Trial Outcomes & Findings for Everolimus for Children With NF1 Chemotherapy-Refractory Radiographic Progressive Low Grade Gliomas (NCT NCT01158651)
NCT ID: NCT01158651
Last Updated: 2020-02-05
Results Overview
Outcome is 2D MRI target tumor volume compared to baseline volume. Success criteria is volume less than 80% of baseline.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
23 participants
Primary outcome timeframe
Approximately 48 weeks
Results posted on
2020-02-05
Participant Flow
Participant milestones
| Measure |
RAD001 (Everolimus) Active Therapy
If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).
You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection.
RAD001 (Everolimus): If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 course
|
|---|---|
|
Overall Study
STARTED
|
23
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Everolimus for Children With NF1 Chemotherapy-Refractory Radiographic Progressive Low Grade Gliomas
Baseline characteristics by cohort
| Measure |
Active Therapy
n=23 Participants
If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks). You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection. If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).
|
|---|---|
|
Age, Continuous
|
8.8 years
STANDARD_DEVIATION 4.4 • n=93 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
21 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
21 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Approximately 48 weeksPopulation: Intent to treat
Outcome is 2D MRI target tumor volume compared to baseline volume. Success criteria is volume less than 80% of baseline.
Outcome measures
| Measure |
RAD001 (Everolimus) Active Therapy
n=22 Participants
If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks). You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection. If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).
|
|---|---|
|
RAD001 Response Rate Based on 2D MRI Change From Baseline
|
15 participants
Interval 11.0 to 19.0
|
SECONDARY outcome
Timeframe: approximately 48 weeksNumber of participants experiencing serious CTCAE events in the study.
Outcome measures
| Measure |
RAD001 (Everolimus) Active Therapy
n=23 Participants
If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks). You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection. If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).
|
|---|---|
|
Common Terminology Criteria for Adverse Events (CTCAE) Events
|
23 participants
|
Adverse Events
RAD001 (Everolimus) Active Therapy
Serious events: 6 serious events
Other events: 21 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
RAD001 (Everolimus) Active Therapy
n=23 participants at risk
If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks). You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection. If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Cardiac disorders
Cardio Respiratory Arrest
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Dehydration
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Endocrine disorders
Diabetes insipidus
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Investigations
Electrolyte disturbance
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Nervous system disorders
Hydrocephalus
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Investigations
Hypernatremia
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Investigations
Hypokalemia
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Infections and infestations
Infection with absolute neutrophil count (ANC)
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
Low hemoglobin
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pelvic Mass
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Nervous system disorders
Shunt Malfunction
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Surgical and medical procedures
Resection of right temporal lobe tumor
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Eye disorders
Blindness
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Nervous system disorders
Seizure
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Surgical and medical procedures
brain surgery
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Surgical and medical procedures
Brain resection
|
4.3%
1/23 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Vomiting
|
4.3%
1/23 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
RAD001 (Everolimus) Active Therapy
n=23 participants at risk
If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks). You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection. If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).
|
|---|---|
|
Blood and lymphatic system disorders
Leukopenia
|
17.4%
4/23 • Number of events 4 • 1 year
|
|
Blood and lymphatic system disorders
Neutropenia
|
17.4%
4/23 • Number of events 4 • 1 year
|
|
Metabolism and nutrition disorders
Hyperlipidemia
|
8.7%
2/23 • Number of events 2 • 1 year
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
8.7%
2/23 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Mucositis
|
39.1%
9/23 • Number of events 9 • 1 year
|
|
Investigations
Other non-hematologic toxicity
|
43.5%
10/23 • Number of events 36 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place