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NCT Number: NCT07318662

Efficacy and Safety of HMAs Combined With Venetoclax Versus HMAs Alone in ND Int-and H-risk MDS or CMML

The aim of this study is to observe and analyze the clinical efficacy of Venetoclax+HMAs regimen in the treatment of newly diagnosed higher-risk MDS and CMML cases, and to compare the efficacy of venetoclax +HMAs regimen with that of HMAS regimen alone, in order to provide a normative scheme and basis for clinical use.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Guangdong Provincial People's Hospital

Guangzhou, Guangdong, 510000, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥18 years old, male or female;
  • MDS or CMML diagnosed by the 2016 or 222 WHO criteria, with a sustained survival of at least 12 weeks;
  • received more than two cycles of Venetoclax plus HMAs or HMAs alone without prior disease specific or allogeneic hematopoietic stem cell therapy (before initiating Venetoclax or HMAs, Medications such as hydroxyurea were allowed to reduce the white-cell count to 1.0×109 per liter or less.) ;
  • bone marrow blast cell count (BM blast > 5%) or IPSS-R score > 3 (intermediate risk, high risk, very high risk);
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤2;
  • complete case information could be obtained.

Exclusion criteria

  • patients who did not meet the inclusion criteria;
  • inability to obtain complete case information or to follow protocol steps or follow up on time;
  • other conditions considered by the investigators to be unsuitable for inclusion.

Treatment and study plan

Venetoclax

Drug

Previous phase Ib studies have fully demonstrated that venetoclax combined with HMAs is well tolerated in patients with MDS or CMML. Patients treated with VEN/AZA had a higher chance of SCT after remission and a higher survival benefit

Primary outcomes

  1. overall response rate

    Time frame: After the first cycles treatment(Day1, Month2)

    CR+mCR+PR+HI

Secondary outcomes

  1. IWG2023 Composite response rate

    Time frame: After the first cycles treatment(Day1, Month2 )

    CR+CR-L+CRh+CRequ

  2. overall survival

    Time frame: through study completion, an average of 1 year

    Refers to the time from the start of treatment to the patient's death or last follow-up.

  3. Blood transfusion dependence

    Time frame: through study completion, an average of 1 year

    Change in proportion of transfusion dependence from the start of treatment

  4. adaverse event rate

    Time frame: Up to 1 years after the last subject enrolled.

    Grade 3-4 adverse events

Sponsors and collaborators

Lead sponsor

Guangdong Provincial People's Hospital

Other

Registry information

Official study title

Efficacy and Safety of HMAs Combined With Venetoclax Versus HMAs Alone in Newly Diagnosed Patients With Intermediate-/High-Risk MDS and CMML : A Retro-prospective, Multicenter Observational Cohort Study

Important dates

Study start
2020
Primary completion
2026
Study completion
2027
First posted
Jan 6, 2026
Registry last updated
Jan 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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