Acute Myeloid Leukemia in ChildrenNCT06233526
Individualized Treatment of Pediatric R/R AML Based on Transcriptomic Profile and in Vitro Drug Sensitivity Test
A study testing Chemotherapy regiments based on the transcriptomic profile and in vitro drug sensitivity test in people with Acute Myeloid Leukemia in Children, run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.
Trial summary
Acute myeloid leukemia (AML) accounts for about 15% to 20% of childhood leukemia, but the death rate accounts for about 50%. About 20-30% of children with AML did not achieve complete response (CR) after 2 induction treatments, and about 30% of children with CR had relapse within 3 years (including recurrence after… Read more about this study
- Who is it for?
- Ages Up to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Acute Myeloid Leukemia in Children
- What is being tested?
- Chemotherapy regiments based on the transcriptomic profile and in vitro drug sensitivity test
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2024to 31 Dec 2025
- Phase
- Interventional
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 Up to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- (1) Refractory recurrent acute myeloid leukemia (AML), and after second-line reinduction therapy, such as C+HAG did not reach complete CR.Criteria for complete response, refractory, and recurrence are as follows.
- <18 years old; (3) The child had good organ function, could tolerate chemotherapy, and had a physical strength score of 0-3 (WHO standard); (4) Understand the research procedures and voluntarily sign written informed consent.
Who cannot join
- (1) Acute promyelocytic leukemia, chronic myelogenous leukemia, acute mixed cell leukemia or known central nervous system leukemia; (2) AML associated with congenital syndromes such as Down syndrome, Fanconi anemia, Bloom syndrome, Cole's syndrome, or congenital aplastic anemia; (3) secondary to immunodeficiency or positive for human immunodeficiency virus (HIV); (4) Cardiac and renal function were obviously abnormal, and left ventricular ejection fraction was <50%.
- There is active systemic infection; (6) any medical history or concomitant condition that the investigator believes would impair the subject's safe completion of the study; (7) The investigator considers that the subject is medically unfit to receive the investigational drug or is unfit for any other reason; (8) a known or suspected allergy to the subject drug or to any drug administered in connection with this test;
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310000
Xu Xiaojun, chief physician, +86571888736617, xuxiaojun@zju.edu.cn
About this study
Official title: Individualized Treatment of Pediatric Relasped and Refractory Acute Myeloid Leukemia Based on Transcriptomic Profile and in Vitro Drug Sensitivity Test
Acute myeloid leukemia (AML) accounts for about 15% to 20% of childhood leukemia, but the death rate accounts for about 50%. About 20-30% of children with AML did not achieve complete response (CR) after 2 induction treatments, and about 30% of children with CR had relapse within 3 years (including recurrence after hematopoietic stem cell transplantation).Relapsed/refractory (R/R) AML is a major cause of treatment failure and refractory survival. Reinduction chemotherapy for R/R-AML to obtain CR again, followed by hematopoietic stem cell transplantation, is the current treatment. At present, there is no recognized reinduction protocol, and the reinduction remission rate of R/R-AML varies greatly among different treatment regimens, ranging from 23 to 81%. Current guidelines recommend a new combination chemotherapy regimen consisting of new drugs without cross-resistance. This method selects sensitive chemotherapeutic drugs, and then forms a new combination chemotherapy regimen according to the characteristics of drugs, which is the choice of R/R-AML reinduction therapy.This study intends to conduct a clinical study on the individualized treatment of R/R AML patients through in vitro drug sensitivity test combined with patient transcriptomic characteristics.
- Conditions
- Acute Myeloid Leukemia in Children
- Being tested
- Chemotherapy regiments based on the transcriptomic profile and in vitro drug sensitivity test
- Keywords
- Acute myeloid leukemia, relapsed/refractory, transcriptomic sequencing, drug sensitivity test
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Chemotherapy regiments based on the transcriptomic profile and in vitro drug sensitivity testOther
- Leukemia cells from newly diagnosed and relapsed children with AML were tested for high throughput in vitro drug sensitivity and resistance.
