Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)

Akari Therapeutics

Status and phase: Completed; Phase 2

Conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)

Treatments: Drug: Coversin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02591862

AK578

Details and patient eligibility

About

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

Full description

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 for 2 years.

Please note, 'Coversin' is used throughout, but Nomacopan is the official name/INN.

Please note, the end points were assessed for 6 months, but the adverse events were measured over the 2 year period.

Enrollment: 1 patient

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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 50 μg/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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

Coversin (Nomacopan)

Other group

Description:

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.

Treatment:

Drug: Coversin