BESPONSA Injection 1 mg Special Investigation

NCT05923112 | Completed Results

• 2 other identifiers: B1931024NCT05923112
• Study Type: observational
• Target: 421
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to learn about the safety and effectiveness of BESPONSA. BESPONSA is approved for treatment of relapsed or refractory CD22-positive acute lymphocytic leukemia. Registration criteria for this study is all patients who starting BESPONSA in Japan from its launch to the market to April 30, 2020. All patients in this study will receive BESPONSA according to the prescriptions. Patients will be followed up as follow.

• 52 weeks for patients who did not have a HSCT (Hematopoietic Stem Cell Transplant) within 52 weeks after starting BESPONSA.
• Up to 52 weeks after a HSCT for patients who had a HSCT within 52 weeks after starting BESPONSA.

Conditions

Acute Lymphocytic Leukemia

Keywords

Drug use investigation in Japan

Outcome Measures

Primary Outcomes (10)

• The Incidence of Adverse Drug Reactions

  An adverse drug reaction (ADR) was a treatment-related adverse event, and any untoward medical occurrence attributed to BESPONSA Injection 1mg in a participant who received BESPONSA Injection 1mg. A serious adverse drug reaction (SADR) was a treatment-related adverse event resulting in any of the following outcomes or deemed significant for any other reason: results in death; is life-threatening; requires inpatient hospitalization or prolongation of hospitalization; results in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); results in congenital anomaly/birth defect.

  From first dose up to 28 days after last dose. VOD/SOS: No HSCT: up to 52 weeks, HSCT: up to 104 weeks

• The Incidence of Liver Disorder Including VOD/SOS (ADRs)/ (All CTCAE Grades)

  Risk ratios for the proportion of participants with ADRs in different subgroups were determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. ADRs meeting definition of liver disorder including safety specification events of VOD/SOS were analyzed by each of the following factors: Age; Hepatic impairment; Medical history (past) - VOD/SOS, Hepatitis or hepatic disease; Medical history (present) - VOD/SOS, Hepatitis or hepatic disease; Eastern Cooperative Oncology Group performance status (ECOG PS); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels; gamma glutamyl transpeptidase (γ-GTP) level; Total bilirubin level; Platelet count; Peripheral blast count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  No HSCT: up to 52 weeks, HSCT: up to 104 weeks

• The Incidence of Liver Disorder Including VOD/SOS (ADRs)/ (CTCAE Grade 3 or Higher)

  Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator. ADRs meeting definition of liver disorder including safety specification events of VOD/SOS were analyzed by each of the following factors: Age; Hepatic impairment; Medical history (past) - VOD/SOS, Hepatitis or hepatic disease; Medical history (present) - VOD/SOS, Hepatitis or hepatic disease; ECOG PS; AST and ALT levels; γ-GTP level; Total bilirubin level; Platelet count; Peripheral blast count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  No HSCT: up to 52 weeks, HSCT: up to 104 weeks

• The Incidence of Myelosuppression (ADRs)/ (All CTCAE Grades)

  Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. ADRs meeting definition of safety specification events of myelosuppression were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle \> 1.8 mg/m2; ECOG PS; White blood cell count; Neutrophil count; Platelet count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants

• The Incidence of Myelosuppression (ADRs)/ (CTCAE Grade 3 or Higher)

  Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. ADRs meeting definition of safety specification events of myelosuppression were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle \> 1.8 mg/m2; ECOG PS; White blood cell count; Neutrophil count; Platelet count; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants

• The Incidence of Infections (ADRs)/ (All CTCAE Grades)

  Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator. In category (B) The risk ratio and confidence interval could not be estimated because no ADRs were observed in either the numerator, the denominator, or both. ADRs meeting definition of safety specification events of infection were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle \> 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants

• The Incidence of Infections (ADRs)/ (CTCAE Grade 3 or Higher)

  Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator. In category (B) The risk ratio and confidence interval could not be estimated because no ADRs were observed in either the numerator, the denominator, or both. ADRs meeting definition of safety specification events of infection were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle \> 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants

• The Incidence of Hemorrhage (ADRs)/ (All CTCAE Grades)

  Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. In category(A) The risk ratio was calculable; however, the confidence interval could not be estimated due to the absence of ADRs in the numerator. In category (B) The risk ratio and confidence interval could not be estimated because no ADRs were observed in either the numerator, the denominator, or both. ADRs meeting definition of safety specification events of hemorrhage were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle \> 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants

• The Incidence of Hemorrhage (ADRs)/ (CTCAE Grade 3 or Higher)

  Risk ratios for the proportion of participants with ADRs in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. ADRs meeting definition of safety specification events of hemorrhage were analyzed by each of the following factors: Age; Hepatic impairment; Renal impairment; Total dose per cycle \> 1.8 mg/m2; ECOG PS; Salvage line of the induction treatment with this drug; HSCT before the start of this drug.

  From the start date of administration to Day 28 post-final dose, approximately 27 weeks for HSCT and no HSCT participants

• Early Death After HSCT

  Risk ratios for the proportion of participants with early death in different subgroups was determined. However, if there was a category with fewer than 10 participants and it was considered difficult to determine the risk ratio for that category after reconsideration, the risk ratio was not determined for that category. Participants in the safety analysis set who underwent HSCT after the start of BESPONSA Injection 1mg and have completed the study were included in the analysis. Early death after HSCT was defined as death occurring within 100 days after the first HSCT following the start of BESPONSA Injection 1mg. Early death was considered to be absent if the participant died on Day 101 or later, or was confirmed alive at the time of study completion/discontinuation.

  Within 100 days or greater than 100 days after the first HSCT after the start of BESPONSA 1 mg injection up to 52 weeks

Secondary Outcomes (2)

• Hematologic Remission Rate

  52 Weeks. However, if subsequent treatment for the target disease was started or HSCT was performed, the best overall response before the start of the subsequent treatment or HSCT, whichever was earlier, was entered.

• Overall Survival (OS)

  No HSCT: up to 52 weeks, HSCT: up to 104 weeks

Eligibility Criteria

• Age: 0 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

All patients who received BESPONSA

You may qualify if:

• All patients prescribed BESPONSA

You may not qualify if:

• None

Sponsors & Collaborators

• Pfizer: lead

Study Sites (1)

Pfizer

Tokyo, Japan

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Limitations and Caveats

Although this study is an all-case surveillance, the informed consent rate for disclosure of study results was 36%. It is considered that the differences observed between the results of the all-case surveillance and those obtained from patients who provided informed consent may be attributed to variations in baseline characteristics and reduced statistical precision. The main factor is presumed to be the smaller sample size, which led to wider confidence intervals and shifts in point estimates.

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 20, 2023
• First Posted: June 28, 2023
• Study Start: July 2, 2018
• Primary Completion: September 6, 2024
• Study Completion: September 6, 2024
• Last Updated: December 5, 2025
• Results First Posted: December 5, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)