Trial Outcomes & Findings for H3.3K27M Peptide Vaccine With Nivolumab for Children With Newly Diagnosed DIPG and Other Gliomas (NCT NCT02960230)
NCT ID: NCT02960230
Last Updated: 2025-01-22
Results Overview
Safety of the vaccine (Strata A and B) or vaccine in combination with nivolumab (Stratum C) will be assessed for participants who received the vaccination. The severity of toxicities will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE)version 5.0. and include any clinically-significant lab abnormalities meeting Grade 3, 4, or 5 criteria according to CTCAE. Grade 1 \& 2 AEs will be summarized if probably, possible, or definitely related to study therapy. Descriptive statistics will be utilized to display the data on toxicity reported by participants.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
50 participants
Primary outcome timeframe
24 months
Results posted on
2025-01-22
Participant Flow
Participant milestones
| Measure |
Stratum A: Newly Diagnosed DIPG
Newly diagnosed children with diffuse intrinsic pontine glioma (DIPG) who are positive for HLA-A2 and the H3.3.K27M mutation that underwent radiation therapy will receive the specific H3.3.K27M peptide vaccine, combined with the tetanus toxoid (TT) peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum B: Newly Diagnosed Glioma (Non-DIPG)
Newly diagnosed children with gliomas other than DIPG who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum C: Newly Diagnosed DIPG or Other Midline Glioma
Newly diagnosed children with DIPG or other midline gliomas (excluding primary spinal cord tumors) who are positive for HLA-A2 (02:01) and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Nivolumab will also be given via IV. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks. Nivolumab will continue to be given every 3 weeks throughout all of treatment.
|
|---|---|---|---|
|
Overall Study
STARTED
|
19
|
10
|
21
|
|
Overall Study
COMPLETED
|
19
|
10
|
21
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
H3.3K27M Peptide Vaccine With Nivolumab for Children With Newly Diagnosed DIPG and Other Gliomas
Baseline characteristics by cohort
| Measure |
Stratum A: Newly Diagnosed DIPG
n=19 Participants
Newly diagnosed children with diffuse intrinsic pontine glioma who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid (TT) peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum B: Newly Diagnosed Glioma (Non-DIPG)
n=10 Participants
Newly diagnosed children with gliomas other than DIPG who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum C: Newly Diagnosed DIPG or Other Midline Glioma
n=21 Participants
Newly diagnosed children with DIPG or other midline gliomas (excluding primary spinal cord tumors) who are positive for HLA-A2 (02:01) and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Nivolumab will also be given via IV. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks. Nivolumab will continue to be given every 3 weeks throughout all of treatment.
|
Total
n=50 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
10.11 years
STANDARD_DEVIATION 3.09 • n=5 Participants
|
12.0 years
STANDARD_DEVIATION 3.62 • n=7 Participants
|
9.38 years
STANDARD_DEVIATION 3.53 • n=5 Participants
|
10.18 years
STANDARD_DEVIATION 3.38 • n=4 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
26 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
24 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
28 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 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
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
35 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
19 participants
n=5 Participants
|
10 participants
n=7 Participants
|
15 participants
n=5 Participants
|
44 participants
n=4 Participants
|
|
Region of Enrollment
Switzerland
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
6 participants
n=5 Participants
|
6 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 24 monthsSafety of the vaccine (Strata A and B) or vaccine in combination with nivolumab (Stratum C) will be assessed for participants who received the vaccination. The severity of toxicities will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE)version 5.0. and include any clinically-significant lab abnormalities meeting Grade 3, 4, or 5 criteria according to CTCAE. Grade 1 \& 2 AEs will be summarized if probably, possible, or definitely related to study therapy. Descriptive statistics will be utilized to display the data on toxicity reported by participants.
Outcome measures
| Measure |
Stratum A: Newly Diagnosed DIPG
n=19 Participants
Newly diagnosed children with diffuse intrinsic pontine glioma who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid (TT) peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum B: Newly Diagnosed Glioma (Non-DIPG)
n=10 Participants
Newly diagnosed children with gliomas other than DIPG who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum C: Newly Diagnosed DIPG or Other Midline Glioma
n=21 Participants
Newly diagnosed children with DIPG or other midline gliomas (excluding primary spinal cord tumors) who are positive for HLA-A2 (02:01) and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Nivolumab will also be given via IV. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks. Nivolumab will continue to be given every 3 weeks throughout all of treatment.
