Coversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 Polymorphisms

Phase 1

• Conditions: Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.; MedDRA version: 18.1Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000004857; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2015-003778-34-NL
• Lead Sponsor: Volution Immuno Pharmaceuticals (UK) Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-12-10
• Last Update Posted: 5 June 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Patients with known paroxysmal nocturnal haemoglobinuria (PNH)
LDH >= 1.5 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 ug/ml
Willing to self-inject Coversin daily of to receive daily subcutaneous injections by a home nurse or in a doctor's office or hospital clinic.
Willing to receive appropriate prophylaxes against neisseria infection by either or both immunisation or continuous or intermittent antibiotics.
Males or females taking adequate contraceptives 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 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Body weight <50kg or >100kg
Pregnancy (females)
Use of tizanidine (if on ciprofloxacin)
Use of eculizumab should be discontinued before coversin therapy is commenced. Ideally this should be 2 of more weeks before.
Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom)
Failure to satisfy the PI off fitness to participate for any other reason.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified