Trial Outcomes & Findings for Buparlisib in Patients With Relapsed and Refractory Chronic Lymphocytic Leukemia (NCT NCT02340780)
NCT ID: NCT02340780
Last Updated: 2023-08-22
Results Overview
To determine the overall response rate (complete + partial response, as defined in the protocol) to oral buparlisib in patients with relapsed and refractory chronic lymphocytic leukemia. Complete Response (CR): CR requires all of the criteria listed on page 31 of the protocol, maintained for a period of at least 8 weeks. Partial Response (PR): To define a PR, at least 1 of the criteria of Group A plus 1 of the criteria of Group B listed on page 32 of the protocol must be met and persist for ≥ 8 weeks, in the absence of any criteria definitive of progressive disease.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
30 months
Results posted on
2023-08-22
Participant Flow
Participant milestones
| Measure |
Buparlisib
100mg daily orally every 28 days
Buparlisib
|
|---|---|
|
Overall Study
STARTED
|
14
|
|
Overall Study
COMPLETED
|
13
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Buparlisib
100mg daily orally every 28 days
Buparlisib
|
|---|---|
|
Overall Study
Ineligible
|
1
|
Baseline Characteristics
Buparlisib in Patients With Relapsed and Refractory Chronic Lymphocytic Leukemia
Baseline characteristics by cohort
| Measure |
Buparlisib
n=13 Participants
100mg daily orally every 28 days
Buparlisib
|
|---|---|
|
Age, Continuous
|
74 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Performance status
Grade 0
|
5 Participants
n=5 Participants
|
|
Performance status
Grade 1
|
8 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 30 monthsPopulation: All eligible patients who received the protocol treatment.
To determine the overall response rate (complete + partial response, as defined in the protocol) to oral buparlisib in patients with relapsed and refractory chronic lymphocytic leukemia. Complete Response (CR): CR requires all of the criteria listed on page 31 of the protocol, maintained for a period of at least 8 weeks. Partial Response (PR): To define a PR, at least 1 of the criteria of Group A plus 1 of the criteria of Group B listed on page 32 of the protocol must be met and persist for ≥ 8 weeks, in the absence of any criteria definitive of progressive disease.
Outcome measures
| Measure |
Buparlisib
n=13 Participants
100mg daily orally every 28 days
Buparlisib
|
|---|---|
|
Overall Response Rate
Response
|
6 Participants
|
|
Overall Response Rate
Non-response
|
7 Participants
|
SECONDARY outcome
Timeframe: 30 monthsPopulation: All eligible patients who received protocol treatment
Progression-free survival (PFS) is defined as the time in months from study entry until disease progression or death.
Outcome measures
| Measure |
Buparlisib
n=13 Participants
100mg daily orally every 28 days
Buparlisib
|
|---|---|
|
Progression Free Survival
|
21.65 months
Interval 5.36 to
Due to small sample size and small number of events, the upper limit of the 95% confidence interval is not available.
|
Adverse Events
Buparlisib
Serious events: 3 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Buparlisib
n=13 participants at risk
100mg daily orally every 28 days
Buparlisib
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
7.7%
1/13 • 30 months
|
|
Renal and urinary disorders
Hematuria
|
7.7%
1/13 • 30 months
|
|
Surgical and medical procedures
Other surgical and medical procedures
|
7.7%
1/13 • 30 months
|
Other adverse events
| Measure |
Buparlisib
n=13 participants at risk
100mg daily orally every 28 days
Buparlisib
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Back pain
|
15.4%
2/13 • 30 months
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
15.4%
2/13 • 30 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
7.7%
1/13 • 30 months
|
|
Musculoskeletal and connective tissue disorders
Other musculoskeletal and connective tissue disorder
|
7.7%
1/13 • 30 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
15.4%
2/13 • 30 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Other neoplasms benign, malignant and unspecified
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Cognitive disturbance
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Dizziness
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Dysgeusia
|
30.8%
4/13 • 30 months
|
|
Nervous system disorders
Headache
|
23.1%
3/13 • 30 months
|
|
Nervous system disorders
Lethargy
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Memory impairment
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Other nervous system disorders
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Paresthesia
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Seizure
|
7.7%
1/13 • 30 months
|
|
Nervous system disorders
Tremor
|
23.1%
3/13 • 30 months
|
|
Psychiatric disorders
Agitation
|
7.7%
1/13 • 30 months
|
|
Psychiatric disorders