|
|---|---|---|---|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Urinary Retention
|
5.26 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Abdominal Pain
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Abducens Nerve Disorder
|
21.0 percentage of participants
|
0 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Acoustic Nerve Disorder
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Adult respiratory distress syndrome
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Agitation
|
10.5 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Alanine aminotransferase increased
|
42.1 percentage of participants
|
20 percentage of participants
|
28.6 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Alkalosis
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Allergic Reaction
|
0 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Alopecia
|
0 percentage of participants
|
0 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Anemia
|
15.8 percentage of participants
|
10 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Anorexia
|
5.26 percentage of participants
|
20 percentage of participants
|
23.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Arthralgia
|
15.8 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Aspartate aminotransferase increased
|
36.8 percentage of participants
|
20 percentage of participants
|
19 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Aspiration
|
5.26 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Ataxia
|
10.5 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hiccups
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Atelectasis
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Blood bicarbonate decreased
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Blood bilirubin increased
|
0 percentage of participants
|
10 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Blurred Vision
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Bruising
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Cardiac troponin T increased
|
0 percentage of participants
|
0 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Chills
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Constipation
|
0 percentage of participants
|
0 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
CPK increased
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Creatinine Increased
|
5.26 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Depressed levels of consciousness
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Diarrhea
|
10.5 percentage of participants
|
10 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Dizziness
|
5.26 percentage of participants
|
0 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Dry Skin
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Dysarthria
|
15.8 percentage of participants
|
0 percentage of participants
|
23.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Dysphagia
|
10.5 percentage of participants
|
0 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Dysphasia
|
5.26 percentage of participants
|
0 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Dyspnea
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Edema Cerebral
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Encephalopathy
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Enterocolitis Infectious
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Eosinophilia
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Epistaxis
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Extraocular muscle paresis
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Eye disorders - Other
|
0 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Facial Nerve Disorder
|
5.26 percentage of participants
|
0 percentage of participants
|
23.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Fatigue
|
36.8 percentage of participants
|
50 percentage of participants
|
61.9 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Fever
|
25.3 percentage of participants
|
30 percentage of participants
|
28.6 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Flu like symptoms
|
5.26 percentage of participants
|
40 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Gait Disturbance
|
15.8 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
General disorders and administration site conditions - Other
|
5.26 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Generalized Muscle Weakness
|
5.26 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
GGT Increased
|
0 percentage of participants
|
10 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Glossopharyngeal nerve disorder
|
5.26 percentage of participants
|
0 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Headache
|
47.3 percentage of participants
|
30 percentage of participants
|
38 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hearing Impaired
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hydrocephalus
|
5.26 percentage of participants
|
10 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hyperglycemia
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypernatremia
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypersomnia
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypertension
|
0 percentage of participants
|
0 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hyperthyroidism
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypoalbuminemia
|
5.26 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypocalcemia
|
5.26 percentage of participants
|
10 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypoglycemia
|
5.26 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypokalemia
|
10.5 percentage of participants
|
20 percentage of participants
|
19 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypomagnesemia
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hyponatremia
|
5.26 percentage of participants
|
0 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypophosphatemia
|
0 percentage of participants
|
10 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypotension
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Hypoxia
|
5.26 percentage of participants
|
10 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Injection Site Reaction
|
68.4 percentage of participants
|
50 percentage of participants
|
38.1 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
International Normalized Ratio (INR) increased
|
0 percentage of participants
|
10 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Intracranial Hemorrhage
|
5.26 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Investigations - Other
|
10.5 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Keratitis
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Lethargy
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Leukocytosis
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Lipase Increased
|
0 percentage of participants
|
0 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Lung Infection
|
10.5 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Lymphocyte count decreased
