Trial Outcomes & Findings for E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients (NCT NCT01194804)
NCT ID: NCT01194804
Last Updated: 2019-09-17
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
27 participants
Primary outcome timeframe
52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Results posted on
2019-09-17
Participant Flow
Participant milestones
| Measure |
Eculizumab
Treatment with eculizumab for patients with paroxysmal nocturnal hemoglobinuria who have successfully completed the C07-001 protocol (NCT01192399)
Eculizumab: 900 mg intravenous every 14 days.
|
|---|---|
|
Overall Study
STARTED
|
27
|
|
Overall Study
COMPLETED
|
26
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Eculizumab
Treatment with eculizumab for patients with paroxysmal nocturnal hemoglobinuria who have successfully completed the C07-001 protocol (NCT01192399)
Eculizumab: 900 mg intravenous every 14 days.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Baseline characteristics by cohort
| Measure |
Eculizumab
n=27 Participants
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Age, Continuous
|
48.3 years
STANDARD_DEVIATION 12.35 • n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Japanese
|
27 Participants
n=5 Participants
|
|
Region of Enrollment
Japan
|
27 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)Outcome measures
| Measure |
Eculizumab
n=26 Participants
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Change From Baseline in Lactate Dehydrogenase
|
-15.9 Units/Liter
Standard Error 1.20
|
SECONDARY outcome
Timeframe: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)The FACIT-Fatigue scale, Version 4.0, is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.
Outcome measures
| Measure |
Eculizumab
n=26 Participants
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Change From Baseline in FACIT-Fatigue Scale Total Score
|
8.0 units on a scale
Standard Error 1.95
|
SECONDARY outcome
Timeframe: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)Outcome measures
| Measure |
Eculizumab
n=26 Participants
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Change From Baseline in PNH Red Blood Cell (RBC) Count
|
0.51 cellsx10^12/L
Standard Error 0.129
|
SECONDARY outcome
Timeframe: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)Baseline is defined as the number of units transfused in 3 months prior to baseline
Outcome measures
| Measure |
Eculizumab
n=26 Participants
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Change From Baseline in Number of Units of Packed RBCs Transfused
|
-3.5 units
Standard Error 1.43
|
SECONDARY outcome
Timeframe: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)Outcome measures
| Measure |
Eculizumab
n=26 Participants
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Change From Baseline in Plasma Free Hemoglobin
|
-13.5 mg/dL
Standard Error 8.55
|
Adverse Events
Eculizumab
Serious events: 12 serious events
Other events: 26 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Eculizumab
n=27 participants at risk
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Blood and lymphatic system disorders
Haemolysis
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Gastrointestinal disorders
Colitis
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
General disorders
Pyrexia
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Hepatobiliary disorders
Cholelithiasis
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Hepatobiliary disorders
Jaundice cholestatic
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Pneumonia
|
14.8%
4/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Bronchitis
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Cellulitis
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Gastroenteritis viral
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Herpes virus infection
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Sepsis
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Upper respiratory tract infection
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Injury, poisoning and procedural complications
Animal bite
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Renal and urinary disorders
Acute kidney injury
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
3.7%
1/27 • From baseline (after first dose) to end of study (1 year).
|
Other adverse events
| Measure |
Eculizumab
n=27 participants at risk
Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol
900 mg intravenous every 14 days.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
11.1%
3/27 • From baseline (after first dose) to end of study (1 year).
|
|
Eye disorders
Conjunctival haemorrhage
|
11.1%
3/27 • From baseline (after first dose) to end of study (1 year).
|
|
Eye disorders
Cataract
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Gastrointestinal disorders
Diarrhoea
|
14.8%
4/27 • From baseline (after first dose) to end of study (1 year).
|
|
Gastrointestinal disorders
Abdominal discomfort
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Gastrointestinal disorders
Dental caries
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Gastrointestinal disorders
Dyspepsia
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Gastrointestinal disorders
Enterocolitis
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Gastrointestinal disorders
Mouth ulceration
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
General disorders
Pyrexia
|
14.8%
4/27 • From baseline (after first dose) to end of study (1 year).
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Nasopharyngitis
|
77.8%
21/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Upper respiratory tract infection
|
22.2%
6/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Gastroenteritis
|
18.5%
5/27 • From baseline (after first dose) to end of study (1 year).
|
|
Infections and infestations
Pharyngitis
|
11.1%
3/27 • From baseline (after first dose) to end of study (1 year).
|
|
Injury, poisoning and procedural complications
Contusion
|
11.1%
3/27 • From baseline (after first dose) to end of study (1 year).
|
|
Injury, poisoning and procedural complications
Laceration
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Investigations
Blood alkaline phosphatase increased
|
22.2%
6/27 • From baseline (after first dose) to end of study (1 year).
|
|
Investigations
Blood bilirubin increased
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Metabolism and nutrition disorders
Diabetes mellitus
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Nervous system disorders
Headache
|
22.2%
6/27 • From baseline (after first dose) to end of study (1 year).
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
|
14.8%
4/27 • From baseline (after first dose) to end of study (1 year).
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
11.1%
3/27 • From baseline (after first dose) to end of study (1 year).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.4%
2/27 • From baseline (after first dose) to end of study (1 year).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place