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An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study

Phase 1
Conditions
Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 21.1Level: PTClassification code 10018932Term: Haemolytic uraemic syndromeSystem Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 21.1Level: PTClassification code 10034042Term: Paroxysmal nocturnal haemoglobinuriaSystem Organ Class: 10038359 - Renal and urinary disorders
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Registration Number
EUCTR2016-004129-18-NL
Lead Sponsor
Akari Therapeutics Plc
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
50
Inclusion Criteria

1)Patients 18 years and above successfully treated with Coversin under other Akari clinical trial protocols and wish to remain on Coversin at the conclusion of that trial.
2)On a stable dose of Coversin for at least 2 weeks prior to entering the AK581 long term safety and efficacy surveillance study.
3)Evidence of sustained total complement inhibition by CH50 assay.
4)An appropriately qualified clinician is willing to keep the patient under observation and treatment whilst on Coversin and to be named as an investigator in this trial.
5)Willing to give informed consent .

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

1)Patient is unwilling to continue on Coversin.
2)Patient is unwilling to complete the Quaity of Life instruments and diary card.
3)Evidence of active meningococcal infection(swab testing not required).
4)On concurrent treatment with another complement inhibitor.
5)Any other reason for which, in the opinion of the investigator, it would not be in the interests of the patient to remain on Coversin

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: The objectives of the study are:<br>1)To observe the long term safety and efficacy of Coversin over periods in excess of 6 months<br>2)To assess the long term patient acceptability of Coversin using the EORTC QLQ-C30 and the EQ-5D-5L instruments<br>3)To observe the changes, if any, in the production of anti-drug antibodies (ADA) and whether such antibodies are, or become, neutralising<br>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:<br>Long term safety of Coversin as assessed by SAEs, AEs, vital signs, immunogenicity assessments, results of appropriate standard laboratory tests (clinical chemistry, haematology, coagulation, urinalysis, and ADA).<br>;Timepoint(s) of evaluation of this end point: 3, 6, 9, 12 months and every 6 monthly thereafter
Secondary Outcome Measures
NameTimeMethod
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