|
31.5 percentage of participants
|
60 percentage of participants
|
28.6 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Lymphocyte Count Increased
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Meningitis
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Movements involuntary
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Muscle Weakness Left-Sided
|
21 percentage of participants
|
0 percentage of participants
|
19 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Muscle Weakness Right-Sided
|
15.8 percentage of participants
|
10 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Musculoskeletal and connective tissue disorder - Other
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Myalgia
|
15.8 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Myositis
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Nasal Congestion
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Nausea
|
21 percentage of participants
|
10 percentage of participants
|
19 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Neck Pain
|
0 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other
|
0 percentage of participants
|
20 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Nervous system disorders - Other
|
10.5 percentage of participants
|
0 percentage of participants
|
23.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Neutrophil count decreased
|
15.8 percentage of participants
|
10 percentage of participants
|
19 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Nystagmus
|
5.26 percentage of participants
|
0 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Oculomotor nerve disorder
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Optic Nerve Disorder
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Pain
|
5.26 percentage of participants
|
0 percentage of participants
|
19 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Pain in extremity
|
10.5 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Paresthesia
|
5.26 percentage of participants
|
10 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Platelet Count Decreased
|
5.26 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Pruritus
|
5.26 percentage of participants
|
10 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Pyramidal tract syndrome
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Rash maculo-papular
|
0 percentage of participants
|
10 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Renal and urinary disorders - Other
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Respiratory failure
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Respiratory, thoracic and mediastinal disorders - Other
|
10.5 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Seizure
|
15.8 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Skin and subcutaneous tissue disorders - Other
|
10.5 percentage of participants
|
0 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Skin induration
|
2.63 percentage of participants
|
10 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Skin Infection
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Somnolence
|
10.5 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Spasticity
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Stomach Pain
|
10.5 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Stroke
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Thromboembolic event
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Thyroid stimulating hormone increased
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Trigeminal Nerve Disorder
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Urinary Tract Infection
|
5.26 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Vaccination Complication
|
0 percentage of participants
|
0 percentage of participants
|
4.8 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Vomiting
|
31.6 percentage of participants
|
60 percentage of participants
|
57.1 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Weight Gain
|
0 percentage of participants
|
10 percentage of participants
|
9.5 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
Weight Loss
|
5.26 percentage of participants
|
10 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Adverse Events (AE) Related to Treatment
White blood cells decreased
|
31.6 percentage of participants
|
60 percentage of participants
|
9.5 percentage of participants
|
PRIMARY outcome
Timeframe: 12 monthsOS12 is defined as the percentage of participants still alive at 12 months, and is the clinical efficacy, primary endpoint for Stratum A. Any eligible participant that received at least one dose of the K27M/TT vaccine will be considered evaluable for clinical efficacy. OS12 will be censored at the last contact date and estimated using the Kaplan-Meier method.
Outcome measures
| Measure |
Stratum A: Newly Diagnosed DIPG
n=19 Participants
Newly diagnosed children with diffuse intrinsic pontine glioma who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid (TT) peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum B: Newly Diagnosed Glioma (Non-DIPG)
Newly diagnosed children with gliomas other than DIPG who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum C: Newly Diagnosed DIPG or Other Midline Glioma
Newly diagnosed children with DIPG or other midline gliomas (excluding primary spinal cord tumors) who are positive for HLA-A2 (02:01) and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Nivolumab will also be given via IV. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks. Nivolumab will continue to be given every 3 weeks throughout all of treatment.
|
|---|---|---|---|
|
Overall Survival Rate at 12 Months (OS12) (Stratum A Only)
|
42 percentage of participants
Interval 25.0 to 71.0
|
—
|
—
|
Adverse Events
Stratum A: Newly Diagnosed DIPG
Serious events: 6 serious events
Other events: 19 other events
Deaths: 17 deaths
Stratum B: Newly Diagnosed Glioma (Non-DIPG)
Serious events: 8 serious events
Other events: 10 other events
Deaths: 7 deaths
Stratum C: Newly Diagnosed DIPG or Other Midline Glioma
Serious events: 13 serious events
Other events: 21 other events
Deaths: 20 deaths
Serious adverse events
| Measure |
Stratum A: Newly Diagnosed DIPG
n=19 participants at risk
Newly diagnosed children with diffuse intrinsic pontine glioma who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid (TT) peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum B: Newly Diagnosed Glioma (Non-DIPG)
n=10 participants at risk
Newly diagnosed children with gliomas other than DIPG who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum C: Newly Diagnosed DIPG or Other Midline Glioma
n=21 participants at risk
Newly diagnosed children with DIPG or other midline gliomas (excluding primary spinal cord tumors) who are positive for HLA-A2 (02:01) and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Nivolumab will also be given via IV. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks. Nivolumab will continue to be given every 3 weeks throughout all of treatment.
