Trial Outcomes & Findings for Ibrutinib as Early Therapy in Chronic Lymphocytic Leukemia (CLL) (NCT NCT03207555)
NCT ID: NCT03207555
Last Updated: 2025-09-02
Results Overview
Complete Remission (CR): Peripheral blood lymphocytes below 4 x 10\^9/L (4000/ƒÝL), Absence of significant lymphadenopathy (lymph nodes \>1.5 cm in diameter) by CT(or PET) examination of neck, thorax, abdomen and pelvis, No hepatomegaly or splenomegaly by physical examination (and CT/PET if assessment was abnormal before therapy or if physical exam is inconclusive), Absence of constitutional symptoms, Neutrophils more than 1.5 x 10\^9/L (1500/ƒÝL) without need for exogenous growth factors, Platelets more than 100 x 10\^9/L (100 000/ƒÝL) without need for exogenous growth factors, Hemoglobin more than 110 g/L (11.0 g/dL) without red blood cell transfusion or need for exogenous erythropoietin, Bone Marrow aspirate and biopsy, demonstrating at least normocellular for age, with less than 30% of nucleated cells being lymphocytes. Lymphoid nodules should be absent. If the bone marrow is hypocellular, a repeat determination should be made in 4 weeks or when peripheral blood counts have recovered.
COMPLETED
PHASE2
23 participants
Up to 24 months
2025-09-02
Participant Flow
Participant milestones
| Measure |
Ibrutinib
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Overall Study
STARTED
|
23
|
|
Overall Study
COMPLETED
|
23
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Ibrutinib as Early Therapy in Chronic Lymphocytic Leukemia (CLL)
Baseline characteristics by cohort
| Measure |
Ibrutinib
n=23 Participants
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
8 Participants
n=5 Participants
|
|
Age, Continuous
|
60 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
23 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
23 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 24 monthsPopulation: Of the 23 participants registered on-study, 18 participants were evaluable.
Complete Remission (CR): Peripheral blood lymphocytes below 4 x 10\^9/L (4000/ƒÝL), Absence of significant lymphadenopathy (lymph nodes \>1.5 cm in diameter) by CT(or PET) examination of neck, thorax, abdomen and pelvis, No hepatomegaly or splenomegaly by physical examination (and CT/PET if assessment was abnormal before therapy or if physical exam is inconclusive), Absence of constitutional symptoms, Neutrophils more than 1.5 x 10\^9/L (1500/ƒÝL) without need for exogenous growth factors, Platelets more than 100 x 10\^9/L (100 000/ƒÝL) without need for exogenous growth factors, Hemoglobin more than 110 g/L (11.0 g/dL) without red blood cell transfusion or need for exogenous erythropoietin, Bone Marrow aspirate and biopsy, demonstrating at least normocellular for age, with less than 30% of nucleated cells being lymphocytes. Lymphoid nodules should be absent. If the bone marrow is hypocellular, a repeat determination should be made in 4 weeks or when peripheral blood counts have recovered.
Outcome measures
| Measure |
Ibrutinib
n=18 Participants
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Participants With Complete Remission (CR) at 24 Months
|
3 Participants
|
PRIMARY outcome
Timeframe: Up to 24 monthsPopulation: Of the 23 participants registered on-study, 18 participants were evaluable.
Partial Remission (PR) at least one of criteria 1-3, and one or more of the features listed in number 4. 1. A decrease lymphocytes by 50% or more from the value before therapy. 2. Reduction in lymphadenopathy, defined by the following: - A decrease in lymph node size by 50% or more either in the sum products of up to 6 lymph nodes, or in the largest diameter of the enlarged lymph node(s) detected prior to therapy. - No increase in lymph node, and no new enlarged lymph node. In small lymph nodes (\</=2 cm), an increase of less than 25% is not considered to be significant. 3. A reduction in enlargement of the spleen or liver by 50% or more. 4. The blood count should show one or more of the following results: - Neutrophils more than 1, without need for exogenous growth factors. - Platelet counts greater than 100 x10\^9/L (100 000/ƒÝL) or 50% improvement over baseline without need for exogenous growth factors. - Hemoglobin greater than 11
Outcome measures
| Measure |
Ibrutinib
n=18 Participants
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Participants With Partial Remission (PR) at 24 Months
|
15 Participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: Of the 23 participants registered on-study, 18 participants were evaluable.
Time from date of treatment start until the date of progression or death from leukemia.
Outcome measures
| Measure |
Ibrutinib
n=18 Participants
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Progression-Free Survival (PFS) at 24 Months
|
NA Months
The Progression Free Survival was not met below the level of detection
|
SECONDARY outcome
Timeframe: Up to 24 monthsPopulation: Of the 23 participants registered on-study, 18 participants were evaluable.
