MDS/MPNPhase 2NCT05817955
Clinical Study of Azacitidine Combined With Ruxolitinib in the Treatment of Higher-risk MDS/MPN
A Phase 2 study testing Azacitidine (AZA) with Ruxolitinib in people with MDS/MPN, run by The First Affiliated Hospital with Nanjing Medical University at 1 site in China.
Trial summary
This study observes the safety and efficacy of Azacitidine (AZA) combined with ruxolitinib to treat higher-risk Myelodysplastic Syndromes∕Myeloproliferative Neoplasms(MDS/MPN) Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- MDS/MPN
- What is being tested?
- Azacitidine (AZA) with Ruxolitinib
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Nov 2022to 30 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- According to WHO (2016) classification, researchers made the diagnosis of CMML based on clinical and morphological characteristics. Other criteria should be met: 1) Neut ≥2x109/L, PLT ≥25x109/L, 2) belongs to the following prognostic risk group according to CPSS-MOL or MMM : CPSS-MOL: inter-2 risk (2 to 3 points); high risk (≥4 points);
- MMM: inter-2 risk (2.5 to 4.5 points); high risk (≥5 points), or:
- According to the WHO (2016) classification standards (Arber 2016), researchers made the diagnosis of other types of MDS/MPN (including aCML and MDS /MPN-U) based on clinical and morphological characteristics. Other criteria should be met: 1) Neut ≥2x109/L, PLT ≥25x109/L, 2) Bone marrow blasts ≥5%;
- Patients who are not suitable for hematopoietic stem cell transplantation (HSCT) according to the local medical standards and treatment guidelines;
- Patients who are suitable for Azacitidine(AZA) treatment according to the local medical standards and treatment guidelines;
- BCR-ABL positive Chronic Myelogenous Leukemia (CML) and Ph chromosomal negative classic myeloproliferative Neoplasms, such as essential thrombocytosis (ET), polycythemia vera (PV) and primary myelofibrosis (PMF) are excluded;
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- Age is between 18 to 80 years old;
- ALT, AST and serum bilirubin is no more than 2 times of the upper limit of normal values (ULN), serum creatinine is no more than 150 μmol/L, and serum myocardial enzyme is less than (the same age) 2 times of normal value upper limit;
- The LVEF determined by the echocardiography is no less than 50%;
- Estimated glomerular filtration rate (EGFR) is no less than 30ml · min · 1.73m2;
- Eastern Tumor Collaboration Group (ECOG) physical states score is 0 to 2;
- Informed Consent Form is signed by patients or legal agents.
Who cannot join
- BCR-ABL positive Chronic Myelogenous Leukemia (CML) and Ph chromosomal negative classic myeloproliferative Neoplasms, such as essential thrombocytosis (ET), polycythemia vera (PV) and primary myelofibrosis (PMF) ;
- Low risk or inter-1 risk CMML patients according to CPSS-MOL or MMM scores; other types of MDS/MPN with less than 5% bone marrow blasts;
- Patients with Neut<2x109/L, PLT<25x109/L;
- Secondary acute leukemia, myeloid sarcoma, and blast phase of aCML;
- Patients who are allergic to any drug involved in the trial;
- Pregnancy, lactating Women, and patients who are unwilling to use contraceptives;
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- Patients with abnormal Liver and kidney function which exceeded the inclusion criteria;
- Patients with organic heart disease with clinical symptoms or heart dysfunction (NYHA ≥ level 2);
- Patients with other malignancies at the same time except the following situations:
- Patients had received treatment for the purpose of cure and had no active malignancies for at least 5 years prior to enrollment; 2)Patients had received sufficient treatment, non-melanoma skin cancer or malignant freckles -like moles with no signs of illness (even if random grouping is less than 3 years); 3)Received sufficient treatment, in situ cancer without signs of illness (even if the random group is less than 3 years);
- Patients with AIDS, syphilis, active hepatitis B (HBV-DNA can be measured) and hepatitis C;
- Patients with cardiovascular diseases with clinical significance, such as arrhythmia that have not been controlled or have symptoms, congestive heart failure or myocardial infarction within 6 months, or level 3 (moderate) or level 4 (Severe) heart disease (NYHA according to the New York Heart Society's functional grading method);