|
|---|---|---|---|
|
Eye disorders
Eye disorders - Other
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Gastrointestinal disorders
Dysphagia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Gastrointestinal disorders
Nausea
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Gastrointestinal disorders
Vomiting
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
30.0%
3/10 • Number of events 4 • Up to 24 months
|
14.3%
3/21 • Number of events 3 • Up to 24 months
|
|
General disorders
Chills
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Fatigue
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
General disorders
Fever
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Malaise
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
General disorders and administration site conditions - Other
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Infections and infestations
Lung infection
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Infections and infestations
Meningitis
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Infections and infestations
Infections and infestations - Other
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Muscle weakness left-sided
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Muscle weakness right-sided
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other
|
0.00%
0/19 • Up to 24 months
|
20.0%
2/10 • Number of events 3 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Ataxia
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Depressed level of consciousness
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Dizziness
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Facial nerve disorder
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Glossopharyngeal nerve disorder
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Nervous system disorders
Headache
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Hydrocephalus
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Nervous system disorders
Intracranial hemorrhage
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Seizure
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Somnolence
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Spasticity
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Stroke
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Nervous system disorders - Other
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
23.8%
5/21 • Number of events 5 • Up to 24 months
|
|
Renal and urinary disorders
Urinary retention
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Renal and urinary disorders
Renal and urinary disorders - Other
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Adult respiratory distress syndrome
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 2 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 4 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Vascular disorders
Thromboembolic event
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
Other adverse events
| Measure |
Stratum A: Newly Diagnosed DIPG
n=19 participants at risk
Newly diagnosed children with diffuse intrinsic pontine glioma who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid (TT) peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum B: Newly Diagnosed Glioma (Non-DIPG)
n=10 participants at risk
Newly diagnosed children with gliomas other than DIPG who are positive for HLA-A2 and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks.
|
Stratum C: Newly Diagnosed DIPG or Other Midline Glioma
n=21 participants at risk
Newly diagnosed children with DIPG or other midline gliomas (excluding primary spinal cord tumors) who are positive for HLA-A2 (02:01) and the H3.3K27M mutation that underwent radiation therapy will receive the specific H3.3K27M peptide vaccine, combined with the tetanus toxoid peptide, emulsified in Montanide. Poly-ICLC, which is a synthetic nucleic acid, will be given concurrently to improve the therapeutic effects of the vaccine. Nivolumab will also be given via IV. Vaccine will be given every 3 weeks for the first 24 weeks, then if there is stable or improved disease, will be given every 6 weeks for a total treatment period of 96 weeks. Nivolumab will continue to be given every 3 weeks throughout all of treatment.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
20.0%
2/10 • Number of events 10 • Up to 24 months
|
19.0%
4/21 • Number of events 7 • Up to 24 months
|
|
Blood and lymphatic system disorders
Eosinophilia
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
14.3%
3/21 • Number of events 3 • Up to 24 months
|
|
Blood and lymphatic system disorders
Leukocytosis
|
10.5%
2/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Cardiac disorders
Sinus bradycardia
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Cardiac disorders
Sinus tachycardia
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Ear and labyrinth disorders
Ear pain
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Ear and labyrinth disorders
Hearing Impaired
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Ear and labyrinth disorders
Tinnitus
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Endocrine disorders
Adrenal insufficiency
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Endocrine disorders
Hyperthyroidism
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Endocrine disorders
Hypothyroidism
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Eye disorders
Blurred vision
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Eye disorders
Conjunctivitis
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Eye disorders
Dry Eye
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Eye disorders
Extraocular muscle paresis
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Eye disorders
Eye disorders - Other
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Eye disorders
Eye Pain
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Eye disorders
Flashing lights
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Eye disorders
Keratitis
|
10.5%
2/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Eye disorders
Optic nerve disorder
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Eye disorders
Papilledema
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Gastrointestinal disorders
Abdominal pain
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Gastrointestinal disorders
Constipation
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
23.8%
5/21 • Number of events 8 • Up to 24 months
|
|
Gastrointestinal disorders
Dental caries
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Gastrointestinal disorders
Diarrhea
|
21.1%
4/19 • Number of events 5 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Gastrointestinal disorders
Dysphagia
|
15.8%
