Trial Outcomes & Findings for Single-arm Study With Bimiralisib in Patients With HNSCC Harboring NOTCH1 Loss of Function Mutations (NCT NCT03740100)
NCT ID: NCT03740100
Last Updated: 2023-07-14
Results Overview
Radiological tumor assessments were performed by computed tomography (CT) or magnetic resonance imaging (MRI) according to a standard protocol. ORR: comprised of all patients who achieved a confirmed partial or a confirmed complete response per RECIST 1.1
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
At 6 and 12 weeks after the start of therapy (± 3 days)
Results posted on
2023-07-14
Participant Flow
Participant milestones
| Measure |
Bimilralisib
Bimiralisib capsules orally.
All patients received 140 mg bimiralisib (PQR309) 140 mg orally once daily on two consecutive days followed by five days without treatment weekly until unacceptable toxicity, tumor progression, patient's request for withdrawal, investigator judgment, or death.
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
Confirmed Partial Response
|
1
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Bimilralisib
Bimiralisib capsules orally.
All patients received 140 mg bimiralisib (PQR309) 140 mg orally once daily on two consecutive days followed by five days without treatment weekly until unacceptable toxicity, tumor progression, patient's request for withdrawal, investigator judgment, or death.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Death
|
1
|
Baseline Characteristics
Single-arm Study With Bimiralisib in Patients With HNSCC Harboring NOTCH1 Loss of Function Mutations
Baseline characteristics by cohort
| Measure |
Bimiralisib Treatment
n=8 Participants
Bimiralisib capsules (140 mg) orally once daily on 2 consecutive days followed by 5 days without treatment weekly.
|
|---|---|
|
Age, Continuous
|
65.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
8 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At 6 and 12 weeks after the start of therapy (± 3 days)Radiological tumor assessments were performed by computed tomography (CT) or magnetic resonance imaging (MRI) according to a standard protocol. ORR: comprised of all patients who achieved a confirmed partial or a confirmed complete response per RECIST 1.1
Outcome measures
| Measure |
Open Single Arm
n=6 Participants
Bimiralisib capsules orally
Bimiralisib: Bimiralisib capsules
|
|---|---|
|
Objective Response Rate (ORR)
|
1 Participants
|
Adverse Events
Open Single Arm
Serious events: 5 serious events
Other events: 8 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Open Single Arm
n=8 participants at risk
Bimiralisib capsules orally
Bimiralisib: Bimiralisib capsules
|
|---|---|
|
General disorders
Disease Progression
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Infections and infestations
Skin Infection
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Infections and infestations
Osteomyelitis
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Infections and infestations
Sepsis
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Investigations
Hyperglycemia
|
25.0%
2/8 • Number of events 2 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Investigations
Alanine aminotransferase increased
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration Pneumonia
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Distress
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal Stenosis
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Renal and urinary disorders
Renal Injury
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Vascular disorders
Hemorrhage
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Blood and lymphatic system disorders
Anemia
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
Other adverse events
| Measure |
Open Single Arm
n=8 participants at risk
Bimiralisib capsules orally
Bimiralisib: Bimiralisib capsules
|
|---|---|
|
Blood and lymphatic system disorders
Platelet count decreased
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Renal and urinary disorders
Blood creatinine increased
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Metabolism and nutrition disorders
Dehydration
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Investigations
Blood glucose increased
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Gastrointestinal disorders
Nausea
|
25.0%
2/8 • Number of events 2 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Gastrointestinal disorders
vomiting
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Gastrointestinal disorders
Diarrhea
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
2/8 • Number of events 2 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
General disorders
Fatigue
|
25.0%
2/8 • Number of events 3 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
|
Ear and labyrinth disorders
Tinnitus
|
12.5%
1/8 • Number of events 1 • From the first dose of study drug (bimiralisib) until 30 days after the last dose of bimiralisib.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place