Bambino Gesù Hospital and Research InstituteUniversity, hospital or other
- Study start
- 7 Jan 2026 (actual)
- Primary completion
- 30 Nov 2032 (estimated)
- Study completion
- 1 Dec 2032 (estimated)
- First posted
- 22 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Current therapeutic strategies for high-risk or relapsed ALL patients often involve intensive treatments, including allogeneic hematopoietic stem cell transplantation (HSCT). HSCT remains a cornerstone of therapy, offering curative potential; however, it is associated with considerable risks, including non-relapse mortality (NRM), significant morbidity, and long-term complications that continue to be major concerns.
In response to these challenges, the FORUM consortium has made substantial progress in improving outcomes for children with ALL undergoing HSCT. The consortium focuses on reducing life-threatening and lifelong complications, ultimately aiming to enhance quality of life for these high-risk patients. Building on the robust evidence generated by FORUM1, the FORUM2 study has been designed to further optimize the role of HSCT in ALL across all age groups and donor settings within a harmonized and internationally coordinated framework.
The FORUM2 study introduces a master protocol structure that encompasses multiple hypothesis-driven substudies, each addressing a specific determinant of HSCT outcomes. This design enables simultaneous or sequential evaluation of novel strategies while ensuring uniform governance, endpoint definitions, and data-quality standards. The overarching objective is to refine the role of HSCT in ALL by reducing treatment-related toxicity while preserving the essential graft-versus-leukemia effect.
Detailed description
The key focus areas and objectives include:
- Optimization of conditioning regimens
- Advancements in GvHD prevention and treatment
- Integration of novel immunotherapies
- Improvement of long-term survivorship
- Harmonization of supportive care and post-transplant monitoring
- Expansion of donor availability The FORUM2 study comprises two randomized comparisons (R1 and R2 substudies), one stratified cohort (S1 substudy), and one pilot cohort (P1 substudy). The protocol is structured as a master protocol, with the R1 substudy serving as the central component because it represents the continuation of the FORUM1 study (which compared TBI-based and chemotherapy-based conditioning) and is expected to enroll the majority of patients.
Patients ineligible for the R1 substudy-due to age (<2 years), donor type, physician discretion, or personal preference-will still be included in the master protocol and monitored accordingly. Patients transplanted from a mismatched family donor will be stratified within the S1 substudy. Additionally, patients younger than 2 years of age with B-ALL are eligible for the P1 pilot substudy, which will investigate the use of post-HSCT blinatumomab to reduce relapse incidence in this high-risk population.
Primary and secondary endpoints, general assessment timelines, and supportive care guidelines will remain consistent for both R1 substudy participants and patients included in the broader master protocol. Additional assessments, endpoints, and interventions specific to other study groups are outlined in their respective protocol sections (or appendices) and will be conducted exclusively for patients enrolled in those specific cohorts.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- The FORUM2 study has a flexible design which consists of: * Two randomized comparison groups (R1 SUBSTUDY and R2 SUBSTUDY), * One stratified cohort (S1 SUBSTUDY), * One pilot cohort (P1 SUBSTUDY).
| Arm | Type | Receives |
|---|---|---|
| R1 substudy | Experimental | Radiation: Total Body Irradiation 8 Gy, Radiation: Total Body Irradiation 12 GyA phase III, randomized, multi-center study comparing TBI 8 Gy versus TBI 12 Gy in children and young adults with ALL for conditioning of allogeneic HSCT |
| R2 substudy | Experimental | Combination Product: Ruxolitinib, Drug: CorticosteroidsA phase III, randomized, open-label multi-center study comparing ruxolitinib plus corticosteroids versus corticosteroids alone in children with ALL and treatment-naïve grade II-IV aGvHD following allogeneic HSCT |
| S1 substudy | Experimental | Drug: Cyclophosphamide, Other: αβ T-cells depletionA prospective stratified cohort study comparing between in vivo PT-Cy and ex vivo αβ T-cell depletion in mismatched donor transplantation for pediatric and young adult ALL. |
| P1 substudy | Active comparator | Drug: BlinatumomabA phase II, open-label, multi-center study of blinatumomab following allogeneic HSCT for B-lineage ALL in children younger than 2 years not receiving TBI as part of the conditioning regimen |
Who may qualify
- Ages
- 3 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Male and female patients with allogenic transplant indication for ALL, as determined by national frontline protocols
- Age ≥3 months to ≤25 years at the time of HSCT.
