Myelodysplastic/Myeloproliferative NeoplasmsPhase 2NCT05282719
Clinical Study of Venetoclax Combined With Azacytiside in the Treatment of Myelodysplastic/Myeloproliferative Neoplasms in Adults
A Phase 2 study testing venetoclax combined with azacitidine in people with Myelodysplastic/Myeloproliferative Neoplasms and Adult, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic/Myeloproliferative Neoplasms, Adult
- What is being tested?
- venetoclax combined with azacitidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteChina
- Study dates
- Apr 2022to Feb 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 9Show fewer
- Subjects with grade 2 or above cardiac failure and those considered unsuitable for inclusion by the investigator
- Subjects who are pregnant or breastfeeding
- Subjects reject to participate in the study
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology
Suzhou, Jiangsu, 215000
About this study
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
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.
Read the 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.
- Being tested
- venetoclax combined with azacitidine
- Medical subject headings
- Myelodysplastic-Myeloproliferative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
venetoclax combined with azacitidineDrug
Also called combination of venetoclax plus azacitidine
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: venetoclax combined with azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
- CR and CCyR are shown in the secondary outcome measures below.
- 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.
- 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.
- CB: Hematology improvement, spleen response and symptom response.
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Secondary outcomes (7)
Complete remission rate
- Bone marrow: ≤5% myeloblasts (including monocytic blast equivalent in case of CMML) with normal maturation of all cell lines and return to normal cellularity Osteomyelofibrosis absent or equal to "mild reticulin fibrosis" (≤grade 1 fibrosis).
- Peripheral blood: Leukocyte≤10×10E9 cells/L; Hemoglobin≥11g/dL; Platelets≥100×10E9/L, ≤450×10E9/L; Neutrophils≥1.0×10E9/L; Blasts 0%; Neutrophil precursors reduced to≤2%; Monocytes ≤1.0× 10E9/L.
- Extramedullary disease: Complete resolution of extramedullary disease present before therapy (eg, cutaneous disease, disease-related serous effusions), including palpable hepatosplenomegaly.
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Complete remission rate of bone marrow morphology
Presence of all marrow criteria necessary for CR without normalization of peripheral blood indices as presented above.
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Hematology improvement (HI) rate
Percentages of participants with HI (erythroid/platelet/neutrophil responses)
- Erythroid response: Hemoglobin increase by≥2.0 g/dL; Transfusion independence (TI) for ≥8 week for patients requiring at least 4 packed red blood cell transfusions in the previous 8 week; Only red blood cell transfusions given based on physician's judgment for a pretreatment Hgb of ≤8.5 g/dL will count in the red blood cell TI response evaluation.
- Platelet response: TI when previously requiring platelet transfusions of at least a rate of 4 platelet transfusions in the previous 8 week; Pretreatment≤20×10E9/L: increase from<20×10E9/L to>20×10E9/L and by at least 100%; Pretreatment>20×10E9/L but≤100×10E9/L: absolute increase of ≥30×10E9/L.
- Neutrophil response: Pretreatment≤0.5×10E9/L at least 100% increase and an absolute increase≥0.5×10E9/L; Pretreatment>0.5×10E9/L and≤1.0×10E9/L, at least 50% increase and an absolute increase ≥0.5×10E9/L.
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Complete cytogenetic remission rate
Resolution of previously present chromosomal abnormality (known to be associated with myelodysplastic, syndrome myeloproliferative neoplasms, or MDS/MPN), as seen on classic karyotyping with minimal of 20 metaphases or FISH.
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Incidence of severe infection (≥grade 3 )
Assessed using CTCAE 5
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Spleen response rate
Either a minimum 50% reduction in palpable splenomegaly of a spleen that is at least 10 cm at baseline or a spleen that is palpable at more than 5 cm at baseline becomes not palpable.
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Symptom response rate
Improvement in symptoms as noted by decrease of ≥50% as per the MPN-SAF TSS scoring<20 were not considered eligible for measuring clinical benefit.
Measured: Study start date to study end date, or death, whichever comes first, up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2022
- Study startApr 2022 (estimated)
- Primary completionAug 2022 (estimated)
- Study completionFeb 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic/Myeloproliferative NeoplasmsStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chen Suning, The First Affiliated Hospital of Soochow University
- Investigators
- Suning Chen, Professor, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05282719
- Sponsor's protocol ID
- MDS/MPN-01
- Status last verified
- Mar 2022
- First submitted
- 21 Feb 2022
- Last updated
- 16 Mar 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05282719
- Acronym / sponsor ID
- MDS/MPN-01
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 33 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- Apr 2022 (estimated)
- Primary completion
- Aug 2022 (estimated)
- Study completion
- Feb 2023 (estimated)
- First posted
- 16 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment regime | Experimental | Drug: venetoclax combined with azacitidineOn 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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 ;
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| venetoclax combined with azacitidineAlso: combination of venetoclax plus azacitidineOn 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 | Treatment regime |
Primary outcome
Overall Response Rate (ORR)
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
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.
7 secondary outcomes
Complete remission rate
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
Complete remission rate of bone marrow morphology
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
Hematology improvement (HI) rate
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
Complete cytogenetic remission rate
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
Incidence of severe infection (≥grade 3 )
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
Spleen response rate
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
Symptom response rate
Time frame: Study start date to study end date, or death, whichever comes first, up to 4 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Suning Chen, Professor principal investigator
The First Affiliated Hospital of Soochow University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing venetoclax combined with azacitidine in people with Myelodysplastic/Myeloproliferative Neoplasms and Adult, run by The First Affiliated Hospital of Soochow University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) (time frame: Study start date to study end date, or death, whichever comes first, up to 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2022; AzTrials last refreshed its copy on 8 Nov 2026.