- Patients with any situations that might interfere with research procedures or results, or have the medical status or disease that will bring a certain risk to participating in this study judged by researchers (such as activity systemic infection);
- Patients who can't understand or follow the research plan;
- Patients who are under 18 or over 80 years old;
- Patients who underwent a major surgery within 4 weeks before the random grouping;
- Patients who are participating in other clinical trials one month before joining the group;
- Patients who rely on illegal drugs;
- Patients with psychological disorders or cognitive disorders
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Nanjing Medical University
Nanjing, Jiangsu, 210000
Zhongxun Shi, MD, +8613502187429, shizhongxun1@hotmail.com
About this study
Official title: Clinical Study of Azacitidine Combined With Ruxolitinib in the Treatment of Higher-risk Myelodysplastic Syndromes∕Myeloproliferative Neoplasms
This study observes the safety and efficacy of Azacitidine (AZA) combined with ruxolitinib to treat higher-risk Myelodysplastic Syndromes∕Myeloproliferative Neoplasms(MDS/MPN)
Read the detailed description
This is an open label, a multi -centered clinical trial, aiming to evaluate the safety and efficacy of Azacitidine with Ruxolitinib to treat MDS/MPN patients Myelodysplastic Syndromes/Myeloproliferative Neoplasms（MDS/MPN） are a set of malignant clonal diseases that originated from hematopoietic stem cells, which have both the characteristics of MDS and MPN. They also have the risk of transforming to Acute Myeloid Leukemia (AML). According to the 2016 World Health Organization (WHO) classification, MDS/MPN include Chronic myelomonocytic leukemia(CMML), Atypical chronic myeloid leukemia(aCML)，Juvenile myelomonocytic leukemia (JMML), MDS/MPN with ring sideroblasts and thrombocytosis(MDS/MPN-RS-T), and MDS/MPN unclassified(MDS/MPN-U). Except for JMML, other types of MDS/MPN mainly happened in persons over 60 years old, and the heterogeneity of the disease is large. At present, the treatment is limited. Although allo hematopoietic stem cell transplantation is the only way to cure the diseases, a recent large series study on CMML showed that allo hematopoietic stem cell transplantation would increase the mortality risk of lower-risk CMML, and did not obtain survival benefit in higher-risk CMML. Therefore, it is currently urgent to explore the new treatment for MDS/MPN.
CMML is the most common subtype of MDS/MPN, manifested the increased monocytes in peripheral blood (absolute cell count ≥1x109/L and percentage ≥10%, which lasts more than 3 months) accompanied with at least one lineage dysplasia and bone marrow blasts ≤20%. The median diagnosis age is (73-75) years old, and the estimated annual incidence in the population is 4/10,000.
The treatment strategy of CMML based on the prognostic stratification. According to the prognostic stratification, low-risk patients (low and inter-1) receive red blood cells transfusion and cytoreductive treatment. For high-risk (inter-2 and high) patients who are unsuitable for stem cell transplantation, hypomethylating agents and clinical trials are recommended. The hypomethylating agents (including Decitabine, Azacitidine, and oral Decitabine) are the only drugs approved by the FDA in the United States for CMML treatment. The approvement were based on MDS clinical trials that included CMML, such as CALGB Studies (including 14 patients with CMML) and European AZA-001 Studies (including 11patients with CMML). Since then, many prospective and retrospective studies containing CMML have confirmed its efficacy. The Overall Response Rates (ORR) is 40-50% for hypomethylating agents, while Complete Remission (CR) is less than 20%. In addition, a recent prospective clinical trial pointed out that hypomethylating agents did not achieved clinical response in MPN-CMML, and no significant difference was found between hypomethylating agents group and Hydroxyurea group in Overall Survivals (OS) and Leukemia Free Survival.
