Trial Outcomes & Findings for Phase 2 Study of PI3K Inhibitor Copanlisib in Combination With Fulvestrant in Selected ER+ and/or PR+ Cancers With PI3K (PIK3CA, PIK3R1) and/or PTEN Alterations (NCT NCT05082025)
NCT ID: NCT05082025
Last Updated: 2026-01-09
Results Overview
A DLT is defined as an intervention-related adverse event that impedes a patient from dosing a full cycle of copanlisib (3 doses), a delay of over 1 week in intitated study intervention occurring secondary to any study related adverse event, or death not clearly attrubuted to an underlying disease or alternate cause. Six out of the seven treated patients were evaluable for DLT. One patient was not evaluable for DLT due to not completing a full cycle of treatment within the first 28-days of treatment.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
7 participants
Primary outcome timeframe
Within the first cycle (28-days) of treatment.
Results posted on
2026-01-09
Participant Flow
Phase 2 single center study performed at MD Anderson Cancer Center. Participants were eligible if they had histologically confirmed ER+ and/or PR+ advanced or metastatic ovarian, endometrial, or breast cancers, defined as ≥10% nuclear staining by immunohistochemistry, and confirmed genomic alterations in PIK3CA, PIK3R1, or PTEN.
Only seven patients were treated on dose level 1 in the Part 1dose confirmation part of the study. The dose expansion phase did not occur.
Participant milestones
| Measure |
Part 1: Dose Confirmation - Dose Level 1
Copanlisib 60 mg IV, Days 1, 8, and 15 + Fulvestrant 500 mg IM, Days 1 and 15.
|
|---|---|
|
Overall Study
STARTED
|
7
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Part 1: Dose Confirmation - Dose Level 1
Copanlisib 60 mg IV, Days 1, 8, and 15 + Fulvestrant 500 mg IM, Days 1 and 15.
|
|---|---|
|
Overall Study
Not evaluable for DLT per protocol (dosing less than 1 full cycle of treatment)
|
1
|
Baseline Characteristics
Phase 2 Study of PI3K Inhibitor Copanlisib in Combination With Fulvestrant in Selected ER+ and/or PR+ Cancers With PI3K (PIK3CA, PIK3R1) and/or PTEN Alterations
Baseline characteristics by cohort
| Measure |
Part 1: Dose Confirmation - Dose Level 1
n=7 Participants
Copanlisib 60 mg IV, Days 1, 8, and 15 + Fulvestrant 500 mg IM, Days 1 and 15.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=8 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=8 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=8 Participants
|
|
Age, Continuous
|
58.7 years
n=8 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=8 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=8 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=8 Participants
|
PRIMARY outcome
Timeframe: Within the first cycle (28-days) of treatment.A DLT is defined as an intervention-related adverse event that impedes a patient from dosing a full cycle of copanlisib (3 doses), a delay of over 1 week in intitated study intervention occurring secondary to any study related adverse event, or death not clearly attrubuted to an underlying disease or alternate cause. Six out of the seven treated patients were evaluable for DLT. One patient was not evaluable for DLT due to not completing a full cycle of treatment within the first 28-days of treatment.
Outcome measures
| Measure |
Part 1: Dose Confirmation - Dose Level 1
n=6 Participants
Copanlisib 60 mg IV, Days 1, 8, and 15 + Fulvestrant 500 mg IM, Days 1 and 15.
|
|---|---|
|
Dose Limiting Toxicity (DLT)
|
0 Participants
|
PRIMARY outcome
Timeframe: Through study completion, an average of 14 weeks.Population: All 7 patients treated in Part 1: dose confirmation.
Treatment-related adverse events that were deemed as possibly, probably, or definitely related to treatment, and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All treatment-related adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initation of a new anticancer therapy, whichever occurred first. The number of patients experiencing each treatment related adverse event (at least once) is reported.
Outcome measures
| Measure |
Part 1: Dose Confirmation - Dose Level 1
n=7 Participants
Copanlisib 60 mg IV, Days 1, 8, and 15 + Fulvestrant 500 mg IM, Days 1 and 15.
|
|---|---|
|
Number of Participants With Treatment-related Adverse Events
Mucositis Oral
|
2 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Rash maculo-papular
|
3 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Anemia
|
1 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Diarrhea
|
1 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Nausea
|
1 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Pancreatitis
|
1 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Flatulence
|
1 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Hyperglycemia
|
3 Participants
|
|
Number of Participants With Treatment-related Adverse Events
Fatigue
|
3 Participants
|
Adverse Events
Part 1: Dose Confirmation - Dose Level 1
Serious events: 2 serious events
Other events: 7 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Part 1: Dose Confirmation - Dose Level 1
n=7 participants at risk
Copanlisib 60 mg IV, Days 1, 8, and 15 + Fulvestrant 500 mg IM, Days 1 and 15.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Gastrointestinal disorders
Pancreatitis
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
Other adverse events
| Measure |
Part 1: Dose Confirmation - Dose Level 1
n=7 participants at risk
Copanlisib 60 mg IV, Days 1, 8, and 15 + Fulvestrant 500 mg IM, Days 1 and 15.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Investigations
Alanine aminotransferase increased
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Blood and lymphatic system disorders
Anemia
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Gastrointestinal disorders
Ascites
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Investigations
Aspartate aminotransferase increased
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Skin and subcutaneous tissue disorders
Burn
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Musculoskeletal and connective tissue disorders
Buttock pain
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Gastrointestinal disorders
Constipation
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Investigations
creatinine increased
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Gastrointestinal disorders
diarrhea
|
28.6%
2/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
General disorders
edema limbs
|
28.6%
2/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
General disorders
Fall
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
General disorders
Fatigue
|
42.9%
3/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Gastrointestinal disorders
Flatulence
|
28.6%
2/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
28.6%
2/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
General disorders
Insomnia
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Vascular disorders
Lymphedema
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Gastrointestinal disorders
Mucositis oral
|
28.6%
2/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Gastrointestinal disorders
Nausea
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
General disorders
Neck edema
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
General disorders
pain
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Cardiac disorders
Pericardian Effusion
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Respiratory, thoracic and mediastinal disorders
Pleuritic pain
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
42.9%
3/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Infections and infestations
Skin Infection
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
|
Infections and infestations
Urinary Tract Infection
|
14.3%
1/7 • Through study completion, an average of 14 weeks
Incidence and causality of all adverse events for all patients treated on-study of all grades and attribution and were graded based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 5. All adverse events were captured from the time of signing consent through 30 days after the last dose of study drug or initiation of a new anticancer therapy, whichever occurred first.
|
Additional Information
Dr. Timothy Yap
The University of Texas MD Anderson Cancer Center
Phone: (713) 839-5458
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place