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ABT-199

Phase 2

Acute Myelogenous Leukemia | Small molecule | Oncology |AbbVie Inc.|Last Updated: Jun 6, 2023

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment32

FDA Designations

No designations recorded

Clinical trial landscape

ABT-199 · 8 trials · 9 indications

Phase 2 1Phase 1 7
NCT01994837A Phase 2 Study of ABT-199 in Subjects With Acute Myelogenous Leukemia (AML)Acute Myelogenous Leukemia
COMPLETED32 Analytics
PHASE2COMPLETED
A Phase 2 Study of ABT-199 in Subjects With Acute Myelogenous Leukemia (AML)
Acute Myelogenous LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Objective Remission Rate
When 19 participants had completed at least 12 weeks of treatment or after all enrolled participants had discontinued venetoclax, whichever was earlier

The objective remission rate (ORR) was defined as the percentage of participants who achieved complete remission (CR), complete remission with incomplete bone marrow recovery (CRi), or partial remission (PR) per the International Working Group criteria for AML. Complete remission (CR) was defined as peripheral neutrophils at least 10˄3/μL, platelets ≥ 10˄5/μL and normocellular bone marrow with ≤ 5% blasts. Complete remission with incomplete bone marrow recovery (CRi) was defined as bone marrow with less than 5% blasts, with peripheral neutrophils of at least 10˄3/μL or platelets ≥ 10˄5/μL. Partial remission (PR) was defined as normalization in peripheral blood neutrophil and platelet counts with at least a 50% decrease in blasts persisting in bone marrow versus baseline.

Change in cardiac assessment findings
Measured from Day 1 up to 6 years after the last subject has enrolled in the study

Electrocardiogram and Multi Gated Acquisition Scan and/or Echocardiogram

Percentage of subjects with adverse events
Measured up to 6 years after the last subject has enrolled in the study

Subjects will be monitored for clinical and laboratory evidence of adverse events throughout the study

Change in clinical laboratory test results
Measured from Day 1 up to 6 years after the last subject has enrolled in the study

Chemistry, coagulation, hematology, lymphocyte enumeration, paraproteins, urinalysis, and, if appropriate, viral polymerase chain reaction and viral serologies

Number of subjects with adverse events
Measured up to 6 years after the last subject has enrolled in the study

Subjects will be monitored for clinical and laboratory evidence of adverse events throughout the study

Change in physical exam finding, including vital signs
Measured from Day 1 up to 6 years after the last subject has enrolled in the study

Body temperature, weight, blood pressure, heart rate

Determination of maximum observed plasma concentration (Cmax), time to Cmax (peak time, Tmax), terminal phase elimination rate constant (beta), terminal phase elimination half-life (t1/2), & area under the plasma concentration-time curve (AUC) of ABT-199
Measured pre-dose and up to 96 hours post-dose ABT-199

Blood samples for pharmacokinetic (PK) analysis of ABT-199 will be collected at designated timepoints to assess the PK parameters for ABT-199 alone relative to ABT-199 with ketoconazole

Determination of peak concentration (Cmax) of ABT-199
Approximately 5 days in Cycle 1 then on Day 1 of Cycles 2,4,6,8

Blood samples for pharmacokinetic analysis of ABT-199 will be collected at designated timepoints

Determine maximum tolerated dose (MTD), and recommended phase two dose (RPTD) of ABT-199
Minimum first cycle of dosing (21 days)

ABT-199 will be dose-escalated until the largest dose is reached that is determined to be safe based on adverse event reporting and dose-limiting toxicities information from all subjects.

Number of participants with adverse events
From subject's first dose of ABT-199 until 30 days after subject's last dose of ABT-199; up to 2 years following last subject first dose.

Collect all adverse events at each visit.

Determination of trough concentration (Ctrough) of ABT-199
Approximately 5 days in Cycle 1 then on Day 1 of Cycles 2,4,6,8

Blood samples for pharmacokinetic analysis of ABT-199 will be collected at designated timepoints

Determination of area under the concentration versus time curve (AUC) of ABT-199
Approximately 5 days in Cycle 1 then on Day 1 of Cycles 2,4,6,8

Blood samples for pharmacokinetic analysis of ABT-199 will be collected at designated timepoints

Determine recommended phase two dose (RPTD) of ABT-199
Minimum first cycle of dosing (21 days

ABT-199 will be dose-escalated until the largest dose is reached that is determined to be safe based on adverse event reporting and dose-limiting toxicities information from all subjects.