3/19 • Number of events 4 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Gastrointestinal disorders
Mucositis oral
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Gastrointestinal disorders
Nausea
|
36.8%
7/19 • Number of events 10 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
28.6%
6/21 • Number of events 10 • Up to 24 months
|
|
Gastrointestinal disorders
Stomach pain
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Gastrointestinal disorders
Vomiting
|
52.6%
10/19 • Number of events 14 • Up to 24 months
|
60.0%
6/10 • Number of events 10 • Up to 24 months
|
52.4%
11/21 • Number of events 15 • Up to 24 months
|
|
General disorders
Chills
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Edema limbs
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Fatigue
|
52.6%
10/19 • Number of events 10 • Up to 24 months
|
50.0%
5/10 • Number of events 10 • Up to 24 months
|
66.7%
14/21 • Number of events 25 • Up to 24 months
|
|
General disorders
Fever
|
31.6%
6/19 • Number of events 10 • Up to 24 months
|
30.0%
3/10 • Number of events 4 • Up to 24 months
|
28.6%
6/21 • Number of events 7 • Up to 24 months
|
|
General disorders
Flu like symptoms
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
40.0%
4/10 • Number of events 7 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Gait disturbance
|
26.3%
5/19 • Number of events 6 • Up to 24 months
|
30.0%
3/10 • Number of events 3 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
General disorders
General disorders and administration site conditions - Other
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Injection site reaction
|
68.4%
13/19 • Number of events 24 • Up to 24 months
|
50.0%
5/10 • Number of events 6 • Up to 24 months
|
38.1%
8/21 • Number of events 10 • Up to 24 months
|
|
General disorders
Malaise
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Non-cardiac chest pain
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
General disorders
Pain
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
19.0%
4/21 • Number of events 4 • Up to 24 months
|
|
Immune system disorders
Allergic reaction
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 3 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Infections and infestations
Enterocolitis infectious
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Infections and infestations
Infections and infestations - Other
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Infections and infestations
Lung infection
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Infections and infestations
Rhinitis infective
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Infections and infestations
Skin infection
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Infections and infestations
Thrush
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Infections and infestations
Tooth infection
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Infections and infestations
Upper respiratory infection
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Infections and infestations
Urinary tract infection
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Injury, poisoning and procedural complications
Bruising
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Injury, poisoning and procedural complications
Vaccination complication
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Injury, poisoning and procedural complications
Wound dehiscence
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
Alanine aminotransferase increased
|
42.1%
8/19 • Number of events 14 • Up to 24 months
|
40.0%
4/10 • Number of events 5 • Up to 24 months
|
33.3%
7/21 • Number of events 15 • Up to 24 months
|
|
Investigations
Alkaline phosphatase increased
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Investigations
Aspartate aminotransferase increased
|
42.1%
8/19 • Number of events 12 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
19.0%
4/21 • Number of events 10 • Up to 24 months
|
|
Investigations
Blood bicarbonate decreased
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Investigations
Blood bilirubin increased
|
5.3%
1/19 • Number of events 3 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Investigations
Cardiac troponin T increased
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Investigations
Cholesterol high
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
CPK increased
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
Creatinine increased
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
4.8%
1/21 • Number of events 3 • Up to 24 months
|
|
Investigations
Fibrinogen decreased
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Investigations
GGT increased
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Investigations
Hemoglobin increased
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
INR increased
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Investigations
Investigations - Other
|
10.5%
2/19 • Number of events 55 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Investigations
Lipase increased
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
14.3%
3/21 • Number of events 4 • Up to 24 months
|
|
Investigations
Lymphocyte count decreased
|
36.8%
7/19 • Number of events 16 • Up to 24 months
|
60.0%
6/10 • Number of events 18 • Up to 24 months
|
52.4%
11/21 • Number of events 21 • Up to 24 months
|
|
Investigations
Lymphocyte count increased
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
Neutrophil count decreased
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
19.0%
4/21 • Number of events 8 • Up to 24 months
|
|
Investigations
Platelet count decreased
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 9 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
Serum amylase increased
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
Thyroid stimulating hormone increased
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Investigations
Weight gain
|
0.00%
0/19 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
23.8%
5/21 • Number of events 8 • Up to 24 months
|
|
Investigations
Weight loss
|
15.8%
3/19 • Number of events 4 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
19.0%
4/21 • Number of events 8 • Up to 24 months
|
|
Investigations
White blood cell decreased
|
31.6%
6/19 • Number of events 9 • Up to 24 months
|
70.0%
7/10 • Number of events 13 • Up to 24 months
|
28.6%
6/21 • Number of events 11 • Up to 24 months
|
|
Metabolism and nutrition disorders
Alkalosis
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Metabolism and nutrition disorders
Anorexia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
20.0%
2/10 • Number of events 3 • Up to 24 months
|
28.6%
6/21 • Number of events 6 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
15.8%
3/19 • Number of events 6 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
