Leukemia, LymphoblasticPhase 2NCT05464836
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
A Phase 2 study testing CB-103 and Venetoclax in people with Leukemia, Lymphoblastic and Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 12 to 60 years
- Conditions the study is for
- Leukemia, Lymphoblastic, Leukemia
- What is being tested?
- CB-103 and Venetoclax
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Apr 2023to 12 Aug 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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)
- Condom or occlusive cap (diaphragm or cervical/vault caps) used with Spermicide
- Abstinence
- Males agree to inform study doctor right away if their partner becomes pregnant or suspects pregnancy. While in this study and for 30 days after the last treatment the participant agrees not to donate sperm for the purposes of reproduction. He agrees to use a condom while in this study and for 30 days after the last treatment.
Who cannot join
- 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
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- 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.
- Known active hepatitis B or C infection or known seropositivity for HIV.
- Liver cirrhosis or other active severe liver disease or suspected active alcohol abuse.
- Have conditions requiring chronic systemic (not inhaled) glucocorticoid use, such as autoimmune disease or severe asthma. Low doses of corticosteroids (10 mg prednisone equivalent a day) are permitted.
- Participants with unresolved nausea, vomiting, or diarrhea of CTCAE version 5.0, grade ˃
- 1 from prior therapy.
- Participants with impairment of GI function or GI disease presence that may significantly alter the absorption of CB-103 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
- Have current or recurrent (within 3 months) gastrointestinal disease, have conditions requiring chronic systemic glucocorticoid use, have active graft versus host disease, or have a second primary or prior malignancy that would affect the interpretation of study results. Low doses of corticosteroids (10 mg prednisone equivalent a day) are permitted.
- Prior chemotherapy/radiotherapy/investigational therapy within 2 weeks before the start of study drugs with the following exceptions: - Up to 5 days of glucocorticoids (10 mg of dexamethasone or equivalent/day) in combination with up to 3 doses of cyclophosphamide (200 mg/m2/day) are allowed as standard pre-phase treatment up to 1 day before start of study treatment.
- Cytarabine up to 2gm/m2 are also allowed as standard pre-phase treatment up to 1 day before start of study treatment.
- Mercaptopurine may be dosed up to 5 days prior to first dose of CB103
- Vinca alkaloids may be dosed up to 5 days prior to first dose of CB103
- Prophylactic intrathecal (IT) chemotherapy may be dosed up to 1 day prior to first dose of CB103
- Females who are pregnant or lactating
- Male or female participants of childbearing potential, unwilling to use an approved, effective means of contraception in accordance with institution's standards.
- Other severe, uncontrolled acute or chronic medical or psychiatric condition or laboratory abnormality that in the opinion of the Investigator may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and/or would make the patient inappropriate for enrollment into this study.
- Participants who are unable or unwilling to comply with all study requirements for clinical visits, examinations, tests, and procedures.
- Participants must be excluded if they are currently enrolled in another ongoing clinical trial with anti-cancer investigational products
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II Study to Evaluate Safety and Efficacy of CB-103 in Combination With Venetoclax in Adolescent and Young Adult Patients With Relapsed/Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL)
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
Read the detailed description
Primary Objectives:
- To characterize the safety and tolerability of CB-103 in combination with Venetoclax in adolescent (12 to 18 years) and adult (19 to 60 years) patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL).
- To assess the efficacy of CB-103 in combination with venetoclax by the overall response rate (ORR), defined as complete remission (CR), plus CR with incomplete blood count recovery (CRi) [1] plus partial remission (PR) [2], in adolescent, and young adult patients with relapsed/refractory T-ALL/T-LBL.
Secondary Objectives:
- To assess whether the ORR to CB-103 in combination with Venetoclax is dependent on pre-treatment expression of Notch and/or BCL2 pathway.
- To determine the preliminary assessment of CB-103 in combination with Venetoclax by other efficacy parameters such as minimal residual disease (MRD), duration of response (DoR), overall survival (OS) and event-free survival (EFS) in adolescent and young adult patients with relapsed/refractory T-ALL/T-LBL.
