Trial Outcomes & Findings for Cytomegalovirus (CMV) RNA-Pulsed Dendritic Cells for Pediatric Patients and Young Adults With WHO Grade IV Glioma, Recurrent Malignant Glioma, or Recurrent Medulloblastoma (NCT NCT03615404)
NCT ID: NCT03615404
Last Updated: 2021-01-28
Results Overview
Percentage of patients for whom three or more vaccines can be generated from the pre-treatment leukapheresis
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
11 participants
Primary outcome timeframe
1 year
Results posted on
2021-01-28
Participant Flow
All patients were enrolled at a single institution, Duke University Medical Center.
All patients were enrolled into one cohort; no randomization occurred.
Participant milestones
| Measure |
CMV-DCs With GM-CSF and Td (Tetanus Toxoid)
CMV-DCs are autologous dendritic cells derived from peripheral blood mononuclear cells (PBMCs) loaded with ribonucleic acid (RNA) encoding the human CMV matrix protein pp65 as a fusion protein with the full-length LAMP protein (pp65-flLAMP) plus GM-CSF and Td vaccine as adjuvants.
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
CMV-DCs With GM-CSF and Td (Tetanus Toxoid)
CMV-DCs are autologous dendritic cells derived from peripheral blood mononuclear cells (PBMCs) loaded with ribonucleic acid (RNA) encoding the human CMV matrix protein pp65 as a fusion protein with the full-length LAMP protein (pp65-flLAMP) plus GM-CSF and Td vaccine as adjuvants.
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
vaccine did not pass quality assurance
|
1
|
Baseline Characteristics
Cytomegalovirus (CMV) RNA-Pulsed Dendritic Cells for Pediatric Patients and Young Adults With WHO Grade IV Glioma, Recurrent Malignant Glioma, or Recurrent Medulloblastoma
Baseline characteristics by cohort
| Measure |
CMV-DCs With GM-CSF and Td (Tetanus Toxoid)
n=11 Participants
CMV-DCs are autologous dendritic cells derived from peripheral blood mononuclear cells (PBMCs) loaded with ribonucleic acid (RNA) encoding the human CMV matrix protein pp65 as a fusion protein with the full-length LAMP protein (pp65-flLAMP) plus GM-CSF and Td vaccine as adjuvants.
|
|---|---|
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Age, Continuous
|
16 years
STANDARD_DEVIATION 5.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearPercentage of patients for whom three or more vaccines can be generated from the pre-treatment leukapheresis
Outcome measures
| Measure |
CMV-DCs With GM-CSF and Td (Tetanus Toxoid)
n=11 Participants
CMV-DCs are autologous dendritic cells derived from peripheral blood mononuclear cells (PBMCs) loaded with ribonucleic acid (RNA) encoding the human CMV matrix protein pp65 as a fusion protein with the full-length LAMP protein (pp65-flLAMP) plus GM-CSF and Td vaccine as adjuvants.
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|---|---|
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Percentage of Patients for Whom 3 or More Vaccines Can be Made
|
90.9 percentage of participants
Interval 58.7 to 99.8
|
PRIMARY outcome
Timeframe: 1 yearPopulation: One eligible patient had a leukapheresis (evaluable for feasibility), but was withdrawn from the study due to clinical decline prior to receiving any treatment (not evaluable for safety).
Percentage of patients who experience unacceptable toxicity from CMV-DC administration
Outcome measures
| Measure |
CMV-DCs With GM-CSF and Td (Tetanus Toxoid)
n=10 Participants
CMV-DCs are autologous dendritic cells derived from peripheral blood mononuclear cells (PBMCs) loaded with ribonucleic acid (RNA) encoding the human CMV matrix protein pp65 as a fusion protein with the full-length LAMP protein (pp65-flLAMP) plus GM-CSF and Td vaccine as adjuvants.
