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

NCT05464836 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: 2021-0547NCI-2022-05687
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

To learn if the combination of 2 study drugs, CB-103 and venetoclax, can help to control T-cell acute lymphoblastic leukemia (T-ALL) or T-cell lymphoblastic leukemia (T-LBL) in adolescent and young adult patients

Conditions

Leukemia, Lymphoblastic; Leukemia

Outcome Measures

Primary Outcomes (1)

• Efficacy of CB-103 in Combination With Venetoclax

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

  Average of 3 months

Study Arms (1)

CB-103+Venetoclax

EXPERIMENTAL

Control T-cell acute lymphoblastic leukemia (T-ALL) or T-cell lymphoblastic leukemia (T-LBL) in adolescent and young adult patients.

Drug: CB-103; Drug: Venetoclax

Interventions

CB-103 DRUG

Given by PO

Also known as: Cellestia

CB-103+Venetoclax

Venetoclax DRUG

Given by PO

Also known as: ABT-199

CB-103+Venetoclax

Eligibility Criteria

• Age: 12 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Adolescent (12 to 18 years) and adult (19 to 60 years) participants who have relapsed or refractory T-cell lymphoblastic leukemia (T-ALL) or T-Cell lymphoblastic lymphoma (T-LBL) according to 2017 WHO classification \[29\] and NCCN v1 2021 \[30\]:
• Participants must have ≥ 5% blasts in the bone marrow as assessed by morphology on standard bone marrow biopsy and aspirate or less than 5% blasts in the bone marrow in presence of extramedullary relapse, excluding isolated central nervous system (CNS) relapse. However, if an adequate bone marrow sample cannot be obtained, participants may be enrolled if there is unequivocal evidence of leukemia with ≥ 5% blasts in the peripheral blood.
• Participants are eligible independently of Notch pathway activation in the leukemic blasts:
• however, a fresh marrow/blood sample must be obtained before starting the study treatment to classify the participants as being either Notch positive or negative.
• Leukemic blasts must express of at least 2 of the following immune phenotyping: CD1a, CD2, CD3, CD4, CD5, CD7, CD8, CD34, TCRαβ, TCRγδ, cyCD3
• Participants have adequate performance status (ECOG ≤2) for participants ≥16 years old, Lansky score \>50 for patients \<16 years old.
• Participants must be 12 to 60 years of age inclusive when signing the informed consent. For participants \< 18 years of age, parent or legally authorized representative (LAR) should be willing and able to give informed consent. Non-English speaking participants are eligible for whom consent process will follow institutional guidelines.
• Participants with asymptomatic CNS disease are eligible
• Participants must have adequate organ function and laboratory results (obtained within 14 days of enrollment):
• Direct bilirubin ≤2 x upper limit of normal (ULN).
• Serum creatinine ≤ 1.5 x ULN; or if serum creatinine \> 1.5 x ULN, then serum creatinine clearance (CrCl) ≥ 50 mL/min (estimated by Cockcroft-Gault formula).
• Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≤3 x ULN; ≤5 x ULN in case of suspected leukemic liver involvement
• Females of childbearing potential must have a negative serum or urine beta-human chorionic gonadotropin (β-HCG) pregnancy test result within 14 days prior to the first dose of study drugs and must agree to use one of the following effective contraception methods during the study and for 30 days following the last dose of study drug. Effective methods of birth control include:
• Birth control pills, skin patches, shots, subdermal implants
• Intrauterine devices (IUDs)

You may not qualify if:

• Mixed phenotype leukemia (excluding T-ALL with myeloid antigen expression)
• History of another primary invasive malignancy that has not been definitively treated and in remission. Participants with non-melanoma skin cancers or with carcinomas in situ are eligible regardless of the time from diagnosis (including concomitant diagnoses).
• Presence of clinically significant uncontrolled CNS pathology such as epilepsy, childhood seizure, paresis, aphasia, stroke, severe brain injuries, organic brain syndrome, or psychosis.
• Participants with a cardiac ejection fraction (as measured by either MUGA or echocardiogram) \< 50% or with a history of absolute decrease in LVEF of ≥ 15 absolute percentage points are excluded.
• Medical history of cardiovascular disease such as
• Clinically significant cardiac disease including congestive heart failure (NYHA class III or IV), arrhythmia or conduction abnormality requiring medication, or cardiomyopathy
• Clinically uncontrolled hypertension Age Blood Pressure 12 to 13 = \>120/80mmHg \>13 = \>140/90mmHg
• Complete left bundle branch block
• Right bundle branch block + left anterior hemiblock
• Congenital long QT syndrome
• History or presence of sustained or symptomatic ventricular tachyarrhythmia, atrial fibrillation, or clinically significant resting bradycardia (\< 50 bpm)
• Corrected QT interval using Fridericia formula (QTcF) \> 450 ms for males and \> 470 ms for females at the screening ECG
• QRS ≥ 110 ms
• History of symptomatic congestive heart failure
• Participants with uncontrolled, active infections (viral, bacterial, or fungal). Infections controlled on concurrent anti-microbial agents are acceptable. Anti-microbial prophylaxis per institutional guidelines is acceptable.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia

Interventions

venetoclax

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Miriam Garcia, DO
• Organization: MD Anderson Cancer Center

mbgarcia@mdanderson.org

(713) 745-4312

Study Officials

• Miriam Garcia, DO

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 7, 2022
• First Posted: July 19, 2022
• Study Start: April 6, 2023
• Primary Completion: August 12, 2024
• Study Completion: August 12, 2024
• Last Updated: November 26, 2025
• Results First Posted: November 26, 2025

Record last verified: 2025-11

Locations

US (1)