Trial Outcomes & Findings for Phase II Study to Evaluate Safety and Efficacy of CB-103 With Venetoclax in Adolescent and Young Adult Patients With Relapsed/Refractory T-ALL or T-LBL (NCT NCT05464836)

NCT ID: NCT05464836

Last Updated: 2025-11-26

Results Overview

Overall response rate (ORR, including CR, CRi, and PR rate) with flow cytometric assessment of minimum residual disease (MRD)

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 2 participants
• Primary outcome timeframe: Average of 3 months
• Results posted on: 2025-11-26

Participant Flow

This study received IRB approval June 30, 2022 and was opened to recruitment August 12, 2024. The study was terminated by the local IRB on August 12, 2024.

Participant milestones

Measure	CB-103 in Combination With Venetoclax Treatment with CB-103 + Venetoclax in Relapsed/Refractory T-ALL or T-LBL
Overall Study STARTED	2
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	CB-103 in Combination With Venetoclax Treatment with CB-103 + Venetoclax in Relapsed/Refractory T-ALL or T-LBL
Overall Study Progression of Disease	1
Overall Study Physician Decision	1

Baseline Characteristics

Phase II Study to Evaluate Safety and Efficacy of CB-103 With Venetoclax in Adolescent and Young Adult Patients With Relapsed/Refractory T-ALL or T-LBL

Baseline characteristics by cohort

Measure	CB-103 in Combination With Venetoclax n=2 Participants Treatment with CB-103 + Venetoclax in Relapsed/Refractory T-ALL or T-LBL
Age, Categorical <=18 years	0 Participants n=492 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=492 Participants
Age, Categorical >=65 years	0 Participants n=492 Participants
Age, Continuous	20.5 years n=492 Participants
Sex: Female, Male Female	0 Participants n=492 Participants
Sex: Female, Male Male	2 Participants n=492 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=492 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=492 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=492 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=492 Participants
Race (NIH/OMB) Asian	0 Participants n=492 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=492 Participants
Race (NIH/OMB) Black or African American	0 Participants n=492 Participants
Race (NIH/OMB) White	2 Participants n=492 Participants
Race (NIH/OMB) More than one race	0 Participants n=492 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=492 Participants
Region of Enrollment United States	2 participants n=492 Participants

PRIMARY outcome

Timeframe: Average of 3 months

Overall response rate (ORR, including CR, CRi, and PR rate) with flow cytometric assessment of minimum residual disease (MRD)

Outcome measures

Measure	CB-103 in Combination With Venetoclax n=2 Participants Treatment with CB-103 + Venetoclax in Relapsed/Refractory T-ALL or T-LBL
Efficacy of CB-103 in Combination With Venetoclax Progressive Disease	1 Participants
Efficacy of CB-103 in Combination With Venetoclax Stable Disease	1 Participants

Adverse Events

CB-103 in Combination With Venetoclax

Serious events: 2 serious events

Other events: 2 other events

Deaths: 2 deaths

Serious adverse events

Measure	CB-103 in Combination With Venetoclax n=2 participants at risk Treatment with CB-103 + Venetoclax in Relapsed/Refractory T-ALL or T-LBL
Respiratory, thoracic and mediastinal disorders Pleural effusion	50.0% 1/2 • Number of events 2 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Infections and infestations Sepsis	50.0% 1/2 • Number of events 2 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.

Other adverse events

Measure	CB-103 in Combination With Venetoclax n=2 participants at risk Treatment with CB-103 + Venetoclax in Relapsed/Refractory T-ALL or T-LBL
Gastrointestinal disorders Adbominal pain	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Investigations Activated partial thromboplastin time	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Investigations Alkaline phosphatase increased	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Blood and lymphatic system disorders Anemia	50.0% 1/2 • Number of events 7 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Respiratory, thoracic and mediastinal disorders Atelectasis	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Respiratory, thoracic and mediastinal disorders Bronchpulmonary hemorrhage	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Cardiac disorders Tachycardia	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Infections and infestations Cytogalovirus infection reactivation	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Gastrointestinal disorders Diarrhea	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Musculoskeletal and connective tissue disorders Pain in extremity	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Eye disorders Eye Redness	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
General disorders Fatigue	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
General disorders Fever	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Respiratory, thoracic and mediastinal disorders Hypokalemia	50.0% 1/2 • Number of events 5 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Metabolism and nutrition disorders Hyponatremia	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Metabolism and nutrition disorders Hypophosphatemia	50.0% 1/2 • Number of events 2 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Infections and infestations Lung infection	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Investigations Lymphocyte count decreased	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
General disorders Neutrophil count decreased	50.0% 1/2 • Number of events 4 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Respiratory, thoracic and mediastinal disorders Respitory Distress	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Blood and lymphatic system disorders Thrombosis	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Gastrointestinal disorders GI Hemorrhage	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Cardiac disorders Pericardial Effusion	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Blood and lymphatic system disorders Platelet count decreased	50.0% 1/2 • Number of events 17 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Investigations White blood cell decreased	50.0% 1/2 • Number of events 9 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Gastrointestinal disorders Toothache	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Cardiac disorders Sinus tachycardia	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.
Eye disorders Optic nerve disorder	50.0% 1/2 • Number of events 1 • Average of 3 months An an adverse event or suspected adverse reaction is considered "serious" if, in the view of either the Investigator or the sponsor, it results in any of the following outcomes: * Death * A life-threatening adverse event * Inpatient hospitalization or prolongation of existing hospitalization * A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. * A congenital anomaly/birth defect.

Additional Information

Miriam Garcia, DO

MD Anderson Cancer Center

Phone: (713) 745-4312

Email: mbgarcia@mdanderson.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place