Higher-risk Myelodysplastic SyndromePhase 3NCT06927232
AZA+Lus VS AZA Monotherapy in HR-MDS
A Phase 3 study testing Azacitidine (AZA) and Luspatercept in people with Higher-risk Myelodysplastic Syndrome, run by Peking Union Medical College Hospital.
Trial summary
This study is a randomized, prospective, single-center, open-label cohort study involving untreated HR-MDS patients. The patients were divided randomized into AZA+Lus cohort and AZA monotherapy cohort. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Higher-risk Myelodysplastic Syndrome
- What is being tested?
- Azacitidine (AZA) and Luspatercept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 86 participants
- Where
- No sites listed yet
- Study dates
- Apr 2025to 31 Jan 2027
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years old
- Diagnosed as higher-risk MDS (IPSS intermediate-2/high-risk, or IPSS-R >3.5, or IPSS-M moderate high-, high-, very high-risk)
- Untreated patients
- Liver and kidney function less than 2 times of upper limit of normal
- ECOG≤2 and expected survival more than 6 months
- Informed consent signed
Who cannot join
- With active infection
- Other malignant tumors
- Obvious abnormal liver and kidney function, or abnormal function of other organs
- Combined with myelofibrosis
- Have undergone bone marrow transplantation
- Pregnant or lactating women, or men who have recent reproductive needs
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- Allergic to azacytidine, Rotercept or excipients
- History of polysorbate 80 allergy
- Refuse to sign informed consent
- Researchers consider it inappropriate to participate in the experiment
About this study
Official title: Azacitidine Plus Luspatercept Versus Azacitidine Monotherapy in Higher-risk Myelodysplastic Neoplasms: a Randomized Prospective Study
This study is a randomized, prospective, single-center, open-label cohort study involving untreated HR-MDS patients. The patients were divided randomized into AZA+Lus cohort and AZA monotherapy cohort.
Read the detailed description
The hypomethylating agents (HMA) azacitidine (AZA) and decitabine (DEC) have been shown to improve survival and delay disease progression in patients with high-risk MDS. They are recommended by the NCCN as first-line treatments for patients with high-risk MDS. Clinical trials have demonstrated an OR rate of approximately 40-50% with AZA in patients with high-risk MDS. Despite the efficacy of HMA therapy, the rate of transfusion independence remains low. Anemia remains the most prominent symptom in refractory patients, with very limited options for subsequent treatment. Prolonged anemia affects every organ function and seriously affects the prognosis of patients.
Luspatercept is currently approved for the treatment of patients with both erythropoiesis receptor agonist ( ESA) treatment failures in transfusion-dependent low-risk MDS-RS patients. In a randomized controlled phase III clinical trial, compared to a placebo group, luspatercept significantly improved transfusion dependence and improved hemoglobin and quality of life in refractory MDS-RS patients. A recent conference report suggested that there was no significant difference in efficacy between low-risk and high-risk patients treated with luspatercept and that the HI rate for high-risk patients treated with luspatercept monotherapy was approximately 50%.
Thus this study aimed to compare the efficacy of AZA+luspatercept and AZA monotherapy.
- Conditions
- Higher-risk Myelodysplastic Syndrome
- Being tested
- Azacitidine (AZA), Luspatercept
- Keywords
- luspatercept, azacitidine, higher-risk myelodysplastic syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Azacitidine (AZA)Drug
Azacitidine 75mg/m/ day *5 days, 28 days for 1 course
LuspaterceptDrug
Luspatercept 1.0 mg/kg subcutaneously every 3 weeks, adjusted according to hemoglobin, up to 1.75mg/kg. If hemoglobin ≥120g/L, luspatercept can be discontinued.
