A Phase 1/2 interventional study of Sonrotoclax and Azacitidine (AZA) in Myelodysplastic Syndromes, High Risk, sponsored by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine. Recruiting at 6 sites in China. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Phase 1/2, Interventional, and Treatment
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.
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.
2,165 studies on the registry are indexed under Myelodysplastic Syndromes; 322 are open to participants now.
This study's planned enrollment of 60 is above the median of 39 across 1,771 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine is the lead sponsor of 182 studies on the registry; 108 are open to participants now.
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Adequate organ function to tolerate study treatment, including:
Exclusion Criteria:
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.
Drug: Sonrotoclax · Drug: Azacitidine (AZA)
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.
Also known as: BGB-11417
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.
Dose-Limiting Toxicities (DLTs)
Incidence of protocol-defined DLTs during the first treatment cycle.
Time frame: 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
Time frame: 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).
Time frame: From the first dose through completion of up to 4 treatment cycles, approximately 4 months
Duration of Response (DoR)
Time from the first documented response to disease progression, relapse, or death from any cause, whichever occurs first.
Time frame: Up to 36 months
Measurable Residual Disease (MRD) Negativity Rate
Proportion of evaluable participants who achieve MRD-negative status during study treatment.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: From the first dose of study treatment through 30 days after the last dose, assessed up to 12 months.
Plan to share: 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.
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drugs tested here: Sonrotoclax · Azacitidine
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine →