Capizzi Escalating Methotrexate Versus High Dose Methotrexate in Children with Newly Diagnosed T-cell Lymphoblastic Lymphoma (T-LBL)

NCT ID: NCT05681260

Last Updated: 2025-02-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-06

Study Completion Date

2029-12-31

Brief Summary

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T-cell lymphoblastic lymphoma (T-LBL) is the second most common subtype of non-Hodgkin lymphoma (NHL) in children and adolescents. With current treatment, event-free survival (EFS) rates vary between 75%\~85%. Two different MTX intensification strategies are used commonly: HD-MTX with leucovorin rescue, and Capizzi-style MTX without leucovorin rescue plus PEG-ASP (C-MTX). Although superior outcome of patients with T-ALL receiving C-MTX compared with HD-MTX on the AALL0434 trial, the 2 approaches had not been compared directly in patients with T-LBL. There remains controversy on PET/CT interpretation in children with NHL. Large prospective studies in pediatric patients with T-LBL regarding PET/CT value for this is scarce. Around 1% pediatric patients with T-LBL will not achieve remission at the end of Induction (induction failure). The optimal treatment for this small subgroup is largely unclear. The BFM HR Blocks usually are applied to these patients even though the efficacy is unknown. Novel targeted therapies are needed for use. Dasatinib is identified as a targeted therapy for T-cell ALL in preclinical drug screening.

Detailed Description

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1. T-cell lymphoblastic lymphoma (T-LBL) which involves 90% of LBL cases is the second most common subtype of non-Hodgkin lymphoma (NHL) in children and adolescents. With current treatment, event-free survival (EFS) rates vary between 75%\~85%. Poor probabilities of survival (10\~15%) for patients after relapse leave no room for treatment de-escalation in frontline protocols. Limitations in numbers of newly diagnosed patients impeded evaluation potential prognostic markers and validation or conducting clinical studies.
2. In the GER-GPOH-NHL-BFM-95 study, the prophylactic cranial radiation was omitted, and the intensity of induction therapy was decreased slightly. There were no significant increases in CNS relapses, suggesting cranial radiation may be reserved for patients with CNS disease at diagnosis. The 5-year EFS was worse in NHL-BFM-95 (82%) than in NHL-BFM-90 (90%). It was proposed that the major difference in EFS between NHL-BFM-90 and NHL-BFM-95 resulted from the increased number of subsequent neoplasms observed in NHL-BFM-95.
3. Two different MTX intensification strategies are used commonly: HD-MTX with leucovorin rescue, and Capizzi-style MTX without leucovorin rescue plus PEG-ASP (C-MTX). Although superior outcome of patients with T-ALL receiving C-MTX compared with HD-MTX on the AALL0434 trial, the 2 approaches had not been compared directly in patients with T-LBL.
4. POG 9404: the small cohort (n = 66) of lymphoma patients who did not receive HD-MTX, the 5-year EFS was 88%. Of note, all of these patients received prophylactic cranial radiation therapy, which has been demonstrated not to be required in T-cell lymphoblastic lymphoma (T-LBL) patients.
5. COG-A5971 evaluated 2 strategies for CNS prophylaxis without CNS irradiation \[5\]. Patients were randomly assigned to receive HD-MTX in interim maintenance (BFM-95) or intrathecal chemotherapy throughout maintenance (CCG-BFM). The overall incidence of CNS relapse was 1.2%, and there was no difference between the treatment arms for CNS relapse, DFS, or OS. Minimal disseminated disease (MDD) \>1% by FLOW at diagnosis was shown to be associated with a worse outcome in this trial (a BFM backbone containing HD-MTX). Measurement of bone marrow MDD at diagnosis with sequential response monitoring through peripheral blood during remission induction to aid treatment stratification was also suggested in an early COG study. The prognostic significance of MDD at End-of-Induction (EOI) or End-of-Consolidation (EOC) for T-LBL patients with positive MDD at diagnosis is still unclear.
6. COG AALL0434: the COG ABFM regimen with C-MTX provided excellent DFS without cranial radiation for patients with standard risk T-LBL (85%, Arm A, n=82, completed 64) and high risk T-LBL (85%, Arm A, n=61, completed 51) although patients with CNS 3 were not included. It appears that C-MTX may have negated the prognostic impact of MDD.
7. Nelarabine is unavailable in mainland China at this time, which did not show benefit in COG AALL0434 study.
8. AALL07P1: 10 patients with T-LBL in first relapse treated with a 4-drug induction regimen adding bortezomib: 7 had a response (1 had a complete response, 2 had unconfirmed complete responses, and 4 had partial responses)
9. COG AALL1231 for T-LBL: the 4-year EFS and OS were better in bortezomib group than the control group (86.4% and 89.5% vs. 76.5% and 78.3%, p=0.041 and 0.009, respectively.). Incorporating bortezomib into standard therapy for de novo T-LBL appears beneficial.
10. A biopsy for pathological examination of a mediastinal residual mass is a clinical dilemma. Currently, conventional imaging is still considered as the "standard" modality for evaluating pediatric patients with NHL at diagnosis and subsequent response. There remains controversy on PET/CT interpretation in children with NHL. Large prospective studies in pediatric patients with T-LBL regarding PET/CT value for this is scarce.
11. Although an overlap in morphology and immune-phenotyping exists in T-LBL and T-cell acute lymphoblastic leukemia (T-ALL), different disease distribution suggests possible different genetic profiles and pathogenesis. Except for stage, none of other parameters is used in the current stratification system outside of clinical trials for T-LBL (several candidates, but none have been validated sufficiently). Little is known about biomarkers with prognostic relevance for T-LBL. To improve risk stratification strategy and better understand biologic rationale for incorporating novel therapies (chemicals, target agents and immunotherapy) into a conventional chemotherapy backbone, translational research to identify molecular markers with prognostic relevance in T-LBL is highly recommended.
12. With the current treatment, around 1% pediatric patients with T-LBL will not achieve remission at the end of Induction (induction failure). The optimal treatment for this small subgroup is largely unclear. The BFM HR Blocks usually are applied to these patients even though the efficacy is unknown. Novel targeted therapies are needed for use. Dasatinib is identified as a targeted therapy for T-cell ALL in preclinical drug screening.

