Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)

• Status: Completed
• Trial end date: 2018-03-20
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AKARI Therapeutics
• Collaborator: Radboud University
• Treatments: Complement System Proteins

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)