Coversin in paroxysmal nocturnal haemoglobinuria (PNH) in patients with resistance to eculizumab due to complement C5 polymorphisms (VIP study 578)

Phase 2

Completed

Conditions: acquired hemolytic anemia
PNH
10018911

Registration Number: NL-OMON42383

Lead Sponsor: Akari Therapeutics Plc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 1

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 50µg/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;Willing to receive appropriate prophylaxis against Neisseria infection by either or both immunisation or continuous or intermittent antibiotics;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 (see Exclusion Criteria);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);Use of tizanidine (if on ciprofloxacin);Use of eculizumab (Soliris®) should be discontinued before coversin therapy is commenced. Ideally this should be 2 or 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

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Significant reduction in mean serum LDH from Day 0 to Day 28 </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Change in LDH from Day 0 (pre-dose) at monthly intervals after Day 28<br /><br><br /><br>Change in proportion of PNH clone Day 0 * Day 90<br /><br><br /><br>Change in mean Hb Day 0 * Day 90<br /><br><br /><br>Change in mean Hp Day 0 * Day 90<br /><br><br /><br>Change in FACIT score Day 0 * Day 28 - Day 90 - Day 180<br /><br><br /><br>Change in EORTC QLQ C30 score Day 0 * Day 28 - Day 90 * Day 180</p><br>