Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)
Paroxysmal Nocturnal Haemoglobinuria (PNH)
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Haemoglobinuria (PNH)
Eligibility Criteria
Inclusion Criteria:
- Patients with known Paroxysmal Nocturnal Haemoglobinuria (PNH)
- LDH >=1.5 Upper Limit of Normal (ULN)
- Resistance to Eculizumab proven by both a recognised C5 polymorphism on genetic screening and complement inhibition on CH50 ELISA of <100% at concentrations of Eculizumab in excess of 5mug/mL
- Willing to self-inject Coversin daily or to receive daily subcutaneous injections by a home nurse or in a doctor's office or hospital clinic
- Males or females taking adequate contraceptive precautions if of childbearing potential, 18 - 80 years of age
- Body weight ≥50kg and ≤ 100kg
- The patient has provided written informed consent.
- Willing to avoid prohibited medications for duration of study
- Must agree to take appropriate prophylactic precautions against Neisseria infection.
- Must be counselled regarding the possible reproductive risks of using Coversin and be advised to use an adequate method of contraception pending further data on reproductive toxicology.
Exclusion Criteria:
- Body weight <50kg or>100kg
- Pregnancy (females)
- Failure to satisfy the PI of fitness to participate for any other reason
- Known allergy to ticks or severe reaction to arthropod venom (e.g., bee or wasp venom)
Sites / Locations
- Dr Saskia Langemeijer
Arms of the Study
Arm 1
Other
Coversin (Nomacopan)
This is an open label, non-comparator study. Patient will be given a single ablating dose of 0.57mg/kg per subject followed by daily repeat maintenance doses. The initial repeat dose will be 25% of the ablating dose. If this is insufficient to maintain complement inhibition at ≤10% of baseline (pre-treatment) level after 5 days of treatment the daily dose will be increased by doubling until that level of inhibition is achieved. In the event of 100% inhibition being achieved the dose may be titrated downwards at the PI's discretion until a satisfactory clinical result is obtained. If at any point in treatment complement inhibition falls to less than 50% of baseline a further ablating dose of 0.57mg/kg should be given. Coversin lyophilised powder in each vial was diluted with 0.6 mL water for injection prior to use.