Physical Exam including vital signs
Prior to the first dose of ABT-199 until 28 days after single dose of ABT-199 and until 21 days after the last multiple dose of ABT-199

Blood pressure, heart rate and body temperature

Clinical Lab Testing
Prior to the first dose of ABT-199 until 28 days after single dose of ABT-199 and until 21 days after the last multiple dose of ABT-199

Hematology, Chemistry, and Urinalysis

Electrocardiogram (ECG) Measurements
For 24 hours after a single dose of ABT-199 and up to 24 hours after the seventh dose of multiple doses of ABT-199

ECGs done in triplicate

Maximum observed serum concentration (Cmax) of ABT-199
For 72 hours after a single dose of ABT-199 and for 24 hours after the seventh dose of multiple doses of ABT-199

Cmax

Time to Cmax (Tmax) of ABT-199
For 72 hours after a single dose of ABT-199 and for 24 hours after the seventh dose of multiple doses of ABT-199

Time to Cmax

The area under the time curve (AUC) of ABT-199
For 72 hours after a single dose of ABT-199 and for 24 hours after the seventh dose of multiple doses of ABT-199

the area under the exposure-time curve of ABT-199 extrapolated to infinite time for single doses and up to 24 hrs for multiple doses of ABT-199

The terminal phase elimination rate constant and the terminal elimination half-life (t1/2) of ABT-199
For 72 hours after a single dose of ABT-199
Assess the safety profile, to determine the maximum tolerated dose and Recommended Phase Two Dose of ABT-199 when administered in combination with rituximab (R) in subjects with relapsed chronic lymphocytic leukemia and small lymphocytic lymphoma.
Continuous dosing at designated dose level up to Month 6. At end of combination treatment, ABT-199 monotherapy may continue up to 8 years following the date of the last subject enrolled. If disease progression occurs, subjects may re-initiate ABT-199.

Protocol-defined events, which are attributed as having a reasonable possibility of being related to the administration of ABT-199 and/or rituximab, or can not be attributed by the investigator to a clearly identifiable cause such as tumor progression, concurrent illness, underlying disease or concomitant medication, will be considered a dose limiting toxicity.

Determination of the maximum tolerated dose of ABT-199 when administered with Bendamustine and Rituximab in subjects with relapsed or refractory non-Hodgkin's lymphoma
3 days of study drug administration within the 28-day cycle at the designated cohort dose

Protocol-defined events, which can not be attributed by the investigator to a clearly identifiable cause such as tumor progression, concurrent illness, or concomitant medication, will be considered a dose limiting toxicity

Determination of peak concentration (Cmax), trough concentration (Ctrough) and/or area under the concentration versus time curve (AUC) of ABT-199, Bendamustine and Rituximab in the dose escalation cohort
Up to Cycle 6 for ABT-199 and Bendamustine, Up to Cycle 11 for Rituximab

Protocol-defined events, which can not be attributed by the investigator to a clearly identifiable cause such as tumor progression, concurrent illness, or concomitant medication, will be considered a dose limiting toxicity

Number of participants with adverse events as a measure of safety and tolerability
First 5 days of study drug administration, weekly through Cycle 2 and then Day 1 of each Cycle for an anticipated maximum duration of 6 months

Adverse event monitoring, vital signs, physical examination, lymphocyte enumeration, 12-lead ECG, 2D echocardiogram/multiple gated acquisition scan (MUGA), and laboratory assessments

Determination of the recommended Phase 2 dose of ABT-199 when administered with Bendamustine and Rituximab in subjects with relapsed or refractory non-Hodgkin's lymphoma
3 days of study drug administration within the 28-day cycle at the designated cohort dose

Protocol-defined events, which can not be attributed by the investigator to a clearly identifiable cause such as tumor progression, concurrent illness, or concomitant medication, will be considered a dose limiting toxicity

Determination of dose limiting toxicity (DLT), maximum tolerated dose (MTD), recommended phase two dose (RPTD), and lead-in period regimen
Lead-in period (2-5 weeks) plus 3 weeks of study drug administration at the designated cohort dose (continuous dosing)

Protocol-defined events, which can not be attributed by the investigator to a clearly identifiable cause such as tumor progression, underlying illness, concurrent illness, or concomitant medication, will be considered a DLT. Dose limiting toxicities of tumor lysis syndrome observed during the lead-in period will be attributed to the lead-in period.

Determination of plasma peak concentration (Cmax) of ABT-199
Up to Week 24 for ABT-199

Blood and urine samples for pharmacokinetic analysis of ABT-199 will be collected at designated time points

