Trial Outcomes & Findings for Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors (NCT NCT03095248)
NCT ID: NCT03095248
Last Updated: 2025-04-22
Results Overview
The primary endpoint for the Stratum 1 is hearing response at 24 weeks, defined as an improvement in word recognition score above the 95% critical difference, taking as reference the baseline word recognition score. Audiology will include measurement of pure tone thresholds and determination of word recognition scores. Word recognition scores measure the ability to recognize (as opposed to detect) auditory information. Patients are presented a list of 50 words at a fully audible level and the percentage identified correctly is the score. This study will use full 50-item monosyllable lists and standardized recordings. This Outcome Measure was pre-specified to be assessed only within the Stratum 1 Arm/Group.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
24 weeks
Results posted on
2025-04-22
Participant Flow
Patients were identified through referrals and from the PIs current patient population. All patients were enrolled at Cincinnati Children's Hospital Medical Center. The study was opened to enrollment on 05/08/2017 and closed to accrual on 06/09/2024,
There were a total of 10 participants consented who met eligibility criteria and received study interventions.
Participant milestones
| Measure |
Stratum 1 - NF2 Related Vestibular Schwannomas
Stratum 1 included patients with NF2 with vestibular schwannomas who exhibit hearing loss. Participants received continuous twice daily dosing of selumetinib. Dosing was based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy continued for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
Stratum 2: Other NF2 Related Tumors (Meningiomas and Ependymoma)
Stratum 2 will include patients who have progressive lesions other than VS (including non-vestibular schwannomas, meningiomas, and spinal cord lesions). Participants will receive continuous twice daily dosing of selumentinib. Dosing is based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy may continue for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
9
|
|
Overall Study
COMPLETED
|
1
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors
Baseline characteristics by cohort
| Measure |
Stratum 1 - NF2 Related Vestibular Schwannomas
n=1 Participants
Stratum 1 included patients with NF2 with vestibular schwannomas who exhibit hearing loss. Participants received continuous twice daily dosing of selumetinib. Dosing was based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy continued for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
Stratum 2: Other NF2 Related Tumors (Meningiomas and Ependymoma)
n=9 Participants
Stratum 2 will include patients who have progressive lesions other than VS (including non-vestibular schwannomas, meningiomas, and spinal cord lesions). Participants will receive continuous twice daily dosing of selumentinib. Dosing is based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy may continue for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
9 participants
n=7 Participants
|
10 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 weeksThe primary endpoint for the Stratum 1 is hearing response at 24 weeks, defined as an improvement in word recognition score above the 95% critical difference, taking as reference the baseline word recognition score. Audiology will include measurement of pure tone thresholds and determination of word recognition scores. Word recognition scores measure the ability to recognize (as opposed to detect) auditory information. Patients are presented a list of 50 words at a fully audible level and the percentage identified correctly is the score. This study will use full 50-item monosyllable lists and standardized recordings. This Outcome Measure was pre-specified to be assessed only within the Stratum 1 Arm/Group.
Outcome measures
| Measure |
Stratum 1 - NF2 Related Vestibular Schwannomas
n=1 Participants
Stratum 1 included patients with NF2 with vestibular schwannomas who exhibit hearing loss. Participants received continuous twice daily dosing of selumetinib. Dosing was based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy continued for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
Stratum 2- NF2 Tumors (Non-vestibular Schwanoma (VS))
Stratum 2 Response and progression for non-VS tumor by MacDonald Criteria as measured by Complete responses, partial response, stable disease or progressive disease.
|
|---|---|---|
|
Stratum 1- Number of Patients With Hearing Response at 24 Weeks as Measured by Word Recognition
|
0 Participants
|
—
|
PRIMARY outcome
Timeframe: Through study completion up to 2 yearsTo determine the radiographic response rate for Stratum 1 and Stratum 2. Specifics for each stratum are defined in the Arms/ Groups.
