A Phase 2 interventional study of Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by AKARI Therapeutics. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-05-04.
Sponsored by AKARI Therapeutics · Phase 2, Interventional, and Treatment
Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.
Coversin, a small protein complement C5 inhibitor which prevents the cleavage of C5 by C5 convertase into C5a and C5b, will be used in an open label, non-comparative clinical trial in patients with PNH and proven resistance to eculizumab due to C5 polymorphisms. Patients will be treated with Coversin by daily subcutaneous injection for 6 months in order to determine the safety and efficacy of the drug in these circumstances. If satisfactory control of the PNH is achieved, and at the discretion of the Principal Investigator (PI), patients will have the option of remaining on Coversin and being entered into the long term follow-up study.
Exclusion Criteria:
Coversin - 22.5mg followed by 45mg for 6 months.
Drug: Coversin
Coversin - 22.5mg followed by 45mg for 6 months.
Measurement of Serum Lactate Dehydrogenase (LDH)
Measurement of serum lactate dehydrogenase (LDH)
Time frame: Baseline to Day 186
Percentage Change in Haemoglobin (Hb) Compared to Screening
Percentage change in the haemoglobin (Hb) value at each study visit compared to screening
Time frame: Baseline to Day 186
Number of Packed Red Blood Cells (PRBC) Transfusions
Number of units of packed red blood cells (PRBC) transfusions
Time frame: Baseline to Day 180
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.
| Milestone | Coversin Treatment |
|---|---|
| Started | 1 |
| Completed | 1 |
| Not completed | 0 |
Measurement of serum lactate dehydrogenase (LDH)
| U/L | Coversin Treatment |
|---|---|
| Screening | 2913 |
| Day 1 | 3056 |
| Day 3 | 1453 |
| Day 7 | 564 |
| Day 14 | 351 |
| Day 21 | 368 |
| Day 29 | 329 |
| Day 36 | 339 |
| Day 43 | 555 |
| Day 60 | 319 |
| Day 90 | 394 |
| Day 120 | 352 |
| Day 150 | 284 |
| Day 183 | 906 |
| Day 184 | 882 |
| Day 186 | 612 |
Percentage change in the haemoglobin (Hb) value at each study visit compared to screening
| %change in Hb value compare to screening | Coversin Treatment |
|---|---|
| Screening | 0 |
| Day 1 | 28 |
| Day 3 | 18 |
| Day 7 | 22 |
| Day 14 | 17 |
| Day 21 | 25 |
| Day 29 | 23 |
| Day 36 | 9 |
| Day 43 | 26 |
| Day 60 | 60 |
| Day 90 | 28 |
| Day 120 | 18 |
| Day 150 | 23 |
| Day 186 | 26 |
Number of units of packed red blood cells (PRBC) transfusions
| PRBC Units | Coversin Treatment |
|---|---|
| Day -35 | 1 |
| Day -10 | 2 |
| Day 38 | 1 |
| Day 39 | 2 |
Collected over All adverse events were collected from the time of informed consent until completion of patient's study participation. From Screening to Day 186. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Coversin Treatment. Ablation Dose: Day 1. 60 mg and 30 mg (12 Hours Apart) | 0/1 (0%) | 0/1 (0%) | 0/1 (0%) |
| Coversin Treatment. Initiation Dose: Days 2 - 28. 22.5 mg Twice a Day (12 Hours Apart) | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Coversin Treatment. Maintenance Dose: Days 29 - 180. 45 mg Once Daily | 0/1 (0%) | 0/1 (0%) | 0/1 (0%) |
| Event | Coversin Treatment. Ablation Dose: Day 1. 60 mg and 30 mg (12 Hours Apart) | Coversin Treatment. Initiation Dose: Days 2 - 28. 22.5 mg Twice a Day (12 Hours Apart) | Coversin Treatment. Maintenance Dose: Days 29 - 180. 45 mg Once Daily |
|---|---|---|---|
| Device related InfectionInfections and infestations | 0/1 | 1/1 | 0/1 |
| Febrile NeutropeniaBlood and lymphatic system disorders | 0/1 | 1/1 | 0/1 |
| Urinary Tract InfectionInfections and infestations | 0/1 | 1/1 | 0/1 |
| Event | Coversin Treatment. Ablation Dose: Day 1. 60 mg and 30 mg (12 Hours Apart) | Coversin Treatment. Initiation Dose: Days 2 - 28. 22.5 mg Twice a Day (12 Hours Apart) | Coversin Treatment. Maintenance Dose: Days 29 - 180. 45 mg Once Daily |
|---|---|---|---|
| Aspartate Aminotransferase IncreasedInvestigations | 0/1 | 1/1 | 0/1 |
| Hot FlushVascular disorders | 0/1 | 1/1 | 0/1 |
| NauseaGastrointestinal disorders | 0/1 | 1/1 | 0/1 |
| Urinary Tract InfectionInfections and infestations | 0/1 | 1/1 | 0/1 |
| VomitingGastrointestinal disorders | 0/1 | 1/1 | 0/1 |
| HypoglycaemiaMetabolism and nutrition disorders | 0/1 | 1/1 | 0/1 |
| HeadacheNervous system disorders | 0/1 | 1/1 | 0/1 |
| Age, Categorical(Participants) | Coversin Treatment |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 1 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Coversin Treatment |
|---|---|
| Female | 1 |
| Male | 0 |
| Race (NIH/OMB)(Participants) | Coversin Treatment |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 0 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
| Height(cm) | Coversin Treatment |
|---|---|
| Number | 155 |
| Weight(kg) | Coversin Treatment |
|---|---|
| Number | 73.3 |
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