A Long-term Treatment Study of ACH-0144471 in Patients with PNH
Research type
Research Study
Full title
An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment with ACH-0144471 in Patients with PNH who Completed Clinical Study ACH471-100
IRAS ID
243774
Contact name
Austin Kulasekararaj
Contact email
Sponsor organisation
Achillion Pharmaceuticals Inc.
Eudract number
2017-000665-79
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
U1111-1196-0653, UTN
Duration of Study in the UK
1 years, 6 months, 15 days
Research summary
The purpose of this study is to evaluate long term safety and effectiveness of ACH-0144471 in patients with paroxysmal nocturnal haemoglobinuria (PNH).
This study is designed to include up to 12 patients with PNH who are not currently receiving eculizumab and have completed Achillion study ACH471-100 and who have demonstrated a clinical benefit as assessed by the investigator. Clinical benefit will be assessed by the investigator, based on an improvement in haemoglobin and/or LDH.
Eligible patients will continue therapy with ACH-0144471 from the ACH471-100 study into this extension study without interruption. Each patient will enter this study at the dose they were receiving at the completion of ACH471-100. If necessary to improve control of haemolysis, the investigator in consultation with the Sponsor may escalate dosing in increments of 25 mg to a maximum of 250 mg three times a day.
Each patient will be asked to attend the hospital once every 4 weeks for the first 6 months, when clinical and laboratory assessments will be completed. After 6 months each subject will be asked to attend the hospital every 8 weeks, starting with Week 25 visit. Blood samples will be drawn at their local laboratory every 4 weeks after clinic visits.Patients may remain in the study until ACH-0144471 is commercially available in their country or the development of ACH-0144471 as a potential therapy for PNH is terminated, or until the therapy is no longer tolerated or effective.
REC name
London - City & East Research Ethics Committee
REC reference
18/LO/0892
Date of REC Opinion
23 Jul 2018
REC opinion
Further Information Favourable Opinion