Outcome measures
| Measure |
Stratum 1 - NF2 Related Vestibular Schwannomas
n=1 Participants
Stratum 1 included patients with NF2 with vestibular schwannomas who exhibit hearing loss. Participants received continuous twice daily dosing of selumetinib. Dosing was based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy continued for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
Stratum 2- NF2 Tumors (Non-vestibular Schwanoma (VS))
n=9 Participants
Stratum 2 Response and progression for non-VS tumor by MacDonald Criteria as measured by Complete responses, partial response, stable disease or progressive disease.
|
|---|---|---|
|
Radiographic Tumor Response
|
1 Participants
|
9 Participants
|
Adverse Events
Stratum 1 - NF2 Related Vestibular Schwannomas
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Stratum 2: Other NF2 Related Tumors (Meningiomas and Ependymoma)
Serious events: 2 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Stratum 1 - NF2 Related Vestibular Schwannomas
n=1 participants at risk
Stratum 1 included patients with NF2 with vestibular schwannomas who exhibit hearing loss. Participants received continuous twice daily dosing of selumetinib. Dosing was based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy continued for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
Stratum 2: Other NF2 Related Tumors (Meningiomas and Ependymoma)
n=9 participants at risk
Stratum 2 will include patients who have progressive lesions other than VS (including non-vestibular schwannomas, meningiomas, and spinal cord lesions). Participants will receive continuous twice daily dosing of selumentinib. Dosing is based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy may continue for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
|---|---|---|
|
Ear and labyrinth disorders
Grade 4 hearing impairment
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Nervous system disorders
Grade 3 hydrocephalus
|
100.0%
1/1 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
0.00%
0/9 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Nervous system disorders
Grade 2 facial nerve disorder
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Infections and infestations
Grade 3 salivary gland infection
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
Other adverse events
| Measure |
Stratum 1 - NF2 Related Vestibular Schwannomas
n=1 participants at risk
Stratum 1 included patients with NF2 with vestibular schwannomas who exhibit hearing loss. Participants received continuous twice daily dosing of selumetinib. Dosing was based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy continued for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
Stratum 2: Other NF2 Related Tumors (Meningiomas and Ependymoma)
n=9 participants at risk
Stratum 2 will include patients who have progressive lesions other than VS (including non-vestibular schwannomas, meningiomas, and spinal cord lesions). Participants will receive continuous twice daily dosing of selumentinib. Dosing is based on BSA calculated at the beginning of each course. One course is equivalent to 28 days. Therapy may continue for up to two years (26 courses) in the absence of disease progression or unacceptable toxicity.
Selumetinib: Continuous twice daily dosing; oral agent
|
|---|---|---|
|
Investigations
CPK increased
|
100.0%
1/1 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Eye disorders
Cataract
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
22.2%
2/9 • Number of events 2 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
22.2%
2/9 • Number of events 2 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Gastrointestinal disorders
Gastrointestinal disorders-other: mouth sores
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Gastrointestinal disorders
Gastrointestinal disorders-other: stomatitis
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
General disorders
Fatigue
|
100.0%
1/1 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
General disorders
Localized Edema
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
General disorders
Edema limbs
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Infections and infestations
Paronychia
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
66.7%
6/9 • Number of events 9 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Infections and infestations
Infections and Infestations-other: erythema toe
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
66.7%
6/9 • Number of events 6 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Investigations
Creatinine increased
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
22.2%
2/9 • Number of events 2 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Investigations
White blood cell decreased
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Investigations
Alkaline phosphatase increased
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)-other: skin papilloma (right foo
|
100.0%
1/1 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
0.00%
0/9 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnea
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
55.6%
5/9 • Number of events 5 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
33.3%
3/9 • Number of events 3 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
22.2%
2/9 • Number of events 2 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
|
Vascular disorders
Hypertension
|
0.00%
0/1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
11.1%
1/9 • Number of events 1 • Patients will be monitored for adverse events beginning on day 1 of study treatment and continue for the duration of time on study treatment, which may continue for up to 26 courses (approximately 2 years). Adverse events will also be monitored for 30 days post discontinuation of study treatment.
Adverse Event Reporting Description for this study uses the same definitions in the link above.
|
Additional Information
Senior Regulatory Specialist
Cincinnati Children's Hospital Medical Center
Phone: 15135350640
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place