Real-world Study of Acalabrutinib (RESA)

A Prospective, National, Multicenter, Observational Real-World Study of Acalabrutinib in Chinese Patients With MCL/CLL/SLL(RESA)

The primary purpose of this study is to describe acotinib treatment patterns in Chinese patients with CLL/SLL and MCL who received acotinib according to the label. Secondary objectives include: 1) To evaluate the safety of acotinib in Chinese patients with CLL/SLL and MCL who received acotinib according to the label. 2) To evaluate the dose of acotinib in Chinese patients with CLL/SLL and MCL who received acotinib according to the label. 3) To describe the baseline clinical and demographic characteristics of Chinese patients with CLL/SLL and MCL who received acotinib according to the label. The exploratory objectives of the study include: 1) To describe the real-world overall survival (rwOS) of Chinese patients with CLL/SLL and MCL who received acotinib according to the label. 2) To describe the real-world clinical progression-free survival (rwPFS) of Chinese patients with CLL/SLL and MCL who received acotinib according to the label. 3) To describe the real-world response rate (rwRR) of Chinese patients with CLL/SLL and MCL who received acotinib according to the label. 4) To describe the real-world measurable residual disease (MRD) negativity rate in Chinese patients with CLL/SLL and MCL who received acotinib according to the label.

Study Overview

• Status: Enrolling by invitation
• Conditions: Mantle Cell Lymphoma (MCL); Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL)

Detailed Description

Acotinib is a highly selective, irreversible second-generation Bruton tyrosine kinase (BTK) inhibitor. NMPA approved acotinib in March 2023 for R/R MCL (≥1 prior therapy), in August 2023 for R/R CLL/SLL (≥1 prior therapy), and in March 2025 for first-line treatment of treatment-naïve CLL.

The phase III ChangE study (Qiu et al., 2024 ASH Poster 642) demonstrated the efficacy of acotinib in Asian patients with treatment-naïve CLL: 155 patients randomized 1:1 to acotinib (100 mg orally twice daily, continuous) or chlorambucil plus rituximab (6 cycles) across 44 study sites in mainland China, Taiwan (China), Vietnam, Thailand, and the Philippines; the Chinese cohort comprised 103 patients from 30 sites. At a median follow-up of 23.5 months (overall) and 18.2 months (Chinese cohort), acotinib reduced the risk of disease progression or death by 92% as assessed by blinded independent central review (HR=0.08; P<0.0001), with median PFS not reached versus 15.5 months and a 24-month PFS rate of 92% versus 25%; no new safety signals were identified.

Despite these pivotal data, real-world evidence on acotinib in routine Chinese clinical practice-including treatment patterns, dose modifications, long-term safety, effectiveness in real-world populations, and minimal residual disease (MRD) outcomes-remains limited.

The RESA study is a prospective, nationwide, multicenter, non-interventional, observational cohort study designed to describe acotinib real-world treatment patterns, dosing, safety, and effectiveness in Chinese patients with CLL/SLL and MCL treated according to the local prescribing label. Approximately 30 study sites are anticipated. The target sample size is approximately 160 patients (~901L CLL/SLL, ~30 R/R CLL/SLL, ~40 R/R MCL); enrollment will terminate at the earlier of ~160 patients enrolled or 46 months of enrollment. The exploratory endpoint of MRD negativity rate was newly added in protocol v2.0; MRD assessment (sample type, sampling method, detection method, and threshold per routine practice) will be captured at baseline and follow-up.