| Study group | Role | What they receive |
|---|---|---|
| Azacitidine+Luspatercept | Experimental | Not described Receives: Drug: Azacitidine (AZA); Drug: Luspatercept |
| Azacitidine | Active comparator | Not described Receives: Drug: Azacitidine (AZA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall response rate
Measured: 3 months, 6 months
Secondary outcomes (6)
complete response rate
Measured: 3 months, 6 months
rate of transfusion independence
Measured: 3 months, 6 months
adverse event rate
Measured: through study completion, an average of 1 year
relapse rate
Measured: through study completion, an average of 1 year
progress-free survival
Measured: through study completion, an average of 1 year
overall survival
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2025
- Study startApr 2025 (estimated)
- Primary completion31 Jan 2026 (estimated)
- Study completion31 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Higher-risk Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 86 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06927232
- Sponsor's protocol ID
- AZA-Lus-HRMDS
- Status last verified
- Apr 2025
- First submitted
- 1 Apr 2025
- Last updated
- 15 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Azacitidine Plus Luspatercept Versus Azacitidine Monotherapy in Higher-risk Myelodysplastic Neoplasms: a Randomized Prospective Study
- ClinicalTrials.gov ID
- NCT06927232
- Acronym / sponsor ID
- AZA-Lus-HRMDS
- Conditions
- Higher-risk Myelodysplastic Syndrome
- Keywords
- luspatercept, azacitidine, higher-risk myelodysplastic syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 86 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Apr 2025 (estimated)
- Primary completion
- 31 Jan 2026 (estimated)
- Study completion
- 31 Jan 2027 (estimated)
- First posted
- 15 Apr 2025 (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 is a randomized, prospective, single-center, open-label cohort study involving untreated HR-MDS patients. The patients were divided randomized into AZA+Lus cohort and AZA monotherapy cohort.
Detailed description
The hypomethylating agents (HMA) azacitidine (AZA) and decitabine (DEC) have been shown to improve survival and delay disease progression in patients with high-risk MDS. They are recommended by the NCCN as first-line treatments for patients with high-risk MDS. Clinical trials have demonstrated an OR rate of approximately 40-50% with AZA in patients with high-risk MDS. Despite the efficacy of HMA therapy, the rate of transfusion independence remains low. Anemia remains the most prominent symptom in refractory patients, with very limited options for subsequent treatment. Prolonged anemia affects every organ function and seriously affects the prognosis of patients.
Luspatercept is currently approved for the treatment of patients with both erythropoiesis receptor agonist ( ESA) treatment failures in transfusion-dependent low-risk MDS-RS patients. In a randomized controlled phase III clinical trial, compared to a placebo group, luspatercept significantly improved transfusion dependence and improved hemoglobin and quality of life in refractory MDS-RS patients. A recent conference report suggested that there was no significant difference in efficacy between low-risk and high-risk patients treated with luspatercept and that the HI rate for high-risk patients treated with luspatercept monotherapy was approximately 50%.
Thus this study aimed to compare the efficacy of AZA+luspatercept and AZA monotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Azacitidine+Luspatercept | Experimental | Drug: Azacitidine (AZA), Drug: Luspatercept |
| Azacitidine | Active comparator | Drug: Azacitidine (AZA) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age ≥18 years old
- Diagnosed as higher-risk MDS (IPSS intermediate-2/high-risk, or IPSS-R >3.5, or IPSS-M moderate high-, high-, very high-risk)
- Untreated patients
- Liver and kidney function less than 2 times of upper limit of normal
- ECOG≤2 and expected survival more than 6 months
Key exclusion criteria (10 listed)
- With active infection
- Other malignant tumors
- Obvious abnormal liver and kidney function, or abnormal function of other organs
- Combined with myelofibrosis
- Have undergone bone marrow transplantation
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)Azacitidine 75mg/m/ day *5 days, 28 days for 1 course | Drug | Azacitidine, Azacitidine+Luspatercept |
| LuspaterceptLuspatercept 1.0 mg/kg subcutaneously every 3 weeks, adjusted according to hemoglobin, up to 1.75mg/kg. If hemoglobin ≥120g/L, luspatercept can be discontinued. | Drug | Azacitidine+Luspatercept |
Primary outcome
overall response rate
Time frame: 3 months, 6 months
6 secondary outcomes
complete response rate
Time frame: 3 months, 6 months
rate of transfusion independence
Time frame: 3 months, 6 months
adverse event rate
Time frame: through study completion, an average of 1 year
relapse rate
Time frame: through study completion, an average of 1 year
progress-free survival
Time frame: through study completion, an average of 1 year
overall survival
Time frame: through study completion, an average of 1 year
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 15 Apr 2025.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Azacitidine (AZA) and Luspatercept in people with Higher-risk Myelodysplastic Syndrome, run by Peking Union Medical College Hospital.
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 6 inclusion and 10 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: opening soon, as of the sponsor's last update on 15 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
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 (time frame: 3 months, 6 months). 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 15 Apr 2025.