Trial Outcomes & Findings for Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms (NCT NCT03427060)
NCT ID: NCT03427060
Last Updated: 2025-05-04
Results Overview
Measurement of serum lactate dehydrogenase (LDH)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
1 participants
Primary outcome timeframe
Baseline to Day 186
Results posted on
2025-05-04
Participant Flow
Patients with paroxysmal nocturnal hemoglobinuria (PNH) who had received eculizumab without apparent benefit and in whom it had been demonstrated that their serum was resistant to eculizumab at any concentration (due to complement C5 polymorphisms that prevent tight binding of eculizumab) were recruited from one medical center between May 2018 and February 2021.
There were no patients excluded from the study following enrolment
Participant milestones
| Measure |
Coversin Treatment
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
Baseline characteristics by cohort
| Measure |
Coversin Treatment
n=1 Participants
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
|
Height
|
155 cm
n=99 Participants
|
|
Weight
|
73.3 kg
n=99 Participants
|
PRIMARY outcome
Timeframe: Baseline to Day 186Measurement of serum lactate dehydrogenase (LDH)
Outcome measures
| Measure |
Coversin Treatment
n=1 Participants
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
|---|---|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Screening
|
2913 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 1
|
3056 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 3
|
1453 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 7
|
564 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 14
|
351 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 21
|
368 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 29
|
329 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 36
|
339 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 43
|
555 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 60
|
319 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 90
|
394 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 120
|
352 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 150
|
284 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 183
|
906 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 184
|
882 U/L
|
|
Measurement of Serum Lactate Dehydrogenase (LDH)
Day 186
|
612 U/L
|
SECONDARY outcome
Timeframe: Baseline to Day 186Percentage change in the haemoglobin (Hb) value at each study visit compared to screening
Outcome measures
| Measure |
Coversin Treatment
n=1 Participants
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
|---|---|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Screening
|
0 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 1
|
28 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 3
|
18 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 7
|
22 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 14
|
17 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 21
|
25 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 29
|
23 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 36
|
9 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 43
|
26 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 60
|
60 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 90
|
28 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 120
|
18 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 150
|
23 %change in Hb value compare to screening
|
|
Percentage Change in Haemoglobin (Hb) Compared to Screening
Day 186
|
26 %change in Hb value compare to screening
|
SECONDARY outcome
Timeframe: Baseline to Day 180Number of units of packed red blood cells (PRBC) transfusions
Outcome measures
| Measure |
Coversin Treatment
n=1 Participants
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
|---|---|
|
Number of Packed Red Blood Cells (PRBC) Transfusions
Day -35
|
1 PRBC Units
|
|
Number of Packed Red Blood Cells (PRBC) Transfusions
Day -10
|
2 PRBC Units
|
|
Number of Packed Red Blood Cells (PRBC) Transfusions
Day 38
|
1 PRBC Units
|
|
Number of Packed Red Blood Cells (PRBC) Transfusions
Day 39
|
2 PRBC Units
|
Adverse Events
Coversin Treatment. Ablation Dose: Day 1. 60 mg and 30 mg (12 Hours Apart)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Coversin Treatment. Initiation Dose: Days 2 - 28. 22.5 mg Twice a Day (12 Hours Apart)
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Coversin Treatment. Maintenance Dose: Days 29 - 180. 45 mg Once Daily
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Coversin Treatment. Ablation Dose: Day 1. 60 mg and 30 mg (12 Hours Apart)
n=1 participants at risk
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
Coversin Treatment. Initiation Dose: Days 2 - 28. 22.5 mg Twice a Day (12 Hours Apart)
n=1 participants at risk
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
Coversin Treatment. Maintenance Dose: Days 29 - 180. 45 mg Once Daily
n=1 participants at risk
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
|---|---|---|---|
|
Infections and infestations
Device related Infection
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
Other adverse events
| Measure |
Coversin Treatment. Ablation Dose: Day 1. 60 mg and 30 mg (12 Hours Apart)
n=1 participants at risk
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
Coversin Treatment. Initiation Dose: Days 2 - 28. 22.5 mg Twice a Day (12 Hours Apart)
n=1 participants at risk
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
Coversin Treatment. Maintenance Dose: Days 29 - 180. 45 mg Once Daily
n=1 participants at risk
This is an open label, non-comparator study.
Patients were to receive coversin at ablation, initiation and maintenance doses:
* Ablation (Day 1): 60 mg and 30 mg (12 hours apart)
* Initiation (Day 2 - 28): 22.5 mg twice a day
* Maintenance (Day 29 - 180): 45 mg once daily
|
|---|---|---|---|
|
Investigations
Aspartate Aminotransferase Increased
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 2 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Vascular disorders
Hot Flush
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 2 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 2 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 2 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 2 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 4 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
|
Nervous system disorders
Headache
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
100.0%
1/1 • Number of events 6 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
0.00%
0/1 • All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place