CLNP023C12303: APPULSE
Research type
Research Study
Full title
A multicenter, single arm, open-label trial to evaluate efficacy and safety or oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan
IRAS ID
1005886
Contact name
Cecile Hauschka
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2022-502148-10
Clinicaltrials.gov Identifier
Research summary
This is a multicenter, single-arm, open label trial, with iptacopan treatment for 24 weeks in adult patients with Paroxysmal Nocturnal Haematoglobinuria (PNH). Participants in this trial must be PNH patients who are currently treated with anti-C5 treatments (standard of care) and who have been stable for at least 6 months on their current treatment. Stability will have been confirmed by demonstrating their average Haemoglobin (Hb) levels are ≥10 g/dL performed up to 12 months prior to screening visit 1 and that patients have not required red blood cell transfusions for the same period.
This study is comprised of two sections: 8-week screening and 24-week treatment and 50 participants are planned to be enrolled in the trial from 45 sites across 8 countries.
The key objective is to confirm switching from anti-c5 to iptacopan results in increased Hb levels. At screening and at the start of treatment, mean Hb assessments will confirm the baseline for the primary and key secondary objectives; baseline for all other objectives is defined as Day 1. Patients will be assessed for safety and efficacy at 10 time points during the treatment phase and followed up twice for safety post-completion. Efficacy is assessed through Hb levels and safety is assessed through routine blood tests, physical examination, vital signs, weight, ECGs, and evaluation of PNH signs and symptoms.
Following completion of the treatment phase, all patients will be allowed to continue treatment through a rollover study, where they will continue to be assessed for safety and efficacy.REC name
London - Westminster Research Ethics Committee
REC reference
23/LO/0255
Date of REC Opinion
17 Aug 2023
REC opinion
Further Information Favourable Opinion