rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Phase 3TerminatedNCT03829449

AKARI Therapeutics15 enrolled

Overview

Long term management of patients with complement related diseases including Paroxysmal Nocturnal Haemoglobinuria and Atypical Haemolytic Uraemic Syndrome

Patients with diseases requiring complement inhibition who have previously taken part in Akari clinical trials and who wish to continue to receive rVA576 (Coversin) after their active participation in the parent trial has completed and patients treated under compassionate use or named patient arrangements who wish to continue on rVA576 (Coversin) therapy

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

rVA576DRUG

The study population will consist of patients who have completed participation in clinical trials under other Akari protocols and who wish to continue to receive rVA576 (Coversin).

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Patients 18 years and above 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 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 and a negative urine pregnancy test per the schedule of visits. Women cannot donate their eggs. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of amenorrhea or 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 ≥50-100kg 7. Willing to receive appropriate prophylaxis against Neisseria meningitidis infection, by both immunisation and continuous or intermittent antibiotics 8. The 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. Exclusion Criteria: 1. Patient experienced any safety event in the previous study protocol, which puts the patient at unacceptable risk in current protocol as judged by the investigator and sponsor. 2. Patient is unwilling to complete the Quality of Life instruments and diary card 3. Active meningococcal infection (section 4.3.1 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 time period. 6. If male, the subject intends to donate sperm while on the study this study or for 90 days after last dose. 7. Failure to satisfy the Investigator of fitness to participate for any other reason or any other condition which, in the opinion of the investigator, could increase the subject's risk by participating in the study or confound the outcome of the study. 8. Use of prohibited medication 9. The subject has a history of drug abuse (defined as any illicit drug use) or a history of alcohol abuse. 10. Participation in other clinical trials with investigational product.

Locations (1)

Instytut Hematologii i Transfuzjologii

Warsaw, Poland

Outcomes

Primary Outcomes

Long Term Safety and Efficacy of rVA576 (Coversin) Therapy Assessed by AEs, SAEs, Standard Lab Tests and ECG Results.

To determine the safety profile of long-term rVA576 (Coversin) treatment as assessed by AEs, SAEs, Standard Lab tests and ECG results.

Time frame: On entry and every 3 months thereafter, for the duration of the study (approximately 3 years and 5 months)

Secondary Outcomes

Proportion of Subjects With Thrombotic and Haemolytic Event Free Status During Each 3month Time Period Since the Start of the Study.

Thrombotic and Haemolytic Events will include, but are not limited to, the following: haemolytic anaemia, thrombocytopenia, red blood cell haemolysis indicated by dark urine, Budd-Chiara syndrome, any other thrombotic or haemolytic event deemed to be associated with PNH. A haemolytic event will be defined as a rise in LDH or other biochemical evidence of haemolysis accompanied by an increase in symptoms and/or frank haemoglobinuria. Increased symptoms without objective haematological or biochemical evidence of haemolysis will not be counted as haemolytic events. In addition, the following signs and symptoms may be reviewed and classified as Thrombotic/Haemolytic events if appropriate: Acute kidney failure, Hypertension, Myocardial infarction, Stroke, Lung complications, Seizure, Coma, Premature death.

Time frame: On entry and every 3 months thereafter, for the duration of the study (approximately 3 years and 5 months)

Time to Thrombotic or Haemolytic Event Since Joining This Study.

Time to thrombotic or haemolytic event since joining this study.

Time frame: Approximately 3 years and 5 months

Proportion of Subjects Who Require PRBC Transfusion During Each 3-month Period Since the Start of the Study and Over the Entire Period of the Study

Proportion of subjects who require PRBC transfusion during each 3-month period since the start of the study and over the entire period of the study

Time frame: On entry and every 3 months thereafter, for the duration of the study (approximately 3 years and 5 months)

Data from ClinicalTrials.gov

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