14.3%
3/21 • Number of events 5 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypernatremia
|
10.5%
2/19 • Number of events 5 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hyperphosphatemia
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
31.6%
6/19 • Number of events 10 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
14.3%
3/21 • Number of events 4 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
9.5%
2/21 • Number of events 4 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
26.3%
5/19 • Number of events 7 • Up to 24 months
|
20.0%
2/10 • Number of events 3 • Up to 24 months
|
23.8%
5/21 • Number of events 7 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
10.5%
2/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
14.3%
3/21 • Number of events 5 • Up to 24 months
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
19.0%
4/21 • Number of events 7 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.5%
2/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Muscle weakness left-sided
|
26.3%
5/19 • Number of events 6 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
19.0%
4/21 • Number of events 5 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Muscle weakness right-sided
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
19.0%
4/21 • Number of events 6 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
15.8%
3/19 • Number of events 4 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 3 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Myositis
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Amnesia
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Ataxia
|
31.6%
6/19 • Number of events 7 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
33.3%
7/21 • Number of events 10 • Up to 24 months
|
|
Nervous system disorders
Dizziness
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
28.6%
6/21 • Number of events 7 • Up to 24 months
|
|
Nervous system disorders
Dysarthria
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
28.6%
6/21 • Number of events 9 • Up to 24 months
|
|
Nervous system disorders
Dysphasia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
14.3%
3/21 • Number of events 4 • Up to 24 months
|
|
Nervous system disorders
Edema cerebral
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Facial muscle weakness
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Facial nerve disorder
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
28.6%
6/21 • Number of events 8 • Up to 24 months
|
|
Nervous system disorders
Glossopharyngeal nerve disorder
|
5.3%
1/19 • Number of events 4 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
19.0%
4/21 • Number of events 5 • Up to 24 months
|
|
Nervous system disorders
Headache
|
57.9%
11/19 • Number of events 14 • Up to 24 months
|
40.0%
4/10 • Number of events 7 • Up to 24 months
|
38.1%
8/21 • Number of events 26 • Up to 24 months
|
|
Nervous system disorders
Hydrocephalus
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Hypersomnia
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Intracranial hemorrhage
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
IVth nerve disorder
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Lethargy
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Memory impairment
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Movements involuntary
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Nervous system disorders - Other
|
10.5%
2/19 • Number of events 5 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Nystagmus
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Nervous system disorders
Oculomotor nerve disorder
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Paresthesia
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 3 • Up to 24 months
|
14.3%
3/21 • Number of events 4 • Up to 24 months
|
|
Nervous system disorders
Pyramidal tract syndrome
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Seizure
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Nervous system disorders
Somnolence
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Spasticity
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Nervous system disorders
Trigeminal nerve disorder
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Psychiatric disorders
Agitation
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
20.0%
2/10 • Number of events 2 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Psychiatric disorders
Confusion
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/19 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Renal and urinary disorders
Glucosuria
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Renal and urinary disorders
Proteinuria
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 3 • Up to 24 months
|
|
Renal and urinary disorders
Renal and urinary disorders - Other
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Renal and urinary disorders
Urinary frequency
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Renal and urinary disorders
Urinary retention
|
10.5%
2/19 • Number of events 3 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
4.8%
1/21 • Number of events 2 • Up to 24 months
|
|
Reproductive system and breast disorders
Vaginal discharge
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
15.8%
3/19 • Number of events 3 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
0.00%
0/21 • Up to 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
10.5%
2/19 • Number of events 2 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.3%
1/19 • Number of events 2 • Up to 24 months
|
10.0%
1/10 • Number of events 1 • Up to 24 months
|
9.5%
2/21 • Number of events 2 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
5.3%
1/19 • Number of events 1 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Scalp pain
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
21.1%
4/19 • Number of events 4 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
9.5%
2/21 • Number of events 3 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Skin induration
|
26.3%
5/19 • Number of events 5 • Up to 24 months
|
10.0%
1/10 • Number of events 2 • Up to 24 months
|
14.3%
3/21 • Number of events 3 • Up to 24 months
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
|
Vascular disorders
Hypertension
|
10.5%
2/19 • Number of events 3 • Up to 24 months
|
40.0%
4/10 • Number of events 4 • Up to 24 months
|
14.3%
3/21 • Number of events 3 • Up to 24 months
|
|
Vascular disorders
Hypotension
|
0.00%
0/19 • Up to 24 months
|
0.00%
0/10 • Up to 24 months
|
4.8%
1/21 • Number of events 1 • Up to 24 months
|
Additional Information
Dr. Sabine Mueller, MD PhD
University of California, San Francisco
Phone: (415) 502-1600
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place