Open-Label, Uncontrolled Extension Study of RA101495
Research type
Research Study
Full title
RA101495-01.202: A Multicenter, Open-Label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed A RA101495 Clinical Study
IRAS ID
217439
Contact name
Petra Muus
Contact email
Sponsor organisation
Ra Pharmaceuticals, Inc.
Eudract number
2016-003523-34
Duration of Study in the UK
2 years, 6 months, 16 days
Research summary
Summary of Research
The purpose of this extension study is to enable continued access to the investigational drug RA101495 to patients with paroxysmal nocturnal haemoglobinuria (PNH) after they complete a Ra Pharmaceuticals Inc. sponsored clinical study with RA101495.
PNH is a rare disease of the blood which is characterised by the destruction of red blood cells (haemolytic anaemia, a condition in which the blood has a lower than normal number of red blood cells) and vein thrombosis (blood clots in veins).
This study will evaluate long-term safety, tolerability, effectiveness, pharmacokinectics (PK, what the body does to the drug), and pharmacodynamics (PD, what the drug does to the body). The participants will continue to receive the drug as long as they continue benefiting from it. The investigational product will continue to be provided by Ra Pharmaceuticals Inc. until it is approved and available in the territory, or until Ra Pharmaceuticals Inc. terminate the development of the drug for PNH. All countries and centres participating in a RA101495 PNH clinical study will take part in this extension study. All the participants (up to 28) in a RA101495 PNH study are eligible to take part in this study.Summary of Results
UCB will be providing plain language summaries of all UCB-sponsored clinical trials conducted in the UK that end in 2022 and beyond. Since this clinical trial ended prior to that date, a plain language summary is not planned at this time.REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
16/YH/0504
Date of REC Opinion
24 Jan 2017
REC opinion
Further Information Favourable Opinion