|
|---|---|
|
Percentage of Patients Who Experience Unacceptable Toxicity
|
0 percentage of patients
|
Adverse Events
CMV-DCs With GM-CSF and Td (Tetanus Toxoid)
Serious events: 0 serious events
Other events: 9 other events
Deaths: 2 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
CMV-DCs With GM-CSF and Td (Tetanus Toxoid)
n=9 participants at risk
CMV-DCs are autologous dendritic cells derived from peripheral blood mononuclear cells (PBMCs) loaded with ribonucleic acid (RNA) encoding the human CMV matrix protein pp65 as a fusion protein with the full-length LAMP protein (pp65-flLAMP) plus GM-CSF and Td vaccine as adjuvants.
|
|---|---|
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Blood and lymphatic system disorders
Anemia
|
44.4%
4/9 • 2 years from study registration
|
|
Cardiac disorders
Sinus tachycardia
|
22.2%
2/9 • 2 years from study registration
|
|
Eye disorders
Eye disorders - Other, specify
|
11.1%
1/9 • 2 years from study registration
|
|
Gastrointestinal disorders
Abdominal pain
|
11.1%
1/9 • 2 years from study registration
|
|
Gastrointestinal disorders
Constipation
|
22.2%
2/9 • 2 years from study registration
|
|
Gastrointestinal disorders
Diarrhea
|
11.1%
1/9 • 2 years from study registration
|
|
Gastrointestinal disorders
Hemorrhoids
|
11.1%
1/9 • 2 years from study registration
|
|
Gastrointestinal disorders
Nausea
|
33.3%
3/9 • 2 years from study registration
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
3/9 • 2 years from study registration
|
|
General disorders
Chills
|
11.1%
1/9 • 2 years from study registration
|
|
General disorders
Fatigue
|
44.4%
4/9 • 2 years from study registration
|
|
General disorders
Fever
|
11.1%
1/9 • 2 years from study registration
|
|
General disorders
Injection site reaction
|
22.2%
2/9 • 2 years from study registration
|
|
Infections and infestations
Infections and infestations - Other, specify
|
22.2%
2/9 • 2 years from study registration
|
|
Infections and infestations
Upper respiratory infection
|
22.2%
2/9 • 2 years from study registration
|
|
Infections and infestations
Urinary tract infection
|
11.1%
1/9 • 2 years from study registration
|
|
Investigations
Alanine aminotransferase increased
|
11.1%
1/9 • 2 years from study registration
|
|
Investigations
Alkaline phosphatase increased
|
11.1%
1/9 • 2 years from study registration
|
|
Investigations
Aspartate aminotransferase increased
|
22.2%
2/9 • 2 years from study registration
|
|
Investigations
Lymphocyte count decreased
|
88.9%
8/9 • 2 years from study registration
|
|
Investigations
Neutrophil count decreased
|
33.3%
3/9 • 2 years from study registration
|
|
Investigations
Platelet count decreased
|
22.2%
2/9 • 2 years from study registration
|
|
Investigations
White blood cell decreased
|
44.4%
4/9 • 2 years from study registration
|
|
Metabolism and nutrition disorders
Anorexia
|
11.1%
1/9 • 2 years from study registration
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
11.1%
1/9 • 2 years from study registration
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
22.2%
2/9 • 2 years from study registration
|
|
Metabolism and nutrition disorders
Hypokalemia
|
22.2%
2/9 • 2 years from study registration
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Dysarthria
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Dysphasia
|
22.2%
2/9 • 2 years from study registration
|
|
Nervous system disorders
Headache
|
55.6%
5/9 • 2 years from study registration
|
|
Nervous system disorders
Memory impairment
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Muscle weakness left-sided
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Muscle weakness right-sided
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Paresthesia
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Pyramidal tract syndrome
|
11.1%
1/9 • 2 years from study registration
|
|
Nervous system disorders
Seizure
|
11.1%
1/9 • 2 years from study registration
|
|
Psychiatric disorders
Depression
|
11.1%
1/9 • 2 years from study registration
|
|
Psychiatric disorders
Insomnia
|
22.2%
2/9 • 2 years from study registration
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
11.1%
1/9 • 2 years from study registration
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
11.1%
1/9 • 2 years from study registration
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
22.2%
2/9 • 2 years from study registration
|
|
Vascular disorders
Hypertension
|
33.3%
3/9 • 2 years from study registration
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place