Trial Outcomes & Findings for Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients (NCT NCT03518203)
NCT ID: NCT03518203
Last Updated: 2023-09-21
Results Overview
Survival at 6 months after the date of TMA diagnosis
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
23 participants
Primary outcome timeframe
6 months
Results posted on
2023-09-21
Participant Flow
Participant milestones
| Measure |
Eculizumab
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Overall Study
STARTED
|
23
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Eculizumab
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Overall Study
The participant died on study prior to receiving 4 doses and was not evaluable.
|
1
|
|
Overall Study
Relapsed with primary malignancy within 6 months of TMA diagnosis making participant unevaluable.
|
1
|
Baseline Characteristics
Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients
Baseline characteristics by cohort
| Measure |
Eculizumab
n=23 Participants
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Age, Categorical
<=18 years
|
20 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 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
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
23 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsSurvival at 6 months after the date of TMA diagnosis
Outcome measures
| Measure |
Eculizumab
n=21 Participants
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Survival
|
15 Participants
|
SECONDARY outcome
Timeframe: 6 monthsNumber of participants with organ dysfunction at 6 months after TMA diagnosis. Organ dysfunction definitions are listed in the protocol Appendix II that is uploaded to ClinicalTrials.gov site.
Outcome measures
| Measure |
Eculizumab
n=15 Participants
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Number of Participants With Organ Dysfunction
|
4 Participants
|
SECONDARY outcome
Timeframe: 1 yearNumber of participants with organ dysfunction at 1 year after TMA diagnosis. Organ dysfunction definitions are listed in the protocol Appendix II that is uploaded to ClinicalTrials.gov site.
Outcome measures
| Measure |
Eculizumab
n=13 Participants
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Number of Participants With Organ Dysfunction
|
2 Participants
|
SECONDARY outcome
Timeframe: 1 yearNon-relapse mortality descriptively compared with historical controls at 1 year
Outcome measures
| Measure |
Eculizumab
n=21 Participants
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Non-relapse Mortality
|
8 Participants
|
Adverse Events
Eculizumab
Serious events: 19 serious events
Other events: 22 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
Eculizumab
n=23 participants at risk
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Cardiac disorders
Cardiac Tamponade
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Cardiac disorders
Cardiac arrest
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Cardiac disorders
Decreased left ventricular ejection fraction
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Cardiac disorders
Pericardial effusion
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Abdominal pain, right upper quadrant
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Nausea
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Vomiting
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
General disorders
Fever
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
General disorders
Graft versus Host Disease (liver and gastrointestinal)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Hepatobiliary disorders
Sinusoidal obstruction syndrome
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Cellulitis
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Multi organ failure
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Possible/Rule Out Sepsis
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Sepsis
|
21.7%
5/23 • Number of events 5 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
Platelet count decreased
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Anorexia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness, lower limbs
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Altered mental status
|
8.7%
2/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Decreased level of consciousness
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Encephalopathy
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Possible seizure
|
4.3%
1/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Seizure
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Stroke
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Renal and urinary disorders
Acute kidney injury
|
34.8%
8/23 • Number of events 8 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Renal and urinary disorders
Renal failure
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Diffuse alveolar hemorrhage
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxemia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Worsening Hypoxia (due to diffuse alveolar damage)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema (related to fluid overload)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory decline
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
34.8%
8/23 • Number of events 10 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Vascular disorders
Hypertension
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Vascular disorders
Hypotension
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Adenovirus infection (viral hepatitis)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhagic shock, refractory
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
Other adverse events
| Measure |
Eculizumab
n=23 participants at risk
All patients will receive eculizumab based on their weight for 24 weeks.
Eculizumab: Eculizumab will be administered as intravenous infusion (IV) over 60 minutes. The dosage form will be 300 mg single-use vials each containing 30 mL of 10 mg/mL sterile, preservative-free solution.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
8.7%
2/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Cardiac disorders
Increased cardiac troponin
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Cardiac disorders
Biventricular dysfunction
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Cardiac disorders
Pericardial effusion
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Eye disorders
Ocular graft versus host disease
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Abdominal pain
|
17.4%
4/23 • Number of events 4 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Diarrhea
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Gastric hemorrhage
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Graft versus host disease
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Ileus
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Lower GI hemorrhage
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Nausea
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Gastrointestinal disorders
Vomiting
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
General disorders
Fever
|
8.7%
2/23 • Number of events 4 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
General disorders
Generalized edema
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
General disorders
TMA reactivation unresponsive to treatment
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Adenoviremia
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Bacteremia
|
17.4%
4/23 • Number of events 7 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
BK viremia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Blood bilirubin increased
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
CMV viremia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
CMV viremia reactivation
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Endocarditis infective
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Enterocolitis infections
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Worsening enterocolitis
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
EBV reactivation
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Fungal infection (right cheek, vaginal area)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Fungemia
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Mucosal infection (eschar around nares)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Persistent C-Diff infection
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Pulmonary, liver and splenic nodules, possible EBV
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Sinusitis
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Urinary tract infection
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Upper respiratory infection
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Viral infection (HSV1 in blood and on skin)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
Creatinine increased
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
Increased alanine aminotransferase
|
34.8%
8/23 • Number of events 11 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
Increased aspartate aminotransferase
|
39.1%
9/23 • Number of events 10 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
Neutrophil count decreased
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
Platelet count decreased
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
Renal tubular acidosis
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Investigations
White blood cell count decreased
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Anorexia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
13.0%
3/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
60.9%
14/23 • Number of events 17 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness/pain trunk (left hip)
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Worsened generalized muscle weakness
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Altered mental status
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Delirium
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Hydrocephalus
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Nervous system disorders
Vasovagal reaction
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Renal and urinary disorders
End stage renal disease (or CKD)
|
17.4%
4/23 • Number of events 4 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Renal and urinary disorders
Hematuria
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Tension pneumothorax
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Skin and subcutaneous tissue disorders
Skin graft versus host disease
|
8.7%
2/23 • Number of events 2 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Skin and subcutaneous tissue disorders
Skin pain
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Vascular disorders
Hypertension
|
8.7%
2/23 • Number of events 3 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Vascular disorders
Hypotension
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Vascular disorders
Vasculitis
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Infections and infestations
Disseminated adenovirus
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
|
Renal and urinary disorders
Acute kidney injury
|
4.3%
1/23 • Number of events 1 • Adverse Events were monitored from enrollment to 60 days after end of treatment, an average of 32 weeks. All-Cause Mortality was monitored up to 1 year.
|
Additional Information
Sonata Jodele, MD
Cincinnati Children's Hospital Medical Center
Phone: (513) 636-5917
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place