Trial Outcomes & Findings for Complement Inhibition Using Eculizumab to Overcome Platelet Transfusion Refractoriness in Patients With Severe Thrombocytopenia (NCT NCT02298933)
NCT ID: NCT02298933
Last Updated: 2019-01-03
Results Overview
To evaluate the safety and efficacy of eculizumab to increase the platelet increment, defined as Corrected Count Increment (CCI) \>7500/μL at 10-60 min together with CCI\>5000/μL at 18-24 hrs post transfusion in patients with platelet refractoriness following treatment with eculizumab and platelet transfusion.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
24 hours
Results posted on
2019-01-03
Participant Flow
Participant milestones
| Measure |
Eculizumab
Eculizumab 1200 mg IV infusion is given over 30-40 min
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Complement Inhibition Using Eculizumab to Overcome Platelet Transfusion Refractoriness in Patients With Severe Thrombocytopenia
Baseline characteristics by cohort
| Measure |
Eculizumab
n=10 Participants
Eculizumab 1200 mg IV infusion is given over 30-40 min
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: 24 hoursPopulation: All patients were given Eculizumab
To evaluate the safety and efficacy of eculizumab to increase the platelet increment, defined as Corrected Count Increment (CCI) \>7500/μL at 10-60 min together with CCI\>5000/μL at 18-24 hrs post transfusion in patients with platelet refractoriness following treatment with eculizumab and platelet transfusion.
Outcome measures
| Measure |
Eculizumab
n=10 Participants
Eculizumab 1200 mg IV infusion is given over 30-40 min
|
|---|---|
|
Number of Subjects With Sustained Platelet Transfusion Responsiveness
|
4 Participants
|
Adverse Events
Eculizumab
Serious events: 6 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Eculizumab
n=10 participants at risk
Eculizumab 1200 mg IV infusion is given over 30-40 min
|
|---|---|
|
Cardiac disorders
Pericardial effusion
|
10.0%
1/10 • 14 days
|
|
Immune system disorders
Anaphylactic reaction
|
10.0%
1/10 • 14 days
|
|
Infections and infestations
Infection
|
30.0%
3/10 • 14 days
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
10.0%
1/10 • 14 days
|
Other adverse events
| Measure |
Eculizumab
n=10 participants at risk
Eculizumab 1200 mg IV infusion is given over 30-40 min
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
20.0%
2/10 • 14 days
|
|
Cardiac disorders
Dyspnoea
|
10.0%
1/10 • 14 days
|
|
Cardiac disorders
Restrictive cardiomyopathy
|
10.0%
1/10 • 14 days
|
|
Eye disorders
Eye disorder
|
10.0%
1/10 • 14 days
|
|
Gastrointestinal disorders
Abdominal pain
|
10.0%
1/10 • 14 days
|
|
Gastrointestinal disorders
Constipation
|
10.0%
1/10 • 14 days
|
|
Gastrointestinal disorders
Enterocolitis
|
10.0%
1/10 • 14 days
|
|
Gastrointestinal disorders
Nausea
|
10.0%
1/10 • 14 days
|
|
Gastrointestinal disorders
Proctalgia
|
10.0%
1/10 • 14 days
|
|
Gastrointestinal disorders
Upper gastrointestinal haemorrhage
|
10.0%
1/10 • 14 days
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
1/10 • 14 days
|
|
General disorders
Chills
|
10.0%
1/10 • 14 days
|
|
General disorders
Decreased appetite
|
10.0%
1/10 • 14 days
|
|
General disorders
Pyrexia
|
10.0%
1/10 • 14 days
|
|
Immune system disorders
Anaphylactic reaction
|
10.0%
1/10 • 14 days
|
|
Injury, poisoning and procedural complications
Contusion
|
10.0%
1/10 • 14 days
|
|
Investigations
Alanine aminotransferase increased
|
10.0%
1/10 • 14 days
|
|
Investigations
Aspartate aminotransferase increased
|
10.0%
1/10 • 14 days
|
|
Investigations
Blood bilirubin increased
|
10.0%
1/10 • 14 days
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
10.0%
1/10 • 14 days
|
|
Nervous system disorders
Tremor
|
10.0%
1/10 • 14 days
|
|
Psychiatric disorders
Delirium
|
10.0%
1/10 • 14 days
|
|
Renal and urinary disorders
Cystitis noninfective
|
10.0%
1/10 • 14 days
|
|
Renal and urinary disorders
Haematuria
|
10.0%
1/10 • 14 days
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.0%
1/10 • 14 days
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
10.0%
1/10 • 14 days
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
10.0%
1/10 • 14 days
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
10.0%
1/10 • 14 days
|
|
Vascular disorders
Hypertension
|
10.0%
1/10 • 14 days
|
|
Vascular disorders
Hypotension
|
10.0%
1/10 • 14 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place