In addition to hypomethylating agents, clinical trials are also recommended for patients with higher risks CMML. At present, several clinical trials including Lenzilumab (GM-CSF monoclonal antibody, NCT02546284), Tipifarnib (Fennec metastases inhibitor, NCT02807272), Ruxolitinib (JAK1/2 inhibitors, NCT03722407) and Cobimetinib (MEK inhibitor, NCT04409639) are carried on in CMML. The results show that they have good efficacy and safety in CMML. In addition, a series of clinical trials combined hypomethylating agents with other drugs to explore the way to increase the efficacy in CMML.
Some MDS/MPN patients have the clinical manifestations of MPNs such as increased blood cells, large spleen sizes, and constitutional symptom. Meanwhile, activated inflammatory cytokine secretion also exists in MDS/MPN. Ruxolitinib is JAK1/JAK2 inhibitor used for the treatment of myelofibrosis. A multi -center phase I clinical trial of ruxolitinib including 20 CMML patients showed that 4 patients obtained hematopoietic improvement, and 1 patient obtained bone marrow PR. Of the 9 patients with splenomegaly, 5 patients received spleen reduction > 50%. 10 of 11 patients with constitutional symptom were significantly improved. The ORR was 35% combining spleen response and MDS/MPN International Working Group (IWG) efficacy criteria. Only one patient had level 3 platelet decrease, indicating that ruxolitinib had less impact on platelet counts in CMML. Based on this, the phase I/II clinical trial was included 50 patients with CMML. The ORR according to MDS/MPN IWG was 38%, and 43% patients achieved spleen reduction. The most common adverse events were anemia (10%) and platelet decrease (6%), further confirming the safety and efficacy of ruxolitinib in the treatment of CMML.
In addition, a phase II clinical trial explores the efficacy of ruxolitinib in aCML (n = 23) and chronic neutrophilic leukemia (CNL) (n = 22). The ORR was 35% (11 PR[9 CNL and 2 aCML], 4 CR [CNL]). The most common 3/4 adverse event was anemia (34%) and platelet decrease (14%). It is worth noting that the efficacy was better in CNL compared with aCML.
Based on previous studies, we intend to further investigate the safety and efficacy of azacitidine combined with ruxolitinib in the treatment of higher-risk myelodysplastic syndromes/myelodysplastic diseases (MDS/MPN).
- Conditions
- MDS/MPN
- Being tested
- Azacitidine (AZA) with Ruxolitinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Azacitidine (AZA) with RuxolitinibDrug
- Antibiotics: Antibiotic treatment is positively given when symptoms related to infection, and other support treatment is strengthened.
- Blood products: Infuse the blood products according to the patient's blood routine test. Drugs that need to be used by other diseases must be recorded in detail in the case report form, including the general name of the drug, the dosage of the medication, and the frequency of administration. Drugs are not allowed to be combined: Avoiding the use of granulocyte colony-stimulating factor (G-CSF), there may be risk of spleen rupture.
| Study group | Role | What they receive |
|---|---|---|
| Azacitidine (AZA) with Ruxolitinib | Experimental | The treatment phase of each course of treatment is based on Azacitidine (AZA)+Ruxolitinib. Specific scheme: (1) Azacitidine+Ruxolitinib scheme: Azacitidine (AZA) 75mg · m-2 · D-1, D1 to D7, subcutaneous injection; Ruxolitinib, D1 to D28, orally, select Ruxolitinib initial dose according to platelet level: ① PLT> 100X10*9/L: 20mg BID orally; ② PLT (50-100) X10*9/L: 15mg BID orally; ③ PLT <50X10*9/L: 10mg Bid orally.
The above schemes are all 28 days of treatment. If the patient has serious adverse reactions (such as infection and bone marrow suppression), appropriately delay the next course of treatment or adjust the amount of RUXOLITINIB. Termination of the scheme treatment. Receives: Drug: Azacitidine (AZA) with Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
complete response rate
Complete remission was achieved according to the remission criteria of MDS/MPN
Measured: From randomization to the end of Cycle 6 (each cycle is 28 days)
overall survival
It is measured from the date of randomization to the date of death from any cause; patients not known to have died at least follow-up are censored on the date they were last known to be alive.
