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Newly-diagnosed Intermediate/High Risk Pediatric B-cell ALL Protocol

Phase 2
Recruiting
Conditions
Acute Lymphoblastic Leukemia ALL
Childhood Leukemia, Acute Lymphoblastic
B Cell Acute Lymphoblastic Leukemia (B-ALL)
Interventions
Drug: Blinatumomab (Group A)
Drug: Blinatumomab (Group B)
Drug: Venetoclax (nonRand Group)
Registration Number
NCT06764238
Lead Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Brief Summary

Building upon the results from the CCCG-ALL-2015, CCCG-ALL-2020 multicenter study cohort, concurrent research findings, and the latest clinical trials, the CCCG-ALL-2025 I/HR-B-ALL is thus developed to further improve the event-free survival (EFS), and overall survival (OS), and quality of life (QoL) of children with intermediate- and high- risk B-cell childhood acute lymphoblastic leukaemia (I/HR-B-ALL), while decreasing adverse reactions and transplantation rates. This trial primarily aims to explore:

1. The efficacy of two randomized Blinatumomab application scheme on I/HR-ALL as determined by MRD negatvitiy rate.

2. The efficacy of modified mini-hyperCVD + Venetoclax in I/HR-ALL cannot afford blinatumomab, in contrast to historical control as determined by MRD negatvitiy rate.

Detailed Description

The study shown above will lead to the following revisions to the CCCG-ALL2025 I/HR-B-ALL plan, which will be based on the CCCG-ALL2020 plan.

1. After the induction remission phase, all I/HR-B-ALL patients can afford blinatumomab will participate in a blinatumomab+HDMTX randomized controlled trial as consolidation.

2. For patients cannot afford blinatumomab will be treated with venetoclax + modified mini-hyperCVD during the induction phase, then subsequently with CAT as consolidation phase. CAT will removed from induction phase.

3. For patients who received blinatumomab randomization, the CAT+ course was canceled.

4. All patients will continued with 6 cycles of alternated 5-day venetoclax or Dauno-based CCCG-2020 continuous therapy regimen.

5. Adding IgH rearrangement NGS MRD as an evaluation indicator.

6. Adding pharmacotyping study for I/HR B-ALL.

7. Three more bone marrow punctures and IT will be added with the aims to evaluate the CR rate with deepen remission during or after consolidation.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
1800
Inclusion Criteria
  1. Age older than 1 month to younger than 18 years.
  2. Diagnosis of acute lymphoblastic leukemia by bone marrow morphology.
  3. Diagnosis of B-ALL by immunophenotyping.
Exclusion Criteria
  1. Low-risk ALL
  2. sIgM+
  3. Acute leukemias of ambiguous lineage diagnosed according to WHO or EGIL criteria.
  4. ALL evolved from chronic myeloid leukemia (CML).
  5. Down's syndrome, or major congenital or hereditary disease with organ dysfunction
  6. Secondary leukemia
  7. Known underlying congenital immunodeficiency or metabolic disease
  8. Congenital heart disease with cardiac insufficiency.
  9. Treated with glucocorticoids for ≥14 days, or ABL kinase inhibitors for > 7 days within one month before enrollment, or any chemotherapy or radiotherapy within 3 months before enrollment (except for emergency radiotherapy to relieve airway compression)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Group ABlinatumomab (Group A)After PVDL+CAT induction remission phase, patients randomized to Group A will be subjected to consolidation phase with continuous 28 days' blinatumomab followed by 3 cycles of high-dose methotrexate (HDMTX)
Group BBlinatumomab (Group B)After PVDL+CAT induction remission phase, patients randomized to Group B will be subjected to consolidation phase with two 14-day cycles of blinatumomab, alternating with 3 cycles of high-dose methotrexate (HDMTX).
NonRandonmized GroupVenetoclax (nonRand Group)Patients who will not be subjected to blinatomomab randomization, will received PVDL + Venetoclax + mini-hyperCVD as induction phase , subsequently receiving CAT as early intensification.
Primary Outcome Measures
NameTimeMethod
To investigate if Group B[2*(14-day blinatumomab + HDMTX*2)] can result in noninferior event-free survival (EFS) compared to Group A [28-day blinatumomab + HDMTX*4Based on the above analysis in this study the investigators will randomize 1800 patients. The analysis will start1.5 years after the last patient is randomized. The expected study duration is approximately 6.5 years.

