NCT02591862

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: AKARI Therapeutics · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Paroxysmal Nocturnal Haemoglobinuria (PNH)

Interventions

Type	Name	Description
DRUG	Coversin	Patients enrolled in this protocol will initially be treated with an ablating dose of Coversin and daily repeat maintenance doses calculated according to body weight, the ablating dose to be 0.57mg/kg. Thereafter the daily repeat dose will be titrated according to clinical response and complement inhibition determined by CH50 ELISA. The initial repeat dose will be 25% of the ablating dose and this will be adjusted up or down if necessary once steady state is reached (5 days).

Timeline

• Start date: 2016-02-01
• Primary completion: 2018-03-20
• Completion: 2018-03-20
• First posted: 2015-10-30
• Last updated: 2023-07-12
• Results posted: 2023-07-12

Locations

1 site across 1 country: Netherlands