Trial Outcomes & Findings for AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas (NCT NCT02831257)
NCT ID: NCT02831257
Last Updated: 2022-06-13
Results Overview
Number of Target Meningiomas with a Decrease in Tumor Volume of at least 20% Compared with Baseline
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
18 participants
Primary outcome timeframe
up to 24 months
Results posted on
2022-06-13
Participant Flow
Participant milestones
| Measure |
AZD2014
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Overall Study
STARTED
|
18
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
12
|
Reasons for withdrawal
| Measure |
AZD2014
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Overall Study
Lack of Efficacy
|
3
|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Withdrawal by Subject
|
6
|
Baseline Characteristics
AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas
Baseline characteristics by cohort
| Measure |
AZD2014
n=18 Participants
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Age, Continuous
|
41 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 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
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
18 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: up to 24 monthsNumber of Target Meningiomas with a Decrease in Tumor Volume of at least 20% Compared with Baseline
Outcome measures
| Measure |
AZD2014
n=18 target meningiomas
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Radiographic Response Rate for Target Meningioma
|
1 target meningiomas
|
SECONDARY outcome
Timeframe: From date of registration until the date of first documented progression assessed up to 24 monthsKaplan-Meier methodology will be used to estimate progression-free survival, with the 95% confidence intervals based on Greenwood's formula.
Outcome measures
| Measure |
AZD2014
n=18 Participants
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Median Progression-free Survival (PFS)
|
NA months
Interval 24.0 to
The median PFS is "not reached" with a 95% confidence interval of (24 months, infinity)
|
SECONDARY outcome
Timeframe: 6 months of treatmentNumber of target meningiomas without progression at 6 months. Progression is defined using Response Evaluation In Neurofibromatosis and Schwannomatosis (REiNS) criteria, as a 20% increase in the volume of target meningioma compared with baseline
Outcome measures
| Measure |
AZD2014
n=18 target meningiomas
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Progression Free Survival at 6 Month
|
16 target meningiomas
|
SECONDARY outcome
Timeframe: up to 24 monthsThe response rate is the number of non-target meningiomas with at least 20% decrease in volume compared to the baseline volume.
Outcome measures
| Measure |
AZD2014
n=14 non-target meningiomas
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Radiographic Response Rate for Non-target Meningiomas
|
2 non-target meningiomas
|
SECONDARY outcome
Timeframe: up to 24 monthsGrade 3 adverse events as assessed by CTCAE version 5 guidelines
Outcome measures
| Measure |
AZD2014
n=18 Participants
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Number of Participants With Grade 3 Adverse Events
Abdominal pain
|
1 Participants
|
|
Number of Participants With Grade 3 Adverse Events
Anorexia
|
1 Participants
|
|
Number of Participants With Grade 3 Adverse Events
Aspartate aminotransferase increased
|
1 Participants
|
|
Number of Participants With Grade 3 Adverse Events
Hypophosphatemia
|
10 Participants
|
|
Number of Participants With Grade 3 Adverse Events
Pruritis
|
1 Participants
|
|
Number of Participants With Grade 3 Adverse Events
Rash, acneifrom
|
1 Participants
|
|
Number of Participants With Grade 3 Adverse Events
Urticaria
|
1 Participants
|
|
Number of Participants With Grade 3 Adverse Events
Weight loss
|
1 Participants
|
SECONDARY outcome
Timeframe: up to 24 monthsRadiographic response rate is defined as the proportion of vestibular schwannomas with at least 20% decrease in tumor volume compared to baseline
Outcome measures
| Measure |
AZD2014
n=16 vestibular schwannomas
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Radiographic Response Rate of Vestibular Schwannomas
|
3 vestibular schwannomas
|
SECONDARY outcome
Timeframe: Baseline, after 3 months of treatment, and off study (up to 24 months)Population: Analysis population is the participants who completed NFTI-QOL questionnaire at baseline
Neurofibromatosis 2 Impact on Quality of Life (NFTI-QOL) questionnaire. The total NFTI-QOL score ranges from 0 to 24. The total NFTI-QOL score is higher in people with higher morbidity.
Outcome measures
| Measure |
AZD2014
n=14 Participants
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Disease-Specific Quality of Life
Baseline
|
9 score on a scale
Interval 3.0 to 17.0
|
|
Disease-Specific Quality of Life
After 3 months
|
6 score on a scale
Interval 2.0 to 16.0
|
|
Disease-Specific Quality of Life
Off study
|
8.5 score on a scale
Interval 2.0 to 20.0
|
SECONDARY outcome
Timeframe: Baseline, after 3 months of treatment, and off study (up to 24 months)Population: Analysis population includes all participants who completed Pan-QOL questionnaires at baseline
Penn Acoustic Neuroma-Quality of Life (PAN-QOL) questionnaire. Scores are normalized to range from 0 to 100 with higher scores indicating better quality of life.