* The randomization is stratified by hospitals and status of flow-cytometry MRD (positive or negative) immediately prior to the blinatumomab-HDMTX treatment.

* Kaplan-Meier (KM) estimates of each EFS function will be computed along with standard error by the default procedure in R. To test noninferiority the investigators consider a noninferiority margin of 0.04 for 5-year EFS as clinically meaningful.

* Assume the EFS in the control group (Arm A) to be the same as the provisional I/HR in the CCCG-ALL2020 trial (preliminary data above), with the 1-year and 3-year EFS possibly 0.837 and 0.768 respectively. A simulation study with 10,000 rounds shows that by randomizing 1800 patients and 1.5 years of follow up, the above decision rule of declaring noninferiority has well controlled probabilities of false positive and false negative errors.

Secondary Outcome Measures
NameTimeMethod
Event-free survival (EFS) in the two randomized arms [2*(14-day blinatumomab + HDMTX*2)] and [28-day blinatumomab + HDMTX*4], in contrast to historical regimens.Up to 5 years for every enrolled case

EFS functions will be estimated using the Kaplan-Meier estimator of survival functions along with 95% confidence interval at 5 year. Standard error will be estimated using the default procedure in R. Follow up of the historical comparison cohort (CCCG-ALL-2020 and 2015) will continue during the course of the current trial. Comparisons of EFS will be performed using two-sided log-rank test. Multivariable regression modeling including trial (CCCGALL-2025 vs. 2020 or 2015) and other known prognostic factors as main effects may also be performed, using the Cox models.

Cumulative incidence of relapse (CIR) in the two randomized arms [2*(14-day blinatumomab + HDMTX*2)] and [28-day blinatumomab + HDMTX*4], in contrast to historical regimen.Up to 5 years for every enrolled case

CIR functions of relapse will be estimated by the Kalbafleisch-Prentice method. Follow up of the historical comparison cohort (CCCG-ALL-2020 and 2015) will continue during the course of the current trial. Comparisons of CIR will be performed by Gray's test. Multivariable regression modeling including trial (CCCGALL-2025 vs. 2020 or 2015) and other known prognostic factors as main effects may also be performed, using the Fine-Gray models.

Overall survival (OS) in the two randomized arms [2*(14-day blinatumomab + HDMTX*2)] and [28-day blinatumomab + HDMTX*4], in contrast to historical regimens.Up to 5 years for every enrolled case

OS functions will be estimated using the Kaplan-Meier estimator of survival functions along with 95% confidence interval at 5 year. Standard error will be estimated using the default procedure in R. Follow up of the historical comparison cohort (CCCG-ALL-2020 and 2015) will continue during the course of the current trial. Comparisons of OS will be performed using two-sided log-rank test. Multivariable regression modeling including trial (CCCGALL-2025 vs. 2020 or 2015) and other known prognostic factors as main effects may also be performed, using the Cox models.

EFS in patients who receive 6 alternated venetoclax/Daunorubincin courses of interim continuation therapy.Up to 5 years for every enrolled case

EFS functions will be estimated using the Kaplan-Meier estimator of survival functions along with 95% confidence interval at 5 year. Standard error will be estimated using the default procedure in R. Follow up of the historical comparison cohort (CCCG-ALL-2020 and 2015) will continue during the course of the current trial. Comparisons of EFS will be performed using two-sided log-rank test.Multivariable regression modeling including trial (CCCGALL-2025 vs. 2020 or 2015) and other known prognostic factors as main effects may also be performed, using the Cox models.

