Myelodysplastic Syndromes, High RiskPhase 1/2NCT07885397
Sonrotoclax Plus Azacitidine in Patients With MDS-IB2
A Phase 1/2 study testing Sonrotoclax and Azacitidine (AZA) in people with Myelodysplastic Syndromes, High Risk, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 6 sites in China.
Trial summary
This study will evaluate the safety and preliminary effectiveness of sonrotoclax combined with azacitidine in adults aged 18-70 years with newly diagnosed myelodysplastic neoplasms with increased blasts-2 (MDS-IB2). Sonrotoclax blocks BCL-2, a protein that helps cancer cells survive. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myelodysplastic Syndromes, High Risk
- What is being tested?
- Sonrotoclax and Azacitidine (AZA)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 6 sitesChina
- Study dates
- 15 Oct 2026to 31 Dec 2029
- Phase
- Phase 1/2
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 to 70 years, male or female.
- Voluntarily willing to participate in the study, able to understand and sign written informed consent, and willing to comply with study treatment, assessments, and follow-up.
- Newly diagnosed with myelodysplastic syndromes with increased blasts-2 (MDS-IB2) according to the current World Health Organization (WHO) diagnostic criteria.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
- Estimated life expectancy of at least 12 weeks.
- Adequate organ function to tolerate study treatment, including:
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- Total bilirubin ≤1.5 × upper limit of normal (ULN);
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3 × ULN;
- Creatinine clearance ≥40 mL/min;
- Left ventricular ejection fraction (LVEF) ≥50%;
- Baseline oxygen saturation >92%.
- Able to take oral medication and without gastrointestinal disorders that would significantly affect drug absorption.
- Considered by the investigator to be able to safely receive sonrotoclax initiation and dose ramp-up.
- Participants of childbearing potential must have a negative pregnancy test and agree to use effective contraception during the study and for the protocol-specified period after the last dose of study treatment.
Who cannot join
- Disease already transformed to acute myeloid leukemia (AML).
- Prior treatment with chemotherapy, sonrotoclax, or other BCL-2 inhibitors.
- Uncontrolled active infection, including severe bacterial, fungal, or viral infection.
- Active hepatitis B, hepatitis C, human immunodeficiency virus (HIV) infection, or active tuberculosis that is not adequately controlled.
- Significant uncontrolled cardiac, hepatic, renal, or other major organ dysfunction that, in the investigator's judgment, would preclude safe study treatment.
- Active graft-versus-host disease (GVHD) after prior allogeneic hematopoietic stem cell transplantation or ongoing requirement for systemic immunosuppressive therapy.
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- Anticipated need for continuous use of a strong CYP3A inhibitor during sonrotoclax initiation and dose ramp-up that cannot be discontinued or replaced.
- Uncontrolled high risk of tumor lysis syndrome (TLS) that, in the investigator's judgment, makes the participant unsuitable for sonrotoclax treatment.
- Pregnant or breastfeeding women.
- Known severe hypersensitivity to sonrotoclax, azacitidine, or any of their excipients.
- Concurrent participation in another interventional clinical study without meeting applicable discontinuation requirements.
- Any other condition that, in the investigator's judgment, makes the participant unsuitable for participation in the study.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
China6 sites, 6 recruiting
- Recruiting
Shanghai General Hospital
Shanghai, Shanghai Municipality, 200080
Shuang Li, +86 13816324008, lishuang0616@163.com
- Not yet recruiting
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality
Xiaoqin Wang
- Not yet recruiting
Shanghai Jiading District Central Hospital
Shanghai
Xiuhua Han
- Not yet recruiting
Shanghai Minhang District Central Hospital
Shanghai
Yunhua Hou
- Not yet recruiting
Shanghai Qingpu District Central Hospital
Shanghai
Fanli Hua
- Not yet recruiting
Xinhua Hospital, Shanghai Jiao Tong University
Shanghai
Jiangbo Wan
About this study
Official title: A Multicenter, Open-Label, Single-Arm Phase Ib/II Study of the Safety and Efficacy of Sonrotoclax in Combination With Azacitidine in Patients With Myelodysplastic Neoplasms With Increased Blasts-2 (MDS-IB2)
This study will evaluate the safety and preliminary effectiveness of sonrotoclax combined with azacitidine in adults aged 18-70 years with newly diagnosed myelodysplastic neoplasms with increased blasts-2 (MDS-IB2).
Sonrotoclax blocks BCL-2, a protein that helps cancer cells survive. Combining sonrotoclax with azacitidine may improve disease control. This study aims to determine whether the combination can induce remission with an acceptable safety profile, facilitate allogeneic stem cell transplantation in suitable patients, and improve long-term clinical outcomes.
Read more
This multicenter, open-label study has two sequential parts. Phase Ib will evaluate different sonrotoclax doses and identify the recommended dose for phase II. Phase II will further assess remission rates and safety at the selected dose. All participants will receive sonrotoclax plus azacitidine; there is no randomized control group.
