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Phase 2 N=1 Treatment

Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Enrolled (actual)
1
Serious AEs
33.3%
Results posted
May 2025
Primary outcome: Primary: Measurement of Serum Lactate Dehydrogenase (LDH) — 2913; 3056; 1453; 564 U/L

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Coversin (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
AKARI Therapeutics
Primary completion
Nov 2018

Outcome Measures

OutcomeResultp-value
PRIMARY
Measurement of Serum Lactate Dehydrogenase (LDH)
2913; 3056; 1453; 564; 351; 368
SECONDARY
Percentage Change in Haemoglobin (Hb) Compared to Screening
0; 28; 18; 22; 17; 25
SECONDARY
Number of Packed Red Blood Cells (PRBC) Transfusions
1; 2; 1; 2

Summary

Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.

Eligibility Criteria

Inclusion Criteria

  • Patients with known PNH.
  • Aged 18 and above. No upper age limit.
  • Lactate dehydrogenase (LDH) ≥1.5 upper limit of normal.
  • Must agree to use two methods of contraception that are ≥99% effective in preventing pregnancy.
  • Resistance to eculizumab (Soliris®).
  • Voluntary written informed consent.
  • Willing to self-inject Coversin daily.
  • Willing to receive appropriate prophylaxis against Neisseria infection.
  • Willing to avoid prohibited medications for duration of study.

Exclusion Criteria

  • Subjects with body weight 100 kg (220 lb).
  • Pregnancy or breast feeding (females).
  • Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom).
  • Unresolved Neisseria meningitidis infection.
  • Patients who have not received adequate immunization against Neisseria meningitides.
  • Impaired hepatic function.
  • Patients with impaired renal function.
  • Failure to satisfy the Principal Investigator (PI) of fitness to participate for any other reason.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03427060). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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