CIR in patients who receive 6 alternated venetoclax/Daunorubincin courses of interim continuation therapy.Up to five years for every enrolled case

CIR functions of relapse will be estimated by the Kalbafleisch-Prentice method. Follow up of the historical comparison cohort (CCCG-ALL-2020 and 2015) will continue during the course of the current trial. Comparisons of CIR will be performed by Gray's test. Multivariable regression modeling including trial (CCCGALL-2025 vs. 2020 or 2015) and other known prognostic factors as main effects may also be performed, using the Fine-Gray models.

OS in patients who receive 6 alternated venetoclax/Daunorubincin courses of interim continuation therapy.Up to five years for every enrolled case

OS will be estimated using the Kaplan-Meier estimator of survival functions along with 95% confidence interval at 5 year. Standard error will be estimated using the default procedure in R. Follow up of the historical comparison cohort (CCCG-ALL-2020 and 2015) will continue during the course of the current trial. Comparisons of OS will be performed using two-sided log-rank test. Multivariable regression modeling including trial (CCCGALL-2025 vs. 2020 or 2015) and other known prognostic factors as main effects may also be performed, using the Cox models.

To compare grade 3 or higher adverse effects (AEs; CTCAE v5.0) and estimate their cumulative incidencesUp to 30 days after last dose of study treatment

Proportions of grade-3 or higher AEs in each treatment phase will be estimated by the sample proportions along with exact 95% confidence intervals. Cumulative incidences of various grade-3 or higher AEs throughout therapy will be estimated by the Kalbafleisch-Prentice method; death, relapse and other events rendering off therapy before completion are regarded as competing risks.

Trial Locations

Locations (26)

Anhui Medical University Second Affiliated Hospital

🇨🇳

Hefei, Anhui, China

Anhui Provincial Children's Hospital

🇨🇳

Hefei, Anhui, China

Fujian Medical University Union Hospital

🇨🇳

Fuzhou, Fujian, China

Guangzhou Women and Children's Medical Center

🇨🇳

Guangzhou, Guangdong, China

Nanfang Hospital, Southern Medical University

🇨🇳

Guangzhou, Guangdong, China

The People's Hospital of Guangxi Zhuang Autonomous Region

🇨🇳

Nanning, Guangxi, China

The Affiliated Hospital of Guizhou Medical University

🇨🇳

Guiyang, Guizhou, China

Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology

🇨🇳

Wuhan, Hubei, China

Union Hospital of Tongji Medical College, Huazhong University of Science and Technology

🇨🇳

Wuhan, Hubei, China

Wuhan Children's Hospital

🇨🇳

Wuhan, Hubei, China

Hunan Children's Hospital

🇨🇳

Changsha, Hunan, China

Xiangya Hospital Central South University

🇨🇳

Changsha, Hunan, China

Nanjing Children's Hospital Affiliated to Nanjing Medical University

🇨🇳

Nanjing, Jiangsu, China

Children's Hospital of Soochow University

🇨🇳

Suzhou, Jiangsu, China

Jiangxi Provincial Children's Hospital

🇨🇳

Nanchang, Jiangxi, China

Qilu Hospital of Shandong University

🇨🇳

Jinan, Shandong, China

Affiliated Hospital of Qingdao University

🇨🇳

Qingdao, Shandong, China

Xi'an Northwest Women and Children Hospital

🇨🇳

Xi'an, Shanxi, China

West China Second University Hospital, Sichuan University

🇨🇳

Chengdu, Sichuan, China

Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC

🇨🇳

Tianjin, Tianjin, China

Chongqing Medical University Affiliated Children's Hospital

🇨🇳

Chongqing, China

Children's Hospital of Fudan University

🇨🇳

Shanghai, China

Shanghai Children's Hospital

🇨🇳

Shanghai, China

Shanghai Children's Medical Cener, Shanghai Jiao Tong University School of Medicine

🇨🇳

Shanghai, China

Shenzhen Children's Hospital

🇨🇳

Shenzhen, China

Hong Kong Children's Hospital

🇭🇰

Hong Kong, Hong Kong

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