Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1 Level: PT Classification code 10018932 Term: Haemolytic uraemic syndrome System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Product Code: rVA576
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: rVA576
Current Sponsor code: rVA576
Other descriptive name: rVA576
Concentration unit: mg milligram(s)
Sponsors
Akari Therapeutics Plc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1)Patients 18 years and above successfully treated with rVA576 (Coversin) under other Akari clinical trial protocols and wish to remain on rVA576 (Coversin)at the conclusion of that trial. 2)In the opinion of the treating responsible clinician patient is receiving clinical benefit from continued treatment with study drug. 3)Evidence of sustained total complement inhibition by CH50 assay. 4)Women of childbearing potential (WOCBP) must agree to use effective contraception consistently throughout the study and have a negative pregnancy test at screening. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of amenorrhea and considered sterile if they have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks previously. 5)Males with a childbearing potential partner must agree to use effective contraception consistently OR have had a vasectomy. 6)Weight >50kg . 7)Received appropriate prophylaxis against Neisseria meningitidis infection, by both immunisation and continuous or intermittent antibiotics 8)Patient is willing to give voluntary written informed consent. 9)The patient is willing in the process of preparation and self administration of the study drug. 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 42 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 8
Exclusion criteria
Exclusion criteria: 1)Patient experienced any safety event in the previous study protocol, which puts the patient at unacceptable risk in the current protocol in the clinical judgement of the investigator and sponsor. 2)Patient is unwilling to complete the Quaity of Life instruments and diary card. 3)Active meningococcal infection (section 4.3.2 for additional information). 4)Any other reason for which, in the opinion of the investigator, it would not be in the interests of the patient to remain on rVA576 (Coversin). 5)If female, the subject is pregnant or lactating or intending to become pregnant before, during, or within 90 days after last dose; or intending to donate ova during such period. 6)If male, the subject intends to donate sperm during this study or for 90 days after last dose. 7)Failure to satisfy the Investigator of fitness to participate for any reason or any condition which, in the opinion of the investigator, could increase the subject's risk from participating in the study or confound the outcome of the study. 8)Use of prohibited medication (e.g. eculizumab (Soliris®), Chemotherapeutic agents, any other drug acting directly on the complement system). 9)The subject has a history of drug abuse (defined as any illicit drug use) or a history of alcohol abuse within one year prior to screening. 10)Participation in other clinical trials with investigational product.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objectives of the study are: 1)To observe the long term safety and efficacy of rVA576 (Coversin) over periods in excess of 6 months 2)To assess the long term patient acceptability of rVA576 (Coversin) using the EORTC QLQ-C30 and the EQ-5D-5L instruments 3)To observe the changes, if any, in the production of anti-drug antibodies (ADA) and whether such antibodies are, or become, neutralising 4)To assess the effects, if any, on any changes in formulation or drug delivery that may be introduced during the study period ;Secondary Objective: Not applicable;Primary end point(s): Primary Efficacy Endpoints: Long term safety of rVA576 (Coversin) as assessed by SAEs, AEs, vital signs, immunogenicity assessments, results of appropriate standard laboratory tests (clinical chemistry, haematology, coagulation, urinalysis, and ADA). ;Timepoint(s) of evaluation of this end point: 3, 6, 9, 12 months and every 6 monthly thereafter until the end of the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy Endpoints: 1)Thrombotic and haemolytic event free survival at 3, 6, 9 and 12months and 6 monthly thereafter from the start of the long term safety and efficacy study. 2)Reduction in requirement for blood or blood product transfusion compared to pre-rVA576 (Coversin) treatment over a similar period if data are available (for complement inhibitor naïve patients). 3)Reduction in requirement for blood or blood product transfusion compared to pre-rVA576 (Coversin) treatment over a similar period (for patients who received treatment with another complement inhibitor before switching to rVA576 (Coversin). 4)Non-inferiority in requirement for blood or blood product transfusion compared to pre-rVA576 (Coversin) treatment over a similar period for patients who received treatment with another complement inhibitor before switching to rVA576 (Coversin). For example there should be no increase in transfusion requests for 3 months post-switch compared to 3 months pre-switch. 5)Quality of life using the EORTC QLQ-C30 and the EQ-5D-5L instruments at 3, 6, 9 and12 months and at each clinic visit thereafter. ;Timepoint(s) of evaluation of this end point: 3, 6, 9, 12 months and 6 monthly thereafter until the end of the study | — |
Countries
Argentina, France, Germany, Kazakhstan, Lithuania, Netherlands, Poland, Sri Lanka, United Kingdom
Contacts
Public ContactMedical Director
Akari Therapeutics Plc
Outcome results
None listed