Measured: From the time of randomization to time for up to 2 years
overall response rate
overall response includes all the response according to the remission criteria of MDS/MPN
Measured: From randomization to the end of Cycle 6 (each cycle is 28 days)
Secondary outcomes (2)
progression-free survival
The progression-free survival was calculated as the time from randomization to disease recurrence or death due to disease progression
Measured: From the time of randomization to time for up to 2 years
Early mortality
Early mortality was defined as death within the first 3 months after initiation of induction therapy
Measured: up to 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2023
- Study start1 Nov 2022
- Primary completion1 Jan 2025 (estimated)
- Study completion30 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MDS/MPNStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital with Nanjing Medical 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Affiliated Hospital of Jiangsu University, The Second Hospital of Nanjing Medical University, The Affiliated Jiangning Hospital of Nanjing Medical University
- Responsible party
- Sponsor
- Investigators
- Zhongxun Shi, MD, Study director, The First Affiliated Hospital with Nanjing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05817955
- Sponsor's protocol ID
- 2022-SR-757
- Status last verified
- Apr 2023
- First submitted
- 2 Mar 2023
- Last updated
- 18 Apr 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Azacitidine Combined With Ruxolitinib in the Treatment of Higher-risk Myelodysplastic Syndromes∕Myeloproliferative Neoplasms
- ClinicalTrials.gov ID
- NCT05817955
- Acronym / sponsor ID
- 2022-SR-757
- Conditions
- MDS/MPN
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital with Nanjing Medical UniversityUniversity, hospital or other
- Collaborators
- Affiliated Hospital of Jiangsu University, The Second Hospital of Nanjing Medical University, The Affiliated Jiangning Hospital of Nanjing Medical University
- Study start
- 1 Nov 2022 (actual)
- Primary completion
- 1 Jan 2025 (estimated)
- Study completion
- 30 Dec 2026 (estimated)
- First posted
- 18 Apr 2023 (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.
This study observes the safety and efficacy of Azacitidine (AZA) combined with ruxolitinib to treat higher-risk Myelodysplastic Syndromes∕Myeloproliferative Neoplasms(MDS/MPN)
Detailed description
This is an open label, a multi -centered clinical trial, aiming to evaluate the safety and efficacy of Azacitidine with Ruxolitinib to treat MDS/MPN patients Myelodysplastic Syndromes/Myeloproliferative Neoplasms（MDS/MPN） are a set of malignant clonal diseases that originated from hematopoietic stem cells, which have both the characteristics of MDS and MPN. They also have the risk of transforming to Acute Myeloid Leukemia (AML). According to the 2016 World Health Organization (WHO) classification, MDS/MPN include Chronic myelomonocytic leukemia(CMML), Atypical chronic myeloid leukemia(aCML)，Juvenile myelomonocytic leukemia (JMML), MDS/MPN with ring sideroblasts and thrombocytosis(MDS/MPN-RS-T), and MDS/MPN unclassified(MDS/MPN-U). Except for JMML, other types of MDS/MPN mainly happened in persons over 60 years old, and the heterogeneity of the disease is large. At present, the treatment is limited. Although allo hematopoietic stem cell transplantation is the only way to cure the diseases, a recent large series study on CMML showed that allo hematopoietic stem cell transplantation would increase the mortality risk of lower-risk CMML, and did not obtain survival benefit in higher-risk CMML. Therefore, it is currently urgent to explore the new treatment for MDS/MPN.
CMML is the most common subtype of MDS/MPN, manifested the increased monocytes in peripheral blood (absolute cell count ≥1x109/L and percentage ≥10%, which lasts more than 3 months) accompanied with at least one lineage dysplasia and bone marrow blasts ≤20%. The median diagnosis age is (73-75) years old, and the estimated annual incidence in the population is 4/10,000.
