Extension study of APL2 safety and efficacy, APL2-307, Amendment 1, v1
Research type
Research Study
Full title
An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
IRAS ID
272478
Contact name
Pascal Deschatelets
Contact email
Sponsor organisation
Apellis Pharmaceuticals, Inc.
Eudract number
2019-001106-23
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 3 months, 6 days
Research summary
The APL2-307 extension study is a multinational phase-III-study sponsored by Apellis Pharmaceuticals Inc. It is an open-label, non-randomized, multi-center study. The purpose of this research study is to evaluate the long-term safety and efficacy of the new investigational drug APL-2 for subcutaneous infusion in treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH). PNH is a rare disorder that causes the red blood cells to break down too early. Many PNH patients suffer from complications such as blood clots, bleeding, or low red blood cell counts (anaemia) even after receiving a standard treatment. As shown by previous clinical studies, APL-2 provides sustained inhibition of haemolytic (destruction of red blood cells) activity in PNH patients. To date, no safety signals have emerged from ongoing studies in PNH patients that preclude further development. Thus, the aim of the proposed study is to confirm efficacy of subcutaneous APL-2 as monotherapy for the treatment of PNH. The study will enroll patients with PNH who have completed another APL-2 clinical study, therefore the study population includes approximately 119 adult, male and female subjects diagnosed with PNH.
Subjects entering this study receiving APL-2 1,080 mg SC twice weekly or every 3 days will continue with their current dose regimen with no interruptions in dosing.
Subjects entering this study who previously received no APL-2 treatment will start treatment with APL-2 1,080 mg SC twice weekly.
For those subjects who enter this extension study receiving APL-2 270 mg/day, the dose of APL-2 is 1,080 mg SC twice weekly.
For those subjects who enter this extension study receiving APL-2 360 mg/day, the planned dose of APL-2 is 1,080 mg SC every three daysDuration of participation on the extension study will be until APL-2 becomes commercially available in the patient´s participating country or until APL-2 development in PNH is discontinued.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0319
Date of REC Opinion
19 Dec 2019
REC opinion
Further Information Favourable Opinion