- Patients must be in complete remission (with <5% blasts and absence of leukemia cells in extramedullary sites) prior to undergoing HSCT.
- Selected donor must be either a matched donor (matched donor category includes 9/10 identical siblings and 10/10 or 9/10 HLA-matched unrelated donors) or a mismatched family donor (≤8/10 HLA match). Either bone marrow or peripheral blood stem cell grafts are permitted. Cord blood is permitted, as well, provided that the unit is at least 6/8 HLA matched and with a cryopreserved cellularity of at least 3x107 nucleated cells/Kg recipient body weight.
- Female patients of childbearing potential must have a negative pregnancy test at screening, and all patients must agree to adhere to effective contraception during the study period.
Key exclusion criteria (28 listed)
- Patients < 3 months and > 25 years of age at the time of HSCT.
- Patients not in complete morphological remission at the time of enrollment.
- Patients with an initial diagnosis of Non-Hodgkin Lymphoma (NHL).
- Patients with ALL as a secondary malignancy.
- Patients with a history of previous autologous or allogeneic HSCT (prior allogeneic transplantation is permitted for subjects receiving post-transplant interventions, such as those enrolled in the R2 and P1 substudies, provided that this is their first allogeneic HSCT).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Total Body Irradiation 8 GyTotal Body Irradiation 8 Gy administered in combination with VP16 as part of the conditioning regimen | Radiation | R1 substudy |
| RuxolitinibRuxolitinib plus corticosteroids in treatment-naïve acute graft-versus-host disease | Combination product | R2 substudy |
| BlinatumomabUp to four cycles of blinatumomab as post-HSCT maintenance therapy | Drug | P1 substudy |
| CyclophosphamideIn vivo T-cells depletion/modulation with post-transplant cyclophosphamide | Drug | S1 substudy |
| Total Body Irradiation 12 GyTotal Body Irradiation 12 Gy administered in combination with VP16 as part of the conditioning regimen | Radiation | R1 substudy |
| CorticosteroidsCorticosteroids alone in treatment-naïve acute graft-versus-host disease | Drug | R2 substudy |
| αβ T-cells depletionEx vivo graft manipulation based on selective depletion of T-cell receptor αβ (TCR αβ+)/CD19+ lymphocytes from the graft (αβ T-cells depletion) | Other | S1 substudy |
Primary outcome
Event Free Survival (EFS)
Time frame: at year 4
EFS is defined as the time from randomization (intention-to-treat analysis) or HSCT (per-protocol/as treated) to first failure event defined as follows: Failure events are: * Relapse * Graft failure * Death from any cause * Diagnosis of a second malignant neoplasm
Overall response rate (ORR) at day 28
Time frame: day 28
Overall response rate (ORR) at day 28 after randomization, defined as the proportion of patients in each arm demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for earlier progression, mixed response or nonresponse.
Event Free Survival (EFS)
Time frame: at year 4
EFS is defined as the time from HSCT to first failure event defined as follows: Failure events are: * Relapse * Graft failure * Death from any cause * Diagnosis of a second malignant neoplasm
Cumulative incidence of relapse (CIR) at 2 years after HSCT
Time frame: 2 years after HSCT
CIR at 2 years after HSCT in blinatumomab-treated patents and historical controls. CIR is calculated from the time of study enrolment until the date of relapse (defined as either bone marrow aspirate or biopsy with ≥ 5% blasts or as appearance of leukemia cells in an extramedullary site) or last follow-up (death from any cause other than leukemia relapse and secondary malignancies will be considered a competing event)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2026.
- Background: PubMed 38721739Oostenbrink LVE, Von Asmuth EGJ, Jol-van der Zijde CM, Jansen-Hoogendijk AM, Vervat C, Bredius RGM, Van Tol MJD, Schilham MW, Sedlacek P, Ifversen M, Balduzzi A, Bader P, Peters C, Moes DJAR, Lankester AC. Anti-T-lymphocyte globulin exposure is associated with acute graft-versus-host disease and relapse in pediatric acute lymphoblastic leukemia patients undergoing hematopoietic stem cell transplantation: a multinational prospective study. Haematologica. 2024 Sep 1;109(9):2854-2863. doi: 10.3324/haematol.2023.284632.
- Background: PubMed 33332189Peters C, Dalle JH, Locatelli F, Poetschger U, Sedlacek P, Buechner J, Shaw PJ, Staciuk R, Ifversen M, Pichler H, Vettenranta K, Svec P, Aleinikova O, Stein J, Gungor T, Toporski J, Truong TH, Diaz-de-Heredia C, Bierings M, Ariffin H, Essa M, Burkhardt B, Schultz K, Meisel R, Lankester A, Ansari M, Schrappe M; IBFM Study Group;; von Stackelberg A; IntReALL Study Group; Balduzzi A; I-BFM SCT Study Group; Corbacioglu S; EBMT Paediatric Diseases Working Party; Bader P. Total Body Irradiation or Chemotherapy Conditioning in Childhood ALL: A Multinational, Randomized, Noninferiority Phase III Study. J Clin Oncol. 2021 Feb 1;39(4):295-307. doi: 10.1200/JCO.20.02529. Epub 2020 Dec 17.
- Background: PubMed 37738088Bader P, Potschger U, Dalle JH, Moser LM, Balduzzi A, Ansari M, Buechner J, Gungor T, Ifversen M, Krivan G, Pichler H, Renard M, Staciuk R, Sedlacek P, Stein J, Heusel JR, Truong T, Wachowiak J, Yesilipek A, Locatelli F, Peters C. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study. Blood Adv. 2024 Jan 23;8(2):416-428. doi: 10.1182/bloodadvances.2023010591.
- Background: PubMed 39602342Kalwak K, Moser LM, Potschger U, Bader P, Kleinschmidt K, Meisel R, Dalle JH, Yesilipek A, Balduzzi A, Krivan G, Goussetis E, Staciuk R, Sedlacek P, Pichler H, Svec P, Gabriel M, Gungor T, Bilic E, Buechner J, Renard M, Vettenranta K, Ifversen M, Diaz-de-Heredia C, Stein J, Toporski J, Bierings M, Peters C, Ansari M, Locatelli F. Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM. Blood Adv. 2025 Feb 25;9(4):741-751. doi: 10.1182/bloodadvances.2024014548.
- Individual participant dataSharing planned: No
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Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
RecruitingObservational study
A Multi-site Study to Evaluate the Persistence of Protective Immunity to Routine Childhood Vaccinations in Participants With B-ALL/Ly Who Have Received Blinatumomab
B-Cell ALL, B-Cell Acute Lymphoblastic Leukaemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Franco Locatelli, Professor principal investigator
IRCSS Ospedale Pediatrico Bambino Gesù
9 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Total Body Irradiation 8 Gy, Ruxolitinib and Blinatumomab in people with Acute Lymphoblastic Leukemia (ALL) and Stem Cell Transplant, run by Bambino Gesù Hospital and Research Institute at 9 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Austria, Czechia, Denmark, Finland, France and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Franco Locatelli, Professor (+39 06 6859 3697, franco.locatelli@opbg.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event Free Survival (EFS) (time frame: at year 4). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07297914, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2026.