Conditions

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T-cell Lymphoblastic Lymphoma

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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Standard risk Arm A

Any pediatric patients with newly diagnosed T-LBL Stage II to IV who achieve at least a PR at the end of Induction (EOI). Induction I followed by consolidation, Capizzi escalating methotrexate (interim maintenance) , delayed intensification and maintenance therapy. Triple intrathecal injections.

Group Type EXPERIMENTAL

Prednisone,Vincristine, Pegylated-asparaginase, Bortezomib,Cytarabine, Cyclophosphamide, Daunorubicin, 6-mercaptopurine, methotrexate, Dexamethasone, Doxorubicin

Intervention Type DRUG

Standard risk Arm A: Induction I followed by Consolidation, extracompartmental Capizzi MTX, delayed intensification and 96 weeks' maintenance therapy. Twenty-one or twenty-six triple intrathecal injections for CNS negative or positive patients, respectively.

Standard risk Arm B

Any pediatric patients with newly diagnosed T-LBL Stage II to IV who achieve at least a PR at the end of Induction (EOI). Induction I followed by consolidation, high dose methotrexate (interim maintenance) , delayed intensification and maintenance therapy. Triple intrathecal injections.

Group Type EXPERIMENTAL

Prednisone,Vincristine, Pegylated-asparaginase, Bortezomib,Cytarabine, Cyclophosphamide, Daunorubicin, 6-mercaptopurine, methotrexate, Dexamethasone, Doxorubicin

Intervention Type DRUG

Standard risk Arm B: Induction I followed by Consolidation, extracompartmental high dose MTX, delayed intensification and 96 weeks' maintenance therapy. Twenty-one or twenty-six triple intrathecal injections for CNS negative or positive patients, respectively.

High Risk T-LBL

Any pediatric patients with newly diagnosed T-LBL Stage II to IV who fail to achieve at least a PR at the end of Induction (EOI). Induction I followed by 6 intensive polychemotherapy blocks (HR1'-HR2'-HR3'-HR1'-HR2'-HR3'), deIayed intensification, and maintenance therapy. Triple intrathecal injections.

Group Type EXPERIMENTAL

Prednisone,Vincristine, Pegylated-asparaginase, Bortezomib,Cytarabine, Cyclophosphamide, Daunorubicin, 6-mercaptopurine, methotrexate, Dexamethasone, Doxorubicin, Vindesine, Etoposide, Ifosfamide

Intervention Type DRUG

High Risk T-LBL: Induction I followed by 2 cycles of BFM HR Blocks, delayed intensification and 96 weeks' maintenance therapy. Twenty-four or twenty-eight triple intrathecal injections for CNS negative or positive patients, respectively.