Outcome measures
| Measure |
AZD2014
n=15 Participants
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Vestibular Schwannoma-Specific Quality of Life
Baseline
|
58.9 score on a scale
Interval 38.7 to 81.6
|
|
Vestibular Schwannoma-Specific Quality of Life
After 3 months
|
65.3 score on a scale
Interval 28.9 to 84.8
|
|
Vestibular Schwannoma-Specific Quality of Life
Off study
|
56.1 score on a scale
Interval 20.5 to 82.1
|
SECONDARY outcome
Timeframe: up to 24 monthsGrade 2 adverse events as assessed by CTCAE version 5 guidelines
Outcome measures
| Measure |
AZD2014
n=18 Participants
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Number of Participants With Grade 2 Adverse Events
Abdominal pain
|
1 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Anorexia
|
2 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Aspartate aminotransferase increased
|
2 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Hypophosphatemia
|
2 Participants
|
|
Number of Participants With Grade 2 Adverse Events
acute kidney injury
|
1 Participants
|
|
Number of Participants With Grade 2 Adverse Events
anemia
|
1 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Tachycardia
|
1 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Weight loss
|
2 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Increased cholesterol
|
2 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Creatine phosphokinase increased
|
1 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Diarrhea
|
6 Participants
|
|
Number of Participants With Grade 2 Adverse Events
dyspareunia
|
1 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Fatigue
|
2 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Increased triglycerides
|
1 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Mucositis
|
2 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Nausea
|
5 Participants
|
|
Number of Participants With Grade 2 Adverse Events
Vomiting
|
1 Participants
|
Adverse Events
AZD2014
Serious events: 7 serious events
Other events: 18 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
AZD2014
n=18 participants at risk
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
General disorders
Heat stroke
|
5.6%
1/18 • 24 months
|
|
Investigations
Weight loss
|
5.6%
1/18 • 24 months
|
|
Nervous system disorders
Seizure
|
5.6%
1/18 • 24 months
|
|
Infections and infestations
Pneumonia
|
5.6%
1/18 • 24 months
|
|
Psychiatric disorders
Psychosis
|
5.6%
1/18 • 24 months
|
|
Gastrointestinal disorders
Abdominal pain
|
11.1%
2/18 • 24 months
|
Other adverse events
| Measure |
AZD2014
n=18 participants at risk
18 patients will be enrolled in this study in a single stage.
* AZD2014 orally, 2 times a day on 2 consecutive day out of every 7 days.
* One cycle will consist of 28 days (1 cycle = 28 days).
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
55.6%
10/18 • 24 months
|
|
Renal and urinary disorders
Acute kidney injury
|
5.6%
1/18 • 24 months
|
|
Investigations
Alanine aminotransferase increased
|
5.6%
1/18 • 24 months
|
|
Blood and lymphatic system disorders
anemia
|
11.1%
2/18 • 24 months
|
|
Metabolism and nutrition disorders
Anorexia
|
38.9%
7/18 • 24 months
|
|
Investigations
aspartate aminotransferase increased
|
27.8%
5/18 • 24 months
|
|
Cardiac disorders
tachycardia
|
5.6%
1/18 • 24 months
|
|
Gastrointestinal disorders
dyspepsia
|
5.6%
1/18 • 24 months
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
16.7%
3/18 • 24 months
|
|
Gastrointestinal disorders
Constipation
|
22.2%
4/18 • 24 months
|
|
Investigations
Creatine phosphokinase increased
|
5.6%
1/18 • 24 months
|
|
Gastrointestinal disorders
Diarrhea
|
83.3%
15/18 • 24 months
|
|
Nervous system disorders
Jaw weakness
|
5.6%
1/18 • 24 months
|
|
Gastrointestinal disorders
Dry mouth
|
38.9%
7/18 • 24 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
5.6%
1/18 • 24 months
|
|
Nervous system disorders
Dysgeusia
|
33.3%
6/18 • 24 months
|
|
Reproductive system and breast disorders
Dyspareunia
|
5.6%
1/18 • 24 months
|
|
General disorders
fatigue
|
66.7%
12/18 • 24 months
|
|
Nervous system disorders
Headache
|
11.1%
2/18 • 24 months
|
|
Gastrointestinal disorders
Hematochezia
|
5.6%
1/18 • 24 months
|
|
Metabolism and nutrition disorders
High cholesterol
|
16.7%
3/18 • 24 months
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
5.6%
1/18 • 24 months
|
|
Investigations
Hyphophosphatemia
|
72.2%
13/18 • 24 months
|
|
Infections and infestations
infection
|
16.7%
3/18 • 24 months
|
|
General disorders
malaise
|
5.6%
1/18 • 24 months
|
|
Nervous system disorders
Difficulty chewing
|
5.6%
1/18 • 24 months
|
|
Gastrointestinal disorders
mucositis
|
38.9%
7/18 • 24 months
|
|
Gastrointestinal disorders
Nausea
|
88.9%
16/18 • 24 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
16.7%
3/18 • 24 months
|
|
Skin and subcutaneous tissue disorders
Rash, acneiform
|
16.7%
3/18 • 24 months
|
|
Reproductive system and breast disorders
amenorrhea
|
5.6%
1/18 • 24 months
|
|
Nervous system disorders
Seizure
|
11.1%
2/18 • 24 months
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
5.6%
1/18 • 24 months
|
|
Reproductive system and breast disorders
vaginal pain
|
5.6%
1/18 • 24 months
|
|
Gastrointestinal disorders
vomiting
|
33.3%
6/18 • 24 months
|
|
Investigations
Weight loss
|
50.0%
9/18 • 24 months
|
|
Investigations
white blood cells decreased
|
5.6%
1/18 • 24 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place