Clinical Study of Venetoclax Combined With Azacytiside in the Treatment of Myelodysplastic/Myeloproliferative Neoplasms in Adults

Soochow University

Status and phase: Unknown; Phase 2

Conditions: Adult; Myelodysplastic/Myeloproliferative Neoplasms

Treatments: Drug: venetoclax combined with azacitidine

Study type: Interventional

Funder types: Other

Identifiers

NCT05282719

MDS/MPN-01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 33 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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33 participants in 1 patient group

Treatment regime

Experimental group

Description:

On day 1 of each cycle, decitabine 75 mg/m2 will be given subcutaneously, and will continue for 5 days. Simultaneously the patient will start out with Venetoclax 100mg and progress to 400mg until the 14 day cycle is finished.

Treatment:

Drug: venetoclax combined with azacitidine