Interventions

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Prednisone,Vincristine, Pegylated-asparaginase, Bortezomib,Cytarabine, Cyclophosphamide, Daunorubicin, 6-mercaptopurine, methotrexate, Dexamethasone, Doxorubicin

Standard risk Arm A: Induction I followed by Consolidation, extracompartmental Capizzi MTX, delayed intensification and 96 weeks' maintenance therapy. Twenty-one or twenty-six triple intrathecal injections for CNS negative or positive patients, respectively.

Intervention Type DRUG

Prednisone,Vincristine, Pegylated-asparaginase, Bortezomib,Cytarabine, Cyclophosphamide, Daunorubicin, 6-mercaptopurine, methotrexate, Dexamethasone, Doxorubicin

Standard risk Arm B: Induction I followed by Consolidation, extracompartmental high dose MTX, delayed intensification and 96 weeks' maintenance therapy. Twenty-one or twenty-six triple intrathecal injections for CNS negative or positive patients, respectively.

Intervention Type DRUG

Prednisone,Vincristine, Pegylated-asparaginase, Bortezomib,Cytarabine, Cyclophosphamide, Daunorubicin, 6-mercaptopurine, methotrexate, Dexamethasone, Doxorubicin, Vindesine, Etoposide, Ifosfamide

High Risk T-LBL: Induction I followed by 2 cycles of BFM HR Blocks, delayed intensification and 96 weeks' maintenance therapy. Twenty-four or twenty-eight triple intrathecal injections for CNS negative or positive patients, respectively.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Newly diagnosed T-lineage lymphoblastic lymphoma (T-LBL) Stage II-IV

Exclusion Criteria

* Patients with Down syndrome or primary immune comprised disease.
* Ph+ T-LBL
* Patients must not have received any prior cytotoxic chemotherapy
* Any steroids pretreatment for \> 5 days in the 7 days or for \>14 days in the 28 days before the initiation of Induction chemotherapy. The dose of prednisone or methylprednisone pretreatment does not affect eligibility. Any steroids exposure that occurred \> 28 days before the initiation of Induction chemotherapy is allowed. Inhalation and topical steroids are not considered pretreatment. A single dose of vincristine is allowed.
Minimum Eligible Age

12 Months

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shanghai Children's Medical Center

OTHER

Sponsor Role collaborator

Beijing Children's Hospital

OTHER

Sponsor Role collaborator

Children's Hospital of Scow University

UNKNOWN

Sponsor Role collaborator

West China Second University Hospital

OTHER

Sponsor Role collaborator

Nanjing Children's Hospital

OTHER

Sponsor Role collaborator

Qilu Hospital of Shandong University

OTHER

Sponsor Role collaborator

Tianjin Medical University Cancer Institute and Hospital

OTHER

Sponsor Role collaborator

Tongji Hospital

OTHER

Sponsor Role collaborator

Xiangya Hospital of Central South University

OTHER

Sponsor Role collaborator

The First Affiliated Hospital of Zhengzhou University

OTHER

Sponsor Role collaborator

Cancer hospital of Shandong Province

UNKNOWN

Sponsor Role collaborator

Shenzhen Children's Hospital

OTHER_GOV

Sponsor Role collaborator

Wuhan Children's Hospital

OTHER

Sponsor Role collaborator

Zhejiang University School of Medicine Children's Hospital

UNKNOWN

Sponsor Role collaborator

Shanghai Children's Hospital

OTHER

Sponsor Role collaborator

Ruijin Hospital

OTHER

Sponsor Role collaborator

Second Affiliated Hospital of Anhui Medical University

UNKNOWN

Sponsor Role collaborator

Children's Hospital of Hebei Province

OTHER

Sponsor Role collaborator

Cancer Hospital of Henan Province

UNKNOWN

Sponsor Role collaborator

Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

OTHER

Sponsor Role collaborator

Qilu Children's Hospital

UNKNOWN

Sponsor Role collaborator

Children's Cancer Group, China

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Yi-Jin Gao, MD

Role: PRINCIPAL_INVESTIGATOR

Shanghai Children's Medical Center

Locations

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Shanghai Children's Medical Center

Shanghai, Shanghai Municipality, China

Site Status RECRUITING

West China Second University Hospital

Chengdu, , China

Site Status NOT_YET_RECRUITING

Countries

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China

Central Contacts

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Yi-Jin Gao, M.D.

Role: CONTACT

0086-21-38087513

Qing Quan, M.D

Role: CONTACT

Facility Contacts

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Yi-Jin Gao, MD

Role: primary

0086-21-38087513

Qing Yuan, MD

Role: backup

0086-21-38626161

Xia Guo, MD

Role: primary

References

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Other Identifiers

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CCCG-T-LBL-2023

Identifier Type: -

Identifier Source: org_study_id

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