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

NCT05282719 | Unknown Phase 2

• 1 other identifier: MDS/MPN-01
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

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.

Conditions

Myelodysplastic/Myeloproliferative Neoplasms; Adult

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR)

  ORR (equals the rates of complete remission \[CR\]+partial remission \[PR\]+complete cytogenetic remission \[CCyR\]+marrow response \[MR\[+clinical benefit \[CB\] )of venetoclax in combination with azacitidine. 1. CR and CCyR are shown in the secondary outcome measures below. 2. PR: Normalization of peripheral counts and hepatosplenomegaly with bone marrow blasts (and blast equivalents) reduced by 50%, but remaining\>5% of cellularity except in cases of MDS/MPN with≤5% bone marrow blasts at baseline. 3. MR: Optimal marrow response: Presence of all marrow criteria necessary for CR without normalization of peripheral blood indices. Partial marrow response: Bone marrow blasts (and blast equivalents) reduced by 50%, but remaining\>5% of cellularity, or reduction in grading of reticulin fibrosis from baseline on at least 2 bone marrow evaluations spaced at least 2 months apart. 4. CB: Hematology improvement, spleen response and symptom response.

  Study start date to study end date, or death, whichever comes first, up to 4 years

Secondary Outcomes (7)

• Complete remission rate

  Study start date to study end date, or death, whichever comes first, up to 4 years

• Complete remission rate of bone marrow morphology

  Study start date to study end date, or death, whichever comes first, up to 4 years

• Hematology improvement (HI) rate

  Study start date to study end date, or death, whichever comes first, up to 4 years

• Complete cytogenetic remission rate

  Study start date to study end date, or death, whichever comes first, up to 4 years

• Incidence of severe infection (≥grade 3 )

  Study start date to study end date, or death, whichever comes first, up to 4 years

Study Arms (1)

Treatment regime

EXPERIMENTAL

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.

Drug: venetoclax combined with azacitidine

Interventions

venetoclax combined with azacitidine DRUG

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.

Also known as: combination of venetoclax plus azacitidine

Treatment regime

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, Age (years) \>= 18；
• 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.
• Eastern Cooperative Oncology Group (ECOG) Performance status of 0,1, 2 ;
• Liver function: Total bilirubin ≤3 upper limit of normal (ULN); aspartate aminotransferase (AST) ≤3 ULN; alanine aminotransferase (ALT)≤3 ULN；
• Renal function#Ccr ≥30 ml/min;
• 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.

You may not qualify if:

• Acute myeloid leukemia
• Myelodysplastic syndrome
• Subjects who had previously been treated with Venetoclax
• Subjects who are known to be allergic to ingredients of the study drug or their analogues
• HIV infection
• HBV-DNA or HCV-RNA positive
• Subjects who are pregnant or breastfeeding
• Subjects reject to participate in the study

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology

Suzhou, Jiangsu, 215000, China

Location

MeSH Terms

Conditions

Myelodysplastic-Myeloproliferative Diseases

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Suning Chen, Professor

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Hematology Laboratory

Study Record Dates

• First Submitted: February 21, 2022
• First Posted: March 16, 2022
• Study Start: April 1, 2022
• Primary Completion: August 1, 2022
• Study Completion: February 1, 2023
• Last Updated: March 16, 2022

Record last verified: 2022-03

Locations

CN (1)