* APPOINT-PNH
Research type
Research Study
Full title
A multicenter, single-arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who are naive to complement inhibitor therapy
IRAS ID
293217
Contact name
Austin Kulasekararaj
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2020-003172-41
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 10 months, 4 days
Research summary
Research Summary: This is a multicentre, single arm, phase 3, open-label trial which aims to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients who have not previously been treated with the standard of care anti-C5 antibody therapies. PNH is a rare disease which causes haemolysis of red blood cells, releasing haemoglobin, which leads to, amongst other symptoms, severe anaemia. The study will treat 40 adult patients (male and female) and the patients will participate in an 8 week screening period, a 24 week core treatment period and a 24 extension treatment period. The aim of the study is to assess whether treatment with iptacopan will achieve a sustained increase in the patients' haemoglobin levels by ≥ 2g/dL. This measurement confirms patients are not affected by haemolysis. In addition clinical assessments will also measure whether patients treated with iptacopan can achieve haemoglobin levels ≥ 12 g/dL (patients would be considered no longer clinically anaemic) in the absence of red blood cell transfusions. In addition to clinical assessments, changes in patients' quality of life are assessed through patient questionnaires and, in some countries (not UK), a patient interview.
Summary of results: This is a multicentre, single arm, phase 3, open-label trial which aims to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients who have not previously been treated with the standard of care anti-C5 antibody therapies. PNH is a rare disease which causes haemolysis of red blood cells, releasing haemoglobin, which leads to, amongst other symptoms, severe anaemia. The study will treat 40 adult patients (male and female) and the patients will participate in an 8 week screening period, a 24 week core treatment period and a 24 extension treatment period. The aim of the study is to assess whether treatment with iptacopan will achieve a sustained increase in the patients' haemoglobin levels by = 2g/dL. This measurement confirms patients are not affected by haemolysis. In addition clinical assessments will also measure whether patients treated with iptacopan can achieve haemoglobin levels = 12 g/dL (patients would be considered no longer clinically anaemic) in the absence of red blood cell transfusions. In addition to clinical assessments, changes in patients' quality of life are assessed through patient questionnaires and, in some countries (not UK), a patient interview.
REC name
London - Westminster Research Ethics Committee
REC reference
21/FT/0071
Date of REC Opinion
16 Jun 2021
REC opinion
Further Information Favourable Opinion