Overall Response is participants who achieve Complete Remission (CR) or Partial Remission (PR) at 24 months of treatment.
Outcome measures
| Measure |
Ibrutinib
n=18 Participants
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Overall Response at 24 Months
|
18 Participants
|
Adverse Events
Ibrutinib
Serious adverse events
| Measure |
Ibrutinib
n=23 participants at risk
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Infections and infestations
Infections and infestations - Other, specify
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
|
13.0%
3/23 • Number of events 3 • through 24 months
|
|
Nervous system disorders
Syncope
|
4.3%
1/23 • Number of events 1 • through 24 months
|
|
Eye disorders
Blurred vision
|
4.3%
1/23 • Number of events 1 • through 24 months
|
Other adverse events
| Measure |
Ibrutinib
n=23 participants at risk
Participants take Ibrutinib by mouth 1 time every day for up to 2 years (24 cycles).
Ibrutinib: 420 mg by mouth once daily.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
34.8%
8/23 • Number of events 10 • through 24 months
|
|
Blood and lymphatic system disorders
Anemia
|
13.0%
3/23 • Number of events 3 • through 24 months
|
|
Psychiatric disorders
Anxiety
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
47.8%
11/23 • Number of events 16 • through 24 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
17.4%
4/23 • Number of events 5 • through 24 months
|
|
Injury, poisoning and procedural complications
Bruising
|
56.5%
13/23 • Number of events 16 • through 24 months
|
|
General disorders
Chills
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Gastrointestinal disorders
Constipation
|
21.7%
5/23 • Number of events 5 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
26.1%
6/23 • Number of events 6 • through 24 months
|
|
Gastrointestinal disorders
Diarrhea
|
21.7%
5/23 • Number of events 7 • through 24 months
|
|
Nervous system disorders
Dizziness
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
17.4%
4/23 • Number of events 4 • through 24 months
|
|
Gastrointestinal disorders
Dysphagia
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders - Other, specify
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
General disorders
Edema limbs
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
17.4%
4/23 • Number of events 5 • through 24 months
|
|
Eye disorders
Eye disorders - Other, specify
|
13.0%
3/23 • Number of events 3 • through 24 months
|
|
General disorders
Fatigue
|
73.9%
17/23 • Number of events 17 • through 24 months
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
13.0%
3/23 • Number of events 3 • through 24 months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
8.7%
2/23 • Number of events 3 • through 24 months
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
17.4%
4/23 • Number of events 4 • through 24 months
|
|
Nervous system disorders
Headache
|
21.7%
5/23 • Number of events 6 • through 24 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
8.7%
2/23 • Number of events 3 • through 24 months
|
|
Vascular disorders
Hypertension
|
30.4%
7/23 • Number of events 11 • through 24 months
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Infections and infestations
Infections and infestations - Other, specify
|
21.7%
5/23 • Number of events 6 • through 24 months
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications - Other, specify
|
30.4%
7/23 • Number of events 8 • through 24 months
|
|
General disorders
Malaise
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Nervous system disorders
Memory impairment
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, specify
|
13.0%
3/23 • Number of events 3 • through 24 months
|
|
Gastrointestinal disorders
Mucositis oral
|
13.0%
3/23 • Number of events 4 • through 24 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
|
13.0%
3/23 • Number of events 3 • through 24 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
39.1%
9/23 • Number of events 13 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
30.4%
7/23 • Number of events 8 • through 24 months
|
|
Gastrointestinal disorders
Nausea
|
13.0%
3/23 • Number of events 3 • through 24 months
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
General disorders
Pain
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Cardiac disorders
Palpitations
|
17.4%
4/23 • Number of events 4 • through 24 months
|
|
Nervous system disorders
Paresthesia
|
26.1%
6/23 • Number of events 6 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Postnasal drip
|
17.4%
4/23 • Number of events 4 • through 24 months
|
|
Psychiatric disorders
Psychiatric disorders - Other, specify
|
34.8%
8/23 • Number of events 9 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
39.1%
9/23 • Number of events 15 • through 24 months
|
|
Ear and labyrinth disorders
Tinnitus
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory infection
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Infections and infestations
Urinary tract infection
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Renal and urinary disorders
Urinary urgency
|
8.7%
2/23 • Number of events 2 • through 24 months
|
|
Gastrointestinal disorders
Vomiting
|
8.7%
2/23 • Number of events 2 • through 24 months
|
Additional Information
Jan Burger, MD/Professor
The University of Texas MD Anderson
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place