The treatment strategy of CMML based on the prognostic stratification. According to the prognostic stratification, low-risk patients (low and inter-1) receive red blood cells transfusion and cytoreductive treatment. For high-risk (inter-2 and high) patients who are unsuitable for stem cell transplantation, hypomethylating agents and clinical trials are recommended. The hypomethylating agents (including Decitabine, Azacitidine, and oral Decitabine) are the only drugs approved by the FDA in the United States for CMML treatment. The approvement were based on MDS clinical trials that included CMML, such as CALGB Studies (including 14 patients with CMML) and European AZA-001 Studies (including 11patients with CMML). Since then, many prospective and retrospective studies containing CMML have confirmed its efficacy. The Overall Response Rates (ORR) is 40-50% for hypomethylating agents, while Complete Remission (CR) is less than 20%. In addition, a recent prospective clinical trial pointed out that hypomethylating agents did not achieved clinical response in MPN-CMML, and no significant difference was found between hypomethylating agents group and Hydroxyurea group in Overall Survivals (OS) and Leukemia Free Survival.
In addition to hypomethylating agents, clinical trials are also recommended for patients with higher risks CMML. At present, several clinical trials including Lenzilumab (GM-CSF monoclonal antibody, NCT02546284), Tipifarnib (Fennec metastases inhibitor, NCT02807272), Ruxolitinib (JAK1/2 inhibitors, NCT03722407) and Cobimetinib (MEK inhibitor, NCT04409639) are carried on in CMML. The results show that they have good efficacy and safety in CMML. In addition, a series of clinical trials combined hypomethylating agents with other drugs to explore the way to increase the efficacy in CMML.
Some MDS/MPN patients have the clinical manifestations of MPNs such as increased blood cells, large spleen sizes, and constitutional symptom. Meanwhile, activated inflammatory cytokine secretion also exists in MDS/MPN. Ruxolitinib is JAK1/JAK2 inhibitor used for the treatment of myelofibrosis. A multi -center phase I clinical trial of ruxolitinib including 20 CMML patients showed that 4 patients obtained hematopoietic improvement, and 1 patient obtained bone marrow PR. Of the 9 patients with splenomegaly, 5 patients received spleen reduction > 50%. 10 of 11 patients with constitutional symptom were significantly improved. The ORR was 35% combining spleen response and MDS/MPN International Working Group (IWG) efficacy criteria. Only one patient had level 3 platelet decrease, indicating that ruxolitinib had less impact on platelet counts in CMML. Based on this, the phase I/II clinical trial was included 50 patients with CMML. The ORR according to MDS/MPN IWG was 38%, and 43% patients achieved spleen reduction. The most common adverse events were anemia (10%) and platelet decrease (6%), further confirming the safety and efficacy of ruxolitinib in the treatment of CMML.
In addition, a phase II clinical trial explores the efficacy of ruxolitinib in aCML (n = 23) and chronic neutrophilic leukemia (CNL) (n = 22). The ORR was 35% (11 PR[9 CNL and 2 aCML], 4 CR [CNL]). The most common 3/4 adverse event was anemia (34%) and platelet decrease (14%). It is worth noting that the efficacy was better in CNL compared with aCML.
