APPELHUS: Safety+efficacy trial of LNP023 in aHUS. Initial submission
Research type
Research Study
Full title
A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult aHUS patients who are naive to complement inhibitor therapy.
IRAS ID
303565
Contact name
David Kavanagh
Contact email
Sponsor organisation
Novartis Pharmaceuticals UK Limited
Eudract number
2020-005186-13
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 0 months, 6 days
Research summary
A phase III, multi-centre, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult atypical hemolytic uremic syndrome patients who have never received treatment with complement inhibitors. The study will assess the effects of iptacopan on a range of efficacy (how well the drug works) assessments relevant to aHUS including blood and kidney parameters, dialysis requirement, changes in chronic kidney disease (CKD) stage, as well as patient reported outcomes (PRO) for fatigue and quality of life. This study will serve as the pivotal trial for the development of iptacopan as a treatment for patients with aHUS.
The primary objective is to assess the proportion of participants treated with iptacopan achieving complete thrombotic microangiopathy response during 26 weeks of study treatment.
This study comprises of a screening period lasting up to 7 days, followed by a Core Treatment period where eligible participants will receive iptacopan study treatment at 200 mg twice daily for 26 weeks. Study participants will then continue receiving iptacopan at 200 mg twice daily for another 26 weeks as part of the Extension Treatment period.
Key inclusion criteria are: adults 18 years plus; evidence of thrombotic microangiopathy based on laboratory findings; platelets, LDH and serum creatinine within range; vaccination against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae.REC name
London - Central Research Ethics Committee
REC reference
21/LO/0692
Date of REC Opinion
10 Nov 2021
REC opinion
Further Information Favourable Opinion