Research & Trials

Current SACC trials

AAV Study: A seroprevalence study of the presence of Adenovirus-Associated Virus Vector–serotypes AAV2, AAV5 and AAV8 neutralizing activity and antibodies in Patients with Haemophilia A

A multi-centre laboratory study using samples collected prospectively from patients with Haemophilia A from collaborating hospitals within the UK. This study’s objective is to study the seroprevalence of AAV2, AAV5 and AAV8 neutralizing factors and antibodies in a 100 previously treated haemophilia A patients.

A-JOINT: Assessing joint health in patients with mild to moderate haemophilia

A multicentre cross sectional study to determine the relationship between joint health and factor VIII levels in patients with mild/moderate haemophilia.
Many studies have shown that regular FVIII concentrate treatment (prophylaxis therapy) prevents joint arthropathy by reducing joint damage and frequencies of bleeding. This has translated into the clinical care universally where patients with severe Haemophilia A are offered prophylaxis therapy with factor VIII concentrate from childhood. In spite of this, the minimal trough level required to prevent joint damage has not been well defined and there is a lack of robust evidence to support any particular target trough level.
Patients with mild to moderate haemophilia A have lived with a variety of FVIII levels. The degree of joint damage in these patients may offer an insight into the consequence of a wide range of FVIII level. This will in turn give us a better understanding on the minimum trough level required to prevent joint damage in patients with severe haemophilia A.

CARES: Haemophilia Carriers’ Experience Study: Life Choices, Psychosocial Needs and Parenting

The primary objective of this study is to explore the experience of being a carrier of haemophilia. We want to develop a better understanding of mood, self-perception, relationships, and general wellbeing, as well as the decision-making process that carriers face when deciding whether to have a family, or further children after a child with haemophilia.
This study uses a mixed-methods design. In Stage 1, focus groups will be carried out and qualitative methods used to undertake an in-depth exploration of the experience of carriers and identify relevant themes. The themes identified will then be used in Stage 2 to develop a quantitative questionnaire measure that will ‘test out’ and look at the significance of the themes across a larger sample of carriers.

Long term outcome of Fanhdi® in Haemophilia A patients in London

Single centre, observational, retrospective study of haemophilia A patients who received pdFVIII/VWF (Fanhdi®). This study is assessing the long-term efficacy, safety and tolerability, and cost effectiveness of long term Fanhdi® use in patients with haemophilia A.

TABI: Thrombin Generation Assay to predict Bypassing products in Haemophilia Patients with Inhibitors

This is a multicentre study to develop a laboratory technique to monitor and predict the efficacy of bypassing agents for each individual haemophilia patient with an inhibitor. This laboratory method in turn can be used to develop an individualised approach to bypassing product treatment in haemophilia A with inhibitors.

UK-PK: National Study of the Implementation of a Pharmacokinetic-Focused Educational Package for patients living with Severe Haemophilia A

In this multi-centre study we want to observe what happens when myPKFiT®, a new dosing tool for people with severe Haemophilia A, is used in day to day practice. We will capture patient reported outcome measures before and after pharmacokinetic (PK) focused dosing discussions, and examine if and how individual treatment regimens may change.

myPKFiT® is being introduced as part of day to day care in haemophilia centres across the UK to help tailor prophylactic treatment. This tool works by calculating what happens to factor VIII treatment once a patient has injected it, this is a process called pharmacokinetics or PK. PK tracks what happens with the factor VIII from the second it enters the body from the syringe to the moment it leaves the body. It lets us know how high factor levels peak and how long it takes until there is no factor VIII left in the blood. PK can be very different between different people. This means that individuals may need very different ways of managing their prophylaxis. myPKFiT® helps individual patients and their haemophilia team to look at the different treatment options available to them.