Based on previous studies, we intend to further investigate the safety and efficacy of azacitidine combined with ruxolitinib in the treatment of higher-risk myelodysplastic syndromes/myelodysplastic diseases (MDS/MPN).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Azacitidine (AZA) with Ruxolitinib | Experimental | Drug: Azacitidine (AZA) with RuxolitinibThe treatment phase of each course of treatment is based on Azacitidine (AZA)+Ruxolitinib. Specific scheme: (1) Azacitidine+Ruxolitinib scheme: Azacitidine (AZA) 75mg · m-2 · D-1, D1 to D7, subcutaneous injection; Ruxolitinib, D1 to D28, orally, select Ruxolitinib initial dose according to platelet level: ① PLT> 100X10*9/L: 20mg BID orally; ② PLT (50-100) X10*9/L: 15mg BID orally; ③ PLT <50X10*9/L: 10mg Bid orally. The above schemes are all 28 days of treatment. If the patient has serious adverse reactions (such as infection and bone marrow suppression), appropriately delay the next course of treatment or adjust the amount of RUXOLITINIB. Termination of the scheme treatment. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- According to WHO (2016) classification, researchers made the diagnosis of CMML based on clinical and morphological characteristics. Other criteria should be met: 1) Neut ≥2x109/L, PLT ≥25x109/L, 2) belongs to the following prognostic risk group according to CPSS-MOL or MMM : CPSS-MOL: inter-2 risk (2 to 3 points); high risk (≥4 points);
- According to the WHO (2016) classification standards (Arber 2016), researchers made the diagnosis of other types of MDS/MPN (including aCML and MDS /MPN-U) based on clinical and morphological characteristics. Other criteria should be met: 1) Neut ≥2x109/L, PLT ≥25x109/L, 2) Bone marrow blasts ≥5%;
- Patients who are not suitable for hematopoietic stem cell transplantation (HSCT) according to the local medical standards and treatment guidelines;
- Patients who are suitable for Azacitidine(AZA) treatment according to the local medical standards and treatment guidelines;
- BCR-ABL positive Chronic Myelogenous Leukemia (CML) and Ph chromosomal negative classic myeloproliferative Neoplasms, such as essential thrombocytosis (ET), polycythemia vera (PV) and primary myelofibrosis (PMF) are excluded;
Key exclusion criteria (19 listed)
- BCR-ABL positive Chronic Myelogenous Leukemia (CML) and Ph chromosomal negative classic myeloproliferative Neoplasms, such as essential thrombocytosis (ET), polycythemia vera (PV) and primary myelofibrosis (PMF) ;
- Low risk or inter-1 risk CMML patients according to CPSS-MOL or MMM scores; other types of MDS/MPN with less than 5% bone marrow blasts;
- Patients with Neut<2x109/L, PLT<25x109/L;
- Secondary acute leukemia, myeloid sarcoma, and blast phase of aCML;
- Patients who are allergic to any drug involved in the trial;
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 |
|---|---|---|
| Azacitidine (AZA) with Ruxolitinib1. Antibiotics: Antibiotic treatment is positively given when symptoms related to infection, and other support treatment is strengthened. 2. Blood products: Infuse the blood products according to the patient's blood routine test. Drugs that need to be used by other diseases must be recorded in detail in the case report form, including the general name of the drug, the dosage of the medication, and the frequency of administration. Drugs are not allowed to be combined: Avoiding the use of granulocyte colony-stimulating factor (G-CSF), there may be risk of spleen rupture. | Drug | Azacitidine (AZA) with Ruxolitinib |
Primary outcome
complete response rate
Time frame: From randomization to the end of Cycle 6 (each cycle is 28 days)
Complete remission was achieved according to the remission criteria of MDS/MPN
overall survival
Time frame: From the time of randomization to time for up to 2 years
It is measured from the date of randomization to the date of death from any cause; patients not known to have died at least follow-up are censored on the date they were last known to be alive.
overall response rate
Time frame: From randomization to the end of Cycle 6 (each cycle is 28 days)
overall response includes all the response according to the remission criteria of MDS/MPN
2 secondary outcomes
progression-free survival
Time frame: From the time of randomization to time for up to 2 years
Early mortality
Time frame: up to 3 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2023.
- Individual participant dataSharing planned: No
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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.
Zhongxun Shi, MD study director
The First Affiliated Hospital with Nanjing Medical 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 Azacitidine (AZA) with Ruxolitinib in people with MDS/MPN, run by The First Affiliated Hospital with Nanjing Medical 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 12 inclusion and 19 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: looking for participants now, as of the sponsor's last update on 18 Apr 2023. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Zhongxun Shi, MD (+86 13502187429, shizhongxun1@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
complete response rate (time frame: From randomization to the end of Cycle 6 (each cycle is 28 days)). The study lists 3 primary outcomes in all. 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 18 Apr 2023; AzTrials last refreshed its copy on 8 Nov 2026.