Anxiety
|
15.4%
2/13 • 30 months
|
|
Psychiatric disorders
Confusion
|
15.4%
2/13 • 30 months
|
|
Psychiatric disorders
Depression
|
7.7%
1/13 • 30 months
|
|
Psychiatric disorders
Insomnia
|
15.4%
2/13 • 30 months
|
|
Psychiatric disorders
Other psychiatric disorders
|
7.7%
1/13 • 30 months
|
|
Psychiatric disorders
Personality change
|
7.7%
1/13 • 30 months
|
|
Renal and urinary disorders
Hematuria
|
7.7%
1/13 • 30 months
|
|
Renal and urinary disorders
Renal calculi
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial obstruction
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
46.2%
6/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
38.5%
5/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
15.4%
2/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
7.7%
1/13 • 30 months
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
7.7%
1/13 • 30 months
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
15.4%
2/13 • 30 months
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
7.7%
1/13 • 30 months
|
|
Skin and subcutaneous tissue disorders
Other skin and subcutaneous tissue disorders
|
38.5%
5/13 • 30 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
7.7%
1/13 • 30 months
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
7.7%
1/13 • 30 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
23.1%
3/13 • 30 months
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
7.7%
1/13 • 30 months
|
|
Surgical and medical procedures
Other surgical and medical procedures
|
7.7%
1/13 • 30 months
|
|
Vascular disorders
Hematoma
|
7.7%
1/13 • 30 months
|
|
Vascular disorders
Hypertension
|
23.1%
3/13 • 30 months
|
|
Vascular disorders
Other vascular disorders
|
7.7%
1/13 • 30 months
|
|
Vascular disorders
Peripheral ischemia
|
7.7%
1/13 • 30 months
|
|
Vascular disorders
Thromboembolic event
|
7.7%
1/13 • 30 months
|
|
Cardiac disorders
Paroxysmal atrial tachycardia
|
7.7%
1/13 • 30 months
|
|
Ear and labyrinth disorders
Hearing impaired
|
7.7%
1/13 • 30 months
|
|
Ear and labyrinth disorders
Other ear and labyrinth disorders
|
15.4%
2/13 • 30 months
|
|
Ear and labyrinth disorders
Tinnitus
|
7.7%
1/13 • 30 months
|
|
Endocrine disorders
Other endocrine disorders
|
7.7%
1/13 • 30 months
|
|
Eye disorders
Blurred vision
|
15.4%
2/13 • 30 months
|
|
Eye disorders
Cataract
|
7.7%
1/13 • 30 months
|
|
Eye disorders
Other eye disorders
|
7.7%
1/13 • 30 months
|
|
Gastrointestinal disorders
Abdominal pain
|
7.7%
1/13 • 30 months
|
|
Gastrointestinal disorders
Constipation
|
7.7%
1/13 • 30 months
|
|
Gastrointestinal disorders
Diarrhea
|
53.8%
7/13 • 30 months
|
|
Gastrointestinal disorders
Dry mouth
|
7.7%
1/13 • 30 months
|
|
Gastrointestinal disorders
Dyspepsia
|
23.1%
3/13 • 30 months
|
|
Gastrointestinal disorders
Dysphagia
|
7.7%
1/13 • 30 months
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
15.4%
2/13 • 30 months
|
|
Gastrointestinal disorders
Hemorrhoids
|
7.7%
1/13 • 30 months
|
|
Gastrointestinal disorders
Mucositis oral
|
23.1%
3/13 • 30 months
|
|
Gastrointestinal disorders
Nausea
|
30.8%
4/13 • 30 months
|
|
Gastrointestinal disorders
Other gastrointestinal disorders
|
7.7%
1/13 • 30 months
|
|
General disorders
Chills
|
7.7%
1/13 • 30 months
|
|
General disorders
Edema face
|
7.7%
1/13 • 30 months
|
|
General disorders
Edema limbs
|
15.4%
2/13 • 30 months
|
|
General disorders
Fatigue
|
61.5%
8/13 • 30 months
|
|
General disorders
Fever
|
7.7%
1/13 • 30 months
|
|
General disorders
Irritability
|
7.7%
1/13 • 30 months
|
|
General disorders
Non-cardiac chest pain
|
7.7%
1/13 • 30 months
|
|
General disorders
Other general disorders, administration site conditions
|
15.4%
2/13 • 30 months
|
|
General disorders
Pain
|
15.4%
2/13 • 30 months
|
|
Infections and infestations
Lung infection
|
7.7%
1/13 • 30 months
|
|
Infections and infestations
Other infections and infestations
|
15.4%
2/13 • 30 months
|
|
Infections and infestations
Otitis media
|
7.7%
1/13 • 30 months
|
|
Infections and infestations
Sinusitis
|
7.7%
1/13 • 30 months
|
|
Infections and infestations
Skin infection
|
7.7%
1/13 • 30 months
|
|
Infections and infestations
Upper respiratory infection
|
7.7%
1/13 • 30 months
|
|
Injury, poisoning and procedural complications
Bruising
|
15.4%
2/13 • 30 months
|
|
Injury, poisoning and procedural complications
Fall
|
7.7%
1/13 • 30 months
|
|
Injury, poisoning and procedural complications
Other injury, poisoning and procedural complications
|
7.7%
1/13 • 30 months
|
|
Investigations
Alanine aminotransferase increased
|
7.7%
1/13 • 30 months
|
|
Investigations
Platelet count decreased
|
7.7%
1/13 • 30 months
|
|
Metabolism and nutrition disorders
Anorexia
|
30.8%
4/13 • 30 months
|
|
Metabolism and nutrition disorders
Dehydration
|
7.7%
1/13 • 30 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
30.8%
4/13 • 30 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place