• Study Type: Observational
• Enrollment (Estimated): 160

Contacts and Locations

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200025
      • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Chinese patients with MCL (with ≥1 prior therapy) or CLL/SLL (treatment-naïve or with ≥1 prior therapy), treated with acotinib according to the local prescribing label, in routine clinical practice

Description

Inclusion Criteria:

• 1: Patients should be ≥18 years old at diagnosis
• 2: Diagnosed as MCL who have received at least one prior therapy, OR diagnosed as CLL/SLL who are treatment-naïve or have received at least one prior therapy (per the local prescribing label for acalabrutinib)
• 3: Eligible for acalabrutinib treatment assessed by investigators (physician's evaluation) in clinical practice
• 4: Patient/legal guardian must be able to read, understand, and sign the informed consent form (ICF)

Exclusion Criteria:

• 1: Ineligible for acalabrutinib treatment assessed by investigators (physician's evaluation)
• 2: Progression after accepting other BTKi treatment before use of acalabrutinib
• 3: Concurrent participation in another interventional clinical study
• 4: Females of childbearing potential must practice highly effective contraception during treatment of acalabrutinib, and for at least 1 week after the last dose of acalabrutinib, and have a negative urine or serum pregnancy test ≤ 7 days before the first dose of study drug(s).
• 5: No requirement to use contraception for male subjects treated with acalabrutinib. a. A sterile male is considered a highly effective contraception method for female patients. b. Males with known "low sperm counts" (consistent with "sub-fertility") are not to be considered sterile for purposes of this study.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
MCL; Chinese patients with MCL treated with acalabrutinib
CLL/SLL; Chinese patients with CLL/SLL treated with acalabrutinib

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
treatment patterns	Acalabrutinib treatment pattern will be summarized by the percentage of patients with acalabrutinib monotherapy and combo-therapies. Among patients with acalabrutinib combo-therapy, the frequency and percentage of patients in each categories (e.g. chemo, anti-CD20mAb, BCL2i, immunomodulator, etc.) will also be summarized. The Clopper-Pearson 95% confidence intervals (CIs) will also be presented.	Oct 2029

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The percentage of patients with AEs, SAEs (safety)	The percentage of patients with AEs, SAEs, will be summarized by System Organ Class and preferred term. The Clopper-Pearson 95% CIs around the incidence rate will also be reported.	Oct 2029
posology	Posology of acalabrutinib in Chinese CLL/SLL and MCL patients who received acalabrutinib according to Chinese label	Oct 2029

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
rwOS - Real-world overall survival	Time from first dose of acotinib to death from any cause. Survival rates will be estimated using the Kaplan-Meier method, with 95% CIs as appropriate.	Oct 2029
rwPFS - Real-world progression-free survival	Time from first dose of acotinib to documented disease progression or death from any cause, whichever occurs first, per investigator assessment per the local prescribing label.	Oct 2029
rwRR - Real-world response rate	The proportion of patients achieving complete response (CR) or partial response (PR) per investigator assessment per the local prescribing label. The Clopper-Pearson 95% confidence intervals (CIs) will also be presented.	Oct 2029
MRD negativity rate	The proportion of Chinese patients with CLL/SLL or MCL achieving undetectable measurable residual disease (MRD) at any post-baseline assessment following treatment with acotinib per the local prescribing label; sample type (peripheral blood and/or bone marrow), sampling method, detection method, and threshold for negativity will be captured per the investigator's routine practice. The Clopper-Pearson 95% confidence intervals (CIs) will be reported.	Oct 2029

Collaborators and Investigators

• Sponsor: Ruijin Hospital
• Collaborators: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Actual): July 2, 2024
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): November 1, 2029

Study Registration Dates

• First Submitted: October 30, 2024
• First Submitted That Met QC Criteria: January 9, 2025
• First Posted (Actual): January 10, 2025

Study Record Updates

• Last Update Posted (Actual): September 8, 2026
• Last Update Submitted That Met QC Criteria: September 2, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: MCL; Acalabrutinib; CLL/SLL; Acotinib
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Chronic Disease; Disease Attributes; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Lymphoma, Non-Hodgkin; Leukemia, B-Cell; Lymphoma; Leukemia, Lymphoid; Leukemia; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Leukemia, Lymphocytic, Chronic, B-Cell; Lymphoma, Mantle-Cell
• Other Study ID Numbers: ESR-22-21992

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No