- Combined with multi-omics sequencing technology such as whole exome and transcriptome, the data of drug sensitivity, drug resistance and genomic characteristics of children AML patients were integrated, and the internal rules between drug sensitivity or drug resistance and molecular characteristics such as gene fusion, gene mutation and abnormal gene expression of children AML patients were deeply analyzed, and the molecular characteristics of drug sensitivity in children AML were mapped
| Study group | Role | What they receive |
|---|---|---|
| Individualized treatment group | Experimental | Treating the R/R AML patients based on the transcriptomic profile and in vitro drug sensitivity Test Receives: Other: Chemotherapy regiments based on the transcriptomic profile and in vitro drug sensitivity test |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete remission rate
Complete remission rate after the course of individualized treatment
Measured: 4 weeks
Event free survival
The patient's event free survival at one and five years after treatment
Measured: one year, five years
Secondary outcomes (1)
Overall Survival
The patient's overall survival at one and five years after treatment
Measured: one year, five years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2024
- Study start1 Jan 2024
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia in ChildrenStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Children's Hospital of Zhejiang University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- College of Pharmaceutical Science at Zhejiang University
- Responsible party
- Principal investigator, Xiaojun Xu, The Children's Hospital of Zhejiang University School of Medicine
- Investigators
- Xiaojun Xu, MD, Principal investigator, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06233526
- Sponsor's protocol ID
- 2023-IRB-0315-P-01
- Status last verified
- Jun 2024
- First submitted
- 23 Jan 2024
- Last updated
- 3 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Newell LF, Cook RJ. Advances in acute myeloid leukemia. BMJ. 2021 Oct 6;375:n2026. doi: 10.1136/bmj.n2026.
PubMed 34615640Kim H. Treatments for children and adolescents with AML. Blood Res. 2020 Jul 31;55(S1):S5-S13. doi: 10.5045/br.2020.S002.
PubMed 32719170Zarnegar-Lumley S, Caldwell KJ, Rubnitz JE. Relapsed acute myeloid leukemia in children and adolescents: current treatment options and future strategies. Leukemia. 2022 Aug;36(8):1951-1960. doi: 10.1038/s41375-022-01619-9. Epub 2022 Jun 6.
PubMed 35668109Leukemia & Lymphoma Group, Chinese Society of Hematology, Chinese Medical Association. [Chinese guidelines for the diagnosis and treatment of relapsed/refractory acute myelogenous leukemia (2021)]. Zhonghua Xue Ye Xue Za Zhi. 2021 Aug 14;42(8):624-627. doi: 10.3760/cma.j.issn.0253-2727.2021.08.002. No abstract available. Chinese.
PubMed 34547866Kamens JL, Nance S, Koss C, Xu B, Cotton A, Lam JW, Garfinkle EAR, Nallagatla P, Smith AMR, Mitchell S, Ma J, Currier D, Wright WC, Kavdia K, Pagala VR, Kim W, Wallace LM, Cho JH, Fan Y, Seth A, Twarog N, Choi JK, Obeng EA, Hatley ME, Metzger ML, Inaba H, Jeha S, Rubnitz JE, Peng J, Chen T, Shelat AA, Guy RK, Gruber TA. Proteasome inhibition targets the KMT2A transcriptional complex in acute lymphoblastic leukemia. Nat Commun. 2023 Feb 13;14(1):809. doi: 10.1038/s41467-023-36370-x.
PubMed 36781850Du W, Xia Z, Luo Z, Chen Y, Bing S, Wang W, Zhang X, Zhou Z, Zhang J, Cao J, Yang B, He Q, Shao X, Xu X, Ying M. A novel gene fusion RUNX1/ZNF423 promotes leukemic relapse of NUP98-rearranged AML. Leukemia. 2023 Nov;37(11):2286-2291. doi: 10.1038/s41375-023-02024-6. Epub 2023 Sep 15. No abstract available.
PubMed 37714925