Secondary Endpoints

Complete Remission Rate
When 19 participants had completed at least 12 weeks of treatment or after all enrolled participants had discontinued venetoclax, whichever was earlier
Duration of Remission
When 19 participants had completed at least 12 weeks of treatment or after all enrolled participants had discontinued venetoclax, whichever was earlier
Time to Progression
When 19 participants had completed at least 12 weeks of treatment or after all enrolled participants had discontinued venetoclax, whichever was earlier
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ABT-199EXPERIMENTALContinuous dosing of venetoclax (ABT-199) QD (once daily) beginning with dose-escalation on Week 1 Day 1. Participants received a dose of 20 mg of ABT-199 on Week 1 Day 1, 50 mg on Day 2, 100 mg on Day 3, 200 mg on Day 4, 400 mg on Day 5, 800 mg on Day 6 and QD thereafter.
Arm A (ABT-199 and ketoconazole)EXPERIMENTAL -
ABT-199 + BTZ/Dex Dose Escalation CohortsEXPERIMENTALEvaluate the safety and pharmacokinetics profile of ABT-199 administered with standard therapy bortezomib and dexamethasone in a dose escalation scheme in approximately 54 subjects.
ABT-199 + BTZ/Dex Safety Expansion CohortEXPERIMENTALSafety expansion cohort to further evaluate recommended phase two dose (RPTD) of ABT-199 administered with standard therapy bortezomib and dexamethasone in approximately 12 subjects.
Single DoseEXPERIMENTALSubjects enrolled in the Single Ascending Dose (SAD) part of the study will receive a single dose of study drug or placebo. (Groups 1, 2, 3, 4, 5 and 6).
Multiple DoseEXPERIMENTALSubjects enrolled in the Multiple Ascending Dose (MAD) part of the study will receive multiple doses of study drug or placebo. (Groups 7, 8, 9, 10 and 11)
Arm 1EXPERIMENTALChronic Lymphocytic Leukemia (CLL), Small Lymphocytic Lymphoma (SLL)
Arm A (CLL/SLL subjects)EXPERIMENTALChronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) subjects
Arm B (NHL subjects)EXPERIMENTALNon-Hodgkin lymphoma (NHL) subjects

Interventions

NameTypeDescription
ABT-199DRUGTablet
KetoconazoleDRUGSubjects will be dosed with ABT-199, then dosed with ABT-199 in combination with ketoconazole
bortezomibDRUGBortezomib at cohort-defined dosing schedules and dose levels. Bortezomib at defined dose and schedule for Safety Expansion cohort
dexamethasoneDRUGDexamethasone at cohort-defined dosing schedules and dose levels. Dexamethasone at defined dose and schedule for Safety Expansion cohort.
PlaceboOTHERTablet
RituximabDRUGRituximab will be given by intravenous infusion on day 1 of Months 1, 2, 3, 4, 5, and 6. May be reinitiated for an additional 6 months.
BendamustineDRUGBendamustine will be given by intravenous infusion for 2 days out of each 28 day cycle.
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Eligibility Criteria

Age Range18 Years to 99 Years
SexALL
Healthy VolunteersNo

Inclusion Criteria: 1. Histological or cytological confirmation of relapsed or refractory acute myelogenous leukemia (AML) (by World Health Organization \[WHO\] classification) or untreated AML in participants who are unfit for intensive therapy. 2. Participant has an Eastern Cooperative Oncology G...

Countries:United StatesAustraliaFranceGermanyMexicoPuerto Rico
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Frequently asked questions about ABT-199

What is ABT-199 used for?

ABT-199 is an investigational small molecule being studied for use in several blood cancers and autoimmune conditions, including chronic lymphocytic leukemia, non-Hodgkin lymphoma, small lymphocytic lymphoma, mantle-cell lymphoma, acute myelogenous leukemia, and lupus erythematosus. It is being developed by AbbVie Inc. and is currently in Phase 2 clinical development.

Who makes ABT-199?

ABT-199 is being developed by AbbVie Inc., a biopharmaceutical company traded on the New York Stock Exchange under the ticker ABBV. The drug is an investigational small molecule in Phase 2 clinical development for oncology and autoimmune indications.

What phase is ABT-199 in?

ABT-199 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. The drug is being studied for multiple conditions, including chronic lymphocytic leukemia, non-Hodgkin lymphoma, and lupus erythematosus.

What clinical trials is ABT-199 in?

ABT-199 has completed three Phase 1 clinical trials. NCT01328626 evaluated safety and pharmacokinetics in 222 patients with relapsed or refractory chronic lymphocytic leukemia and non-Hodgkin lymphoma. NCT01682616 studied ABT-199 in combination with rituximab in 49 patients with relapsed chronic lymphocytic leukemia and small lymphocytic lymphoma. NCT01686555 evaluated the drug in 97 female patients with systemic lupus erythematosus.

Is ABT-199 being studied in mantle-cell lymphoma?

Yes, ABT-199 is being studied in mantle-cell lymphoma. A completed Phase 1 trial, NCT02419560, evaluated optimal dosing of ABT-199 in combination with ibrutinib in 37 patients with recurrent mantle-cell lymphoma. The trial was conducted in the United States.

What is the development status of ABT-199 in lupus erythematosus?

ABT-199 has been studied in lupus erythematosus. A completed Phase 1 trial, NCT01686555, evaluated the safety, tolerability, and pharmacokinetics of ABT-199 in 97 female patients with systemic lupus erythematosus. The trial was conducted in the United States, Germany, Mexico, and Puerto Rico.