Clinical trial NCT05282719: Clinical Study of Venetoclax Combined With Azacytiside in the Treatment of Myelodysplastic/Myeloproliferative Neoplasms in Adults
Official title: A Multi-center, Phase II, Single-arm Clinical Study of Venetoclax Combined With Azacytiside in the Treatment of Myelodysplastic/Myeloproliferative Neoplasms in Adults
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Myelodysplastic/Myeloproliferative Neoplasms
Adult
Lead sponsor: The First Affiliated Hospital of Soochow University
Sponsor class: OTHER
First posted: 2022-03-16
Enrollment: 33
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: To explore the efficacy of venetoclax combined with azacytidine in Myelodysplastic / myeloproliferative neoplasms（MDS/MPN）, so as to improve the overall survival and treatment status of MDS/MPN patients.

Detailed description: At present, there is no standardized treatment strategy for MDS/MPN. The purpose of our study is to explore the efficacy of venetoclax combined with azacytidine in the treatment of MDS/MPN, so as to improve the overall survival and treatment status of patients with MDS/MPN. After the participants were treated with four cycles of venetoclax combined with azacytidine, the efficacy was evaluated according to the 2015 adult MDS/MPN response criteria to determine the disease status. Participants with disease progression and intolerance withdrew from the study during treatment.

Eligibility criteria: Inclusion Criteria:

1. Male or female, Age (years) \>= 18；
2. Patients newly diagnosed or previously treated with MDS/MPNs (CMML, MDS/MPN-U, aCML) according to 2016 WHO diagnostic criteria:

   Initial diagnosis: CMML: CPSS-mol intermediate risk 2 and above; aCML; MDS/MPN-U.

   Previous treatment: HMA treatment failed.
3. Eastern Cooperative Oncology Group (ECOG) Performance status of 0,1, 2 ;
4. Liver function: Total bilirubin ≤3 upper limit of normal (ULN); aspartate aminotransferase (AST) ≤3 ULN; alanine aminotransferase (ALT)≤3 ULN；
5. Renal function#Ccr ≥30 ml/min;
6. Patients who sign the informed consent must have the ability to understand and be willing to participate in the study and sign the informed consent.

Exclusion Criteria:

1. Acute myeloid leukemia
2. Myelodysplastic syndrome
3. Subjects who had previously been treated with Venetoclax
4. Subjects who are known to be allergic to ingredients of the study drug or their analogues
5. HIV infection
6. HBV-DNA or HCV-RNA positive
7. Subjects with grade 2 or above cardiac failure and those considered unsuitable for inclusion by the investigator
8. Subjects who are pregnant or breastfeeding
9. Subjects reject to participate in the study

Primary outcomes:
measure: Overall Response Rate (ORR)
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
Secondary outcomes:
measure: Complete remission rate
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
measure: Complete remission rate of bone marrow morphology
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
measure: Hematology improvement (HI) rate
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
measure: Complete cytogenetic remission rate
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
measure: Incidence of severe infection (≥grade 3 )
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
measure: Spleen response rate
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
measure: Symptom response rate
timeFrame: Study start date to study end date, or death, whichever comes first, up to 4 years
Other outcomes:
Locations:
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology
state: Jiangsu
Study officials:
affiliation: The First Affiliated Hospital of Soochow University
name: Suning Chen, Professor
role: PRINCIPAL_INVESTIGATOR
References: