Trial Outcomes & Findings for Umbralisib Plus Ublituximab (U2) in Progressive CLL After Novel Therapy (NCT NCT04149821)
NCT ID: NCT04149821
Last Updated: 2023-06-15
Results Overview
Number of subjects who achieve a partial or complete response
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
1 participants
Primary outcome timeframe
11 months
Results posted on
2023-06-15
Participant Flow
Participant milestones
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
|---|---|---|
|
Overall Study
STARTED
|
0
|
1
|
|
Overall Study
COMPLETED
|
0
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Umbralisib Plus Ublituximab (U2) in Progressive CLL After Novel Therapy
Baseline characteristics by cohort
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
n=1 Participants
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Total
n=1 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
—
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 11 monthsPopulation: 0 subjects were enrolled in Cohort A.
Number of subjects who achieve a partial or complete response
Outcome measures
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
n=1 Participants
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
|---|---|---|
|
Efficacy of Umbralisib in Combination With Ublituximab (U2) as Measured by Overall Response Rate (ORR) in Patients With CLL Who Have Progressed on a BTKi or BCL-2 Inhibitor
|
—
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 year and 4 monthsPopulation: 0 subjects were enrolled in Cohort A.
Rate of subjects who experience 1 or more adverse events
Outcome measures
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
n=1 Participants
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
|---|---|---|
|
Safety of Umbralisib in Combination With Ublituximab (U2) as Measured by the Percentage of Subjects Who Experience 1 or More Adverse Events
|
—
|
1 Participants
|
SECONDARY outcome
Timeframe: 1 year and 4 monthsPopulation: 0 subjects were enrolled in Cohort A.
Number of subjects who achieve complete response on study
Outcome measures
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
n=1 Participants
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
|---|---|---|
|
Complete Remission Rate as Measured by the Number of Subjects Who Achieve Complete Response as Their Best Response
|
—
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 year and 4 monthsPopulation: 0 subjects were enrolled in Cohort A.
Median interval of time between subjects' first objective response to the first sign of disease progression
Outcome measures
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
n=1 Participants
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
|---|---|---|
|
Duration of Response
|
—
|
0 years
|
Adverse Events
Cohort A Post BTKi Therapy
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Cohort B Post BCL-2 Therapy
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
n=1 participants at risk
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
|---|---|---|
|
Gastrointestinal disorders
Vomiting
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Metabolism and nutrition disorders
Hyponatremia
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Nervous system disorders
Syncope
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
Other adverse events
| Measure |
Cohort A Post BTKi Therapy
Patients who progress after a BTKi containing regimen
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
Cohort B Post BCL-2 Therapy
n=1 participants at risk
* Patients who progress after BCL-2 containing regimens
* Patients who progress on a regimen containing both a BTKi and a BCL-2 inhibitor
Umbralisib: -Lead-in cycle of 28 days (Cycle -1) of umbralisib monotherapy, 800mg oral daily
-Continued 800mg oral daily dosing in all subsequent cycles (combination therapy cycles 1-6, then continued monotherapy cycle 7 and beyond)
Ublituximab: -Co-administered on days 1 and 2 (dose split over days 1 and 2), 8, 15 of cycle 1 then on day 1 for subsequent cycles 2-6
|
|---|---|---|
|
Eye disorders
Blurred vision
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 3 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Nervous system disorders
Dizziness
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 5 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Nervous system disorders
Headache
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 7 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
General disorders
Fatigue
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 13 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Investigations
Lactate dehydrogenase increased
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 6 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Blood and lymphatic system disorders
Anemia
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 7 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Investigations
Hypocalcemia
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 2 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Investigations
Creatinine increased
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 3 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Investigations
Thrombocytopenia
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 5 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Hepatobiliary disorders
Dysuria
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Metabolism and nutrition disorders
Dehydration
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
General disorders
Tongue numbness
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 3 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Gastrointestinal disorders
Nausea
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 4 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Gastrointestinal disorders
Vomiting
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
General disorders
Numbness
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 2 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Nervous system disorders
Neuropathy
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 3 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
General disorders
Axillary Lymphadenopathy
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 2 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
General disorders
Abdominal Pain
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 2 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Metabolism and nutrition disorders
Constipation
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Psychiatric disorders
Insomnia
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 2 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
|
Skin and subcutaneous tissue disorders
Mouth Sores
|
—
0/0 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
100.0%
1/1 • Number of events 1 • 1 year and 3 months
0 subjects were enrolled in Cohort A."
|
Additional Information
Dr. John Allan, Associate Professor of Medicine
Weill Cornell Medicine
Phone: 2127463481
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place