Exploratory Objectives:
- To explore potential correlations of ORR to treatment and additional pharmacodynamic (PD) markers, i.e., other oncogenic pathway activations that may co-occur at the start of treatment.
- To evaluate how many patients are able to transition to Hematopoietic Stem Cell
Transplant (HSCT):
- Either in the patients achieving CR after the induction and reinduction cycles;
- Or in the patients with PR or stable disease (SD) after the induction and reinduction.
- Conditions
- Leukemia, Lymphoblastic, Leukemia
- Being tested
- CB-103, Venetoclax
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CB-103Drug
Also called Cellestia
Given by PO
VenetoclaxDrug
Also called ABT-199
Given by PO
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: CB-103; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Average of 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2022
- Study start6 Apr 2023
- Primary completion12 Aug 2024
- Study completion12 Aug 2024
Why it stopped: The sponsor requested termination due to internal reprioritization of resources.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Leukemia, LymphoblasticStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Miriam Garcia, DO, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05464836
- Sponsor's protocol ID
- 2021-0547
- Other IDs
- NCI-2022-05687
- Status last verified
- Nov 2025
- First submitted
- 7 Jul 2022
- Last updated
- 26 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study to Evaluate Safety and Efficacy of CB-103 in Combination With Venetoclax in Adolescent and Young Adult Patients With Relapsed/Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL)
- ClinicalTrials.gov ID
- NCT05464836
- Acronym / sponsor ID
- 2021-0547
- Conditions
- Leukemia, Lymphoblastic, Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The sponsor requested termination due to internal reprioritization of resources.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 6 Apr 2023 (actual)
- Primary completion
- 12 Aug 2024 (actual)
- Study completion
- 12 Aug 2024 (actual)
- First posted
- 19 Jul 2022 (actual)
- Results
- Posted 26 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
Primary Objectives:
- To characterize the safety and tolerability of CB-103 in combination with Venetoclax in adolescent (12 to 18 years) and adult (19 to 60 years) patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL).
- To assess the efficacy of CB-103 in combination with venetoclax by the overall response rate (ORR), defined as complete remission (CR), plus CR with incomplete blood count recovery (CRi) [1] plus partial remission (PR) [2], in adolescent, and young adult patients with relapsed/refractory T-ALL/T-LBL.
Secondary Objectives:
- To assess whether the ORR to CB-103 in combination with Venetoclax is dependent on pre-treatment expression of Notch and/or BCL2 pathway.
- To determine the preliminary assessment of CB-103 in combination with Venetoclax by other efficacy parameters such as minimal residual disease (MRD), duration of response (DoR), overall survival (OS) and event-free survival (EFS) in adolescent and young adult patients with relapsed/refractory T-ALL/T-LBL.
Exploratory Objectives:
- To explore potential correlations of ORR to treatment and additional pharmacodynamic (PD) markers, i.e., other oncogenic pathway activations that may co-occur at the start of treatment.
- To evaluate how many patients are able to transition to Hematopoietic Stem Cell
Transplant (HSCT):
- Either in the patients achieving CR after the induction and reinduction cycles;
- Or in the patients with PR or stable disease (SD) after the induction and reinduction.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CB-103+Venetoclax | Experimental | Drug: CB-103, Drug: VenetoclaxControl T-cell acute lymphoblastic leukemia (T-ALL) or T-cell lymphoblastic leukemia (T-LBL) in adolescent and young adult patients. |
Who may qualify
- Ages
- 12 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- 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
Key exclusion criteria (32 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CB-103Also: CellestiaGiven by PO | Drug | CB-103+Venetoclax |
| VenetoclaxAlso: ABT-199Given by PO | Drug | CB-103+Venetoclax |
Primary outcome
Efficacy of CB-103 in Combination With Venetoclax
Time frame: Average of 3 months
Overall response rate (ORR, including CR, CRi, and PR rate) with flow cytometric assessment of minimum residual disease (MRD)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- MD Anderson Cancer CenterLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Miriam Garcia, DO principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CB-103 and Venetoclax in people with Leukemia, Lymphoblastic and Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Nov 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of CB-103 in Combination With Venetoclax (time frame: Average of 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2025.