Trial Outcomes & Findings for Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2 (NCT NCT01419639)
NCT ID: NCT01419639
Last Updated: 2017-07-18
Results Overview
To estimate the objective response rates to RAD001 in patients with NF2-related tumors including cranial nerve schwannomas, meningiomas and ependymomas. Radiographic response for study purposes = greater than or equal to 15% reduction in tumor volume in any of the target tumors (partial response). Complete disappearance of any of the target tumors = complete response. MRI of the brain and spine will be performed every 3 months. If an objective response (15% reduction in tumor volume compared to baseline) is observed in any target tumor or stable disease, drug will be continued.
COMPLETED
PHASE2
10 participants
1 Year
2017-07-18
Participant Flow
10 people enrolled; a total of 9 completed study
Participant milestones
| Measure |
RAD001
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2
Baseline characteristics by cohort
| Measure |
RAD001
n=10 Participants
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
|
|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
27 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 1 YearTo estimate the objective response rates to RAD001 in patients with NF2-related tumors including cranial nerve schwannomas, meningiomas and ependymomas. Radiographic response for study purposes = greater than or equal to 15% reduction in tumor volume in any of the target tumors (partial response). Complete disappearance of any of the target tumors = complete response. MRI of the brain and spine will be performed every 3 months. If an objective response (15% reduction in tumor volume compared to baseline) is observed in any target tumor or stable disease, drug will be continued.
Outcome measures
| Measure |
RAD001
n=12 Tumors
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
|
|---|---|
|
Radiographic Response
|
0 participants
|
PRIMARY outcome
Timeframe: 1 YearOutcome measures
| Measure |
RAD001
n=12 Tumors
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
|
|---|---|
|
Change in Tumor Size From Baseline
|
5.72 % of change in tumor size from baseline
Interval -14.0 to 25.71
|
SECONDARY outcome
Timeframe: 1 YearDefined as improvement in speech discrimination score (SDS), defined as an improvement in the score above the 95% critical difference threshold, compared to baseline audiogram at initiation of treatment. Audiologic worsening: decrease in SDS score below the 95% critical difference threshold, compared to baseline audiogram at initiation of treatment. Patients with vestibular schwannomas will receive baseline audiograms within 28 days before enrollments and subsequent audiograms at the time of each MRI.
Outcome measures
| Measure |
RAD001
n=9 Participants
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
|
|---|---|
|
Audiologic Response
|
0 participants
|
Adverse Events
RAD001
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
RAD001
n=10 participants at risk
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
|
|---|---|
|
Investigations
alkaline phosphatase increased
|
20.0%
2/10 • Continuously throughout 1 year of study
|
|
Blood and lymphatic system disorders
anemia
|
50.0%
5/10 • Continuously throughout 1 year of study
|
|
Psychiatric disorders
anxiety
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Investigations
AST increased
|
30.0%
3/10 • Continuously throughout 1 year of study
|
|
Reproductive system and breast disorders
azoospermia
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Injury, poisoning and procedural complications
bruising
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
General disorders
chills
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Investigations
cholesterol high
|
70.0%
7/10 • Continuously throughout 1 year of study
|
|
Gastrointestinal disorders
constipation
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Psychiatric disorders
depression
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Gastrointestinal disorders
diarrhea
|
40.0%
4/10 • Continuously throughout 1 year of study
|
|
Skin and subcutaneous tissue disorders
dry skin
|
30.0%
3/10 • Continuously throughout 1 year of study
|
|
Nervous system disorders
dysgeusia
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Respiratory, thoracic and mediastinal disorders
epistaxis
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
General disorders
fatigue
|
60.0%
6/10 • Continuously throughout 1 year of study
|
|
General disorders
fever
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Nervous system disorders
headache
|
60.0%
6/10 • Continuously throughout 1 year of study
|
|
Metabolism and nutrition disorders
hyperglycemia
|
50.0%
5/10 • Continuously throughout 1 year of study
|
|
Metabolism and nutrition disorders
hypernatremia
|
50.0%
5/10 • Continuously throughout 1 year of study
|
|
Vascular disorders
hypertension
|
20.0%
2/10 • Continuously throughout 1 year of study
|
|
Metabolism and nutrition disorders
hypertriglyceridemia
|
40.0%
4/10 • Continuously throughout 1 year of study
|
|
Metabolism and nutrition disorders
hypoglycemia
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Metabolism and nutrition disorders
hyponatremia
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Metabolism and nutrition disorders
hypophosphatemia
|
20.0%
2/10 • Continuously throughout 1 year of study
|
|
Reproductive system and breast disorders
irregular menstruation
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Investigations
lymphocyte count decreased
|
50.0%
5/10 • Continuously throughout 1 year of study
|
|
Gastrointestinal disorders
mucositis oral
|
100.0%
10/10 • Continuously throughout 1 year of study
|
|
Musculoskeletal and connective tissue disorders
muscle weakness- right sided
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Respiratory, thoracic and mediastinal disorders
nasal congestion
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Gastrointestinal disorders
nausea
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Musculoskeletal and connective tissue disorders
neck pain
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Investigations
neutrophil count decreased
|
30.0%
3/10 • Continuously throughout 1 year of study
|
|
General disorders
non-cardiac chest pain
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Musculoskeletal and connective tissue disorders
pain (to bone; right clavicula)
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Musculoskeletal and connective tissue disorders
pain (to extremity; left 4th finger)
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Musculoskeletal and connective tissue disorders
pain (to right flank)
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Investigations
platelet count decreased
|
60.0%
6/10 • Continuously throughout 1 year of study
|
|
Skin and subcutaneous tissue disorders
pruritis
|
40.0%
4/10 • Continuously throughout 1 year of study
|
|
Skin and subcutaneous tissue disorders
rash
|
50.0%
5/10 • Continuously throughout 1 year of study
|
|
Skin and subcutaneous tissue disorders
rash acneiform
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Skin and subcutaneous tissue disorders
rash maculo-papular
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Skin and subcutaneous tissue disorders
scalp pain
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Infections and infestations
skin infection
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Gastrointestinal disorders
toothache
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Gastrointestinal disorders
vomiting
|
10.0%
1/10 • Continuously throughout 1 year of study
|
|
Investigations
white blood cell decreased
|
40.0%
4/10 • Continuously throughout 1 year of study
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place