Participants will undergo regular blood tests, bone marrow examinations, and monitoring for side effects. Suitable participants may proceed to transplantation based on disease response and clinical condition. Those who are not proceeding to transplantation and continue to benefit may continue treatment according to the protocol. Follow-up will assess the duration of remission, disease progression, subsequent treatment, survival, and quality of life.
- Conditions
- Myelodysplastic Syndromes, High Risk
- Being tested
- Sonrotoclax, Azacitidine (AZA)
- Keywords
- MDS-IB2, Sonrotoclax, Azacitidine
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SonrotoclaxDrug
Also called BGB-11417
Sonrotoclax is administered orally once daily. Phase Ib target doses are 40, 80, 160, and 320 mg; phase II uses the recommended phase II dose. In cycle 1, a four-day ramp-up administers one-eighth, one-quarter, one-half, and the full target dose on days 1, 2, 3, and 4, respectively. Treatment at the target dose is then continued for 14 days. From cycle 2 onward, sonrotoclax is generally administered on days 1-14 of each 28-day cycle. Dose interruptions, reductions, and adjustments to treatment duration or concomitant medications follow the protocol.
Azacitidine (AZA)Drug
Azacitidine is administered at 75 mg/m² once daily by subcutaneous injection or intravenous infusion for seven consecutive days per 28-day cycle. In cycle 1, azacitidine is administered on days 4-10, beginning when sonrotoclax reaches its target dose. From cycle 2 onward, azacitidine is administered on days 1-7. Treatment delays and dose modifications are permitted according to protocol-defined toxicity and recovery criteria.
| Study group | Role | What they receive |
|---|---|---|
| Sonrotoclax Plus Azacitidine | Experimental | Participants receive sonrotoclax in combination with azacitidine in 28-day cycles. Phase Ib evaluates sonrotoclax target doses of 40, 80, 160, and 320 mg daily using a modified dose-escalation design. Phase II evaluates the combination at the recommended phase II dose established after completion of phase Ib safety assessments. Treatment may continue according to protocol-defined response and safety criteria until disease progression, unacceptable toxicity, initiation of another anticancer treatment, transition to allogeneic hematopoietic stem cell transplantation, withdrawal of consent, or another protocol-defined stopping condition. Receives: Drug: Sonrotoclax; Drug: Azacitidine (AZA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-Limiting Toxicities (DLTs)
Incidence of protocol-defined DLTs during the first treatment cycle.
Measured: Cycle 1 Day 1 through Cycle 1 Day 28; up to Day 42 if required for assessment of unresolved hematologic toxicity.
Maximum Tolerated Dose / Recommended Phase 2 Dose
Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of Sonrotoclax
Measured: Through completion of the Phase Ib dose-escalation stage, approximately 12 months
Composite Response Rate (CRR)
Proportion of participants achieving complete remission (CR), complete remission with limited count recovery (CRL), or complete remission with partial hematologic recovery (CRh).
Measured: From the first dose through completion of up to 4 treatment cycles, approximately 4 months
Secondary outcomes (6)
Duration of Response (DoR)
Time from the first documented response to disease progression, relapse, or death from any cause, whichever occurs first.
Measured: Up to 36 months
Measurable Residual Disease (MRD) Negativity Rate
Proportion of evaluable participants who achieve MRD-negative status during study treatment.
Measured: From baseline through the end of study treatment, up to approximately 12 months
Progression-Free Survival (PFS)
Time from the first dose of study treatment to the first documented disease progression, transformation to acute myeloid leukemia, or death from any cause, whichever occurs first.
Measured: From the date of the first dose of study treatment to the date of the first documented disease progression, transformation to acute myeloid leukemia, or death from any cause, whichever occurs first, assessed up to 36 months.
Overall Survival (OS)
Time from the first dose of study treatment to death from any cause.
Measured: Up to 36 months
Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) Rate
Proportion of participants who undergo allogeneic hematopoietic stem cell transplantation after treatment with sonrotoclax plus azacitidine.
Measured: Up to 36 months
Incidence of Treatment-Emergent Adverse Events
Incidence and severity of treatment-emergent adverse events, serious adverse events, Grade 3 or higher adverse events, infections, and tumor lysis syndrome.
Measured: From the first dose of study treatment through 30 days after the last dose, assessed up to 12 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2026
- Study start15 Oct 2026 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic Syndromes, High RiskStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- BeiGene
- Responsible party
- Principal investigator, Xianmin Song, MD, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07885397
- Sponsor's protocol ID
- SON-AZA-MDSIB2-2026
- Status last verified
- Oct 2026
- First submitted
- 4 Oct 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data will not be made publicly available. Study data will be managed and retained in accordance with the protocol, institutional policies, and applicable regulations, with protection of participant confidentiality.