Trial Outcomes & Findings for INCB018424 in Patients With Advanced Hematologic Malignancies (NCT NCT00674479)

NCT ID: NCT00674479

Last Updated: 2025-06-15

Results Overview

"Time of Response " defined as the period of time from the date of first study drug administration until the first objective documentation of response. Clinical response is defined as Complete remission (CR) + Partial remission (PR) + CRp + Hematologic Improvement (HI). Participants in CR must be free of all symptoms related to leukemia and have an absolute neutrophil count (ANC) \>/= 1x10\^9/L, platelet count ./+ 100x10\^9, normal marrow differential (\</= 5% blasts). Partial remission (PR) is CR with 6-25% abnormal cells in the marrow or 50% decrease in marrow blasts. CRp is CR but platelet count \< 100x10\^9/L. HI is defined as for patients with pretreatment hemoglobin less than 11 g/dL, greater than 2 g/dL increase in hemoglobin; for RBC transfusion-dependent patients, transfusion independence.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

51 participants

Primary outcome timeframe

Patients will be evaluated after each full cycle of therapy (28 days) for response.

Results posted on

2025-06-15

Participant Flow

51 participants received study medication.

Participant milestones

Participant milestones
Measure
INCB018424
The starting dose of INCB018424 will be 25 mg by mouth twice daily. INCB018424: Starting dose: 25 mg by mouth (po) twice daily for 7 days each week for 4 weeks.
Overall Study
STARTED
51
Overall Study
COMPLETED
51
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

INCB018424 in Patients With Advanced Hematologic Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
INCB018424
n=51 Participants
The starting dose of INCB018424 will be 25 mg by mouth twice daily. INCB018424: Starting dose: 25 mg by mouth (po) twice daily for 7 days each week for 4 weeks.
Age, Categorical
<=18 years
0 Participants
n=99 Participants
Age, Categorical
Between 18 and 65 years
16 Participants
n=99 Participants
Age, Categorical
>=65 years
35 Participants
n=99 Participants
Age, Continuous
69 years
n=99 Participants
Sex: Female, Male
Female
15 Participants
n=99 Participants
Sex: Female, Male
Male
36 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
7 Participants
n=99 Participants
Race (NIH/OMB)
White
44 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Region of Enrollment
United States
51 participants
n=99 Participants

PRIMARY outcome

Timeframe: Patients will be evaluated after each full cycle of therapy (28 days) for response.

"Time of Response " defined as the period of time from the date of first study drug administration until the first objective documentation of response. Clinical response is defined as Complete remission (CR) + Partial remission (PR) + CRp + Hematologic Improvement (HI). Participants in CR must be free of all symptoms related to leukemia and have an absolute neutrophil count (ANC) \>/= 1x10\^9/L, platelet count ./+ 100x10\^9, normal marrow differential (\</= 5% blasts). Partial remission (PR) is CR with 6-25% abnormal cells in the marrow or 50% decrease in marrow blasts. CRp is CR but platelet count \< 100x10\^9/L. HI is defined as for patients with pretreatment hemoglobin less than 11 g/dL, greater than 2 g/dL increase in hemoglobin; for RBC transfusion-dependent patients, transfusion independence.

Outcome measures

Outcome measures
Measure
INCB018424
n=51 Participants
The starting dose of INCB018424 will be 25 mg by mouth twice daily. INCB018424: Starting dose: 25 mg by mouth (po) twice daily for 7 days each week for 4 weeks.
Response Rate
7 Participants

SECONDARY outcome

Timeframe: Up to 3 months

Population: INCB018424 PK profile was not done because there was no historical data to compare it to. Data for this Outcome were not collected.

Exploratory sampling will be done to determine the INCB018424 PK profile. The PK parameters of INB018424 will be summarized using descriptive statistics, and the log-transformed INCB018424 PK parameters will be compared using a 1-factor analysis of variance. The mean values of the PK parameters may be compared to historical data in healthy volunteers to determine if the INCB018424 PK profile is different between patients with hematological malignancies and healthy patients.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 3 months

Population: The lab data was not collected so the analysis could not be done.

The PD parameters will be calculated to explore preliminary evidence of PD activity by assessing the effect of INCB018424 on pre- and post-dose. If the p-STAT3/5 signaling data are sufficiently robust, an exploratory PK/PD analysis will be performed.

Outcome measures

Outcome data not reported

Adverse Events

INCB018424

Serious events: 20 serious events
Other events: 0 other events
Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure
INCB018424
n=51 participants at risk
The starting dose of INCB018424 will be 25 mg by mouth twice daily. INCB018424: Starting dose: 25 mg by mouth (po) twice daily for 7 days each week for 4 weeks.
Cardiac disorders
Cerebrovascular Accident
2.0%
1/51 • Number of events 1 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
General disorders
Death
11.8%
6/51 • Number of events 6 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Infections and infestations
Neutropenic Fever
7.8%
4/51 • Number of events 5 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
General disorders
Fever
3.9%
2/51 • Number of events 3 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Vascular disorders
Left Basilic Vein Thrombus
2.0%
1/51 • Number of events 1 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Gastrointestinal disorders
Gastroenteritis
2.0%
1/51 • Number of events 1 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Blood and lymphatic system disorders
Hemorrhage CNS
2.0%
1/51 • Number of events 1 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Infections and infestations
Infection
5.9%
3/51 • Number of events 3 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
General disorders
Fall
2.0%
1/51 • Number of events 1 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Infections and infestations
Pneumonia
7.8%
4/51 • Number of events 4 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Infections and infestations
Sepsis
3.9%
2/51 • Number of events 2 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.
Musculoskeletal and connective tissue disorders
Joint-Effusion
2.0%
1/51 • Number of events 1 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, up to 5 years.

Other adverse events

Adverse event data not reported

Additional Information

Farhad Ravandi-Kashani, MD/Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-745-0394

Results disclosure agreements

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