Clinical trial NCT05175508: Aza With or Without ATRA in Newly Diagnosed Unfit AML or Intermediate,High or Very High Risk MDS
Official title: Azacitidine in Combination With or Without All-trans Retinoic Acid in Newly Diagnosed Unfit Acute Myeloid Leukemia or Intermediate,High or Very High Risk Myelodysplastic Syndromes (MDS) as Per IPSS-R Criteria
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: AML
MDS
Old Age; Debility
Hematologic Cancer
Keywords: acute myeloid leukemia
myelodysplastic syndromes
all-trans retinoic acid
azacitidine
Lead sponsor: The First Affiliated Hospital of Soochow University
Sponsor class: OTHER
First posted: 2022-01-03
Enrollment: 180
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a randomized, open-label, multicenter study to compare the efficacy and safety of AZA with or without ATRA in newly diagnosed unfit AML or Intermediate,High or Very High Risk MDS

Detailed description: Newly diagnosed unfit AML and Intermediate,High or Very High Risk Myelodysplastic Syndromes (MDS) as Per IPSS-R Criteria are unable to tolerate the intensive chemo-therapy regimens due to their old age and poor physical condition, resulting in limited overall survival. Nowadays, AZA are recommended for unfit acute myeloid leukemia or myelodysplastic syndromes patients with remission rate of 30%\~34%. AZA with or without all-trans retinoic acid (ATRA) can cooperatively inhibit leukemia cell proliferation , induce apoptosis and differentiation.

Eligibility criteria: Inclusion Criteria:

* Chinese guidelines for the diagnosis and treatment of acute myeloid leukemia (2017 edition)，excludes acute promyelocytic leukemia (M3、APL) and myelodysplastic syndromes(2017 edition)
* Be at least 18 years of age on day of signing informed consent
* Not suitable for newly diagnosed patients with intensive chemotherapy
* Not suitable for newly diagnosed patients with receiving hematopoietic stem cell transplantation
* The proportion of blast cells was below 50% in bone marrow
* Total white blood cell (WBC) count ≤10,000/µL；Must be able to swallow tablets

Exclusion Criteria:

* Malignant neoplasms with other progression
* Serious mental illness uncooperative
* Refusal to join the study

Primary outcomes:
measure: Overall Response Rate （ORR)
timeFrame: 6 months
measure: Overall survival (OS)
timeFrame: 24months
measure: Progression-free survival (PFS)
timeFrame: 24 months
Secondary outcomes:
measure: Percentage of Participants Achieving Transfusion Independence (TI) Who are Transfusion Dependent at Baseline
timeFrame: 6 months
measure: Incidence of systemic infections
timeFrame: 6 months
Other outcomes:
Locations:
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University
state: Jiangsu
Study officials:
affiliation: The First Affiliated Hospital of Soochow University
name: Han Yue, Ph.D
role: PRINCIPAL_INVESTIGATOR
References:
citation: Chinese Society of Hematology, Chinese Medical Association. [Chinese guidelines for diagnosis and treatment of myelodysplastic syndromes (2019)]. Zhonghua Xue Ye Xue Za Zhi. 2019 Feb 14;40(2):89-97. doi: 10.3760/cma.j.issn.0253-2727.2019.02.001. No abstract available. Chinese.
citation: Leukemia & Lymphoma Group, Chinese Society of Hematology, Chinese Medical Association. [Chinese guidelines for diagnosis and treatment of adult acute myeloid leukemia (not APL) (2017)]. Zhonghua Xue Ye Xue Za Zhi. 2017 Mar 14;38(3):177-182. doi: 10.3760/cma.j.issn.0253-2727.2017.03.001. No abstract available. Chinese.
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