AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02831257|
Recruitment Status : Completed
First Posted : July 13, 2016
Results First Posted : December 22, 2020
Last Update Posted : December 22, 2020
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis 2 Meningioma||Drug: AZD2014||Phase 2|
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.
The goal of this clinical research study is to learn if the study drug AZD2014 can shrink growing or symptomatic meningiomas. Based on laboratory research, the cellular pathways which are blocked by AZD2014 are important for the growth and survival of meningiomas. Further treatment of meningioma cells in the laboratory setting has resulted in decreased survival of tumor cells. As such, the purpose of this research is to see whether treating your meningioma with AZD2014 will result in tumor shrinkage. The safety of AZD2014 will also be studied. Your physical state, your symptoms, changes in the size of the tumor, and laboratory findings obtained while on-study will help the research team decide if AZD2014 is safe and effective in patients with your condition.
AZD2014 is being studied in patients with various cancers as a single agent (a drug that is used alone to treat the cancer) or in combination with a number of anticancer therapies. Previous studies have also allowed investigators to determine the best dose and frequency of AZD2014 to achieve anti-tumor effects while reducing the likelihood of side effects.
The FDA (the U.S. Food and Drug Administration) has not approved AZD2014 as a treatment for any disease.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||18 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Single Arm Phase 2 Study of the Dual mTORC1/mTORC2 Inhibitor AZD2014 Provided on an Intermittent Schedule for Neurofibromatosis 2 Patients With Progressive or Symptomatic Meningiomas|
|Actual Study Start Date :||August 31, 2016|
|Actual Primary Completion Date :||May 31, 2019|
|Actual Study Completion Date :||October 1, 2020|
18 patients will be enrolled in this study in a single stage.
- Radiographic Response Rate for Target Meningioma [ Time Frame: up to 24 months ]Number of Target Meningiomas with a Decrease in Tumor Volume of at least 20% Compared with Baseline
- Median Progression-free Survival (PFS) [ Time Frame: From date of registration until the date of first documented progression assessed up to 24 months ]Kaplan-Meier methodology will be used to estimate progression-free survival, with the 95% confidence intervals based on Greenwood's formula.
- Progression Free Survival at 6 Month [ Time Frame: 6 months of treatment ]Number 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
- Radiographic Response Rate for Non-target Meningiomas [ Time Frame: up to 24 months ]The response rate is the number of non-target meningiomas with at least 20% decrease in volume compared to the baseline volume.
- Number of Participants With Grade 3 Adverse Events [ Time Frame: up to 24 months ]Grade 3 adverse events as assessed by CTCAE version 5 guidelines
- Radiographic Response Rate of Vestibular Schwannomas [ Time Frame: up to 24 months ]Radiographic response rate is defined as the proportion of vestibular schwannomas with at least 20% decrease in tumor volume compared to baseline
- Disease-Specific Quality of Life [ Time Frame: Baseline, after 3 months of treatment, and off study (up to 24 months) ]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.
- Vestibular Schwannoma-Specific Quality of Life [ Time Frame: Baseline, after 3 months of treatment, and off study (up to 24 months) ]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.
- Number of Participants With Grade 2 Adverse Events [ Time Frame: up to 24 months ]Grade 2 adverse events as assessed by CTCAE version 5 guidelines
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02831257
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02115|
|Dana Farber Cancer Institute|
|Boston, Massachusetts, United States, 02215|
|Principal Investigator:||Scott Plotkin, MD, PhD||Massachusetts General Hospital|