5674-0001_IPIG Registry_UK
Research type
Research Study
Full title
The International PNH Interest Group PNH Registry
IRAS ID
335520
Contact name
Richard Kelly
Contact email
Sponsor organisation
International PNH Interest Group (IPIG)
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a very rare blood condition where blood cells are attacked by part of the body’s immune system (the complement system). This leads to destruction of the red blood cells (haemolysis) and can cause blood clots due to activation of platelets. Patients may experience dark urine due to the removal of broken down red blood cells, difficulty swallowing, abdominal pain, breathlessness, erectile dysfunction, anaemia and fatigue. Available treatments prevent complement from attacking the blood cells, reducing symptoms and the risk of complications such as blood clots and kidney failure.
As PNH is rare, long-term follow-up data in large numbers of patients is vital to improve the understanding of PNH, enhance its management and collect information on the safety and effectiveness of treatments. As new therapies for PNH emerge, several pharmaceutical companies will need data on their treatments and this presents a risk of multiple registries or data collection efforts. To avoid the risk of break-up of long-term data if patients switch between registries and to reduce the burden on PNH centres and patients, a single registry for all PNH patients is being established.
The International PNH Interest Group (IPIG) PNH Registry is an international, observational database collecting real-world health information about PNH patients over time. The registry is owned and managed by IPIG, a not-for-profit network of international PNH experts dedicated to improving care and treatment for PNH patients. Several pharmaceutical partners are collaborating with IPIG to fund the registry, which will provide data to support their post-approval regulatory commitments. All patients with a diagnosis of PNH will be eligible, regardless of their treatment status or type of treatment they receive. Data, including optional patient reported outcomes (PROs), will be collected at six monthly intervals in accordance with the natural course of patients’ healthcare for at least 5 years.
REC name
Yorkshire & The Humber - South Yorkshire Research Ethics Committee
REC reference
24/YH/0067
Date of REC Opinion
4 Apr 2024
REC opinion
Further Information Favourable Opinion